FDA reverses an embarrassing rejection of Sanofi's Lemtrada, OKs MS drug

Late on Friday the FDA put out the word that it has reversed its earlier decision against Sanofi's ($SNY) multiple sclerosis drug Lemtrada, agreeing to allow the pharma giant the right to market the treatment--with some tight controls on just who will get this drug for the relapsing form of the disease.

Like other MS drugs on the market, Lemtrada will come with a boxed warning. Theirs includes cautions against "autoimmune conditions, serious and life-threatening infusion reactions and also noting Lemtrada may cause an increased risk of malignancies including thyroid cancer, melanoma and lymphoproliferative disorders." Access to the therapy will be controlled through a restricted distribution program.

And company officials tell reporters that the drug will be priced at a premium to the rival drug Rebif. Lemtrada will be sold for $158,000 for a two-treatment regimen, compared to about $135,000 for a similar course of Rebif from Merck KGaA.

Instead of challenging the FDA's rejection of Lemtrada--a monoclonal antibody--close to a year ago, Sanofi execs opted to present a new analysis to deal with regulators' concerns about the trial design. The company handed over additional information on the studies, and Sanofi's Genzyme division has since also delivered more efficacy data from studies of the treatment, which may help negotiations with payers.

But Sanofi faced an uphill climb at the FDA. An internal FDA review highlighted the data on an increased risk of autoimmune diseases, serious infections and treatment-emergent malignancies among the patients taking the drug in Sanofi's studies. To be approvable, the FDA initially ruled, Lemtrada would have to demonstrate substantial clinical benefits or the drug's "serious and potentially fatal safety issues" would scuttle the marketing application. A panel review followed with a mixed vote, with a majority saying the safety issues didn't preclude an approval but that Sanofi's clinical trials were inadequate.

The win now marks a major turnaround for Sanofi, which was embarrassed by the setback at the FDA. Lemtrada was one of the key reasons why CEO Chris Viehbacher engineered the $20 billion acquisition of Cambridge, MA-based Genzyme. Its rejection had left a lingering black eye. But Lemtrada can now be sold alongside Aubagio in the U.S., which won an approval about two years ago.

The MS market is huge and fast-changing. Biogen Idec ($BIIB) has had some signal accomplishments, quickly ramping up sales of the oral drug Tecfidera. Teva ($TEVA), meanwhile, relies on Copaxone for much of its revenue but faces the loss of patent protection.

Genzyme CEO David Meeker

Viehbacher, though, won't be around to join the celebrations at Sanofi. The board in Paris ousted the CEO weeks ago after growing disenchanted with his management style. But David Meeker, the Genzyme veteran who stayed on to run the subsidiary, is still in place to toast their success.

"Today's approval is the culmination of more than a decade of work by Genzyme to develop Lemtrada," said Meeker. "Lemtrada demonstrated superior efficacy over Rebif on annualized relapse rates in the two studies which were the basis for approval. A comprehensive risk evaluation and mitigation strategy (REMS) will be instituted in order to help detect and manage the serious risks identified with treatment."

- here's the release (PDF)
- read the story from Bloomberg

Suggested Articles

Solid Bio fixed the manufacturing issue that led to a partial clinical hold on its DMD trial, but a full hold remains.

Commissioner Scott Gottlieb has laid out the reasons why he thinks the FDA needs a double-digit budget bump.

GW's cannabis-based epilepsy drug scored a favorable review from FDA staff ahead of an expert panel meeting.