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.

Working in the rare disease space has never been more rewarding than it is today. Technology is turning what was once thought to be impossible into actual, tangible realities for physicians, patients, and families. There are many exciting advancements happening now – in real time – and even more on the horizon that demand our attention and advocacy to help bring them to light.

Vertex Pharmaceuticals Inc. will apply for regulatory approval during 2019 for the company’s triple combination of drugs to treat the life-shortening genetic condition cystic fibrosis.

Shares of North Carolina-based BioCryst Pharmaceuticals were down more than 53 percent after the company released data from a Phase III trial that underwhelmed analysts.