FDAnews Drug Daily Bulletin
Pharmaceuticals / Submissions and Approvals

EMA Committee Recommends 12 Orphan Drugs

Feb. 8, 2019

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.

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