Perkinelmer Inc., of Waltham, Mass., has won a thumbs up from the U.S. FDA for its GSP Neonatal Creatine Kinase-MM kit. The assay is the first test approved in the U.S. for help in screening newborns for Duchenne muscular dystrophy (DMD), a rare but devastating genetic disorder. While there is no known cure for DMD, earlier screening and diagnosis could lead to improvements in quality of life by enabling earlier and more personalized treatment of symptoms. The kit’s approval opens the door for states to include DMD among the battery of tests newborns typically undergo, such as phenylketonuria, cystic fibrosis and congenital heart disease.
In August the FDA was skeptical about Sarepta Therapeutics Inc.’s injectable Vyondys 53 (golodirsen), but that changed swiftly Friday with the agency’s accelerated approval for the Duchenne muscular dystrophy (DMD) follow-on therapy, the first treatment specifically for this subtype.
As expected, Horizon Pharma plc sailed through the meeting of the FDA’s Dermatologic and Ophthalmic Drugs Advisory Committee regarding the BLA for teprotumumab in thyroid eye disease (TED) with few surprises but much discussion.
DUBLIN – The European Medicines Agency’s Committee for Medicinal Products for Human Use closed out its year’s work with positive opinions on two applications, Novartis AG’s Beovu (brolucizumab) in wet age-related macular degeneration and Merck & Co. Inc.’s Recarbrio for treating gram negative infections in adults.
LONDON – Over the next decade pharmacovigilance will move on from a sole focus on adverse drug reactions (ADRs) to encompass monitoring of the in-market efficacy of medicines, according to Guido Rasi, executive director of EMA.
Hitches for Horizon Pharma plc seem unlikely in the upcoming FDA advisory panel to mull the BLA for teprotumumab in thyroid eye disease (TED), though regulators did take issue with the clinical activity score (CAS) as calculated by the company.