Santhera Pharmaceuticals has asked the European Medicines Agency (EMA) to approve vamorolone to treat Duchenne muscular dystrophy (DMD). The agency’s assessments of new approval requests — in the form of marketing authorization applications — take up to 210 days, but can be longer if the Committee for Medicinal Products…

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.

The European Medicines Agency (EMA) has validated a marketing authorization application for Puldysa (idebenone) for treating respiratory dysfunction in Duchenne muscular dystrophy (DMD) patients who do not take glucocorticoids. This means that the submission, which was made as a conditional marketing authorization (potentially granted to therapies whose immediate benefit…

Biophytis SA is collaborating with AFM-Telethon to advance an oral pediatric formulation of Sarconeos (BIO101) for treating Duchenne muscular dystrophy (DMD). The pact calls for the French muscular dystrophy organization AFM-Telethon to give €400,000 (about $452,000) to France-based Biophytis  for further preclinical study. The funds also will go…

Santhera Pharmaceuticals has submitted a marketing authorization request to the European Medicines Agency (EMA) for Puldysa (idebenone) as a treatment for respiratory dysfunction in patients with Duchenne muscular dystrophy (DMD). The request is made as a conditional marketing authorization (CMA), which may be granted to medicines whose benefit…

Santhera Pharmaceuticals intends to file for a conditional marketing authorization with the European Medicines Agency (EMA) for idebenone — to be marketed as Puldysa — as a treatment for respiratory dysfunction in Duchenne muscular dystrophy (DMD). As it continues to gather data on the therapy, the company…

The Committee for Medicinal Products for Human Use (CHMP) — part of the European Medicines Agency (EMA) — is recommending against Exondys 51 (eteplirsen) as a therapy for Duchenne muscular dystrophy (DMD) patients amenable to skipping exon 51 of the dystrophin gene, the treatment’s developer, Sarepta Therapeutics, has announced. The company…

A European Medicines Agency (EMA) committee is beginning a review of  Sarepta Therapeutics‘ request for conditional approval of eteplirsen as a treatment for Duchenne muscular dystrophy (DMD) patients with a confirmed mutation of the dystrophin gene that can be overcome by exon 51 skipping. The Committee for Human Medicinal Products (CHMP) validated a Marketing Authorization application (MAA)…