European Commission Grants Chelsea Therapeutics Two Orphan Medicinal Product Designations for Droxidopa
CHARLOTTE, N.C., Aug. 14 -- Chelsea Therapeutics International, Ltd. announced today that Droxidopa has been granted two orphan medicinal product designations by the European Commission for the treatment of orthostatic hypotension in patients with Pure Autonomic Failure (PAF) and patients with Multiple Systems Atrophy (MSA). These designations are based on a recommendation from the Committee for Orphan Medicinal Products of the European Medicines Agency (EMEA) and follow the previously announced Orphan Drug designation granted by the U.S. FDA in January 2007.
According to the EMEA, orphan medicinal products are for diagnosing, preventing or treating life-threatening or very serious conditions that are rare and affect less than five of every 10,000 persons in the European Union (EU). An Orphan Drug designation provides 10 years of market exclusivity upon receipt of EU marketing approval. It also allows for regulatory assistance in preparing the marketing application, free protocol assistance to optimize clinical development, reduced regulatory fees associated with applying for marketing approval and direct access to the centralized procedure for Marketing Authorization Application through the EMEA.
"Obtaining Orphan Drug designations from the EMEA for both PAF and MSA significantly increases the value of our Droxidopa development program," said Simon Pedder, President and CEO of Chelsea Therapeutics. "The 10-year exclusivity afforded by these designations combined with the 10-year NCE exclusivity for the treatment of OH in Parkinson's Disease in the EU complement our existing U.S. orphan designation and firmly establishes global exclusivity for Droxidopa."
About Droxidopa and Symptomatic Neurogenic Orthostatic Hypotension (NOH)
Symptomatic NOH is a neurogenic disorder resulting from a deficient release of norepinephrine, the neurotransmitter used by sympathetic autonomic nerves to send signals to the blood vessels and the heart. This deficiency results in decreased blood pressure when a person assumes a standing position and is characterized by lightheadedness, dizziness, blurred vision and syncope. Droxidopa, an orally active synthetic precursor of norepinephrine, increases the supply of norepinephrine available for delivery to its receptors to improve orthostatic blood pressure and alleviate symptoms of orthostatic hypotension.
Chelsea estimates that nearly 300,000 patients suffer from chronic symptomatic NOH in the U.S. and EU combined. In addition to creating significant health care costs, symptomatic NOH has a dramatic impact on the quality of patient life. Midodrine, currently the only FDA approved treatment for orthostatic hypotension, not only fails to treat the underlying cause of symptomatic NOH but is limited in its use by a pronounced side-effect profile and black box warning for supine hypertension. Given the chronic nature of symptomatic NOH and the proven safety and tolerability of Droxidopa, Chelsea expects that daily oral treatment with Droxidopa should provide a significant improvement in the long-term treatment of symptomatic NOH.
Droxidopa, developed by and licensed from Dainippon Sumitomo Pharma Co., Ltd. (DSP), initially received Japanese approval in 1989 for the treatment of frozen gait and dizziness on standing associated with Parkinson's Disease and for the treatment of orthostatic hypotension, syncope or dizziness on standing associated with Shy-Drager syndrome and Familial Amyloidotic Polyneuropathy. In 2000, Droxidopa received expanded marketing approval to include prevention of vertigo, dizziness and weakness associated with orthostatic hypotension in hemodialysis patients. Droxidopa has historically generated annual revenues of approximately $50 million in Japan.
About Chelsea Therapeutics
Chelsea Therapeutics is a biopharmaceutical development company that acquires and develops innovative products for the treatment of a variety of human diseases. The Company is currently developing a library of metabolically inert antifolate compounds engineered to have potent anti-inflammatory and anti-tumor activity to treat a range of immunological disorders. Early clinical data suggests that Chelsea's lead antifolate compound, CH-1504, is a safe and effective treatment alternative to methotrexate for RA and may have further applications for psoriasis, IBD and certain cancers. Chelsea's antifolate program is complemented by a strategic partnership with Active Biotech AB for the joint development of a portfolio of therapeutics targeting immune-mediated inflammatory disorders and transplantation. In addition to its autoimmune pipeline, Chelsea is pursing an Orphan Drug strategy for the development of Droxidopa, an orally active synthetic precursor of norepinephrine, for the treatment of neurogenic orthostatic hypotension. Currently approved and marketed in Japan, Droxidopa has accumulated over 15 years of proven safety and efficacy data, generating annual revenue of approximately $50 million in Japan.
This press release contains forward-looking statements regarding future events. These statements are just predictions and are subject to risks and uncertainties that could cause the actual events or results to differ materially. These risks and uncertainties include reliance on collaborations and licenses, risks and costs of drug development, regulatory approvals, intellectual property risks, our reliance on our lead drug candidate CH-1504, our history of losses and need to raise more money, competition, market acceptance for our products if any are approved for marketing, reliance on key personnel including specifically Dr. Pedder, management of rapid growth, and the need to acquire or develop additional products.