FDA Places Clinical Hold on Translate Bio’s Application to Begin Trials for mRNA Therapy

January 23, 2019

Rare Daily Staff

The U.S. Food and Drug Administration has placed a clinical hold on Translate Bio’s application to begin human clinical trials of its messenger RNA (mRNA) therapy MRT5201 for the treatment of ornithine transcarbamylase (OTC) deficiency, a rare, inherited urea cycle disorder.

OTC is the most common urea cycle disorder in humans. It is caused by the lack of the OTC enzyme. As a result, toxic levels of ammonia build up in the blood, which can cause delirium, erratic behavior, a reduced level of consciousness, headaches, vomiting, and seizures.

The company submitted the application in December 2018 to support the initiation of a phase 1/2 clinical trial in patients with OTC deficiency. The FDA has completed its review of the company’s application for MRT5201 but has additional clinical and nonclinical questions.

The company said it expects to receive formal written communication with additional information from the FDA in the near future and plans to work with the FDA in an effort to resolve its questions as promptly as possible.


January 23, 2019



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