IL-1 TRAP AND INFLAMMATORY DISEASES
Regeneron Files NDA for its lead drug rilonacept
This submission by Regeneron (REGN) of a Biologics License Application (BLA) to the FDA for its lead product, Interleukin-1 (IL-1) Trap (rilonacept), for the long-term treatment of Cryopyrin-Associated Periodic Syndromes (CAPS) marks a major corporate milestone for Regeneron. If approved, the drug will become Regeneron’s first marketed product. It will validate the firm’s trap technology and will be tried for other inflammatory diseases where IL-1 seems to play a major role in their etiologies.
CAPS, the diseases targeted by rilonacept, consists of rare genetic inflammatory diseases caused by a variety of mutations in one gene known as CIAS1 and also known as NALP3. This gene encodes a the protein cryopyrin. The diseases resulting from the gene mutations include Familial Cold Autoinflammatory syndrome (FCAS) Muckle Wells syndrome (MWS) and Neonatal Onset Multisystem Infalammatory Diseasev – all classified as autoinflammatory disorders.
Interleukin-1 (IL-1) is a protein secreted by certain cells in the body. Normally, IL-1 acts as a messenger to help regulate immune and inflammatory responses by attaching to cell-surface receptors in cells that participate in the body’s immune system. In excess, though, this protein becomes harmful. As more pathways of inflammatory diseases in various body systems are unfolding, IL-1 is found to be a key driver of inflammation in many diseases, not only CAPS. So, neutralizing the excess IL-1 could become a major approach to treating several inflammatory diseases that affect several organs.
Regeneron’s IL-1 Trap is designed to attach to and neutralize IL-1 in the blood stream before it can attach to cell-surface receptors and generate signals that can trigger inflammation. Once trapped (attached to the Trap), IL-1 cannot bind to the cell surface receptors and, together with the Trap, is flushed from the body.
Having said so, one can imagine how large could become the market of Regeneron’s IL-1 Trap drug rilonacept. As a matter of fact, the product is in experimentation for other diseases, starting now with gout.
Regeneron has also 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.
Cryopyrin-Associated Periodic Syndromes (CAPS) are characterized by spontaneous systemic inflammation. Inflammatory symptoms in patients with FCAS and MWS include fever, chills, rash, fatigue, joint pain, and eye redness. Currently, there are no medicines approved for the treatment of CAPS.