The European Medicines Agency (EMA) has recommended the approval of two medicines that could offer new therapeutic options for people living with rare and serious conditions.
Duvyzat® has received a conditional marketing authorisation recommendation for the treatment of Duchenne Muscular Dystrophy (DMD) in children aged six and over who are still able to walk. DMD is a life-threatening genetic condition that leads to progressive muscle weakening. Duvyzat® is an oral suspension containing givinostat, a histone deacetylase (HDAC) inhibitor to be used alongside standard corticosteroid therapy, that helps modulate abnormal HDAC activity in dystrophic muscles. By doing so, it may help slowing the progression of the disease by reducing inflammation, tissue scarring and muscle deterioration.
Tepezza® has been recommended for marketing authorisation for the treatment of moderate to severe thyroid eye disease (TED) in adults. TED is a rare autoimmune condition that causes inflammation and swelling around the eyes, often leading to bulging, pain, double vision and, in severe cases, vision loss. Tepezza® contains teprotumumab, a monoclonal antibody (a type of protein) that targets the orbital fibroblasts (specialised cells of the eye sockets) blocking their autoimmune activation and thus potentially inhibiting the development and progression of TED.
While Tepezza® has been recommended for standard approval, Duvyzat® conditional authorisation is a regulatory mechanism designed to provide earlier access to promising therapies where urgent medical needs exist. This type of approval allows medicines to reach patients more quickly, while requiring the company to provide additional data over time to confirm long-term benefits and safety.
Both opinions now go to the European Commission for a final decision. If authorised, each EU Member State will then decide how the treatments will be made available in their national healthcare systems through pricing and reimbursement procedures.
More information available here for Duvyzat®, and here for Tepezza®.