FDA Approves Kiniksa’s Pericarditis Therapy Arcalyst

Rare Daily Staff

The U.S. Food and Drug Administration approved Kiniksa Pharmaceuticals Arcalyst for the treatment of recurrent pericarditis and reduction in risk of recurrence in adults and children 12 years and older.

Recurrent pericarditis is a painful and debilitating autoinflammatory cardiovascular disease that typically presents with chest pain and is often associated with changes in electrical conduction and sometimes buildup of fluid around the heart, called pericardial effusion. Patients who have additional pericarditis episodes following a symptom-free period of 4-6 weeks are identified as having recurrent pericarditis. Recurrent pericarditis symptoms have an impact on quality of life, limit physical activities, and lead to frequent emergency department visits and hospitalizations.

Arcalyst is a weekly, subcutaneously injected, recombinant dimeric fusion protein that blocks IL-1α and IL-1β signaling. Arcalyst was discovered by Regeneron and is approved by the FDA for recurrent pericarditis, CAPS, including familial cold autoinflammatory syndrome and Muckle-Wells syndrome, and DIRA. The FDA granted Breakthrough Therapy designation to Arcalyst for the treatment of recurrent pericarditis in 2019 and Orphan Drug designation to Arcalyst for the treatment of pericarditis in 2020. The commercial launch is expected in April 2021.

“The approval of Arcalyst in recurrent pericarditis offers patients the first and only FDA-approved therapy for this devastating disease and also represents a transformational event for Kiniksa,” said Sanj Patel, CEO and chairman of the board of Kiniksa.

The FDA approval of Arcalyst in recurrent pericarditis follows positive data from RHAPSODY, a pivotal Phase 3 trial of Arcalyst in recurrent pericarditis. RHAPSODY met its prespecified primary endpoint of time-to-first adjudicated pericarditis recurrence in the randomized withdrawal period and all major secondary efficacy endpoints with statistical significance.

The data showed that Arcalyst treatment in the trial improved clinically meaningful outcomes associated with the significant unmet medical need in recurrent pericarditis. There were rapid and sustained reductions in both reported pain and inflammation as early as after the first dose. Median time to treatment response was five days, with a 97 percent treatment response rate.

Patients randomized to Arcalyst experienced a 96 percent reduction in the risk for a recurrent pericarditis event, with 92 percent of trial days being pain free or at most experiencing minimal pain, compared to 40 percent of trial days on placebo.

The most common adverse events were injection site reactions and upper respiratory tract infections.

Photo: Sanj Patel, CEO and chairman of the board of Kiniksa