Three orphan medicines recommended for approval by EMA/CHMP

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) at its September 2018 meeting recommended 13 medicines for approval, including three orphan medicines, with the latter being the following.

The Committee recommended granting a marketing authorization for the gene therapy Luxturna (voretigene neparvovec), developed by US biotech firm Spark Therapeutics (Nasdaq: ONCE)for the treatment of adults and children with inherited retinal dystrophy caused by RPE65 gene mutations, a rare genetic disorder which causes vision loss and usually leads to blindness. Luxturna was designated as an orphan medicine during its development.

This one-time therapy, which Swiss pharma giant Novartis (NOVN: VX) has acquired the rights to outside of the USA, is seen as a breakthrough for these individuals who currently face inevitable blindness and a life time of challenges.