Regeneron Pharmaceuticals, Inc. has received marketing approval from the US Food and Drug Administration (FDA) for Arcalyst (rilonacept) injection for subcutaneous use, an interleukin-1 blocker, for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS), including Familial Cold Auto-inflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS) in adults and children 12 and older.
Arcalyst is the only therapy approved for patients with CAPS, a group of rare, inherited, auto-inflammatory conditions characterized by life-long, recurrent symptoms of rash, fever/chills, joint pain, eye redness/pain, and fatigue. Intermittent, disruptive exacerbations or flares can be triggered at any time by exposure to cooling temperatures, stress, exercise, or other unknown stimuli.
Arcalyst is a targeted inhibitor of interleukin-1 (IL-1), the key driver of inflammation in CAPS. In the pivotal clinical development program, patients treated with Arcalyst experienced a greater improvement in overall symptom scores than patients treated with placebo. These improvements were sustained over time with continued Arcalyst treatment. The most commonly reported adverse reactions reported with Arcalyst were injection-site reaction and upper respiratory tract infection.
"The approval of Arcalyst represents a major advance in the treatment of CAPS patients," said Hal Hoffman, M.D., Associate Professor, University of California, San Diego and a leading expert on CAPS. "Much-needed treatment will now be available to patients suffering from debilitating CAPS symptoms. I hope that the approval of Arcalyst will also contribute to increased awareness of this rare disease which currently is frequently misdiagnosed and insufficiently treated."
Regeneron expects to launch Arcalyst, its first commercial product, within the next 30 days.
"This approval exemplifies Regeneron's commitment to discover, develop, and commercialize important medicines for patients suffering from serious diseases, such as CAPS. I would like to take this opportunity to thank the clinical investigators and CAPS patients participating in our studies, the FDA, and everyone at Regeneron for their collaborative effort in making Arcalyst available to patients who need it," said Leonard S. Schleifer, M.D., Ph.D., Regeneron's president and chief executive officer. "We recognize that Arcalyst may help address a significant unmet medical need that exists among CAPS patients and are therefore committed to helping these patients obtain access to this new treatment."
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