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The U.S. FDA issued a new draft guidance for drugmakers aiming their resources at Fabry disease, a rare genetic disease.
Cranbury, N.J.-based Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania are expanding their gene therapy collaboration.
Asklepios BioPharmaceutical (AskBio) secured a $235 million investment from Vida Ventures and TPG Capital.
Pfizer acquired a 15 percent stake in Vivet Therapeutics as well as an exclusive option to fully acquire the business, a deal that will give the New York-based pharma giant access to the French company’s experimental gene therapy for a rare genetic disorder.
An advisory committee of the EMA recommended conditionally approving a drug from Akcea Therapeutics and Ionis Pharmaceuticals that aims to treat a rare genetic disease.