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.
Sanofi is doubling down on its RNAi development deal with Alnylam. The pharma giant says it will pay a hefty premium to buy up a 12% stake in the biotech, investing $700 million and greatly expanding its rights to Alnylam's lead drug along with a portfolio of current and prospective therapies.
Sanofi has been slammed against the regulatory wall at the FDA, picking up a stinging rejection of its multiple sclerosis drug Lemtrada with orders to go back to the clinic for a major round of new trial work if the company ever expects to get the drug over the U.S. finish line at some point.