Benzinga | Nov 23, 2020 08:01AM EST

Kiniksa Announces U.S. FDA Acceptance of sBLA and Priority Review for Rilonacept in Recurrent Pericarditis

- PDUFA goal date of March 21, 2021 -

- Filing based on positive data from RHAPSODY, which achieved its primary and all major secondary endpoints -

- Rilonacept BLA for CAPS transferred to Kiniksa from Regeneron -

HAMILTON, Bermuda, Nov. 23, 2020 (GLOBE NEWSWIRE) -- Kiniksa Pharmaceuticals, Ltd. (NASDAQ:KNSA) ("Kiniksa"), a biopharmaceutical company with a pipeline of assets designed to modulate immunological pathways across a spectrum of diseases, today announced that the U.S. Food and Drug Administration (FDA) accepted the supplemental Biologics License Application (sBLA) for rilonacept in recurrent pericarditis. The FDA granted priority review to the application and assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 21, 2021. Rilonacept is a weekly, subcutaneously-injected, recombinant dimeric fusion protein that blocks interleukin-1 alpha (IL-1?) and interleukin-1 beta (IL-1?) signaling. The FDA granted Breakthrough Therapy designation to rilonacept for the treatment of recurrent pericarditis in 2019 and Orphan Drug designation to rilonacept for the treatment of pericarditis in 2020.

The regulatory submission was based on 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 treatment in the trial improved clinically meaningful outcomes associated with the significant unmet medical need in recurrent pericarditis. RHAPSODY data were recently published in The New England Journal of Medicine simultaneously with a late-breaking scientific presentation at the American Heart Association's Scientific Sessions 2020.

"We are thrilled to receive the FDA's acceptance of the sBLA submission for rilonacept in recurrent pericarditis with priority review, said Sanj K. Patel, Chief Executive Officer and Chairman of the Board of Kiniksa. "Rilonacept has the potential to become the first FDA-approved therapy for this painful and debilitating autoinflammatory disease. We are committed to bringing this potential treatment option to patients as soon as possible."

Rilonacept was discovered and developed by Regeneron Pharmaceuticals, Inc. (Regeneron) and is approved by the FDA for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS) under the brand name ARCALYST(r). Kiniksa licensed rilonacept from Regeneron in 2017 for evaluation in diseases believed to be mediated by both IL-1? and IL-1?, including recurrent pericarditis. Based on positive RHAPSODY data, the Biologic License Application (BLA) for CAPS transferred to Kiniksa. If approved by the FDA for recurrent pericarditis, Kiniksa will take responsibility for sales and distribution of rilonacept for all the approved indications in the United States and evenly split profits with Regeneron, as described in the Rilonacept License Agreement.

Kiniksa is obligated to pay regulatory milestones to Regeneron of up to an aggregate of $27.5 million through the time of a potential approval of rilonacept in recurrent pericarditis, of which $7.5 million is expected to be paid in the fourth quarter of 2020.

Kiniksa continues to prepare for the potential commercial launch of rilonacept in recurrent pericarditis. The company has been generating evidence on disease burden, building disease awareness with payers, physicians, and advocacy groups, and establishing core capabilities such as distribution, patient services and data management.

About RHAPSODY

RHAPSODY is the global, randomized withdrawal design, pivotal Phase 3 clinical trial of rilonacept in recurrent pericarditis. Eligible patients presented at screening with at least a third pericarditis episode, defined as at least 1 day with pericarditis pain of ? 4 on the 11-point Numerical Rating Scale (NRS) and a C-reactive protein (CRP) value ? 1 mg/dL within the 7-day period prior to first study drug administration. Patients could be receiving concomitant nonsteroidal anti-inflammatory drugs (NSAIDs) and/or colchicine and/or oral corticosteroid treatment in any combination. The study was comprised of 4 periods: a screening period; a single-blind run-in period during which patients received a loading dose of rilonacept 320 mg injected subcutaneously (SC) followed by 160 mg SC weekly while background pericarditis medications were tapered and discontinued; a double-blind, placebo-controlled randomized withdrawal period during which clinical responders to rilonacept were randomized 1:1 and received 160 mg SC weekly rilonacept or placebo; and a long-term extension treatment period with up to 24 months of open-label rilonacept 160 mg SC weekly. The primary efficacy endpoint was time-to-first pericarditis-recurrence in the randomized withdrawal period. The Clinical Endpoint Committee adjudicated all suspected pericarditis recurrences for inclusion in the primary efficacy endpoint analysis. Kiniksa will continue to follow patients in the long-term extension treatment period for up to 24 months. The co-principal investigators are Dr. Allan Klein of Cleveland Clinic and Dr. Massimo Imazio of the University of Torino, Italy. For more information, refer to ClinicalTrials.gov Identifier: NCT03737110.