On May 10th, 2018 the Endocrinologic and Metabolic Drugs Advisory Committee met to review new drug application (NDA) 210645.
On May 10th, 2018 the Endocrinologic and Metabolic Drugs Advisory Committee met to review new drug application (NDA) 210645, for Akcea Therapeutics’ volanesoren. The drug is a subcutaneous injection proposed as an adjunct to diet for the treatment of familial chylomicronemia syndrome (FCS). A starting dose of 300 mg once weekly with dose adjustments based on body weight and tolerability was considered.
The sponsor discussed that there is no approved therapy for the rare disease and the insufficiency of diet modifications or triglyceride lowering medications. Seven clinical studies were presented to substantiate the safety and efficacy of the volanesorsen. The studies showed a significant decrease in fasting triglyceride levels for subjects who were given volanesorsen relative to the placebo groups. The committee voiced concerns over risk of low blood platelet count, serious bleeding with the drug and whether a risk evaluation and mitigation strategy would be appropriate.
During this meeting, 6 discussion questions and 1 voting question were posed to the committee. When asked whether the applicant provided sufficient efficacy and safety data to support approval of volanesorsen, the panel voted 12 (Yes) to 8 (No). Those who voted “Yes” noted that the study met its primary endpoint of decreasing triglyceride levels. Panelists who voted “No” indicated that the drug did not demonstrate a favorable risk/benefit ratio. The agency is not obligated to follow its panel’s recommendation, but normally does so.
ISS has almost two decades of experience in developing regulatory strategies including support for FDA Advisory Committee meetings. We are involved in more FDA AdComms per year than even the largest pharmaceutical companies. For more information on how ISS can help you prepare for your next meeting, contact firstname.lastname@example.org
This post was authored by Sami Mourad. Sami is a summer intern at ISS and a 3rd year pharmacy student at the Rutgers University Ernest Mario School of Pharmacy.
Over the past few years, the number of ex-US manufacturers submitting a Class II exempt 510k device registration has increased substantially. For companies with medical devices that are approved and successful in a foreign market, the logical next step is to introduce their product into the US, which has one of the most lucrative healthcare markets in the world. Read more
FDA advisory committee meetings are important regulatory events for many manufacturers on the path to marketing approval. These meetings can quite literally result in the approval or rejection of a product based on the panel’s vote. Read more
One of the main challenges our clients face is determining which regulatory pathway is best for their products. For example, what if an OTC product has a Monograph active ingredient but that ingredient is present at a different amount than the permitted amount? Or what if the label claims a novel indication not specified in a Monograph? If you are debating pursuit of either regulatory pathway for your OTC drug, it is critical to consider cost, timeline, and label claims to ensure your products has a unique stance in today’s market. Read more
US distributors and agents trying to import rapid antibody tests for coronavirus face a number of obstacles. The good news is that FDA has opened up the Emergency Use Authorization (EUA) program to include SARS-CoV-2 (the virus) and COVID-19 (the disease), expediting time to market. Read more
The journey to scientific and commercial success is often complex and always critical, if you are looking for an expert partner to help steer you to confident solutions, contact us todayContact us