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.
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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.