Catalyst Pharmaceutical Partners Granted Orphan Medicinal Product Designation in European Union for CPP-115 for the Treatment of West Syndrome

Catalyst Pharmaceutical Partners, Inc.CPRX +2.80% today announced that the European Commission (EC) has granted orphan medicinal product designation for the company's investigational drug, CPP-115, a novel GABA aminotransferase inhibitor, for the treatment of West Syndrome (infantile spasms). The EC designation is based on the recommendation by the European Medicines Agency (EMA) Committee on Orphan Medicinal Products (COMP) after their review of all relevant preclinical data for CPP-115, which showed that it may have a longer duration of action, improved benefits and fewer retinal side effects than the existing first-line treatment."We have hit another major milestone in our efforts to advance this clinically important treatment for patients around the world," said Patrick J. McEnany, Chief Executive Officer of Catalyst. "Obtaining an orphan designation for CPP-115 in the European Union (EU) is an important step that builds upon our recent progress with CPP-115, including the grant of Fast Track development program designation by the U.S. Food and Drug Administration (FDA) for cocaine dependency and commencement of a first-in-man Phase I(a) safety study.""Our orphan designations in the EU and U.S. support our global development strategy for CPP-115 and our goal of providing improved therapies for infants and children with West Syndrome and infantile spasms," said Steven R. Miller, Ph.D., Catalyst's Chief Operating Officer and Chief Scientific Officer. "Our preclinical experience with CPP-115 to-date has demonstrated its potential in treating infantile spasms (West Syndrome) with greater safety and efficacy than existing therapies. Currently, there are limited treatment options for this serious pediatric disease, all of which have significant side effects. We hope to offer providers and their patients a more effective and safer therapy than is currently available."Orphan medicinal product designation is granted by the EC, in conjunction with the EMA's COMP, to promote the development of products to treat life-threatening or very serious conditions that are rare and affect not more than 5 in 10,000 persons in the EU. Key benefits include 10 years of market exclusivity if CPP-115 is approved for the treatment of West Syndrome, as well as possible EU-funded research, protocol assistance and fee reductions for centralized activities like a Marketing Authorization Application (MAA).Similarly, orphan drug designation is granted by the FDA Office of Orphan Drug Products to promote the development of drugs and biologics for the treatment of rare diseases and disorders that affect fewer than 200,000 persons in the United States. The key benefit includes a 7-year period of market exclusivity if CPP-115 is the first of its type approved for the specified indication or if it demonstrates superior safety, efficacy or a major contribution to patient care versus another drug of its type previously granted the designation for the same indication. Other potential benefits include tax credits for clinical research costs, annual grant funding, clinical trial design assistance and waiver of Prescription Drug User Fee Act (PDUFA) filing fees.Catalyst filed its application for orphan medicinal product designation in the EU through Catalent Pharma Solutions, Ltd., its representative in Europe. The approved grant of orphan medicinal product designation will be held in the representative's name until Catalyst instructs transfer of it to Catalyst or to another biopharmaceutical company with a presence in the EU.