EU orphan status for Raptor's cysteamine bitartrate in Huntington's disease

US drugmaker Raptor Pharmaceutical (Nasdaq: RPTP) says that the European Commission has approved its 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 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 Starr, Raptor's chief executive, adding: "Orphan designation provides the company with 10 years of marketing exclusivity in the EU after the Marketing Authorization Application approval. Together with our intellectual property estate, this designation continues to strengthen our proprietary position in the EU."