A U.S. Food and Drug Administration (FDA) advisory committee met to discuss the application for marketing approval of Lemtrada™ (alemtuzumab, Genzyme, a Sanofi Company) as a disease-modifying therapy for people with relapsing forms of MS. While the panel is typically asked by the FDA for a yes or no vote about its recommendation for approval, that was not the case at this meeting. Instead, the panel was asked to vote on several questions related to the company’s application. These votes yielded mixed results. It is important to note that this is not a formal decision by the FDA, but rather the committee’s advice to the agency. It is expected that the FDA will make an approval decision by the end of the calendar year.
During an all-day meeting held November 13, 2013, the FDA advisory committee reviewed data about the effectiveness and safety of alemtuzumab, as well as a proposed plan designed to monitor and mitigate risks associated with the therapy – called Risk Evaluation Mitigation Strategies (REMS) that would likely be mandated to monitor safety if the agent were to be approved. (Lemtrada is given by infrequent intravenous infusion – for 5 days initially and for 3 days one year later. Details of the results of two phase III trials of alemtuzumab are available here
The committee also heard public testimony from people living with MS and patient advocacy groups, including the National MS Society. The Society’s testimony addressed the need for more therapeutic options for people with MS and the importance of empowering people with MS to make their own informed treatment decisions.
The committee was asked to vote on questions related to the treatment’s effectiveness, safety, and the proposed REMS. The committee’s votes on these question were mixed. For example, when asked whether the sponsor had conducted adequately controlled studies, most committee members voted no. When asked whether the sponsor had provided substantial evidence of effectiveness, most committee members voted yes.
The next step is that the FDA will consider the advisory committee’s discussion as it continues its review of data submitted by the company to support its application for marketing approval. The FDA will likely announce a final decision on the application by the end of the year.
“We urge the FDA to keep people with MS at the center of their decision-making process and consider the uncertainty that people living with this disease face every day as the agency reviews the data and makes a decision,” stated Dr. Timothy Coetzee, Chief Advocacy, Services and Research Officer. “It’s vital that the FDA continues to advance a regulatory environment that is supportive of innovation in MS therapy development.”
Lemtrada is a trademark of Genzyme, a Sanofi Company.