FDA Approves Lemtrada for the Treatment of Multiple Sclerosis

Sanofi announced on Nov. 15, 2014 that FDA approved their drug Lemtrada (alemtuzumab) for the treatment of relapsing multiple sclerosis (MS). The approval follows a denial in December 2013, when FDA said through a Complete Response Letter that the drug was not ready for approval and that “one or more additional active comparator clinical trials of different design and execution are needed prior to the approval of Lemtrada.” At the point of its rejection in the US, Lemtrada had been approved in Europe, Australia, and Canada. It is currently available in more than 40 countries.

Rather than conduct new trials to satisfy FDA, Sanofi submitted enhanced efficacy data on their previously run CARE-MS I and CARE-MS II trials. In those trials, Lemtrada outperformed Rebif (interferon beta-1a). In both CARE-MS I and CARE-MS II trials, Lemtrada 12 mg was given intravenously a total of eight times over the course of the two-year study. In the CARE-MS I trial, the drug reduced the number of relapses by 78% and reduced the risk of disability by 71%, a number that Sanofi admitted was significant, but not statistically significant in terms of disease progression. In CARE-MS II, Lemtrada was "significantly more effective than interferon beta-1a at reducing annualized relapse rates and accumulation of disability was significantly slowed," according to a press release.

Genzyme executives said Lemtrada will be priced at $158,000 for two courses of treatment over two years, compared with Rebif, which costs $134,600 for a similar regimen. A 2012 study by Prime Therapeutics predicted that MS drug costs would exceed $50,000 per person per year in 2016—but drugs such as Lemtrada may push prices well beyond these expectations.

Genzyme also has another option to treat MS, oral medication Aubagio (teriflunomide). The approval of Lemtrada allows Genzyme to hold a bigger portion on the market in the treatment of MS. According to Genzyme President and CEO, David Meeker, the approval was “the culmination of more than a decade of work by Genzyme to develop Lemtrada.”

Because of the concern surrounding its safety, Lemtrada will come with a boxed warning, will only be recommended for patients who have had an inadequate response to two or more drugs indicated for the treatment of multiple sclerosis, and will only be available through a limited distribution model with a Risk Evaluation and Mitigation Strategy program.