European marketing approval for this rare orphan condition
anticipated in late 2009
Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) today announced that the
Committee for Medicinal Products for Human Use (CHMP) of the European
Medicines Agency (EMEA) has issued a positive opinion for the marketing
authorization in the European Union of 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. The positive opinion recommends the granting
of a marketing authorization for rilonacept under exceptional
circumstances. Such authorizations are 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.
“We are very pleased to receive the EMEA's positive opinion for
rilonacept,” said Leonard S. Schleifer, M.D., Ph.D., Regeneron's
president and chief executive officer. “We recognize that rilonacept may
help address a significant unmet medical need that exists among CAPS
patients in the European Union and are therefore committed to helping
these patients obtain access to this new treatment.”
CAPS are 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. Patient-reported symptoms assessment, using a validated daily
diary instrument, represents 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. Serious, life-threatening infections have been reported in
patients taking rilonacept. Treatment should not be initiated in
patients with active or chronic infections.
