FDA Approves Mounjaro (tirzepatide) Injection for the Treatment of Adults with Type 2 Diabetes

The U.S. Food and Drug Administration (FDA) has approved Mounjaro (tirzepatide) injection, Eli Lilly and Company's (NYSE: LLY) new once-weekly GIP (glucose-dependent insulinotropic polypeptide) and GLP-1 (glucagon-like peptide-1) receptor agonist indicated as an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes. Mounjaro has not been studied in patients with a history of pancreatitis and is not indicated for use in patients with type 1 diabetes mellitus.

As the first and only FDA-approved GIP and GLP-1 receptor agonist, Mounjaro is a single molecule that activates the body's receptors for GIP and GLP-1, which are natural incretin hormones.1

"Mounjaro delivered superior and consistent A1C reductions against all of the comparators throughout the SURPASS program, which was designed to assess Mounjaro's efficacy and safety in a broad range of adults with type 2 diabetes who could be treated in clinical practice. The approval of Mounjaro is an exciting step forward for people living with type 2 diabetes given the results seen in these clinical trials," said Juan Pablo Frías, M.D., Medical Director, National Research Institute and Investigator in the SURPASS program.

Mounjaro will be available in six doses (2.5 mg, 5 mg, 7.5 mg, 10 mg, 12.5 mg, 15 mg) and will come in Lilly's well-established auto-injector pen with a pre-attached, hidden needle that patients do not need to handle or see.

The approval was based on results from the phase 3 SURPASS program, which included active comparators of injectable semaglutide 1 mg, insulin glargine and insulin degludec. Efficacy was evaluated for Mounjaro 5 mg, 10 mg and 15 mg used alone or in combination with commonly prescribed diabetes medications, including metformin, SGLT2 inhibitors, sulfonylureas and insulin glargine. Participants in the SURPASS program achieved average A1C reductions between 1.8% and 2.1% for Mounjaro 5 mg and between 1.7% and 2.4% for both Mounjaro 10 mg and Mounjaro 15 mg. While not indicated for weight loss, mean change in body weight was a key secondary endpoint in all SURPASS studies. Participants treated with Mounjaro lost between 12 lb. (5 mg) and 25 lb. (15 mg) on average.1

Side effects reported in at least 5% of patients treated with Mounjaro include nausea, diarrhea, decreased appetite, vomiting, constipation, indigestion (dyspepsia), and stomach (abdominal) pain. The labeling for Mounjaro contains a Boxed Warning regarding thyroid C-cell tumors. Mounjaro is contraindicated in patients with a personal or family history of medullary thyroid carcinoma or in patients with Multiple Endocrine Neoplasia syndrome type 2.1

"Lilly has a nearly 100-year heritage of advancing care for people living with diabetes – never settling for current outcomes. We're not satisfied knowing that half of the more than 30 million Americans living with type 2 diabetes are not reaching their target blood glucose levels," said Mike Mason, president, Lilly Diabetes. "We are thrilled to introduce Mounjaro, which represents the first new class of type 2 diabetes medication introduced in almost a decade and embodies our mission to bring innovative new therapies to the diabetes community."

More at 

https://www.rxlist.com/mounjaro-drug.htm

FDA Approves Radicava ORS (edaravone) for the Treatment of Amyotrophic Lateral Sclerosis (ALS)

Mitsubishi Tanabe Pharma Corporation (MTPA)   announced the U.S. Food and Drug Administration (FDA)   approval of Radicava ORS (edaravone), the oral form of edaravone, for the treatment of amyotrophic lateral sclerosis (ALS), a neurodegenerative disease that currently has no cure and can progress rapidly.  Radicava ORS offers the same efficacy as Radicava (edaravone), an FDA-approved intravenous (IV) treatment shown in a pivotal trial to help slow the loss of physical function in ALS. 

"At MTPA, patients have been the driving force behind our work as we strive to develop meaningful treatment options for ALS and continue to tackle unmet needs," said Atsushi Fujimoto, President, MTPA. "Five years ago, we proudly launched Radicava as a treatment option for patients with ALS in the U.S. Now, we continue to push the boundaries of innovation with Radicava ORS, an orally administered option allowing patients flexibility in how they take their medicine."

Radicava ORS is specifically formulated for patients with ALS and provides a flexible administration option (taken orally or via feeding tube) with a small, 5 mL dose, a portable bottle, an oral dosing syringe and no need for patients to refrigerate or reconstitute before taking.1 With appropriate instruction from a healthcare provider (HCP), Radicava ORS may take only a few minutes to administer on treatment days.1 Radicava ORS should be taken in the morning after overnight fasting.1 To learn more about Radicava ORS, visit Radicava.com/update.

"ALS is a progressive disease that, due to its heterogenous nature, impacts patients at different rates with varying symptoms," said Tulio Bertorini, M.D., Professor of Neurology, The University of Tennessee Health Science Center. "Therefore, it is crucial that patients have treatment and formulation options that accommodate their own unique needs, and Radicava ORS provides HCPs who have prescribed their ALS patients edaravone with an alternate delivery option."

The comprehensive clinical development program for edaravone in ALS has spanned over a decade and included multiple clinical trials for the IV and oral formulations. The FDA approval of Radicava ORS is supported by several studies, including data from the pivotal Phase 3 clinical trial (MCI186-19) evaluating 137 ALS patients that showed treatment with Radicava slowed the loss of physical function by 33 percent (approximately one-third) compared to placebo at 24 weeks, as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R), a validated rating instrument for monitoring disease progression in patients.1Additionally, seven Phase 1 clinical pharmacology studies were conducted to examine the pharmacokinetics, safety, drug-to-drug interactions, dosing, bioavailability and bioequivalence of Radicava ORS in healthy individuals and ALS patients with and without a percutaneous endoscopic gastrostomy (PEG) tube/nasogastric (NG) tube, as well as a global Phase 3, 24-week trial demonstrating the safety and tolerability profile of the treatment in 185 patients with ALS. Lastly, there is an ongoing Phase 3 study evaluating the long-term safety and tolerability of Radicava ORS for up to 96 weeks.

The most common adverse events that occurred in greater than 10 percent of patients treated with Radicava were bruising (contusion), problems walking (gait disturbance) and headache. Fatigue was observed in 7.6 percent of patients taking Radicava ORS.  Radicava and Radicava ORS are contraindicated in patients with a history of hypersensitivity to edaravone or any of the inactive ingredients of this product.1 Hypersensitivity reactions and anaphylactic reactions have been reported in patients treated with Radicava.

More at..

https://en.wikipedia.org/wiki/Edaravone

Voquezna Triple Pak (amoxicillin, clarithromycin, and vonoprazan) FDA Approval

The Voquezna Triple and Dual Paks offer physicians the flexibility of two different treatment options. The Voquezna Triple Pak contains clarithromycin that is not included in the Voquezna Dual Pak.

Voquezna Triple Pak (amoxicillin, clarithromycin, and vonoprazan) is a co-packaged product containing amoxicillin (penicillin class antibacterial), clarithromycin (macrolide antimicrobial), and vonoprazan (potassium-competitive acid blocker (PCAB)) indicated for the treatment of Helicobacter pylori (H. pylori) infection in adults.

More 

https://en.wikipedia.org/wiki/Amoxicillin

https://en.wikipedia.org/wiki/Clarithromycin

https://en.wikipedia.org/wiki/Vonoprazan

FDA Approves Camzyos (mavacamten) for the Treatment of Symptomatic NYHA Class II-III Obstructive Hypertrophic Cardiomyopathy

In continuation of my update on mavacamten

Bristol Myers Squibb (NYSE: BMY)  announced  the U.S. Food and Drug Administration (FDA) approval of  Camzyos (mavacamten, 2.5 mg, 5 mg, 10 mg, 15 mg capsules) for the treatment of adults with symptomatic New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (obstructive HCM) to improve functional capacity and symptoms. Camzyos is the first and only FDA-approved allosteric and reversible inhibitor selective for cardiac myosin that targets the underlying pathophysiology of obstructive HCM.

“This is a first-in-class medicine specifically for patients living with symptomatic obstructive HCM,” said Milind Desai, M.D., MBA, director of the Hypertrophic Cardiomyopathy Center and director of clinical operations in Cleveland Clinic’s Heart Vascular & Thoracic Institute. "With this FDA approval, U.S. cardiologists now have a new pharmacological option for eligible patients that targets the underlying pathophysiology of the disease.”

The full U.S. Prescribing Information for Camzyos includes a Boxed WARNING for the risk of heart failure. Camzyos reduces left ventricular ejection fraction (LVEF) and can cause heart failure due to systolic dysfunction. Echocardiogram assessments of LVEF are required prior to and during treatment with Camzyos. Initiation of Camzyos in patients with LVEF <55% is not recommended. Interrupt Camzyos if LVEF is <50% at any visit or if the patient experiences heart failure symptoms or worsening clinical status. Concomitant use of Camzyos with certain cytochrome P450 inhibitors or discontinuation of certain cytochrome P450 inducers may increase the risk of heart failure due to systolic dysfunction; therefore, the use of Camzyos is contraindicated with moderate to strong CYP2C19 inhibitors or strong CYP3A4 inhibitors, and moderate to strong CYP2C19 inducers or moderate to strong CYP3A4 inducers. Because of the risk of heart failure due to systolic dysfunction, Camzyos is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Camzyos REMS PROGRAM. Please see additional Important Safety Information including Boxed WARNING below.

“This approval builds on decades of cardiovascular leadership and reflects our steadfast commitment to people impacted by cardiovascular disease,” said Samit Hirawat, M.D., executive vice president and chief medical officer, Global Drug Development, Bristol Myers Squibb. “We are proud to bring this first-of-its kind medicine to patients, which may help to address an unmet need in the U.S. in the symptomatic NYHA class II-III obstructive HCM treatment landscape.”

This approval is based on data from the Phase 3 EXPLORER-HCM trial. At baseline, approximately 73% of the randomized patients were NYHA class II and 27% were NYHA class III. The mean LVEF was 74%, and the mean Valsalva left ventricular outflow tract (LVOT) gradient was 73 mmHg. The baseline mean Kansas City Cardiomyopathy Questionaire-23 (KCCQ-23) Clinical Summary Score (CSS) was 71. At Week 30, 37% (n=45/123) of patients taking Camzyos achieved the composite primary endpoint, defined as the proportion of patients who achieved either improvement of mixed venous oxygen tension (pVO2) by ≥1.5 mL/kg/min plus improvement in NYHA class by at least 1 or improvement of pVO2 by ≥3.0 mL/kg/min plus no worsening in NYHA class, versus 17% (n=22/128) treated with placebo. The difference was 19% (95% CI: 9, 30; p=0.0005). Additionally at Week 30, patients receiving Camzyos had greater improvement compared to placebo group across all secondary endpoints, including:

• Mean change from baseline post-exercise LVOT peak gradient [-47 mmHg vs -10 mmHG; -35 difference (95% CI: -43, -28; p<0.0001)]
• Mean change from baseline in pVO2 [1.4 mL/kg/min vs -0.1 mL/kg/min; 1.4 difference (95% CI: 0.6, 2.1; p<0.0006)]
• Number (%) of patients with improvement of NYHA class ≥ 1 [80 (65%) vs 40 (31%); difference of 34% (95% CI; 22%, 45%; p<0.0001)]
• Mean change from baseline in KCCQ-23 † CSS [14 vs 4; difference of 9 (95% CI: 5, 13); p<0.0001]
  • Mean change in baseline in KCCQ-23 Total Symptom Score (TSS) (12 vs 5)
  • Mean change in baseline in KCCQ-23 Physical Limitations (PL) (15 vs 4)

  •  The KCCQ-23 CSS is derived from the Total Symptom Score (TSS) and the Physical Limitations (PL) score of the KCCQ-23. The CSS ranges from 0 to 100 with higher scores representing less severe symptoms and/or physical limitations.

    Missing data were not imputed to summarize the baseline and change from baseline to Week 30 values. Difference in mean change from baseline between treatment groups was estimated using a mixed model for repeated measures.

  • In the EXPLORER-HCM trial, adverse reactions occurring in >5% of patients and more commonly in the Camzyos group than in the placebo group were dizziness (27% vs 18%) and syncope (6% vs 2%). Mean (SD) resting LVEF was 74% (6) at baseline in both treatment groups. Mean (SD) absolute change from baseline in LVEF was -4% (8) in the Camzyos group and 0% (7) in the placebo group over the 30-week treatment period. At Week 38, following an 8-week interruption of trial drug, mean LVEF was similar to baseline for both treatment groups. Additionally, 7 (6%) patients in the Camzyos group and 2 (2%) patients in the placebo group experienced reversible reductions in LVEF to <50% (median 48%: range 35-49%) while on treatment. In all 7 patients treated with Camzyos, LVEF recovered following interruption of Camzyos.

    “The approval of Camzyos represents a significant milestone for appropriate symptomatic obstructive HCM patients and their families, who have long awaited a new treatment option for this chronic and progressive disease,” said Anjali T. Owens, MD, Medical Director of the Center for Inherited Cardiac Disease and an Assistant Professor of Medicine at the Perelman School of Medicine at the University of Pennsylvania. “As a lead U.S. investigator on the EXPLORER-HCM study, I’m grateful to the patients and their families whose participation in the trial played a key role in this approval.”

    Bristol Myers Squibb offers various programs and resources to address the needs of patients and caregivers, and provide support that allows for access to therapies, including Camzyos. For additional information, call 855-Camzyos (855-226-9967) 8 am to 11 pm ET, Monday through Friday.

https://en.wikipedia.org/wiki/Mavacamten

FDA Approves Vtama (tapinarof) Cream for the Treatment of Plaque Psoriasis in Adults

Dermavant Sciences, a biopharmaceutical company dedicated to developing and commercializing innovative therapeutics in immuno-dermatology,  announced that the U.S. Food and Drug Administration (FDA) has approved Vtama (tapinarof) cream, 1%, an aryl hydrocarbon receptor agonist, indicated for the topical treatment of plaque psoriasis in adults. This approval makes Vtama cream the first and only FDA-approved steroid-free topical medication in its class.

“We are delighted with our FDA-approved label for Vtama cream, which is for adults with psoriasis, regardless of disease severity, and with an unlimited duration of use. In anticipation of today’s approval, we have a fully built commercial infrastructure in place, and I am excited to say we will have product in the channel in the first week of June. As the first and only approved drug in its class in the U.S., the FDA’s approval of Vtama cream provides an effective new non-steroidal treatment option for millions of adults living with plaque psoriasis and represents a major milestone for Dermavant and its stakeholders,” said Todd Zavodnick, Chief Executive Officer of Dermavant. “At Dermavant, we are committed to advancing novel, patient-focused innovation in immuno-dermatology. As such, we are proud to have developed a topical treatment in Vtama cream that provides not only efficacy over 52 weeks but can also be used on all body areas, including on sensitive locations, such as face, skin folds, neck, genitalia, anal crux, inflammatory areas, and axillae. In addition, an approximately four month off-treatment remittive effect (median time to first worsening), leads us to believe that Vtama cream has the potential to become the preferred topical option for this chronically underserved patient population and among the physicians who treat them.”

“Following 20-plus years of minimal innovation in the topical psoriasis treatment space1,2,3,4, I believe the approval of Vtama cream is an important step in establishing a new treatment option for adults with mild, moderate and severe plaque psoriasis ,” said Mark Lebwohl, MD, FAAD, Dean for Clinical Therapeutics and Waldman Professor and Chairman Emeritus of the Kimberly and Eric J. Waldman Department of Dermatology, Icahn School of Medicine at Mount Sinai in New York and lead author of the Phase 3 studies of Vtama cream published in The New England Journal of Medicine. “As a clinician, I’m excited to finally have a versatile, once-daily, steroid-free topical treatment that is backed by extensive clinical trial data supporting its favorable safety and efficacy profile and a demonstrated remittive effect of approximately four months in patients off therapy.”

Across PSOARING 1 and PSOARING 2, Vtama cream demonstrated highly statistically significant improvement in Physician Global Assessment (PGA)6 score of “clear” (PGA=0) or “almost clear” (PGA=1) with a minimum 2-grade improvement compared with vehicle from baseline at week 12. Vtama cream also demonstrated a highly statistically significant improvement in all secondary endpoints versus vehicle, including ≥75% Improvement in Psoriasis Area and Severity Index (PASI) score (PASI-75) from baseline at week 127. The adverse event (AE) profile of Vtama cream reported in both PSOARING 1 and PSOARING 2 demonstrated that the majority of AEs were localized to the site of application and were mild to moderate in nature. The most common AEs of subjects treated with Vtama cream were folliculitis, nasopharyngitis, and contact dermatitis.

More... at 

https://en.wikipedia.org/wiki/Tapinarof#

FDA Approves Tlando (testosterone undecanoate) for Male Hypogonadism

Antares Pharma, Inc., (NASDAQ: ATRS) (the “Company”), a specialty pharmaceutical company, today announced that the U.S. Food and Drug Administration granted final approval for TLANDO™ (testosterone undecanoate), an oral treatment for testosterone replacement therapy (“TRT”) indicated for conditions associated with a deficiency or absence of endogenous testosterone, or hypogonadism in adult males.

Robert F. Apple, President and Chief Executive Officer of Antares Pharma, commented, “The FDA approval of TLANDO brings to market an oral formulation of testosterone that we believe will prove beneficial to physicians and their patients. We have recently expanded our commercial organization to 108 sales representatives and expect to leverage our relationships with urologists and endocrinologists to drive adoption of TLANDO. This approval also reinforces the opportunity for Antares to continue to drive share gains in the TRT market with both TLANDO and XYOSTED and support our future growth with an expanded commercial portfolio. We look forward to launching TLANDO commercially, which will provide a complementary treatment option to patients and clinicians in the second quarter of this year.”

“We are excited with the opportunity to commercialize TLANDO and reinforce our commitment to the TRT market. Our existing commercial capabilities and presence in the market with XYOSTED provide an important foundation for the potential commercial success of TLANDO. With an expanded commercial footprint, we expect to continue to foster our strong physician relationships to support their patient-centric care and preference for different treatment options. We believe TLANDO’s oral formulation and convenient dosing, which requires no titration, differentiates it from other treatment options. As we prepare for the commercial launch, we look forward to our sales representatives detailing a differentiated portfolio of products consisting of XYOSTED, TLANDO and NOCDURNA that will continue to deliver solutions for improved patient care,” added Joe Renda, Senior Vice President, Commercial of Antares Pharma.