On the 25th of April, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) gave givinostat (Duvyzat), a novel histone deacetylase (HDAC) inhibitor, a positive opinion. The recommendation is for granting a conditional marketing authorisation for givinostat for the treatment of ambulant patients with Duchenne Muscular Dystrophy (DMD) aged six […]
Read MoreThe Committee for Orphan Medicinal Products (COMP) organized their Plenary Meeting last week (13-15 February), that was chaired by Regulatory Scientific Committee (RSC) Vice Chair, Violeta Stoyanova-Beninska, and Armando Magrelli. Among the topics discussed were the new applications for orphan medicinal product designation (including amendments of existing designations), requests for protocol assistance with significant benefit […]
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There are over 6,000 different rare diseases identified to date that affect 3.5% – 5.9% of the worldwide population. They have a big impact not only on the person diagnosed, but also on their families, friends, care takers and society as a whole. This is why development of medicines addressing rare diseases, also known as […]
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