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.
U.S. Supreme Court justices appeared sympathetic to Merck & Co. in the company’s bid to fend off hundreds of lawsuits accusing the company of failing to adequately warn patients of the risks of thigh bone fractures associated with the osteoporosis drug Fosamax.
Acer Therapeutics in-licensed the clinical-stage, selective, non-peptide tachykinin NK3 receptor antagonist osanetant from Sanofi. MyoKardia regained global rights to all the programs that the company had inked with Sanofi.
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.