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  Overview Antifolates I-3D Droxidopa

Chelsea Therapeutics Droxidopa:

Overview

Orphan Drug Strategy

Therapeutic Areas

Chemistry

Publications


Droxidopa: Orphan Drug Strategy

In January 2007, the FDA granted orphan drug status for droxidopa for the treatment of symptomatic NOH in patients with primary autonomic failure (Parkinson’s disease, multiple system atrophy, and pure autonomic failure), dopamine-ß-hydroxylase deficiency, or nondiabetic autonomic neuropathy. In August 2007, the drug was granted orphan medicinal product designation by the European Medicines Agency (EMA) for the treatment of patients with Pure Autonomic Failure (PAF) and patients with multiple system atrophy (MSA). Orphan Drug Status, granted for rare diseases afflicting less than 200,000 patients per year in the United States and a similar population in Europe, should provide Chelsea with considerable strategic advantages for accelerating the development of droxidopa by reducing clinical trial size, development costs, facilitating global regulatory filings and providing 7 years of marketing exclusivity in the United States and 10 years in the European Union.