FDA Lifts Clinical Hold on LogicBio’s Pediatric Trial
The FDA has lifted a clinical hold on Lexington, Mass.-based LogicBio’s early-stage study in pediatric patients with methylmalonic acidemia (MMA) — a rare inherited disorder in which the body cannot properly break down certain proteins and fats.
The agency placed the clinical trial on hold in February when two patients treated with the investigational gene editing drug LB-001 experienced thrombotic microangiopathy (TMA), a condition that affects small blood vessels and can cause blood clots. Both cases were resolved within weeks, the company said.
LogicBio has adjusted the clinical trial protocol, including adding frequent testing and the provision of a complement inhibitor if TMA is detected.
The company plans to release interim clinical data on the phase 1/2 study by the end of the second quarter of 2022 and to resume dosing in the third quarter.
The FDA has granted LB-001 Fast Track, Rare Pediatric Disease and Orphan Drug designations for treatment of MMA.
May 10, 2022
