The QMN Weekly Bulletin
Pharmaceuticals / Regulatory Affairs / Submissions and Approvals

Sanofi Pipeline Woes Continue with FDA Rejection of Lemtrada sBLA

Jan. 3, 2014

The FDA has rejected Sanofi’s plans for an expanded indication in relapsing multiple sclerosis for its drug Lemtrada, the second pipeline setback for the company since November.

The agency based its decision on the design of completed Phase III active comparator studies supporting the sBLA for Lemtrada (alemtuzumab), Sanofi subsidiary Genzyme said Dec. 30. The FDA’s complete response letter to the company asks that additional active comparator clinical trials of a different design and execution be conducted to support approval, it added.

CEO David Meeker said the company plans to appeal the decision, arguing the Phase III results it submitted provide robust evidence of efficacy and a favorable benefit-risk profile. The data have been used to support approval for Lemtrada for relapsing multiple sclerosis in the EU and elsewhere, Meeker added.

Lemtrada is currently approved in the U.S. as a treatment for B-cell chronic lymphocytic leukemia and was approved in the EU as a treatment for adults with relapsing-remitting multiple sclerosis. The drug also has marketing approval in Canada and Australia.

The agency’s Peripheral and Central Nervous System Drugs Advisory Committee in November highlighted the drug’s potential risks of autoimmune and thyroid diseases and potential to cause certain adverse events. The panel urged the application not be approved.

U.S. rejection of Sanofi’s Lemtrada sBLA follows on the heels of the French drugmaker’s November decision to discontinue development of its blood-cancer drug candidate fedratinib, a JAK2 kinase inhibitor, after new safety concerns came to the fore.

The drugmaker has about 10 candidates in Phase III development, about half of which are vaccines. Positive first Phase III results for alirocumab in hypercholesterolemia were published recently, boosting Sanofi’s hopes that its pipeline will have better luck in 2014.

Need help ensuring that your next application to the FDA will be accepted? Check the Pharmahandbook 2013: United States to make sure all your ducks are in a row. Order your copy here.