Sanofi's Genzyme snagged a 6-month priority review from the FDA for what could become the first oral treatment for Gaucher disease, a rare genetic disorder that causes abnormal fat buildup in cells, enlarging organs in the body.
Less than a month ago, Sanofi's multiple sclerosis treatment Lemtrada ran into problems with FDA staffers who were unconvinced of the drug's safety and efficacy. Now, it looks as if the U.K. may share some of those doubts. Its cost-effectiveness gatekeeper has asked the French pharma for more data, giving the company just over a month to submit the supplementary information.
Gene-silencing expert Alnylam is $7 million richer thanks to some positive mid-stage results for its Genzyme-licensed RNA-interference candidate, and the Cambridge, MA, company is looking to be the first to market with the difficult-to-deliver class of treatments.
After a scathing staff review, Sanofi walked away from an FDA panel with mixed messages on its long-delayed multiple sclerosis drug, as agency advisers said the injection wasn't too risky to approve but took issue with the drugmaker's trial design.
Sanofi's multiple sclerosis drug Lemtrada may be too dangerous to warrant FDA approval, agency staff said, potentially damning news for an injection that has slogged through 25 years of back-and-forth development.
Genzyme's Framingham, MA, plant came to the rescue last year, supplying its rare-disease drug Fabrazyme that had been in short supply after viral contamination forced the closure of another facility in 2009. Now, the drugmaker intends to invest $80 million in downstream processing at the facility.
Sales of Sanofi's rare-disease drug Fabrazyme have been on an upward trajectory since it got on top of supply issues last year with FDA approval of a new U.S. manufacturing plant. Sales are so good that the French drugmaker will plow another $80 million into expanding production at the Framingham, MA, facility.
No buyer's remorse for Sanofi CEO Chris Viehbacher. Acquiring Genzyme for $20.1 billion in 2011 has paid off handsomely, Viehbacher told the Boston Globe in an interview--and not only in terms of drug sales and new regulatory approvals.
The U.K.'s cost-effectiveness analysts aren't sure Sanofi's new multiple sclerosis pill is worth the price--even at a discount. In a new review, the National Institute for Health and Care Excellence found information on Aubagio wanting, and so it asked the French drugmaker's Genzyme unit for more.
Sanofi's Genzyme got a double dip of good news Friday for its expansion in the multiple sclerosis market. First Europe's drug regulator gave a nod to Lemtrada for treating the disease. Then, as a bonus, it gave a designation to Genzyme's potential blockbuster Aubagio that will keep its patent protected longer.