May CHMP meeting sees five novel medicines backed for approval

Following the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) meeting in May, positive opinions were issued on five novel medicinal products. These opinions will now be sent to the European Commission, which usually makes a final decision in two to three months.

First up, a recommendation for marketing authorization was adopted for Cevenfacta (eptacog beta (activated)) for the treatment of bleeding episodes in patients with congenital hemophilia, from Laboratoire Francais du Fractionnement et des Biotechnologies. The benefit of Cevenfacta is its ability to effectively control bleeding episodes, as observed in a Phase III, multicenter, open-label trial.

The CHMP gave a positive opinion under exceptional circumstances for a new gene therapy, Upstaza (eladocagene exuparvovec), from PTC Therapeutics (Nasdaq: PTCT). It is the first medicine intended to treat adult and pediatric patients with aromatic L‑amino acid decarboxylase (AADC) deficiency, an ultra-rare genetic disorder affecting the nervous system. Approval would make Upstaza the first disease-modifying treatment for AADC.