Regeneron Pharmaceuticals, Inc has received marketing authorization in the European Union for rilonacept, an interleukin-1 blocker, for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS) with severe symptoms, including Familial Cold Auto-inflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS), in adults and children aged 12 years and older. Marketing authorization for rilonacept was granted by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMEA) under exceptional circumstances.

"Rilonacept, which is known as Arcalyst in the United States, has been prescribed to treat the majority of patients documented to have been treated for CAPS symptoms to date in the US Arcalyst is the only treatment for CAPS in the U.S. that has demonstrated a significant improvement in CAPS symptoms as reported by patients in a pivotal clinical program and is approved for self-administration at home," said Robert Terifay, senior vice president, Commercial at Regeneron. "Currently, rilonacept is available to patients in the European Union through their prescribers on a named-patient or individualized case review basis. We are evaluating our broader commercialization options for this very small, ultra-orphan patient population, which is dispersed across Europe."

CAPS include 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. Rilonacept 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 rilonacept reported a greater improvement in overall symptom scores than patients treated with placebo. These improvements were sustained over time with continued rilonacept treatment. Patients reported their symptoms using a validated daily diary instrument. These assessments represent a critical measure of effectiveness in a disease characterized by frequent, unpredictable symptom flares of variable severity and duration. Unlike other agents used in the treatment of CAPS, rilonacept is supported by patient-reported symptoms data using a validated assessment instrument.

Rilonacept has been developed as a once-weekly injection which can be administered at home by the patient or their care giver following appropriate training. The most commonly reported adverse reactions with rilonacept were injection-site reaction and upper respiratory tract infection. IL-1 blockade may interfere with immune response to infections and treatment should not be initiated in patients with active or chronic infections. There have been reports of serious, life-threatening infections in patients taking rilonacept. Rilonacept should be discontinued if a patient develops a serious infection.

The authorization for approval of rilonacept in the EU under exceptional circumstances is permissible for products for which a company can demonstrate that comprehensive data cannot be provided, for example because of the rarity of the condition. Each year, Regeneron will need to provide the EMEA with any new information that may become available for review.

Recently, medical researchers have identified and described a group of rare, inherited, auto-inflammatory disorders, known as Cryopyrin-Associated Periodic Syndromes or CAPS.

Rilonacept is a targeted inhibitor of interleukin-1 (IL-1), the key driver of inflammation in Cryopyrin-Associated Periodic Syndromes (CAPS).

Regeneron is a fully integrated biopharmaceutical company that discovers, develops, and commercializes medicines for the treatment of serious medical conditions.