In August, Sanofi's Genzyme won the FDA's blessing to take brand-new orphan drug Cerdelga into the U.S. Gaucher disease market, where the company hoped the pill could show up older, intravenous treatments. And now, it could be on its way to the same opportunity in the EU.
Late on Friday the FDA put out the word that it has reversed its earlier decision against Sanofi's 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.
Alnylam's in-development RNAi treatment successfully staved off nerve damage related to a rare disease in an ongoing study, the biotech said, early but promising results as the company heads into Phase III with partner Sanofi in tow.
Despite an FDA rejection and changing winds in the field of multiple sclerosis, Sanofi believes its Genzyme unit is on an upward trajectory, talking up potential deals and could-be blockbusters for its pricey acquisition.
Here's why Big Pharma wants to be in rare diseases--and why Sanofi made its $20.1 billion play for Genzyme.
Here's why Big Pharma wants to be in rare diseases--and why Sanofi made its $20.1 billion play for Genzyme. The company has slapped a price tag on its new oral treatment for Gaucher disease, Cerdelga: $310,250 per year.
Sanofi's rare ailment-focused Genzyme unit won FDA approval for a new Gaucher disease treatment, providing an oral alternative to its own trailblazing intravenous therapy.
Late last year, Sanofi's MS drug Lemtrada was slapped down by the FDA. Sanofi investigators now tell FierceBiotech that two years after patients in Phase III studies received their last course of treatment, many are still clearly benefiting from the drug.
Sanofi is walking away from any plans to appeal the FDA's emphatic rejection of the multiple sclerosis drug Lemtrada, at least for now. But rather than launching the added trial that the FDA demanded ahead of any possible approval, the company thinks it has a shot at answering the FDA's concerns with a new application.
Sanofi's Genzyme unit isn't offering details about its sales of Kynamro, the rare disease treatment approved last January. It's not allowing its development partner, Isis Pharmaceuticals, to dole out specifics, either. What is certain, according to Isis COO Lynne Parshall, is that the launch started off slowly, and Genzyme has expanded its sales force to step up the pace.