By Marie Rosenthal
In a 10-to-1 vote, the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee said more data are needed to establish the effectiveness of ataluren (Translarna, PTC Therapeutics) before it can be recommended for approval.
The committee agreed with the FDA reviewers that the data presented by PTC Therapeutics were not conclusive enough to prove the drug was effective for the treatment of dystrophinopathies (most notably Duchenne muscular dystrophy [DMD]