Regeneron Pharmaceuticals, Inc. has completed the rolling submission of a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for the Interleukin-1 (IL-1) trap (rilonacept) for the long-term treatment of Cryopyrin-Associated Periodic Syndromes (CAPS).
CAPS is a spectrum of rare inherited inflammatory conditions, including Familial Cold Autoinflammatory Syndrome and Muckle-Wells Syndrome.
As part of the BLA submission, Regeneron has requested a Priority Review from the FDA, which, if granted, would provide a target for the FDA to complete their review within six months from their receipt of the submission under current guidelines. The FDA has previously granted Orphan Drug status and fast track designation to the IL-1 Trap for the treatment of CAPS.
"Currently, there are no medicines approved for patients suffering from CAPS, and we are hopeful that the IL-1 Trap will play an important role in the future treatment of this rare condition, said Leonard S Schleifer, MD, PhD, Regeneron's president and chief executive officer. "This submission marks a major corporate milestone for Regeneron. I want to express my appreciation for the hard work and dedication shown by the many people at Regeneron who discovered and developed the IL-1 Trap and completed the submission of this, our first BLA."
Regeneron has completed a phase III programme evaluating self-administration by weekly subcutaneous injection of the IL-1 Trap in patients with CAPS. It is also conducting a proof-of-concept study of the IL-1 Trap in patients with gout.