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).

KalVista Pharmaceuticals Inc. inked a collaboration deal with Merck & Co. for potential therapeutic treatments for diabetic macular edema.

London-listed drugmaker Shire successfully tested a new medicine that prevents attacks in patients with hereditary angioedema (HAE).

FDA granted orphan-drug exclusivity for Haegarda for the prevention of hereditary angioedema attacks.

Investors are cooling off on Biocryst Pharmaceuticals despite the company’s strong interim reports for its Phase II hereditary angioedema (HAE) treatment. After rising about 45 percent in premarket trading, shares of Biocryst were down more than 14 percent.