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Theravance Biopharma, Inc. Announces Orphan Drug Designation Granted to Ampreloxetine for the Treatment of Symptomatic Neurogenic Orthostatic Hypotension in Patients with Multiple System Atrophy
DUBLIN, May 9, 2023 /PRNewswire/ -- Theravance Biopharma, Inc. ("Theravance Biopharma" or the "Company") (NASDAQ: TBPH) today announced that the US Food and
About this update from Theravance Biopharma, Inc.
[{"type":"text","content":"DUBLIN, May 9, 2023 /PRNewswire/ -- Theravance Biopharma, Inc. (\"Theravance Biopharma\" or the \"Company\") (NASDAQ: TBPH) today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) status to ampreloxetine for the treatment of symptomatic neurogenic orthostatic hypotension (nOH) in patients with multiple system atrophy (MSA). MSA is a rare neurodegenerative disorder in which patients suffer autonomic dysfunction affecting movement, balance, heart rate and blood pressure. Many patients with MSA suffer from the debilitating symptoms of nOH including dizziness, disrupted vision, muscle weakness, head and neck discomfort, and fatigue, which adversely affect quality of life. \n\n \n \n \n \n \n \n\n \n\"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, Chief Executive Officer. \"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.\"\nAmpreloxetine is a once-daily norepinephrine reuptake inhibitor discovered at Theravance Biopharma. Previous clinical studies have demonstrated that ampreloxetine increased norepinephrine levels, prevented a drop in blood pressure, and prevented symptom worsening in patients with MSA. Approximately 80% of the 50,000 MSA patients in the U.S. suffer from nOH.1,2\nThe Orphan Drug Act of 1983 (ODA) was created to incentivize the pharmaceutical and medical communities to develop products to treat rare diseases, defined as those for which the U.S. prevalence is less than 200,000 individuals. Under the ODA, companies can request Orphan Drug Designation status for an experimental therapy and stand to receive a 50% tax credit toward the cost of qualified clinical trial and seven years of marketing exclusivity, if approved. Orphan Drugs are also exempt from user fees associated with regulatory review.\nAbout Ampreloxetine\nAmpreloxetine, an investigational, once-daily norepinephrine reupt...