Massachusetts-headquartered Genzyme, a subsidiary of the French pharmaceutical company Sanofi, yesterday announced that, following constructive discussions with the U.S. Food and Drug Administration (FDA), the company plans to resubmit its supplemental Biologics License Application (sBLA), seeking approval of Lemtrada (alemtuzumab) for the treatment of relapsing forms of multiple sclerosis.
The resubmission, which should occur in the second quarter of 2014, will provide information to specifically address issues previously noted by the FDA in its December 27, 2013 Complete Response Letter.
Last year, the FDA raised issues about Lemtrada in treatment of multiple sclerosis. Even though the drug had been approved by regulators in the European Union, Australia, and Canada, the FDA formally rejected Lemtrada in December. A month earlier, an FDA panel of medical advisors warned of rashes, bleeding, and even thyroid cancer as possible side effects.
Earlier this year, Genzyme had already announced its intention to appeal the FDA’s Complete Response Letter. At the time of FDA’s rejection, Genzyme President and CEO, David Meeker, wrote in a press release that the company was “extremely disappointed with the outcome of the review and the implications for patients in the U.S. suffering with multiple sclerosis who remain in need of alternative therapies to manage a devastating disease.”
Meeker stressed that the company “strongly” believed 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. This evidence was also the basis for the approvals of Lemtrada by other regulatory agencies around the world.”
In light of the planned resubmission, the company does not expect to pursue an appeal at this time.
More than 2.3 million people have been diagnosed with multiple sclerosis throughout the world, of which, about 400,000 live in the United States alone. The potential of that market, and the promise of Lemtrada’s approval in the United States, was a major attraction for Sanofi, which paid $20.1 billion to acquire Genzyme in 2011.
For over 30 years, Genzyme has pioneered the development and delivery of transformative therapies for patients affected by rare and debilitating diseases. The company holds the worldwide rights to alemtuzumab and has primary responsibility for its development and commercialization in multiple sclerosis. Bayer HealthCare holds the right to co-promote alemtuzumab in MS in the United States. Upon commercialization, Bayer will receive contingent payments based on global sales revenue.