FDA Approves Tepylute (thiotepa) Ready-to-Dilute Injectable Formulation to Treat Breast Cancer and Ovarian Cancer

Shorla Oncology (‘Shorla’), a U.S.-Ireland specialty pharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has approved the company’s New Drug Application (NDA) for Tepylute, a ready-to-dilute formulation to treat breast and ovarian cancer in an easier to prepare, injectable product that enables dosing accuracy.

Shorla Oncology (‘Shorla’), a U.S.-Ireland specialty pharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has approved the company’s New Drug Application (NDA) for Tepylute, a ready-to-dilute formulation to treat breast and ovarian cancer in an easier to prepare, injectable product that enables dosing accuracy.1

‘’This approval fulfills an unmet need by addressing the shortcomings and handling complexities of the current lyophilized powder formulation,” said Sharon Cunningham, Chief Executive Officer and Co-Founder of Shorla Oncology. “We have taken a vital oncology drug and made it easier for oncology clinics and hospitals to use, while also reducing medical personnel exposure to a hazardous drug.”

Tepylute, formerly SH-105, is the third FDA-approved drug for Shorla, and a significant milestone for the company as it seeks approval for several cancer-fighting drugs for the U.S. market.

“The approval of Tepylute represents an important milestone for Shorla as our first in-house developed NDA,” said Orlaith Ryan, Chief Technical Officer and Co-Founder of Shorla Oncology.

Tepylute is a liquid form of a well-established, standard of care oncology drug, thiotepa. The new formulation eliminates the need for complex and time-consuming reconstitution. It provides consistent dosing accuracy and allows for “just in time” preparation.2

“Among Tepylute’s many benefits, it removes the necessity to reconstitute which can introduce additional risks of drug preparation errors,” emphasized Rayna Herman, Chief Commercial Officer. “We look forward to providing an update on our launch plans for Tepylute in the near future.”

The American Cancer Society estimates that more than 300,000 women will be diagnosed with breast cancer in the U.S in 2024.3 About 19,680 women will be diagnosed with ovarian cancer in the United States.4

Shorla Oncology is currently marketing two products with a robust pipeline including SH-201, the first palatable oral liquid treatment for certain forms of leukemia and other cancers. In April, the company announced the FDA had accepted SH-201 for an NDA review with an expected action date of November 30, 2024.

‘’This approval fulfills an unmet need by addressing the shortcomings and handling complexities of the current lyophilized powder formulation,” said Sharon Cunningham, Chief Executive Officer and Co-Founder of Shorla Oncology. “We have taken a vital oncology drug and made it easier for oncology clinics and hospitals to use, while also reducing medical personnel exposure to a hazardous drug.”

Tepylute, formerly SH-105, is the third FDA-approved drug for Shorla, and a significant milestone for the company as it seeks approval for several cancer-fighting drugs for the U.S. market.

“The approval of Tepylute represents an important milestone for Shorla as our first in-house developed NDA,” said Orlaith Ryan, Chief Technical Officer and Co-Founder of Shorla Oncology.

Tepylute is a liquid form of a well-established, standard of care oncology drug, thiotepa. The new formulation eliminates the need for complex and time-consuming reconstitution. It provides consistent dosing accuracy and allows for “just in time” preparation.2

“Among Tepylute’s many benefits, it removes the necessity to reconstitute which can introduce additional risks of drug preparation errors,” emphasized Rayna Herman, Chief Commercial Officer. “We look forward to providing an update on our launch plans for Tepylute in the near future.”

The American Cancer Society estimates that more than 300,000 women will be diagnosed with breast cancer in the U.S in 2024.3 About 19,680 women will be diagnosed with ovarian cancer in the United States.4

Shorla Oncology is currently marketing two products with a robust pipeline including SH-201, the first palatable oral liquid treatment for certain forms of leukemia and other cancers. In April, the company announced the FDA had accepted SH-201 for an NDA review with an expected action date of November 30, 2024.

FDA Approves Tepylute (thiotepa) Ready-to-Dilute Injectable Formulation to Treat Breast Cancer and Ovarian Cancer

FDA Approves Ohtuvayre (ensifentrine) for the Maintenance Treatment of Chronic Obstructive Pulmonary Disease (COPD)

Verona Pharma plc (Nasdaq: VRNA) (“Verona Pharma” or the “Company”), announces the US Food and Drug Administration (“FDA”) approved Ohtuvayre (ensifentrine) for the maintenance treatment of chronic obstructive pulmonary disease (COPD) in adult patients. Ohtuvayre is the first inhaled product with a novel mechanism of action available for the maintenance treatment of COPD in more than 20 years.

Ohtuvayre is a first-in-class selective dual inhibitor of the enzymes phosphodiesterase 3 and phosphodiesterase 4 (“PDE3 and PDE4”) that combines bronchodilator and non-steroidal anti-inflammatory effects in one molecule. Ohtuvayre is delivered directly to the lungs through a standard jet nebulizer without the need for high inspiratory flow rates or complex hand-breath coordination.

“The approval of Ohtuvayre is a significant advance in COPD care, and we believe Ohtuvayre’s novel profile can change the treatment paradigm for COPD,” said David Zaccardelli, Pharm. D., President and Chief Executive Officer of Verona Pharma. “We plan to launch Ohtuvayre in the third quarter 2024, ensuring Ohtuvayre is available to help the millions of patients who still experience daily COPD symptoms.”

Michael Wells, MD, Associate Professor in the Division of Pulmonary, Allergy, and Critical Care Medicine at the University of Alabama Birmingham, commented: “In my experience, despite maintenance therapy, most patients report grappling with daily symptoms, including breathlessness and persistent coughing. COPD has a significant impact on both mortality and morbidity in the US, and until today, innovation in inhaled treatment modalities has been limited to combinations of existing treatment classes for over two decades. Ohtuvayre, as a first-in-class PDE3 and PDE4 inhibitor, offers a needed, unique approach and is an important advance in the treatment of COPD.”

The US approval of Ohtuvayre was based on extensive data including the Phase 3 ENHANCE trials, the results of which were published in the American Journal of Respiratory and Critical Care Medicine. In the ENHANCE trials, Ohtuvayre demonstrated clinical benefits both alone and when used with other maintenance therapies. Ohtuvayre was well-tolerated in a broad population of subjects with moderate to severe COPD.

The Company is fully staffed to launch and expects Ohtuvayre to be available in the third quarter 2024 through an exclusive network of accredited specialty pharmacies.

About Ohtuvayre (ensifentrine)

Ohtuvayre is the first inhaled therapy for the maintenance treatment of COPD that combines bronchodilator and non-steroidal anti-inflammatory activities in one molecule. Verona has evaluated nebulized Ohtuvayre in its Phase 3 clinical program ENHANCE (“Ensifentrine as a Novel inHAled Nebulized COPD thErapy”) for COPD maintenance treatment. Ohtuvayre met the primary endpoint in both ENHANCE-1 and ENHANCE-2, demonstrating statistically significant and clinically meaningful improvements in lung function. A fixed-dose combination of ensifentrine and glycopyrrolate, a LAMA, is currently under development for the maintenance treatment of COPD. Ensifentrine has potential applications for development in non-cystic fibrosis bronchiectasis, cystic fibrosis, asthma and other respiratory diseases.

REF: https://en.wikipedia.org/wiki/Ensifentrine

FDA Approves Ohtuvayre (ensifentrine) for the Maintenance Treatment of Chronic Obstructive Pulmonary Disease (COPD)

FDA Approves Sofdra (sofpironium) Topical Gel for the Treatment of Primary Axillary Hyperhidrosis

Clinical dermatology company, Botanix Pharmaceuticals Ltd. (ASX:BOT, Botanix or the Company), is pleased to announce the US Food and Drug Administration (FDA) approval of Sofdra™ (sofpironium) gel, 12.45%. Sofdra is a prescription medicine used to treat primary axillary hyperhidrosis (excessive underarm sweating) in adults and children 9 years and older.

Sofdra is the first and only new chemical entity approved by the FDA to treat primary axillary hyperhidrosis and presents a novel safe and effective solution for patients who have lacked treatment options for this socially challenging medical condition.

Botanix Chief Executive Officer, Dr Howie McKibbon, commented: "We are pleased to share this accomplishment with our dedicated Botanix team and dermatologist partners, patients who participated in the clinical studies and our shareholders who made this approval possible."

"This is a transformative event for Botanix as we transition from a development stage to a revenue generating dermatology company."

Hyperhidrosis is a condition characterised by abnormally increased sweating, beyond that required to regulate body temperature.1 The disproportionate sweat production that characterizes hyperhidrosis, results in a disabling medical condition with profound effects on the patient’s quality of life. Hyperhidrosis affects work productivity, daily routine activities, emotional well-being and personal relationships.2 Hyperhidrosis is the third largest dermatology condition (after acne and atopic dermatitis), with approximately 10 million patients in the US with primary axillary hyperhidrosis.3

David Pariser, MD, leading expert on hyperhidrosis, founding board member of the International Hyperhidrosis Society and past President of the American Academy of Dermatology commented: "The approval of Sofdra is terrific news for the hyperhidrosis community, which has been frustrated by the lack of effective and convenient treatment options."

"The availability of a new treatment alternative that is topical, well-tolerated, effective and easy to use is truly exciting and would be welcomed amongst patients and physicians."

The FDA approval of Sofdra was supported by results from the two pivotal Phase 3 ‘CARDIGAN’ studies which evaluated the efficacy and safety of Sofdra versus vehicle and enrolled 701 patients with primary axillary hyperhidrosis. In the studies, treatment with Sofdra successfully met all primary and secondary endpoints with clinically and statistically meaningful changes from baseline in Gravimetric Sweat Production (GSP) and the Hyperhidrosis Disease Severity Measure-Axillary, 7-item (HDSM-AX7) score.

An early patient experience program is planned to be launched by the Company in Q3 CY2024 to enable highly qualified patients to gain early access to Sofdra. These patients will be guided through the telemedicine and payer reimbursement process to be the first commercial users of the product. Broader launch of Sofdra is expected to follow in early Q4 CY2024 and Botanix expects to receive first revenues from sales in Q4 CY2024.

Botanix Executive Chairman, Mr Vince Ippolito, commented: "We are very excited to provide a new option for the 10 million patients with primary axillary hyperhidrosis in the United States."

"As the first and only new chemical entity, Sofdra represents a new therapeutic approach for dermatologists to treat patients with this disabling medical condition."

REF: https://en.wikipedia.org/wiki/Sofpironium_bromide

FDA Approves Sofdra (sofpironium) Topical Gel for the Treatment of Primary Axillary Hyperhidrosis

FDA Approves Vigafyde (vigabatrin) as the First and Only Ready-to-Use Vigabatrin Oral Solution

Pyros Pharmaceuticals, Inc. (Pyros or the Company), a leader in the development of enhanced specialty pharmaceuticals for rare diseases, announced today that the U.S. Food and Drug Administration (FDA) has approved Vigafyde™, the only ready-to-use vigabatrin oral solution. Vigafyde™ (vigabatrin) oral solution, is indicated as a monotherapy for the treatment of pediatric patients 1 month to 2 years of age with infantile spasms (IS), where the potential benefits outweigh the potential risk of vision loss.  

Infantile spasms, a rare but severe form of epilepsy, poses significant challenges for patients and their families. Pyros is committed to providing dependable services and an affordable treatment option to help ensure that families facing an IS diagnosis, and whose children are prescribed vigabatrin can quickly obtain this essential therapy.

"The approval of Vigafyde™ marks the first new drug indicated for infantile spasms in fifteen years and underscores our unwavering commitment to supporting families facing this challenging condition," stated Michael Smith, co-founder and Chief Executive Officer of Pyros.

“This is a momentous day for patients and caregivers who have long awaited the first ready-to-use vigabatrin. I believe this approval will bring renewed energy and momentum to the infantile spasms community, enhancing our efforts to improve disease education, develop care pathways, and expand investment in infantile spasms research,” stated Edwin Urrutia, co-founder and Chief Operating Officer at Pyros.

Vigafyde™ is expected to be available in the second half of 2024. Additionally, Pyros Total Care™, the Company’s personalized comprehensive support program, is available to assist families throughout the treatment journey.

About Pyros Total Care™

As part of the Company’s commitment to prioritizing patient access to treatments for those who need them most, Pyros provides ongoing personalized support to caregivers through Pyros Total Care™ for those prescribed Vigafyde™. The support program offers personal assistance and financial resources to caregivers whose child is starting or continuing therapy.

Our dedicated support team includes a nurse educator, reimbursement support, and a clinical pharmacist. For more information, visit PyrosTotalCare.com or call 1-888-760-8330, Monday to Friday, 8 a.m. to 5 p.m. Central Time.

About Infantile Spasms

Infantile spasms (IS) is a rare, severe form of epilepsy that typically begins in children less than one year old.1 Infantile spasms can appear as subtle but repetitive movements that can often be overlooked or misdiagnosed. IS can lead to long-term permanent issues such as continued seizures, other forms of epilepsy, autism spectrum disorder, and developmental issues.2 The American Academy of Neurology conducted a systematic review of treatment for IS and concluded that successful treatment of IS can improve the long-term prognosis.3

About Vigabatrin

Vigabatrin is a medication used in the treatment of infantile spasms and is designed to inhibit the enzyme GABA transaminase, consequently increasing gamma-aminobutyric acid (GABA) levels in the brain.4,5 This release is thought to enhance seizure control for patients by modulating neuronal excitability.6 Vigabatrin's mechanism of action underscores its importance in treating seizure disorders.7 Its impact may extend beyond seizure management, with emerging research indicating potential benefits in mitigating neurodevelopmental complications associated with certain epilepsy syndromes

FDA Approves Vigafyde (vigabatrin) as the First and Only Ready-to-Use Vigabatrin Oral Solution

FDA Grants Accelerated Approval to Iqirvo (elafibranor) for the Treatment of Primary Biliary Cholangitis

Ipsen (Euronext: IPN; ADR: IPSEY) today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Iqirvo (elafibranor) 80 mg tablets for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA. Iqirvo may be prescribed immediately in the U.S. for eligible patients.

This indication is approved under accelerated approval based on the reduction of alkaline phosphatase (ALP). Improvement in survival or prevention of liver decompensation events has not been demonstrated. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). Iqirvo is not recommended for people who have or develop decompensated cirrhosis (e.g., ascites, variceal bleeding, hepatic encephalopathy).

“For a significant number of people living with PBC, available treatments do not control the condition and may exacerbate symptoms of PBC. Left unmanaged, PBC can progress, leading to liver failure and in some cases, the need for a liver transplant,” said Christelle Huguet, Executive Vice President, Head of Research and Development at Ipsen. “Iqirvo demonstrated statistically significant improvements in biochemical response compared to UDCA alone. Iqirvo is, therefore a much-needed treatment option and the first new medicine for PBC in nearly a decade.”

Iqirvo is a first-in-class oral, once-daily peroxisome proliferator-activated receptor (PPAR) agonist. Iqirvo was in-licensed from GENFIT in 2021. The accelerated approval of Iqirvo is based on data from the Phase III ELATIVE trial published in the New England Journal of Medicine1. The ELATIVE trial demonstrated that 13 times more patients achieved the composite primary endpoint of biochemical response when treated with Iqirvo plus UDCA (n=108) versus placebo plus UDCA (=53) (respectively 51% versus 4% for a 47% treatment difference). ALP is a biochemical marker and is used as a surrogate endpoint in PBC trials. Secondary endpoints showed normalization in ALP levels in only Iqirvo-treated patients (15% for Iqirvo plus UDCA (n=108) versus 0% for placebo plus UDCA (n=53)). Most patients (95%) received study treatment (Iqirvo or placebo) in combination with UDCA.

The most common adverse reactions with Iqirvo reported in ≥10% of study participants were weight gain, abdominal pain, diarrhea, nausea, and vomiting. Some study participants treated with Iqirvo experienced myalgia, myopathy and rhabdomyolysis; fractures; adverse effects on fetal and newborn development; drug-induced liver injury; hypersensitivity reactions; or biliary obstruction. See full Important Safety Information below.

“Data from the pivotal Phase III ELATIVE clinical trial demonstrated that Iqirvo is an effective second-line treatment for patients with PBC with favorable benefit and risk data,” said Dr. Kris Kowdley, Director at Liver Institute Northwest, Washington, and a primary investigator on the ELATIVE study. “The approval of Iqirvo will allow healthcare providers in the U.S. to address an unmet need with the potential to significantly reduce ALP levels for our patients with PBC.”

PBC is a rare, autoimmune, cholestatic liver disease where a build-up of bile and toxins (cholestasis) and chronic inflammation causes irreversible fibrosis (scarring) of the liver and destruction of the bile ducts. Impacting approximately 100,000 people in the U.S.2, the majority being women, PBC is a lifelong condition that can worsen over time if not effectively treated, leading to liver transplant and in some cases, premature death. PBC also affects day-to-day life, with people most commonly experiencing severe fatigue symptoms and debilitating itch (pruritus).

“People living with PBC can feel like the symptoms they experience are dismissed by family members, friends, or even their doctors because they have not experienced something similarly disruptive in their lives. People with PBC may also feel uncertainty around the disease progression and if, or when, their liver health may deteriorate,” said Carol Roberts, Executive President of PBCers, a patient advocacy organization in the U.S. providing support to people living with PBC. “Earlier diagnosis and education about PBC, along with new treatment options are important to meet the current needs of people living with PBC.”

Iqirvo has been submitted to the European Medicines Agency (EMA) and the UK Medicines and Healthcare Products Regulatory Agency (MHRA), seeking authorization for PBC, with final EMA and MHRA regulatory decisions anticipated in the second half of 2024. The FDA approval of Iqirvo further strengthens Ipsen’s portfolio of treatments for rare cholestatic liver diseases available to patients in the U.S. This includes our FDA-approved medicine for the treatment of pruritus in patients three months and older with progressive familial intrahepatic cholestasis (PFIC) and for the treatment of cholestatic pruritus in patients from 12 months of age with Alagille syndrome (ALGS).

REF : https://en.wikipedia.org/wiki/Elafibranor

FDA Grants Accelerated Approval to Iqirvo (elafibranor) for the Treatment of Primary Biliary Cholangitis

FDA Approves Lumisight (pegulicianine) Optical Imaging Agent to Illuminate Residual Breast Cancer Post-Lumpectomy

Lumicell, Inc., a privately held company focused on developing innovative fluorescence-guided imaging technologies for cancerous tissue detection during surgery, today announced the U.S. Food & Drug Administration (FDA) approved the company’s New Drug Application (NDA) for its Lumisight™ (pegulicianine) optical imaging agent and its Premarket Approval (PMA) application for Lumicell™ Direct Visualization System (DVS), together referred to as LumiSystem™.

With 84% diagnostic accuracy, LumiSystem enables surgeons to scan the breast cavity post-lumpectomy, in real-time, to detect and resect residual cancer that may have otherwise been missed, potentially sparing some patients from second surgeries.1-2 The LumiSystem combination is indicated for fluorescence imaging in adults with breast cancer as an adjunct for the intraoperative detection of cancerous tissue within the resection cavity following removal of the primary specimen during lumpectomy surgery.

“We are immensely proud of the dual approval of Lumisight and Lumicell DVS – we believe this is the first drug-device combination product approved in over a decade to have followed both of the FDA's most stringent NDA and PMA review processes,”3 said Howard Hechler, President and Chief Operating Officer, Lumicell. “With the FDA’s approval, LumiSystem is now the first and only imaging combination product capable of detecting cancerous tissue where it matters most, inside the breast cavity.”

“Breast cancer is all too common, and sadly, 1 in 8 women will develop it during their lifetime,”4 said Kelly Hunt, MD, Chair of the Department of Breast Surgical Oncology at MD Anderson Cancer Center and President of the Society of Surgical Oncology. “Our most common surgical procedure to treat these women is lumpectomy. Unfortunately, the intraoperative tools we have are limited and do not identify the extent of tumor accurately enough, making it challenging to achieve a complete tumor resection, leading to as many as 36% of patients needing a second surgery.”5

“Up to 65% of the time, we do not find residual cancer in the second surgery and are left wondering if we performed an unnecessary surgery due to a false positive margin assessment or if the cancer was missed again in the second surgery,”6 said Irene Wapnir, MD, Breast Surgical Oncologist and Professor of Surgery, Stanford University School of Medicine.

“During lumpectomy surgery, surgeons still struggle to identify and remove all of the tumor during the first operation,”7 said Barbara Smith, MD, PhD, Director of the Breast Program at Massachusetts General Hospital and Professor of Surgery at Harvard Medical School. “With LumiSystem, we will now have a technology that is clinically proven to achieve a more complete cancer resection during lumpectomy that could help some patients avoid a second surgery.”2

The safety of the system was established using data from more than 700 breast cancer patients across five clinical studies at top academic and regional community cancer centers across the U.S. The most common side effects with Lumisight are hypersensitivity and an abnormal color in urine. Lumisight may cause serious hypersensitivity reactions, including anaphylaxis. Results of the Investigation of Novel Surgical Imaging for Tumor Excision (INSITE) pivotal trial, used to support the efficacy of the system, were published in NEJM Evidence.

“We want to thank our clinical investigators and the hundreds of women who participated in our breast program for Lumisight and Lumicell DVS,” said Jorge Ferrer, Chief Scientific Officer, Lumicell. “Due to your important contributions, Lumisight and Lumicell DVS are now approved and will be available in the United States shortly.”

FDA Approves Lumisight (pegulicianine) Optical Imaging Agent to Illuminate Residual Breast Cancer Post-Lumpectomy

FDA Approves Xromi (hydroxyurea) Oral Solution for Use in Pediatric Patients with Sickle Cell Anemia

The U.S. Food and Drug Administration (FDA) has approved Xromi, an oral solution formulation of hydroxyurea indicated to reduce the frequency of painful crises and reduce the need for blood transfusions in pediatric patients aged 6 months of age to less than 2 years with sickle cell anemia with recurrent moderate to severe painful crises.

Sickle cell anemia is caused by an abnormal version of hemoglobin called hemoglobin S, which leads to sickle-shaped red blood cells that can form painful clumps inside the blood vessels. These painful episodes are called sickle cell crises, and are one of the most common and distressing symptoms of sickle cell disease.

Hydroxyurea has been shown to reduce the frequency of painful episodes associated with sickle cell disease. It is thought to work by increasing levels of hemoglobin F (also called fetal hemoglobin because it is present in newborn babies) to make the red blood cells bigger, rounder, more flexible, and less likely to turn into a sickle shape.

Hydroxyurea was first approved in 1967 in an oral capsule formulation as a cancer treatment and has been used by doctors to treat sickle cell anemia since the 1980s. It was approved by the FDA for treating adults with sickle cell anemia in 1998, and children in 2017.

Xromi is supplied as a strawberry-flavored oral solution containing 100 mg/mL hydroxyurea. The Xromi package contains two dosing oral dosing syringes (a red oral dosing syringe graduated to 3 mL and a white oral dosing syringe graduated to 12 mL) for accurate measurement of the prescribed dose.

FDA Approves Xromi (hydroxyurea) Oral Solution for Use in Pediatric Patients with Sickle Cell Anemia

FDA Approves Rytelo (imetelstat) for the Treatment of Adult Patients with Lower-Risk MDS with Transfusion-Dependent Anemia

Geron Corporation (Nasdaq: GERN), a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer, today announced that the U.S. Food and Drug Administration (FDA) has approved Rytelo™ (imetelstat) for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent (TD) anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESA).

“With the approval and availability of Rytelo, we believe eligible patients with lower-risk MDS can potentially experience meaningful clinical benefit, particularly the potential for greater than 24 weeks of freedom from the burden of red blood cell transfusions and symptomatic anemia,” said John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer. “The approval of Rytelo as the first telomerase inhibitor is a testament to the power of our science and the passion of our people to innovate in the field of blood cancer. As we celebrate today’s momentous milestone, I would like to thank the patients and families, advocates, clinicians, study coordinators and site personnel, scientists, and Geron employees and collaborators past and present whose participation was integral to this achievement and to supporting our transformation into a commercial company.”

Lower-risk myelodysplastic syndromes (LR-MDS) is a blood cancer that often progresses to require increasingly intensified management of key symptoms such as anemia and resulting fatigue1. These symptomatic LR-MDS patients frequently become red blood cell transfusion dependent, which has been shown to be associated with short- and long-term clinical consequences that reduce quality of life and shorten survival2,3. There is a high unmet need for many LR-MDS patients, particularly those with characteristics having poorer prognosis. Current treatment options for those failing ESA are limited to select sub-populations and there is an unmet need for treatments that can provide extended and continuous red blood cell transfusion independence.

Approval Based on Results from IMerge Phase 3 Clinical Trial

“For patients with lower-risk MDS and anemia who are transfusion dependent, we have very few options today and often cycle through available therapies, making the approval of Rytelo potentially practice changing for us,” said Rami Komrokji, MD, Vice Chair, Malignant Hematology Department, Moffitt Cancer Center, who was an investigator of the pivotal IMerge clinical trial. “What is exciting about Rytelo is the totality of the clinical benefit across LR-MDS patients irrespective of ring sideroblast status or high transfusion burden, including sustained and durable transfusion independence and increases in hemoglobin levels, all within a well-characterized safety profile of generally manageable cytopenias. The treatment goal for patients with LR-MDS and anemia is transfusion-independence and before today, this wasn’t possible for many patients.”

The FDA approval of Rytelo is based on results from the IMerge Phase 3 clinical trial, published in The Lancet 4. The IMerge trial met its primary and key secondary endpoints, with Rytelo demonstrating significantly higher rates of red blood cell transfusion independence (RBC-TI) versus placebo for at least eight consecutive weeks (Rytelo 39.8% [95% CI 30.9–49.3]; placebo 15.0% [7.1–26.6]; p<0.001) and for at least 24 weeks (Rytelo 28.0% [95% CI 20.1-37.0]; placebo 3.3% [95% CI 0.4-11.5]; p<0.001). RBC-TI was durable and sustained in the Rytelo treated population, with a median RBC-TI duration for 8-week responders and 24-week responders of approximately 1 year and 1.5 years, respectively.

In an exploratory analysis of Rytelo-treated patients achieving ≥8-week RBC-TI, median increases in hemoglobin were 3.6 g/dL for Rytelo and 0.8 g/dL for placebo. Clinically meaningful efficacy results were observed across key MDS subgroups irrespective of ring sideroblast (RS) status, baseline transfusion burden and IPSS risk category.

In the IMerge trial, the safety profile of Rytelo was well-characterized with generally manageable and short-lived thrombocytopenia and neutropenia, which are familiar side effects for hematologists who are experienced with managing cytopenias. The most common Grade 3/4 adverse reactions were neutropenia (72%) and thrombocytopenia (65%), which lasted a median duration of less than two weeks, and in more than 80% of patients were resolved to Grade < 2 in under four weeks. Cytopenias were generally manageable with dose modifications. The intravenous administration of Rytelo every four weeks aligns to routine blood count monitoring for these patients.

The most common adverse reactions (incidence ≥10% with a difference between arms of >5% compared to placebo), including laboratory abnormalities, were decreased platelets (thrombocytopenia), decreased white blood cells, decreased neutrophils (neutropenia), increased aspartate aminotransferase (AST), increased alkaline phosphatase (ALP), increased alanine aminotransferase (ALT), fatigue, prolonged partial thromboplastin time, arthralgia/myalgia, COVID-19 infections, and headache. Clinically relevant adverse reactions in < 5% of patients who received Rytelo included febrile neutropenia, sepsis, gastrointestinal hemorrhage, and hypertension.

REF: https://en.wikipedia.org/wiki/Imetelstat

FDA Approves Rytelo (imetelstat) for the Treatment of Adult Patients with Lower-Risk MDS with Transfusion-Dependent Anemia

FDA Grants Accelerated Approval for Ojemda (tovorafenib) for Relapsed or Refractory BRAF-altered Pediatric Low-Grade Glioma

Day One Biopharmaceuticals, Inc. (Nasdaq: DAWN) (“Day One” or the “Company”), a commercial-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved Ojemda (tovorafenib), a type II RAF inhibitor, for the treatment of patients 6 months of age and older with relapsed or refractory pLGG harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. This indication is approved under accelerated approval based on response rate and duration of response. With the approval, Day One received a rare pediatric disease priority review voucher from the FDA.

“Ojemda ushers in a new day for children living with relapsed or refractory pLGG, and we are pleased that we can deliver a new medicine for these patients in desperate need of new treatment options. Moreover, Ojemda is the first and only FDA-approved medicine for children with BRAF fusions or rearrangements, which are the most common molecular alteration in pLGG,” said Jeremy Bender, Ph.D., chief executive officer of Day One. “We are very proud that our first approved medicine addresses this serious and life-threatening disease of childhood and adolescence. We are grateful to the pLGG community, including patients and their families, study investigators, non-profit organizations, and advocacy groups, for their collaboration and support as we strive to close the innovation gap for children with cancer awaiting new treatments.”

pLGG is the most common brain tumor diagnosed in children, with patients suffering profound tumor- and treatment-associated morbidities that can impact their life trajectory. BRAF is the most commonly altered gene in pLGG, with up to 75 percent of children having a BRAF alteration. Until now, there had been no medicines approved for patients with pLGG driven by BRAF fusions.

“pLGG is a chronic and relentless cancer that can devastate children and their families, often stealing their vision, balance and speech,” said Dr. Sabine Mueller, pediatric neuro-oncologist, University of California San Francisco Benioff Children’s Hospitals. “The goal of pLGG treatment is to stabilize or shrink the tumor without further disrupting the child’s and family’s life. Historically, there has been no standard of care for children with pLGG who have relapsed. We are excited to welcome a new targeted treatment option with once-weekly oral dosing designed specifically for these kids and their families.”

Ojemda is the only systemic therapy for pLGG that offers once-weekly dosing, with or without food, as a tablet or oral suspension.

REF: https://en.wikipedia.org/wiki/Tovorafenib

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FDA Grants Accelerated Approval for Ojemda (tovorafenib) for Relapsed or Refractory BRAF-altered Pediatric Low-Grade Glioma

FDA Approves Onyda XR (clonidine hydrochloride) Non-Stimulant Liquid Treatment for ADHD

Tris Pharma, Inc. (Tris), a commercial-stage biopharmaceutical company focused on attention deficit hyperactivity disorder (ADHD), pain, addiction and neurological disorders, today announced the U.S. Food and Drug Administration (FDA) has approved Onyda XR (clonidine hydrochloride), a once-a-day extended-release oral suspension with nighttime dosing, for the treatment of ADHD as a monotherapy or as an adjunctive therapy to approved central nervous system (CNS) stimulant medications in pediatric patients six years and older.

Onyda XR is the first non-stimulant ADHD medication in Tris’ portfolio, the first-and-only liquid non-stimulant ADHD medication approved in the United States and the only approved non-stimulant ADHD medication with nighttime dosing. Non-stimulant ADHD therapies are an important option for patients who do not respond adequately to stimulant medication or experience negative side effects from them, and they are increasingly used as an effective alternative to stimulant treatments. Onyda XR is expected to be available in pharmacies in the second half of 2024.

“People with ADHD require a range of therapeutic options that are designed for their individual needs, because not every medication or type of therapy works for every patient,” said Ann Childress, M.D. “The approval of Onyda XR, the only liquid non-stimulant ADHD medication, with nighttime dosing that shifts the release profile, is a convenient option for patients needing better ADHD control.”

Tris Pharma harnessed the flexibility of its proprietary LiquiXR® technology to develop Onyda XR, a liquid non-stimulant medication with a smooth, extended-release profile that physicians can use to treat ADHD patients either alone or in combination with stimulant therapy. This product adds to Tris’ comprehensive and expanding portfolio of leading ADHD therapies that enhance patient care for individuals with the disorder. The company’s ADHD therapies are available in both oral suspension (liquid) and solid (tablet) forms for administration to children and adults. Tris continues to grow its ADHD offerings with a pipeline of new medications that could have a substantial impact for those with the disorder.

“Securing FDA approval for Onyda XR is not just an important milestone, but a testament to our unwavering commitment to innovating and improving outcomes for this patient population,” said Ketan Mehta, Founder and CEO at Tris Pharma. “Our relentless pursuit to offer a range of ADHD medicines to patients of all ages does not stop here, and we look forward to continuing to expand our portfolio in other ADHD indications.”

The U.S. FDA approval of Onyda XR is based on adequate and well-controlled studies of clonidine hydrochloride extended-release tablets

REF: https://en.wikipedia.org/wiki/Clonidine

FDA Approves Onyda XR (clonidine hydrochloride) Non-Stimulant Liquid Treatment for ADHD

FDA Approves Risvan (risperidone) for the Treatment of Schizophrenia

Today, Laboratorios Farmacéuticos Rovi, S.A. (“ROVI” or the “Company”) has announced that the U.S. Food and Drug Administration (FDA) has authorized the marketing of Risvan® (Risperidone ISM®) for the treatment of schizophrenia in adults.

Risperidone ISM® is a prolonged-release injectable antipsychotic developed and patented by ROVI for the treatment of schizophrenia in adults, which, as of the first injection, provides immediate and sustained plasmatic drug levels and does not require loading doses or supplementation with oral risperidone.

This approval is based on the positive results of the pivotal PRISMA-3 study on the efficacy and safety of Risperidone ISM® in schizophrenia patients. The results obtained in this study show that the two different doses (75 mg and 100 mg once a month) have achieved the prespecified primary and secondary efficacy endpoints for treatment of patients with moderate to severe symptoms of schizophrenia. The primary efficacy endpoint, the PANSS total score (mean difference, CI: 95%), improved significantly with Risperidone ISM® 75 mg and 100 mg from the beginning until day 85, with adjusted differences of -13.0 (17.3 to -8-8; p <0.0001) and -13.3 (-17.6 to -8.9; p<0.0001), respectively, in comparison with the placebo. Significantly improved mean changes for the secondary endpoint, the CGI-S score, were also obtained for Risperidone ISM® in comparison with the placebo, -0.7 (-1.0 to -0.5; p<0.0001), for both doses, from the beginning until day 85. The significant statistical improvement for both efficacy results was observed as early as 8 days after the first injection. The most frequently reported treatment-emergent adverse events were increased blood prolactin (7.8%), headaches (7.3%), hyperprolactinemia (5%) and weight increase (4.8%). No important new or unexpected safety information was reported. Likewise, patients who successfully completed the double-blind period were offered the opportunity to continue in a long-term, open-label 12-month extension phase with once every four weeks injections of Risperidone ISM® (75 mg or 100 mg). New, clinically stable patients ("de novo" patients) were also able to enter this open phase of the study. Long-term treatment was observed to be effective, safe and well tolerated in adult patients with schizophrenia, regardless the initial severity of the disease or whether they had been treated previously with Risperidone ISM® during an acute exacerbation or switched from stable doses of oral risperidone. Likewise, Risperidone ISM® provided a swift and sustained improvement in functioning (both social and personal) and health-related quality of life. These findings, together with a quick onset of effectiveness, could help strengthen the therapeutic alliance and possibly lead to an earlier hospital discharge. Furthermore, the patient’s functioning either continued to improve or remained stable with long-term treatment.

Ref: https://en.wikipedia.org/wiki/Risperidone

FDA Approves Risvan (risperidone) for the Treatment of Schizophrenia

FDA Approves Zevtera (ceftobiprole medocaril sodium) for Bacteremia, Skin and Skin Structure Infections, and Pneumonia

Basilea Pharmaceutica Ltd, Allschwil (SIX: BSLN), a commercial-stage biopharmaceutical company committed to meeting the needs of patients with severe bacterial and fungal infections, announced today that the US Food and Drug Administration (FDA) approved Zevtera® (ceftobiprole medocaril sodium for injection), for the treatment of adult patients with Staphylococcus aureus bloodstream infections (bacteremia) (SAB), including those with right-sided infective endocarditis, and adult patients with acute bacterial skin and skin structure infections (ABSSSI) and for adult and pediatric patients (3 months to less than 18 years old) with community-acquired bacterial pneumonia (CABP).

David Veitch, Chief Executive Officer of Basilea, said: “We are excited with the US approval of Zevtera. The positive decision by the FDA is a key milestone towards bringing Zevtera to patients in the US. Zevtera has 10 years of market exclusivity from the date of approval and we believe the US provides the most important global commercial opportunity for the brand.”

Dr. Marc Engelhardt, Chief Medical Officer of Basilea, stated: “We are very pleased that the FDA approved Zevtera for all three indications that were submitted with the NDA, including a pediatric labelling. This approval is a landmark for ceftobiprole and reflects its broad clinical utility. The indication in adult patients with Staphylococcus aureus bacteremia, including those with right-sided infective endocarditis, caused by methicillin-susceptible and methicillin-resistant isolates, MSSA and MRSA, addresses a real medical need, as current treatment options are limited.”

The New Drug Application (NDA) was supported by clinical efficacy and safety data from the phase 3 studies ERADICATE (SAB)1 and TARGET (ABSSSI),2 and a phase 3 study in CABP.3 The ERADICATE study was the largest double-blind randomized registrational study conducted for a new antibiotic treatment in SAB.

Vance G. Fowler, Jr., M.D., Professor in the Departments of Medicine and Molecular Genetics & Microbiology at the Duke University School of Medicine and academic lead investigator of the ERADICATE study, commented: “Complicated Staphylococcus aureus infections have a high mortality rate and are associated with substantial morbidity. We need more options for treating these infections, especially if MRSA is involved.”

Thomas Holland, M.D., Associate Professor in the Department of Medicine at the Duke University School of Medicine and chair of the data review committee of the ERADICATE study, added: “There is a high medical need in Staphylococcus aureus bacteremia, therefore, the first approval of a therapy for this indication in over 15 years is highly welcome.”

Adesh Kaul, Chief Financial Officer of Basilea, added: “As we were moving towards completion of the regulatory review, especially with increasing visibility on the expected label, the external interest for commercial partnering increased. Whilst our initial goal was to have announced a commercial partnership by the time of approval of Zevtera in the US, in order to explore fully all potential partnering opportunities, we now expect to complete the process around mid-year. In parallel, we are also taking preparatory steps to shorten the launch timelines, once we have entered into a commercialization partnership.”

Basilea’s phase 3 program for ceftobiprole is funded in part with federal funds from the US Department of Health and Human Services (HHS); Administration for Strategic Preparedness and Response (ASPR); Biomedical Advanced Research and Development Authority (BARDA), under contract number HHSO100201600002C. Through this partnership, Basilea has been awarded approximately USD 112 million, or approximately 75 percent of the costs related to the SAB and ABSSSI phase 3 studies, regulatory activities and non-clinical work.

About Zevtera® (ceftobiprole medocaril sodium for injection)

Ceftobiprole, the active moiety of the prodrug ceftobiprole medocaril, is an advanced generation cephalosporin antibiotic for intravenous administration, with rapid bactericidal activity against a wide range of Gram-positive bacteria, such as Staphylococcus aureus, including methicillin-resistant strains (MRSA), and Gram-negative bacteria.4 In several countries in Europe and beyond, the brand is currently approved and marketed as Zevtera® and Mabelio® for the treatment of adult patients with hospital-acquired bacterial pneumonia (HABP), excluding ventilator-associated bacterial pneumonia (VABP), and for the treatment of community-acquired bacterial pneumonia (CABP). Basilea has entered into license and distribution agreements covering more than 80 countries. In the United States, Zevtera is indicated for the treatment of adult patients with Staphylococcus aureus bloodstream infections (bacteremia) (SAB), including right-sided infective endocarditis, and adult patients with acute bacterial skin and skin structure infections (ABSSSI) and for adult and pediatric patients (3 months to less than 18 years old) with community-acquired bacterial pneumonia (CABP).

Ref: https://en.wikipedia.org/wiki/Ceftobiprole

FDA Approves Zevtera (ceftobiprole medocaril sodium) for Bacteremia, Skin and Skin Structure Infections, and Pneumonia

FDA Approves Myhibbin (mycophenolate mofetil) Oral Suspension for Prophylaxis of Organ Rejection

Azurity Pharmaceuticals, a pharmaceutical company focused on developing innovative dose forms and formulations of products to serve the needs of overlooked patients, announced today that the U.S. Food and Drug Administration (FDA) has approved Myhibbin™, the only ready-to-use mycophenolate mofetil oral suspension. Mycophenolate mofetil is an antimetabolite immunosuppressant used to protect a donated organ from being rejected from the body’s immune response. In 2023, there were over 46,000 transplants in the US and these patients need to take medication every day to fight against rejection. Myhibbin is indicated for the prophylaxis of organ rejection in adult and pediatric recipients 3 months of age and older of allogeneic kidney, heart, or liver transplants, in combination with other immunosuppressants.

“We are very pleased that adult and pediatric organ transplant recipients will soon have access to the only FDA-approved ready-to-use oral liquid formulation of mycophenolate,” said Richard Blackburn, CEO of Azurity Pharmaceuticals. “Patients are our priority, and our purpose is to bring them new formulations that help them benefit from established medicines. Myhibbin’s ready-to-use formulation provides patients, pharmacists, and caregivers an alternative to other mycophenolate dosage forms.”

REF : https://en.wikipedia.org/wiki/Mycophenolic_acid

FDA Approves Myhibbin (mycophenolate mofetil) Oral Suspension for Prophylaxis of Organ Rejection

FDA Approves Libervant (diazepam) Buccal Film for the Treatment of Seizure Clusters in Pediatric Patients Ages 2-5 Years

Aquestive Therapeutics, Inc. (NASDAQ: AQST) (“Aquestive” or the “Company”), a pharmaceutical company advancing medicines to bring meaningful improvement to patients' lives through innovative science and delivery technologies, today announced the U.S. Food and Drug Administration (FDA) has approved Libervant™ (diazepam) Buccal Film for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity (i.e., seizure clusters, acute repetitive seizures) that are distinct from a patient’s usual seizure pattern in patients with epilepsy between 2 to 5 years of age.

“We are thrilled to have received FDA approval for Libervant™ in patients between the ages of two and five,” said Daniel Barber, Chief Executive Officer of Aquestive. “Patients have been waiting years for Libervant, the first and only FDA approved orally-administered rescue product for the treatment of seizure clusters. Our first priority is to provide and maintain availability of Libervant to the intended patient population. I am pleased to announce that we are currently able to accept and fill non-Medicaid prescriptions. We expect to expand our distribution capabilities over the coming weeks and months. I am also pleased with our continued track record of success with the FDA. We respect the FDA’s mission to protect public health and we will always seek to partner with the FDA wherever possible.”

“Libervant provides a new way to deliver diazepam for the treatment of acute repetitive seizure emergencies in children aged two to five,” said Michael Rogawski, M.D., Ph.D, distinguished professor of neurology and pharmacology, University of California, Davis, “The film is placed onto the buccal mucosa inside the cheek where it adheres firmly and dissolves quickly, delivering a consistent dose of diazepam. Studies show that the film is easy to administer and performs reliably in children as young as 2 years of age. Libervant is packaged in a compact foil pouch that is convenient to carry so that the treatment can be available wherever these children may be.”

In 2023, over 55,000 prescriptions were filled for patients between the ages of 2 and 5. This was an increase of 10.8% over the previous year and an average increase of 9.3% over the last three years for this patient population. Over 90% of filled prescriptions in 2023 for this patient population were for diazepam rectal gel. Prescription writing for this indication is highly concentrated among pediatric epileptologists and pediatric neurologists.

REF: https://en.wikipedia.org/wiki/Diazepam

FDA Approves Libervant (diazepam) Buccal Film for the Treatment of Seizure Clusters in Pediatric Patients Ages 2-5 Years