The European Commission has approved Agamree (vamorolone), a dissociative corticosteroid — a treatment expected to have fewer side effects than standard steroid medications — for patients ages 4 and older with Duchenne muscular dystrophy (DMD). The decision applies to all member states of the European Union, plus Iceland,…

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…

The European Commission has granted Orphan Drug Designation to Benitec Biopharma’s BB-301, an investigational treatment for oculopharyngeal muscular dystrophy (OPMD). The decision represents a major advance for OPMD, a rare congenital myopathy, for which the currently used therapeutic strategies involve repetitive surgical interventions that have limited effectiveness. Orphan Drug…

The European Commission (EC) has decided to renew the conditional marketing authorization for Translarna (ataluren) to treat certain nonsense mutation Duchenne muscular dystrophy (nmDMD) patients. PTC Therapeutics can now market Translarna for nmDMD patients who are 5 or older, mobile, and living in the European Union, Iceland, Liechtenstein, and Norway. The decision follows a…

The U.S. Food and Drug Administration (FDA) and the European Commission (EC) have granted Orphan Drug designations to Solid Biosciences’ SGT-001, a micro-dystrophin gene therapy under development for the treatment of Duchenne muscular dystrophy (DMD). Clinical trials should commence in 2017, the company announced…

Biotherapeutics company aTyr Pharma, Inc. was recently awarded orphan drug designation from the European Commission (EC) for its Resolaris therapy, designed to treat facioscapulohumeral muscular dystrophy (FSHD). The investigational drug is the company’s first Physiocrine-based therapy, and is currently being developed to become a first-in-class intravenous protein treatment for…