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.
The U.S. Food and Drug Administration accepted Copenhagen, Denmark-based Orphazyme’s New Drug Application (NDA) for arimoclomol for the treatment of Niemann-Pick disease Type C.
Alnylam Pharmaceuticals Inc. priced the company’s gene silencing drug to treat patients with a rare genetic disorder that can cause severe pain at $575,000 per year after receiving an early U.S. approval.
The U.S. FDA issued a new draft guidance for drugmakers aiming their resources at Fabry disease, a rare genetic disease.