Martin Pharmaceuticals, a clinical stage pharmaceutical company focused on repurposing already-approved drugs to offer life-changing advances to patients afflicted with rare (orphan) diseases or challenging medical conditions, has been granted Orphan Drug Designation by the FDA for Livantra in the treatment of Acute on Chronic Liver Failure.
Orphan drug designation is granted to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity, as well as tax credits for clinical research costs.
Acute-on-chronic liver failure (ACLF) is a syndrome characterized by acute decompensation of chronic liver disease associated with organ failures and high short- term mortality. The greater the number of organ systems involved, the higher the mortality rate. Despite current aggressive medical management, mortality at 30 and 90 days in ACLF remains high, ranging from about 50 percent in milder cases to nearly 80 percent in patients with three or more organ system failures.
Each year in the U.S. approximately 41,000 people are rushed to hospital with ACLF. Costs of ACLF patient care can range from $116,000 to $180,000 per episode.
Medical management is often viewed as a bridge to transplantation, but many patients don’t qualify for transplantation (age, co-morbidities, etc.) and demand greatly exceeds the supply (50 percent die before getting a transplant and only 25 percent of patients are successfully transplanted).
Livantra is a potential game changer as it may help patients to survive ACLF without transplantation, or allow patients to live long enough to receive a new liver.
(Source: Martin Pharmaceuticals, Inc.)
Filed Under: Drug Discovery