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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.
" 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."
Ampreloxetine 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
The 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.
About Ampreloxetine
Ampreloxetine, an investigational, once-daily norepinephrine reuptake inhibitor in development for the treatment of symptomatic neurogenic orthostatic hypotension (nOH) in patients with multiple system atrophy (MSA). The unique benefits of ampreloxetine treatment reported in MSA patients from Study 0170 included an increase in norepinephrine levels, a favorable impact on blood pressure, clinically meaningful and durable symptom improvements, and no signal for supine hypertension. The Company presented three scientific platform presentations at the 2022 American Autonomic Society meeting. 3
About Multiple System Atrophy (MSA) and Symptomatic Neurogenic Orthostatic Hypotension (nOH)
MSA is a progressive brain disorder that affects movement and balance and disrupts the function of the autonomic nervous system. The autonomic nervous system controls body functions that are mostly involuntary. One of the most frequent autonomic symptoms associated with MSA is a sudden drop in blood pressure upon standing (nOH). 4 There are approximately 50,000 MSA patients in the US 1 and 70-90% of MSA patients experience nOH symptoms. 2 Despite available therapies, many MSA patients remain symptomatic with nOH.
Neurogenic orthostatic hypotension (nOH) is a rare disorder defined as a fall in systolic blood pressure of ⩾20 mm Hg or diastolic blood pressure of ⩾10 mm Hg, within 3 minutes of standing. Severely affected patients are unable to stand for more than a few seconds because of their decrease in blood pressure, leading to cerebral hypoperfusion and syncope. A debilitating condition, nOH results in a range of symptoms including dizziness, lightheadedness, fainting, fatigue, blurry vision, weakness, trouble concentrating, and head and neck pain.
About Theravance Biopharma
Theravance Biopharma, Inc.'s focus is to deliver Medicines that Make a Difference ® in people's lives. In pursuit of its purpose, Theravance Biopharma leverages decades of expertise, which has led to the development of FDA-approved YUPELRI ® (revefenacin) inhalation solution indicated for the maintenance treatment of patients with chronic obstructive pulmonary disease (COPD). Ampreloxetine, its late-stage investigational norepinephrine reuptake inhibitor in development for symptomatic neurogenic orthostatic hypotension, has the potential to be a first in class therapy effective in treating a constellation of cardinal symptoms in multiple system atrophy patients. The Company is committed to creating/driving shareholder value.
For more information, please visit www.theravance.com .
THERAVANCE BIOPHARMA ® , THERAVANCE ® , and the Cross/Star logo are registered trademarks of the Theravance Biopharma group of companies (in the U.S. and certain other countries).
YUPELRI ® is a registered trademark of Mylan Specialty L.P., a Viatris company. Trademarks, trade names or service marks of other companies appearing in this press release are the property of their respective owners.
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1 UCSD Neurological Institute ( 25K - 75K , with ~10K new cases per year); NIH National Institute of Neurological Disorders and Stroke ( 15K - 50K ). 2 Delveinsight MSA Market Forecast (2023); Symptoms associated with orthostatic hypotension in pure autonomic failure and multiple systems atrophy, CJ Mathias (1999). 3 November 2022, Biaggioni I, et al. Abstract 34 / Virtual Poster 106; Kaufmann H, et al. Abstract 33 / Virtual Poster 117; Freeman R, et al. Abstract 30 / Virtual Poster 4 4 https://medlineplus.gov/genetics/condition/multiple-system-atrophy/
SOURCE Theravance Biopharma, Inc.