Pharmaceutical company Sanofi, which owns Genzyme, will appeal a U.S. Federal Drug Administration (FDA) decision not to approve its multiple-sclerosis treatment Lemtrada at this time, according to a press release today, Dec. 30.
The FDA in a letter to Genzyme said its drug Lemtrada application can't immediately be approved because Genzyme hasn't submitted evidence showing its benefits outweigh its side effects.
"We strongly believe that the clinical development program--which was designed to demonstrate how Lemtrada compares against an active comparator as opposed to placebo--provides robust evidence of efficacy and a favorable benefit-risk profile, Genzyme Chairman and Chief Executive David Meeker said on Monday in a press release.
Lemtrada already has European Union, Canadian and Australian approval and is under review in other countries.
U.S. Food and Drug Administration staff said Lemtrada increased the risk of autoimmune and thyroid diseases, including diabetes and cancer.
Annual Lemtrada sales worldwide is $752 million by 2018, according to forecasts compiled by Thomson Reuters Pharma.
Multiple sclerosis affects more than 2.1 million people globally.
Lemtrada is indicated for the treatment of adult patients with relapsing remitting multiple sclerosis with active disease defined by clinical or imaging features. Lemtrada 12 mg 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.
Genzyme's own press release in September said the "most common side effects of Lemtrada are infusion associated reactions, infections (upper respiratory tract and urinary tract), lymphopenia and leukopenia. Serious autoimmune conditions can occur in patients receiving Lemtrada. A comprehensive risk management program will support early detection and management of these autoimmune events."
Sanofi is the parent company of Genzyme, which has offices in Cambridge and Framingham. French drug maker Sanofi purchased Genzyme for $20.1 billion in 2011 based largely on anticipated sales of Lemtrada.