The U.S. Food and Drug Administration granted full approval to Orphalan’s Cuvrior (trientine tetrahydrochloride) for the treatment of Wilson’s disease, a rare genetic disorder. The approval announcement marks the first new treatment available for patients in over five decades.
The U.S. health regulator approved Marinus Pharmaceuticals Inc.’s lead drug to treat seizures associated with a rare genetic disorder in patients two years of age and older, the company said on March 18, sending its shares soaring around 36 percent.
Shares of Taysha Gene Therapies were down more than 10 percent after a patient’s death marred positive interim data for the company’s experimental gene therapy treatment for Sandhoff and Tay-Sachs diseases, which are two forms of GM2 gangliosidosis.
Mirum Pharmaceuticals Inc. said on Sept. 29 the company’s drug became the first U.S. approved therapy for treating itching in patients with a rare liver disorder called Alagille syndrome (ALGS).
Merck won U.S. Food and Drug Administration approval for a first-of-its-kind oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor that is used to treat a rare genetic disease that causes benign and cancerous tumors in blood vessels associated with renal cell carcinoma.
The U.S. Food and Drug Administration granted marketing clearance for Ryplazim as the first FDA-approved therapy for the treatment of patients with the plasminogen deficiency type 1 (hypoplasminogenia), a rare genetic disorder.
Amicus Therapeutics announced topline results from the Phase III PROPEL pivotal trial of AT-GAA (cipaglucosidase alfa and miglustat) for late-onset Pompe disease.
The U.S. Food and Drug Administration’s PDUFA dates for the last week of November 2020 include a review of Liquidia Technologies’ NDA for LIQ861 for the treatment of pulmonary arterial hypertension (PAH).
While the world has largely been focused on the development of vaccines and therapeutics for Covid-19, the U.S. Food and Drug Administration has remained busy lining up potential approvals of medications for other diseases and illnesses.
Novartis said new interim data from an ongoing phase clinical trial for the company’s $2.1 million-per-patient gene therapy Zolgensma showed spinal muscular atrophy patients experienced significant therapeutic benefit.