CHMP Recommends Three Orphan Drugs at September Meeting

The EMA’s Committee for Medicinal Products for Human Use recommended 13 drugs for human use at its September meeting including three orphan drugs.

The orphan products recommended for EU approval included Bayer’s hemophilia treatment Jivi (damoctocog alfa pegol), Kyowa Hakko’s Sezary syndrome drug Poteligeo (mogamulizumab) and Spark Therapeutics’ retinal disease gene therapy Luxturna (voretigene neparvovec).

Luxturna is the first treatment to be recommended by CHMP for the rare genetic disease. It is intended for patients who have inherited the mutation from both parents and who have enough viable retinal cells.