The U.S. Food and Drug Administration (FDA) approved Bristol Myers Squibb’s oral heart disease drug, making Camzyos (mavacamten) the first cardiac myosin inhibitor to be permitted for use in the country.

Bristol Myers Squibb announced results from an extended study to evaluate the long-term effects of taking the cardiovascular drug mavacamten.

BioMarin updated several of the company’s gene therapy programs on February 17 following a September 2021 update, when the drug manufacturer reported the U.S. Food and Drug Administration had placed a clinical hold on the BMN 307 Phearless Phase I/II trial.

BioCryst Pharmaceuticals, a global biopharmaceutical company that focuses on developing treatments for rare diseases, canceled plans to hold a public offering to raise funds. 

The U.S. Food and Drug Administration placed a clinical hold on KalVista Pharmaceuticals’ proposed Phase II trial of KVD824, a prophylactic treatment of hereditary angioedema. The regulatory agency requested additional information and analysis of preclinical studies regarding KVD824 before giving the go-ahead to the Phase II trial. 

The U.S. Food and Drug Administration approved BioCryst Pharmaceuticals Inc.’s Orladeyo (berotralstat) for prophylaxis to prevent attacks of hereditary angioedema in adults and young patients 12 years and older.

Only a year after buying cancer specialist Celgene for $74 billion, Bristol Myers Squibb announced the acquisition of cardiology company MyoKardia for about $13.1 billion.

San Diego-based Ligand Pharmaceuticals is acquiring San Diego-based Pfenex for a total deal transaction of up to $516 million.

AstraZeneca and Merck & Co. announced that the U.S. Food and Drug Administration approved the kinase inhibitor Koselugo (selumetinib) for the treatment of pediatric patients 2 years of age and older with neurofibromatosis type 1 who have symptomatic, inoperable plexiform neurofibromas.

Rockville, Maryland-based RegenxBio entered a license deal with Pfizer for a gene therapy for Friedreich’s ataxia, the most common hereditary ataxia.