Amicus Therapeutics announced topline results from the Phase III PROPEL pivotal trial of AT-GAA (cipaglucosidase alfa and miglustat) for late-onset Pompe disease.
Data with Sanofi’s investigational enzyme replacement therapy avalglucosidase alfa in late-onset Pompe disease and infantile-onset Pompe disease will be featured as platform and poster presentations at the 17th annual WORLDSymposiumTM, to be held February 8-12, 2021.
Shares of Viela Bio skyrocketed more than 52% in trading after Ireland’s Horizon Therapeutics announced the acquisition of the rare disease medicines company in a $3.05 billion deal.
The U.S. Food and Drug Administration approved Janssen Pharmaceuticals’ Darzalex Faspro for adults with newly diagnosed light chain amyloidosis.
Alexion Pharmaceuticals Inc. paused enrollment in a late-stage study testing the company’s rare blood-disorder drug in adults with severe Covid-19 requiring mechanical ventilation.
AstraZeneca is acquiring Alexion Pharmaceuticals for $39 billion, with the deal expected to close in the third quarter of 2021.
The U.S. Food and Drug Administration approved Rhythm Pharmaceuticals’ Imcivree (setmelanotide) for chronic weight management in adult and pediatric patients aged 6 years and older with obesity due to proopiomelanocortin, proprotein convertase subtilisin/kexin type 1 or leptin receptor deficiency confirmed by genetic testing.
The U.S. Food and Drug Administration (FDA) approved Alnylam Pharmaceuticals’ Oxlumo (lumasiran) for primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in children and adults.
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.