Genzyme's Cerdelga gets FDA approval for Gaucher disease

The US Food and Drug Administration late yesterday approved Cerdelga (eliglustat) for the long-term treatment of adult patients with the type 1 form of Gaucher disease, a rare genetic disorder. The drug was developed and will be marketed by US biotech firm Genzyme, a subsidiary of French pharma major Sanofi (Euronext: SAN).

Cerdelga is a hard gelatin capsule containing eliglustat that is taken orally. In patients with Gaucher disease type 1, the drug slows down the production of the fatty materials by inhibiting the metabolic process that forms them. type 1 Gaucher disease is estimated to affect about 6,000 people in the USA.

“Today’s approval offers another important treatment option for patients with type 1 Gaucher disease,” said Amy Egan, deputy director of the Office of Drug Evaluation III in FDA’s Center for Drug Evaluation and Research, adding: “In addition, Cerdelga received orphan drug designation from the FDA, reflecting the agency’s focus and commitment to the development of treatments for rare diseases.”