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. Chelsea has also submitted requests
for Orphan Status to the EMEA for symptomatic
NOH associated with Parkinson's Disease; multiple systems atrophy;
and pure autonomic failure respectively.
Orphan
Drug Status, granted for rare diseases afflicting less than 200,000
patients per year in the United States and 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.
