Chelsea Therapeutics International, Ltd. has announced that the FDA has granted Orphan Drug designation to its drug candidate Droxidopa for the treatment of symptomatic neurogenic orthostatic hypotension (NOH) in patients with Primary Autonomic Failure, a group of diseases that includes Parkinson's Disease, Pure Autonomic Failure (PAF) and Multiple Systems Atrophy (MSA).
The Orphan Drug Act provides for economic incentives to encourage the development of drugs for diseases affecting fewer than 200,000 people in the United States. Orphan Drug designation will entitle Chelsea to seven years of market exclusivity for Droxidopa in the treatment of symptomatic NOH. Additional benefits include tax credits related to clinical trial expenses, a possible exemption from the FDA-user fee, and assistance in clinical trial protocol design.
"Because of the U.S. Orphan Drug Act, there are incentives for companies like Chelsea to bring desperately needed drug therapies like Droxidopa to underserved patients who suffer from neurogenic orthostatic hypotension," said Dr. Horacio Kaufmann, the Alex and Shirley Aidekman Professor of Neurology, Mount Sinai School of Medicine and Director, Autonomic Disorders Research and Treatment Program. "Given its clearly delineated mechanism of action, extensive body of efficacy data and favorable safety reputation in the Japanese market, Droxidopa will help improve the health and quality of life for many patients with neurogenic orthostatic hypotension."
Chelsea plans to initiate a double-blind pivotal Phase III trial comparing Droxidopa to placebo at multiple sites in the U.S. and Europe during the second half of 2007. The trial is intended to assess the safety and efficacy of Droxidopa in patients suffering from symptomatic NOH associated with Parkinson's Disease, Pure Autonomic Failure and Multiple Systems Atrophy with the primary efficacy endpoint being defined as improvement in orthostatic blood pressure over time.
"Receiving this designation is an important step in both our clinical development and planned commercialization of Droxidopa, providing Chelsea with considerable strategic advantages by providing market exclusivity, reducing clinical development costs and facilitating future regulatory filings," said Dr. Simon Pedder, President and Chief Executive Officer of Chelsea. "With 7-years of exclusivity in place in the U.S. and 10-years in the EU, either under Orphan designation or as a new chemical entity, we have secured the necessary exclusivity to move aggressively ahead in our planned development of Droxidopa. We are pleased to have reached this critical milestone and look forward to working with the FDA to finalize our trial design and initiate our pivotal Phase III study later this year."
As part of its Orphan Drug strategy for Droxidopa, Chelsea also filed an application for Orphan designation for the treatment of symptomatic NOH in patients with Primary Autonomic Failure with the EMEA. Based on the timing of this filing, Chelsea expects to receive a determination regarding EU Orphan status late in the first quarter 2007.