Shares of Vertex Pharmaceuticals fell in trading after the company announced it will not advance a novel small molecule corrector of the Z-AAT protein into a Phase III study for a rare disease that can lead to complications of the liver and lungs.

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.

Several biopharmaceutical companies submitted New Drug Applications (NDAs) to the U.S. Food and Drug Administration in recent weeks, covering treatment indications ranging from an opioid overdose to bipolar disorder and rare disease.

A first-of-its-kind organization launched on May 13 unifying the voices of life science companies committed to delivering desperately needed treatments to rare disease patients. The Rare Disease Company Coalition’s 10 founding members have brought 22 treatments to market and have more than 160 rare disease programs in the works. 

Amryt inked an all-stock acquisition deal with Chiasma. The combined company will focus on rare and orphan diseases with three marketed products and a strong development pipeline.

On the back of a $135 million public offering in February, rare disease gene therapy company Krystal Biotech Inc. launched subsidiary Jeune Inc. to promote gene therapies for aesthetic skin conditions.

Prescription drug watchdog GoodRx compiled a list of the 10 most expensive drugs on the market.

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.