EMA Committee Recommends 12 Orphan Drugs

The European Medicine Agency’s Committee for Orphan Medical Products adopted 12 positive opinions for orphan treatments during its Jan. 22-24 meeting.

The recommendations included PharmaMar’s lurbinectedin for treating small cell lung cancer; Fondazione Telethon’s hemophilia B treatment; 3R Pharma Consulting’s losartan for treating epidermolysis bullosa; and Roche Registration’s risdiplam for treatment of spinal muscular atrophy.

The committee also recommended: FGK Representative Service’s infantile neuroaxonal dystrophy treatment; Enzyvant Therapeutics Ireland’s treatments for DiGeorge syndrome, CHARGE syndrome and severe combined immunodeficiency due to FOXN1 deficiency; Common Services Agency’s treatment of post-transplant lymphoproliferative disorder; and Clinical Network Services’ non-traumatic osteonecrosis treatment.