Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq:RPTP), announced that the European Medicines Agency's ("EMA") Committee for Orphan Medicinal Products ("COMP") has issued a positive opinion recommending orphan medicinal product designation for DR Cysteamine for the potential treatment of cystinosis.
DR Cysteamine is Raptor's proprietary, delayed-release, oral formulation of cysteamine bitartrate. Immediate-release cysteamine bitartrate is the current standard of care for treating cystinosis.
"The granting of orphan medicinal product designation by EMA is a significant milestone in the development and eventual commercialization of DR Cysteamine for the potential treatment of cystinosis as it allows for ten years of market exclusivity in the European Union ("E.U.") once the drug is approved," said Ted Daley, President of Raptor. "Our progress with European regulators dovetails well with our work with the U.S. Food and Drug Administration's ("FDA") Office of Orphan Product Development and is an important aspect of our orphan product strategy. Our recent Phase IIb study with DR Cysteamine in cystinosis patients demonstrated improved tolerability and the potential to reduce total daily dosage and administration frequency compared to immediate-release cysteamine bitartrate. Raptor is now poised to initiate its pivotal, Phase III clinical trial in cystinosis patients at multiple sites in the U.S. and Europe in Q2 2010."
The EMA grants orphan medicinal product designations to advance the development of drugs intended to treat, prevent or diagnose life-threatening or very serious conditions that are rare and affect not more than 5 in 10,000 persons in the E.U. With this designation, Raptor will have market exclusivity in the E.U. for ten years in the event that DR Cysteamine receives marketing approval as a treatment for cystinosis. The designation also provides for special benefits, including research support, eligibility for protocol assistance, and possible exemptions or reductions in certain regulatory fees during development or at the time of application for marketing approval.
Raptor's European clinical development subsidiary, Raptor Pharmaceuticals Europe B.V., has been granted Small and Medium-Sized Enterprises status ("SME") by the EMA which, in Europe, provides Raptor with administrative and procedural assistance, fee reductions for scientific advice and inspections, fee exemptions for certain administrative services of EMA, deferral of the fee payable for an application for marketing authorization or related inspection, conditional fee exemption where scientific advice is followed and a marketing authorization application is not successful and assistance with translations of product information documents submitted in the application for marketing authorization. SME status is renewed bi-annually and represents a significant cost savings for small companies, like Raptor, in developing potential therapeutics in the E.U. In cases where orphan medicinal product designation and SME status provide similar cost savings, the most beneficial savings to Raptor would prevail.
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