Sanofi snags a 'breakthrough' tag for a new rare disease drug

Sanofi's ($SNY) rare disease-focused Genzyme picked up the FDA's coveted breakthrough-therapy designation for a candidate designed to treat a form of Niemann-Pick disease.

Genzyme's treatment, olipudase alfa, is an enzyme-replacement therapy for acid sphingomyelinase deficiency, or Niemann-Pick disease type B (NPB). The disease results from an unhealthy buildup of a lipid called sphingomyelin, caused by a lack of sphingomyelinase. Olipudase alfa works by replacing that missing enzyme, restoring the body's ability to break down sphingomyelin, Genzyme said.

The FDA's breakthrough tag guarantees Genzyme a speedy trip through the regulatory process, and the agency is promising access to its top officials as the company moves forward in the clinic. The FDA granted the breakthrough nod based on Phase Ib data from 5 patients showing early signs of efficacy for the therapy. There are no approved treatments for NPB.

Olipudase alfa is now in a Phase I/II study in children, and Genzyme is planning to kick off a Phase II/III trial in adults later this year.

NPB is a lysosomal storage disease, part of a group of ailments that result from genetic mutations that impair cell metabolism. Genzyme has a long history of success in the field, winning approval with enzyme-replacement therapies for the diseases Gaucher, Fabry, Pompe and mucopolysaccharidosis.

Thanks to some top-down cuts to R&D at Sanofi, Genzyme has focused its research efforts solely on rare diseases and multiple sclerosis, working to build on its string of FDA victories. Sanofi tightened the reins on its $20 billion acquisition in February, at the same time cutting 100 researchers and restructuring its own oncology division.

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