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Dec 30, 2013

Genzyme Vows to Appeal FDA Letter on Lemtrada Phase III Trial Design

  • Genzyme said today it will challenge the FDA’s complete response letter over the company’s supplemental NDA seeking approval of Lemtrada™ (alemtuzumab) for relapsing forms of multiple sclerosis (MS).

    The dispute precludes Lemtrada from meeting the drug’s first contingent value right (CVR) milestone of winning FDA approval by March 31, 2014, Genzyme acknowledged.

    The FDA, according to the company, is taking issue with the design of recently completed Phase III active comparator studies of Lemtrada in relapsing-remitting MS patients. The agency contends that in order to approve Lemtrada, Genzyme needs to complete one or more additional active comparator clinical trials of different design and execution than its current active comparator studies, which the FDA says have not produced evidence demonstrating that Lemtrada’s benefits outweigh its adverse effects.

    “Genzyme strongly disagrees with the the FDA’s conclusions and plans to appeal the agency’s decision,” the company said in a statement in which CEO, David Meeker, M.D., added, “We are extremely disappointed with the outcome of the review and the implications for patients in the United States suffering with multiple sclerosis who remain in need of alternative therapies to manage a devastating disease.”

    “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. This evidence was also the basis for the approvals of Lemtrada by other regulatory agencies around the world,” Dr. Meeker added.

    Lemtrada is approved for marketing in the European Union, Canada, and Australia, with additional applications under review worldwide.

    Lemtrada was among key assets that prompted Sanofi to acquire Genzyme in 2011 for $20.1 billion—with 17% of the purchase price, or about $3.4 billion, set to be paid out as CVRs entitling holders to additional cash tied in part to the performance of Lemtrada.

    CVR holders were entitled to receive $1 per CVR if the FDA approved on or before March 31. Instead, CVRs plunged this morning on NASDAQ to 25 cents, down almost 70% from Friday’s close of 82 cents, before inching up to 30 cents in before-hours trading.

    The Sanofi-Genzyme merger included four additional CVR produce-sales milestones entitling holders to additionally receive:

    • $2 per CVR if net sales for Lemtrada total $400 million or more on a global basis during specified periods following product launch.
    • $3 per CVR if global net sales for Lemtrada total $1.8 billion during any four consecutive calendar quarters. But if that level of sales is achieved despite FDA approval occurring after March 31, 2014, CVR holders can still receive an additional $1 per CVR.
    • $4 per CVR if global net sales for Lemtrada total $2.3 billion during any four consecutive calendar quarters.
    • $3 per CVR if global net sales for Lemtrada total $2.8 billion during any four consecutive calendar quarters.

     



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