FDA Slaps Clinical Hold on Translate Bio’s mRNA Therapy for Rare Liver Disease

Translate Bio had been hoping that it would initiate clinical trials for its rare liver disease treatment asset MRT5201 this year. However, that hope has been delayed after the U.S. Food and Drug Administration (FDA) placed a hold on the trial. This morning, Lexington, Mass.-based Translate Bio announced the hold in a very brief statement. And, as could be expected, investors are not happy. Share prices have begun to slide as the market opens. As of 9:50, share prices have fallen more than 7 percent to $5.10. Translate Bio filed an Investigational New Drug Application in December, ahead of the partial government shutdown that has gummed up much of the FDA’s regulatory work. The company wanted to launch a Phase I/II trial to study MRT5201 for the treatment of ornithine transcarbamylase (OTC) deficiency, a rare disease that causes too much ammonia to accumulate in the blood. The most severe forms of OTC typically occur in newborns, according to the U.S. National Library of Medicine. Translate Bio said that after completing its review of the proposed study, the FDA has some “additional clinical and nonclinical questions.” Until those questions can be answered, the proposed trial was placed on hold, Translate Bio said. The initial notification of the clinical hold was sent verbally. A written notification with more details about the FDA’s questions will be sent to the company. Translate Bio plans to work with the FDA in an effort to resolve its questions as promptly as possible, the company said this morning.