WALTHAM, Mass.
(AP) — Repligen will be awarded incentives by both the U.S. and Europe
as it develops a drug to treat the neuromuscular disease spinal muscular
atrophy, the company said today.
The drug RG3039 has already received fast track status in
the U.S.
That means the Food and Drug Administration will make faster decisions about
Repligen’s marketing application. Regulators in the European Union gave the
drug orphan drug status, which comes with up to 10 years of marketing
exclusivity if approved. Orphan drug and fast track status are used to
encourage treatments for rare diseases, or conditions that have few treatment
options.
RG3039 received orphan drug status in the U.S., which
includes up to seven years of marketing exclusivity.
The company said it has received approval for an early stage
clinical trial of the drug. Repligen Corp. plans to test RG3039 on 40 healthy
people to evaluate its safety and its effects on the body.
The company said RG3039 is the first drug developed
specifically to treat spinal muscular atrophy.
