Genzyme Corp., a Cambridge-based biotech and pharmaceutical company with significant operations in Central Massachusetts, announced that the FDA is planning an accelerated approval for a drug to treat Pompe disease.
Pompe disease is a progressive, debilitative and life-threatening inherited disorder that affects about 2,000 people in the U.S., according to the company.
Genzyme already has approval to produce the drug, Myozyme, through one process, at the 160 L bioreactor scale. The new FDA approval that the company is now seeking would allow it to make the drug on the 200L scale, allowing it to make larger quantities at one time.
The company and the FDA now need to agree on the design of a post-approval verification study, and the FCA must complete its review of the risk evaluation and mitigation strategy for the product.
