Rare disease specialist Genzyme talked up the results of a study published in The Journal of the American Medical Association concluding that the only first-line oral therapy for Gaucher disease reduced spleen size 28% compared to placebo after 9 months.
Three years out, French drugmaker Sanofi continues to reap benefits from having opened a U.S. biologics plant that added capacity for some of the rare disease meds developed by its Genzyme unit.
Sanofi subsidiary Genzyme is tying up with gene therapy upstart Voyager Therapeutics, gambling $100 million upfront on the hot field and promising up to $745 million in milestones for their partnership on a full slate of development programs.
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.