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CHMP Adopts Positive Opinion for Alexion's Soliris(TM) in Europe
Friday April 27, 8:01 am ET
- CHMP recommends marketing authorization for Soliris(TM) (eculizumab) to treat all patients with paroxysmal nocturnal hemoglobinuria (PNH) -
- First medicinal product to receive positive opinion from CHMP under Accelerated Assessment Procedure -
CHESHIRE, Conn., April 27 /PRNewswire-FirstCall/ -- Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN - News) today announced that the Committee for Human Medicinal Products (CHMP) of the European Medicines Agency (EMEA) adopted a positive opinion recommending marketing authorization for Soliris(TM) (eculizumab) for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). Based upon the CHMP's positive recommendation, marketing authorization by the European Commission is expected in two to three months.
Soliris, a designated orphan medicinal product, is intended to reduce hemolysis (destruction of red blood cells) in patients with paroxysmal nocturnal hemoglobinuria (PNH). The CHMP recommended a broad label for Soliris covering all patients with PNH. According to the adopted CHMP opinion, "Soliris (eculizumab) is indicated for the treatment of patients with paroxysmal nocturnal haemoglobinuria (PNH)." An additional sentence describing the PNH patient population that was studied in the Phase III clinical trials states, "Evidence of clinical benefit of Soliris in the treatment of patients with PNH is limited to patients with history of transfusions." Soliris was previously approved by the U.S. Food and Drug Administration on March 16, 2007 for the treatment of PNH to reduce hemolysis, and is currently being marketed in the United States.
The CHMP evaluated Soliris under the European Accelerated Assessment procedure, the fastest evaluation timeframe for full approval awarded by EMEA. According to the EMEA, Soliris is the first medicinal product to receive a positive CHMP opinion within the Accelerated Assessment procedure.
There are no therapies specifically available for the treatment of PNH in Europe. PNH is a rare, disabling and life-threatening blood disorder defined by chronic red blood cell destruction, or hemolysis. Hemolysis can cause one or more of the following symptoms in patients with PNH: severe anemia, disabling fatigue, recurrent pain, shortness of breath, pulmonary hypertension, intermittent episodes of dark colored urine (hemoglobinuria), kidney disease, impaired quality of life and blood clots (thromboses).
"Soliris brings real hope to people who live daily with the devastating effects of PNH," said Leonard Bell, MD, chief executive officer of Alexion Pharmaceuticals. "The positive opinion adopted by CHMP is an important step in Alexion's mission to improve PNH patients' lives in Europe and around the globe. Importantly, the adopted CHMP opinion recommends marketing authorization for Soliris to treat all patients with PNH."
CHMP based its opinion on clinical data from three multi-national clinical studies involving 195 patients. In these studies Soliris reduced hemolysis in every treated patient, thereby reducing symptoms, stabilizing hemoglobin and significantly reducing transfusions. Soliris patients reported markedly less fatigue and improved health-related quality of life. Additionally, there were fewer thrombotic events during Soliris treatment than during the same period of time prior to treatment.
A summary of the CHMP opinion can be accessed at emea.europa.eu .
Important Safety Information
Soliris is generally well tolerated. The most frequent adverse events observed in clinical studies were headache, nasopharyngitis (a runny nose), back pain and nausea. Treatment with Soliris should not alter anticoagulant management because the effect of withdrawal of anticoagulant therapy during Soliris treatment has not been established.
In the United States, the product label for Soliris also includes a boxed warning: "Soliris increases the risk of meningococcal infections. Vaccinate patients with a meningococcal vaccine at least 2 weeks prior to receiving the first dose of Soliris; revaccinate according to current medical guidelines for vaccine use. Monitor patients for early signs of meningococcal infections, evaluate immediately if infection is suspected, and treat with antibiotics if necessary." Two out of 196 vaccinated PNH patients treated with Soliris experienced a serious meningococcal infection.
Prior to beginning Soliris therapy, all patients and their prescribing physicians in the United States, and following approval those in Europe as well, will be enrolled in the Soliris Safety Registry which is part of a special risk management program that involves initial and continuing education and long-term monitoring for detection of new safety findings.
Please see full prescribing information for Soliris in the United States at www.soliris.net. |
