FDA approves Genzyme MS treatment
CAMBRIDGE, Mass. — Genzyme, a Sanofi company, on Friday announced that the Food and Drug Administration approved Lemtrada (alemtuzumab) for the treatment of patients with relapsing forms of multiple sclerosis. Because of its safety profile, the use of Lemtrada should generally be reserved for patients who have had an inadequate response to two or more drugs indicated for the treatment of MS.
“Today’s approval is the culmination of more than a decade of work by Genzyme to develop Lemtrada,” said Genzyme president and CEO David Meeker. “Lemtrada demonstrated superior efficacy over Rebif on annualized relapse rates in the two studies,which were the basis for approval. A comprehensive risk evaluation and mitigation strategy will be instituted in order to help detect and manage the serious risks identified with treatment.”
The FDA approval of Lemtrada is based on two randomized Phase 3 open-label rater-blinded studies comparing treatment with Lemtrada to Rebif (high-dose subcutaneous interferon beta-1a) in patients with relapsing remitting MS who were either new to treatment (CARE-MS I) or who had relapsed while on prior therapy (CARE-MS II).
In CARE-MS I, Lemtrada was significantly more effective than interferon beta-1a at reducing annualized relapse rates; the difference observed in slowing disability progression did not reach statistical significance. In CARE-MS II, Lemtrada was significantly more effective than interferon beta-1a at reducing annualized relapse rates, and accumulation of disability was significantly slowed in patients given Lemtrada versus interferon beta-1a. The clinical development program for Lemtrada involved nearly 1,500 patients with more than 6,400 patient years of safety follow-up.
“The unmet need in MS remains high,” said Edward Fox, director of the Multiple Sclerosis Clinic of Central Texas. “It is a great day for people living with relapsing forms of MS in the United States, who will now have access to this new meaningful treatment.”
The Lemtrada label includes a boxed warning, noting a risk of serious, sometimes fatal autoimmune conditions, serious and life-threatening infusion reactions and also, noting Lemtrada may cause an increased risk of malignancies, including thyroid cancer, melanoma and lymphoproliferative disorders.
Lemtrada is only available through a restricted distribution program, the Lemtrada REMS (Risk Evaluation and Mitigation Strategy). This program has been developed to ensure that access to Lemtrada in the United States is only through certified prescribers, healthcare facilities and specialty pharmacies, and to also ensure that patients are enrolled in the REMS program. The program is intended to help educate healthcare providers and patients on the serious risks associated with Lemtrada and the appropriate periodic monitoring required to support the detection of these risks for 48 months after the last infusion. The REMS is based on a developmental risk-management program that was successfully implemented in the Phase 2 and Phase 3 trials, and allowed for early detection and management of some of the serious risks associated with Lemtrada.
Lemtrada has a unique dosing and administration schedule of two annual treatment courses. The first treatment course is administered via intravenous infusion on five consecutive days, and the second course is administered on three consecutive days, 12 months later.
First approved in September 2013 in the European Union, Lemtrada is approved in more than 40 countries.
The FDA approval of Lemtrada marks Genzyme’s second MS treatment approval in the United States. Genzyme received FDA approval of its once-daily, oral Aubagio (teriflunomide) for the treatment of relapsing forms of MS in September 2012. Aubagio is approved in more than 50 countries and is under review by additional regulatory agencies. Between clinical trials and commercial use, approximately 30,000 patients have been treated with Aubagio.