Trials for an investigational gene therapy developed by Massachusetts-based biotech company LogicBio Therapeutics have been placed under clinical hold by the U.S. FDA after two pediatric cases of thrombotic microangiopathy (TMA) were reported.
The clinical trials were studying the use of the drug LB-001 for treating a metabolic disorder called methylmalonic acidemia (MMA) which prevents the body from properly breaking down certain proteins and fats. LB-001 is an AAV-delivered gene therapy, which has been associated with other cases of TMA and other serious adverse events during clinical trials. TMA is characterized by microscopic blood clots, organ damage and low platelets.
In the trials, two of the four patients, aged six months to two years old, fell ill. Both are responding well to treatment, the company said.
Currently, LogicBio says it does not have enough information to comment on the timeline of the drug’s development moving forward.
“We look forward to working closely with the FDA and the DMSB to determine the next steps for the trial and the program,” said Fred Chereau, president and CEO of LogicBio.
