FDA Grants Orphan Drug Designation to Ampreloxetine for the Treatment of Symptomatic Neurogenic Orthostatic Hypotension in Patients with Multiple System Atrophy
The Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) status to ampreloxetine (Theravance Biopharma, Dublin, Ireland) for the treatment of symptomatic neurogenic orthostatic hypotension (nOH) in patients with multiple system atrophy (MSA). MSA, also known as Shy-Drager syndrome, olivopontocerebellar atrophy, and striatonigral degeneration, is a rare progressive neurodegenerative disorder that affects the autonomic system and movement. An estimated 80% of the 50,000 patients with MSA in the United States experience nOH, which is associated with debilitating symptoms that adversely affect quality of life, including dizziness, muscle weakness, head and neck discomfort, vision disturbances, and fatigue.
"We are very pleased to have received Orphan Drug status for ampreloxetine in the treatment of nOH in patients with MSA," said Rick E. Winningham, CEO, Theravance Biopharma. "Ampreloxetine has the potential to improve MSA patients' and their caregivers' quality of life significantly. We are diligently progressing our registrational Phase 3 study (CYPRESS) study of ampreloxetine for the treatment of symptomatic nOH in patients with MSA and today's Orphan Drug Designation status approval represents an important milestone towards our goal of bringing this potential therapy to patients with limited treatment options."
Ampreloxetine is a norepinephrine reuptake inhibitor administered one-daily orally which has been shown in clinical trials to prevent blood pressure drops and prevent symptom worsening in patients with MSA.
The Orphan Drug Act of 1983 (ODA) was created to promote the development of therapies to treat rare diseases, defined as those for which the US prevalence is less than 200,000 individuals. There are a number of benefits to ODA status, including tax credits and marketing exclusivity in the United States for 7 years.