Thursday, June 23, 2011

Repligen's SMA treatment gets fast track designation

By James M. Connolly

Waltham biotech Repligen Corp. (Nasdaq: RGEN) has received U.S. Food and Drug Administration Fast Track designation for RG3039, a potential treatment for Spinal Muscular Atrophy (SMA), according to a press release issued by the company.

That designation is designed to expedite the review of drugs that treat serious diseases and fill an unmet medical need.

Repligen also said that the European Medicines Agencies has delivered a positive opinion for orphan medicinal product designation for RG3039.

“Receipt of Fast Track designation and a positive opinion for European orphan medicinal product designation for RG3039 demonstrates the FDA and EMA commitment to the study and development of treatments for rare and serious diseases,” said Repligen CEO Walter C. Herlihy in the press release. “This regulatory support adds momentum to our efforts to develop a novel treatment for patients with Spinal Muscular Atrophy.”

Repligen noted that it has received approval from the FDA to initiate a Phase 1 clinical trial of RG3039, saying that it is the first clinical trial of a novel drug specifically designed to treat SMA. The company said the double-blind study will evaluate the pharmacokinetic and safety profile of escalating doses of RG3039 in up to 40 healthy volunteers.

In December, Repligen received $1.4 million from the Muscular Dystrophy Association to aid research in a treatment for SMA.

SMA is an inherited neurodegenerative disease that leads to progressive damage to motor neurons, loss of muscle function and, in many patients, early death.