Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the European Medicine Agency's (EMA's) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the marketing authorization of Praluent® (alirocumab), recommending its approval for use in certain adult patients with hypercholesterolemia. Praluent is an investigational fully human monoclonal antibody targeting PCSK9 (proprotein convertase subtilisin/kexin type 9).
"We are very pleased to receive a positive opinion from the CHMP for Praluent, and look forward to bringing Praluent to those in greatest need across Europe," said Elias Zerhouni, M.D., President, Global R&D, Sanofi. "Despite statins and other lipid-lowering therapies, many patients are unable to reach their LDL cholesterol goals, and may benefit from new therapeutic options such as Praluent."
The CHMP recommended Praluent in both a 75 mg and 150 mg dose be approved for the treatment of adult patients with primary hypercholesterolemia (heterozygous familial hypercholesterolemia [HeFH] and non-familial) or mixed dyslipidemia as an adjunct to diet: a) in patients unable to reach their low density lipoprotein cholesterol (LDL-C) goals with a maximally-tolerated statin, Praluent would be used in combination with a statin, with or without other lipid-lowering therapies; and b) for patients who are statin intolerant, or for whom a statin is contraindicated, Praluent would be used alone or in combination with other lipid-lowering therapies. The effect of Praluent on cardiovascular morbidity and mortality has not been determined. The most common adverse reactions were injection site reactions, upper respiratory tract signs and symptoms, and pruritus.
The European Commission (EC) is expected to make a final decision on the Marketing Authorization Application for Praluent in the European Union in late September. The CHMP opinion was based on the benefit-risk profile of Praluent, following review of efficacy and safety data from more than 5,000 patients across 10 pivotal Phase 3 double-blind trials ranging from six months to two years. Clinical data from the ODYSSEY Phase 3 program show consistent, positive results in reducing LDL-C.
"In our clinical trial program, Praluent significantly reduced LDL cholesterol among patients with high unmet needs, including those with high or very high cardiovascular risk and those with an inherited form of high cholesterol called familial hypercholesterolemia," said George D. Yancopoulos, M.D., Ph.D., Chief Scientific Officer of Regeneron and President, Regeneron Laboratories. "In these trials, patients received Praluent as a single subcutaneous injection once every two weeks using either a 75 mg or 150 mg dose, providing flexible dosing options that can be tailored to an individual's cholesterol lowering needs."
The U.S. Food and Drug Administration has set a target action date of July 24 for the Biologics License Application (BLA) of Praluent. The safety and efficacy of Praluent have not been fully evaluated by any other regulatory authority.