EMA's CHMP issues positive opinion for approval of Genzyme's LEMTRADA in Europe

Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion for approval of LEMTRADA™ (alemtuzumab) for the treatment of adult patients with relapsing remitting multiple sclerosis (RRMS) with active disease defined by clinical or imaging features.

“The superior efficacy of Lemtrada vs. Rebif in the clinical trials, which was sustained despite infrequent administration, represents an approach to treatment that promises to reshape the future for many people with active relapsing-remitting multiple sclerosis.”

In addition, the CHMP issued a positive opinion on new active substance designation (NAS) for AUBAGIO^® (teriflunomide). Earlier this year, the CHMP issued a positive opinion recommending the approval of AUBAGIO for the treatment of adult patients with relapsing remitting MS.

The European Commission (EC) is expected to render a final decision to grant marketing authorizations for LEMTRADA and AUBAGIO in the EU in the coming months.

"Today's CHMP opinions set the stage for the approval of two important new treatment options for MS patients. Treatments to-date have addressed some of the unmet needs in MS, but still have limitations," said David Meeker, MD, Genzyme President and CEO. "Upon approval, physicians will have the ability to prescribe LEMTRADA for appropriate relapsing remitting patients based on their impressions of clinical or imaging characteristics regardless of duration of disease or treatment history. Expectations among the MS community are high for LEMTRADA and with today's positive CHMP opinion we are a step closer to making this very innovative treatment available for MS patients in Europe."

The positive CHMP opinion for approval of LEMTRADA was based on data from the CARE-MS I and CARE-MS II trials, in which LEMTRADA was significantly more effective than Rebif^® (subcutaneous interferon beta-1a 44 mcg three times weekly) at reducing relapse rates. In CARE-MS II, accumulation of disability was significantly slowed in patients given LEMTRADA vs. Rebif, and importantly, patients treated with LEMTRADA were significantly more likely to experience improvement in pre-existing disability.

"Today's announcement from Genzyme represents a key milestone in the extensive program evaluating LEMTRADA in multiple sclerosis," said Professor Alastair Compston, Head of the Department of Clinical Neurosciences at the University of Cambridge, United Kingdom. "The superior efficacy of Lemtrada vs. Rebif in the clinical trials, which was sustained despite infrequent administration, represents an approach to treatment that promises to reshape the future for many people with active relapsing-remitting multiple sclerosis."

LEMTRADA has a novel dosing and administration schedule of two annual treatment courses. The first treatment course of LEMTRADA is administered via intravenous infusion on five consecutive days, and the second course is administered on three consecutive days, 12 months later.

The LEMTRADA clinical development program included two randomized Phase III studies comparing treatment with LEMTRADA to Rebif in patients with relapsing-remitting MS who had active disease and were either new to treatment (CARE-MS I) or who had relapsed while on prior therapy (CARE-MS II), as well as an ongoing extension study. A large randomized Phase II study provided the foundation for the Phase III program.

Safety results were consistent across both the CARE-MS I and CARE-MS II studies. The most common adverse events associated with LEMTRADA were infusion-associated reactions, including headache, rash, fever, nausea and hives. Infections were common in both the LEMTRADA and Rebif groups. Infections more common on LEMTRADA treatment included upper respiratory and urinary tract infections, herpes viral infections, and influenza. Most infusion-associated reactions and infections were mild to moderate in severity and responded to standard treatments.

In both CARE-MS I and CARE-MS II, the incidence of serious adverse events was similar between the two treatment arms. As previously reported, autoimmune disorders were more frequent in patients treated with LEMTRADA, primarily autoimmune thyroid disease which was observed in an estimated 36% of patients during extended follow-up. Immune thrombocytopenia (ITP) developed in 1.4 percent of LEMTRADA-treated patients through extended follow-up and 0.3% developed glomerulonephritis. Autoimmune disorders were detected soon after onset through a monitoring program, and were generally managed using standard treatments.

A comprehensive risk management program has been proposed to support early detection and management of adverse events.

In the U.S. the FDA has accepted for review the company's supplemental Biologics License Application (sBLA) file seeking approval of LEMTRADA (alemtuzumab) for the treatment of relapsing multiple sclerosis (RMS). FDA recently extended the review cycle for LEMTRADA^TM by three months; no additional clinical studies have been requested, therefore FDA action on the application is expected in late 2013.