The European Commission (EC) has approved Raptor Pharmaceutical's application for orphan drug designation of cysteamine bitartrate, the active ingredient in RP103, for the treatment of Huntington's disease. The decision follows a positive recommendation from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) in June.
In February of this year, Raptor announced encouraging top line results from a planned 18-month analysis of the blinded, randomized, placebo-controlled phase of its ongoing CYST-HD clinical trial of RP103, its proprietary delayed release cysteamine bitartrate capsule, in patients with early-stage Huntington's disease.
"This designation is an important milestone as Raptor moves through the regulatory process for the potential approval of RP103 as a treatment for Huntington's disease in the EU," said Christopher M Starr, Raptor's chief executive officer. "Orphan designation provides the company with 10 years of marketing exclusivity in the EU after the Marketing Authorisation Application approval. Together with our intellectual property estate, this designation continues to strengthen our proprietary position in the EU."
RP103 was designated an Orphan Medicinal Product for the treatment of Huntington's disease because it meets the conditions established by the EC under council Regulation 141/2000. Orphan Drug Exclusivity is designed to provide regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or debilitating conditions with a prevalence in the EU of not more than five in 10,000. Huntington's disease prevalence meets the criteria as defined by this regulation. The COMP also considers whether there is any satisfactory method of prevention, diagnosis or treatment or if the medicinal product being considered will be of significant benefit to those affected by the condition. Raptor submitted the 18-month CYST-HD results as part of the orphan application.
Raptor Pharmaceutical Corp. is a global biopharmaceutical company focused on the development and commercialization of life-altering therapeutics that treat rare, debilitating and often fatal diseases.