Ipsen Announces U.S. FDA Approval for Newly Designed Pre-Filled Syringe for Somatuline Depot (lanreotide)

 

Ipsen Biopharmaceuticals, an affiliate of Ipsen , announced today that the United States Food and Drug Administration (FDA) has approved a new pre-filled syringe for Somatuline Depot (lanreotide). The syringe includes updated features, such as larger flanges, designed to help make it easier for healthcare providers to administer the injection.1 The indications remain the same as those for the previous pre-filled syringe and include the treatment of adult patients with unresectable, well- or moderately-differentiated, locally advanced or metastatic gastroenteropancreatic neuroendocrine tumors (GEP-NETs) to improve progression-free survival; treatment of adults with carcinoid syndrome; when used, it reduces the frequency of short-acting somatostatin analog rescue therapy; and the long-term treatment of patients with acromegaly who have had an inadequate response to surgery and/or radiotherapy, or for whom surgery and/or radiotherapy is not an option. Please see Important Safety Information below and accompanying full Prescribing Information.

“The conditions of GEP-NETs and acromegaly can be associated with a number of uncomfortable and unpleasant symptoms, and innovation aimed at improving the injection process is a step forward,” said Daphne Adelman, Clinical Nurse Specialist, Northwestern University, Chicago, and one of the authors of the study.

Ipsen conducted five separate but complementary studies in partnership with patients, their caregivers, nurses and other healthcare professionals to better understand the current use of the existing Somatuline® Depot pre-filled syringe and to evaluate ways to improve the features of the device.1 The result of this collaboration is a redesigned delivery system intended to make it easy to grip the syringe and administer the injection. The new syringe features a needle shield removal system, more stable plunger and thermoform tray that has recessed areas designed to help prevent accidental plunger depression. The built-in safety system, which may help to prevent needle stick injury by locking in place following the administration, has not been changed.

“We consistently look for opportunities to respond to the needs of the communities we serve, and this approval would not have been possible without the direct involvement of nurses and the patients with GEP-NETs and acromegaly whom they treat,” said Bradley Bailey, SVP, and Franchise Head Oncology/Endocrinology Business Unit at Ipsen. “We listened and collaborated to enhance the existing pre-filled syringe, making it sturdier for healthcare providers when administering treatment, with the intention of improving the injection process. We look forward to bringing this innovation to healthcare providers for their patients soon.”

The new pre-filled syringe is for deep subcutaneous injection and is intended for administration by a healthcare professional. Healthcare providers can expect to receive the new syringe during Q3 2019. The device is approved for use in the U.S., EU and additional ex-U.S. markets.

Dova Pharmaceuticals Announces FDA Approval of Doptelet (avatrombopag) for Treatment of Chronic Immune Thrombocytopenia (ITP)

In continuation of my update on avatrombopag

Dova Pharmaceuticals, Inc.  a pharmaceutical company focused on acquiring, developing and commercializing drug candidates for diseases where there is a high unmet need, today announced the U.S. Food and Drug Administration (FDA) approved a supplemental New Drug Application (sNDA) that expands the use of Doptelet (avatrombopag) to include the treatment of thrombocytopenia in adults with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. 

Doptelet is also FDA-approved for the treatment of thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo a procedure.  Earlier this week, Dova announced the marketing authorization granted by the European Commission for Doptelet for the treatment of severe thrombocytopenia in adult patients with CLD who are scheduled to undergo an invasive procedure.

“Dova is pleased to provide Doptelet to patients and physicians in the United States for the treatment of chronic ITP in adult patients who have had an insufficient response to a previous treatment,” said Dr. David Zaccardelli, president and CEO of Dova. “In addition to offering patients with ITP a new treatment option, we expect Doptelet will also address an important unmet medical need in the market.  We sincerely thank the patients and dedicated researchers who participated in our clinical program as well as FDA for their collaboration during the review of this application.”

Doptelet is an oral, thrombopoietin receptor agonist (TPO-RA) administered with food.  In the pivotal Phase 3 study, Doptelet administration resulted in a platelet count of at least 50,000 per µL at day eight of therapy in the majority of patients, with efficacy superior to placebo in maintaining platelet counts in the target range during the 6-month treatment period.  Additional supportive efficacy data for the ITP sNDA were provided by two Phase 2 ITP clinical trials, as well as two Phase 3 trials for the treatment of thrombocytopenia in patients with CLD. 

Safety data for 128 patients with ITP, and more than 1,000 subjects treated across 24 studies in the Doptelet clinical development program across multiple indications, support the safety and tolerability of Doptelet. 

“ITP patients should work with their clinician to choose a therapy that supports their lifestyle and aims to achieve the best possible result to treat their ITP. That’s why having additional treatment options are so important,” said Caroline Kruse, president and CEO of the Platelet Disorder Support Association, a patient advocacy organization dedicated to ITP patients. “We are thrilled to have a new, oral TPO-RA available for adult patients with ITP.  Every new treatment provides more choices and new hope to our community.”

Dova is committed to enabling patient access to Doptelet.  Doptelet will be priced similarly to other TPO-RAs used to treat ITP, and Dova will continue to offer Patient Assistance and Co-Pay programs. The commercial launch of Doptelet for ITP is anticipated to occur in mid-July 2019. 

Dova also entered into an expanded partnership in the United States with Salix. Starting on July 1, 2019, in addition to the gastroenterology, colorectal surgery, and proctology segments, Salix will have the exclusive right to co-promote the CLD indication for Doptelet to the hepatology and interventional radiology segments.  Dova will continue to pay Salix a commission based on a percentage of net sales in these specialties, which will be in the mid-thirties beginning on July 1, 2019.  In addition, the co-promotion agreement was extended to September 2023.

Dr. Zaccardelli added, “The expanded partnership with Salix builds additional momentum for Doptelet and enables the Dova team to focus on a successful launch of the ITP indication. As a growing leader in the treatment of thrombocytopenia, we are committed to realizing Doptelet’s significant market opportunity in CLD, ITP and potentially chemotherapy-induced thrombocytopenia (CIT) for which we expect Phase 3 trial top-line results in the first half of 2020.”

https://pubchem.ncbi.nlm.nih.gov/compound/AVATROMBOPAG#section=3D-Conformer

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

Experimental Drug, Voxelotor, Shows Early Promise Against Sickle Cell Disease

An experimental drug for sickle cell disease reduced anemia and boosted the health of red blood cells in patients, according to a new study.

Whether the drug, voxelotor, will have long-term health benefits to patients remains to be seen.

But if approved for use by the U.S. Food and Drug Administration, "people living with sickle cell disease might have a new, once daily, tolerable oral medication that increases their hemoglobin level in the near future," noted Dr. Banu Aygun, who wasn't involved in the new trial.

She is associate chief of hematology at Cohen Children's Medical Center in New Hyde Park, N.Y.

As Aygun explained, sickle cell disease is an inherited blood disorder affecting more than 100,000 Americans. Black Americans, especially, are prone to the illness.

"The disease is the result of a change in a single gene leading to the production of an abnormal hemoglobin called sickle hemoglobin [HbS]," Aygun said.

"Due to this abnormal hemoglobin, red blood cells take the shape of a sickle and die much sooner than normal red blood cells. The sickle cells block small blood vessels, causing pain and affecting many organs throughout the body, leading to premature death," she said.

Right now, patients with sickle cell -- many of them children -- have few treatment options. "So far, there are only two FDA-approved drugs for sickle cell disease: hydroxyurea and glutamine," Aygun noted.

The new 17-month, phase 3 clinical trial was designed to see if a third treatment might be on the horizon. It was funded by voxelotor's maker, Global Blood Therapeutics, and included 274 patients, ages 12 to 65, in 12 countries.

Patients were divided into three groups that received either a 900-mg or 1,500-mg daily dose of the drug voxelotor, or a "dummy" placebo pill.

The study found that 51% of patients who took the higher dose of voxelotor had a significant increase in their hemoglobin levels after six months of treatment, compared with 7% of those who received the placebo.

Another finding was that 41% of patients who took the higher dose of the drug reached hemoglobin levels of more than 10g/dl at 24 weeks. A normal, non-anemic hemoglobin count ranges between 11.5 to 17.5 g/dl, depending on age and gender, the study authors noted.

"Chronic organ failure, which is predicted by the severity of anemia, is a leading cause of death for patients with sickle cell disease," said study lead researcher Dr. Elliott Vichinsky, a professor at the University of California, San Francisco.
"These patients are susceptible to strokes, renal failure and other complications that lead to early death," he said in a UCSF news release. "We believe this drug has the potential to decrease chronic organ failure in patients with this condition."

For her part, Aygun said the new drug does seem to hold promise, but gains for patients were so far not dramatic.

She noted that patient pain "events" didn't change, regardless of whether people received voxelotor or the placebo. The most common side effects with the new drug were headache and diarrhea.

And Aygun stressed that it remains to be seen "whether taking this medication for longer duration will lead to a decrease in pain events or organ damage caused by sickle cell disease."

https://pubchem.ncbi.nlm.nih.gov/compound/Voxelotor#section=2D-Structure

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