Droxidopa for the treatment of neurogenic orthostatic hypotension and other symptoms of neurodegenerative disorders

SANTIAGO PÉREZ LLORET; MARIA VERÓNICA REY; ANNE PAVY; OLIVIER RASCOL

Expert Opinion in Orphan Drugs

Informa Healthcare

Año: 2013

2167-8707

Introduction: L-threo-3,4-dihydroxyphenylserine (droxidopa) is a pro-drug that is metabolized to norepinephrine (NE) in nerve endings and in other tissues. It is commercialized in Japan since 1989 for treatment of orthostatic hypotension symptoms in Parkinson´s disease (PD) with Hoehn & Yahr stage III, Shy-Drager syndrome, familial amyloid polyneuropathy; and hemodialytic patients. It has also been recently approved by the FDA for symptomatic neurogenic orthostatic hypotension. Areas covered: In this review, clinical uses of droxidopa in neurogenic orthostatic hypotension and other neurodegenerative disorders will be reviewed. Results from a MEDLINE search with the keywords ?droxidopa? OR ?L-threo-3,4-dihydroxyphenylserine? OR ?L-DOPS? will be discussed. Abstracts from international congresses were also explored. Expert opinion: Results from a few small and short placebo-controlled trials clinical studies in neurogenic OH showed significant reductions in manometric drop of blood pressure (BP) after posture changes or meals. Larger Phase III studies suggest a positive effect of the drug on dizziness, which has only been shown in the short-term (i.e. less than 2 weeks). FDA approval was granted in the context of an ?accelerated approval program?, which means that further studies have to be conducted in order to assess the long-term effects of droxidopa on OH symptoms. Droxidopa has also been studied for the treatment of other causes of OH, other symptoms of PD or other indications. Nonetheless, the results of such pilot studies remain preliminary, as randomized placebo-controlled clinical trials are generally lacking.