The U.S. FDA has lifted its clinical hold on LogicBio Therapeutics phase 1/2 trials for a rare pediatric disorder, just months after stopping them after complications with children involved in the study.
The biotech has been cleared to resume enrolling patients in its SUNRISE study of a new genetic medicine for methylmalonic acidemia (MMA), a life-threatening disorder where the body cannot break down certain proteins and fats.
Trials were stopped in February after two cases of thrombotic microangiopathy (TMA) in children dosed in the study were reported. Both patients have since recovered and now LogicBio will move forward with new guidelines that include frequent testing for symptoms of TMA. TMA is characterized by microscopic blood clots, organ damage and low platelets. The patients who fell ill were aged six months to two years old. The first case of TMA was reported in 2020, but the study was not paused until the second case was reported almost two years later.
There is currently no approved medical therapy addressing the underlying cause of MMA. To manage the symptoms, patients go on a severely restrictive, low-protein, high-calorie diet, often through a feeding tube.
The company expects to dose its next patient by the end of September, said Frederic Chereau, president and CEO of LogicBio. The FDA has said that the biotech will need to implement protocol changes before it can continue, including enhanced monitoring measures of its patients.
The SUNRISE trials look at the use of LogicBio’s MMA drug LB-001 in pediatric patients aged six months to twelve years old. New patients in the study, who may be as young as six months old, will receive the lower dose. The company expects to present interim clinical data from the trial by the end of June.
