Kiniksa Announces U.S. Orphan Drug Designation for Rilonacept for the Treatment of Pericarditis

HAMILTON, Bermuda, July 16, 2020 (GLOBE NEWSWIRE) -- Kiniksa Pharmaceuticals, Ltd. (Nasdaq: KNSA) (“Kiniksa”), a biopharmaceutical company focused on discovering, acquiring, developing and commercializing therapeutic medicines for patients with significant unmet medical need, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug designation for rilonacept for the treatment of pericarditis, which includes recurrent pericarditis. Rilonacept is a weekly, subcutaneously-injected, recombinant fusion protein that blocks interleukin-1 alpha (IL-1α) and interleukin-1 beta (IL-1β) signaling.

“Orphan Drug designation for rilonacept for the treatment of pericarditis, along with the Breakthrough Therapy designation for recurrent pericarditis and the recent highly statistically significant pivotal Phase 3 data, represent important advances toward bringing a potential treatment solution to patients suffering from this debilitating autoinflammatory disease,” said Sanj K. Patel, Chief Executive Officer and Chairman of the Board of Kiniksa. “We look forward to submitting an sBLA in recurrent pericarditis to the FDA later this year and are actively preparing for commercialization.”

Kiniksa recently reported positive data from RHAPSODY, a pivotal Phase 3 trial of rilonacept in recurrent pericarditis. RHAPSODY met its prespecified primary and all major secondary efficacy endpoints, showing that rilonacept improved clinically-meaningful outcomes associated with the unmet medical need in recurrent pericarditis.

Rilonacept was discovered and developed by Regeneron Pharmaceuticals, Inc. (Regeneron) and is approved by the FDA under the brand name ARCALYST for the treatment of for Cryopyrin-Associated Periodic Syndromes (CAPS). Kiniksa licensed rilonacept from Regeneron in 2017 for evaluation in diseases believed to be mediated by both IL-1α and IL-1β, including recurrent pericarditis. The FDA granted Breakthrough Therapy designation to rilonacept for recurrent pericarditis in 2019. Based on the Phase 3 RHAPSODY data, the Biologic License Application (BLA) for CAPS will transfer to Kiniksa, and the company plans to submit a supplemental Biologic License Application (sBLA) in recurrent pericarditis to the FDA later this year. Upon receipt of FDA approval for rilonacept in recurrent pericarditis, Kiniksa would assume the sales and distribution of rilonacept for the approved indications in the United States and would evenly split profits on sales with Regeneron.