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.
Rockville, Maryland-based RegenxBio entered a license deal with Pfizer for a gene therapy for Friedreich’s ataxia, the most common hereditary ataxia.
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.
An EMA panel recommended approving a potential blockbuster drug from Shire Plc to treat a rare hereditary disease.
The U.S. FDA issued a Complete Response Letter to Netherlands-based Pharming Group for the company’s supplemental Biologics License Application for Ruconest.
The U.S. FDA approved a first-of-its-kind drug from Shire Plc to treat patients aged 12 and older suffering from a rare hereditary disease that causes swelling.
Shire plc announced the U.S. Food and Drug Administration accepted the Biologics License Application and granted priority review for lanadelumab (SHP643).