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

doctor in scrubs holding up hand in halt motion

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.

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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.

MRT5201 is an mRNA therapeutic designed to deliver functional OTC enzymes to the liver to enable the hepatocytes, the predominant type of liver cell, to produce the normal OTC enzyme. The therapeutic was being planned as an intravenous medication, according to data on the company’s website. MRT5201 has been granted orphan drug designation for the treatment of OTC deficiency in the United States and European Union.

Last year, Translate Bio and Sanofi teamed up to develop mRNA vaccines for up to five infectious diseases. The companies did not disclose what diseases they would target when they announced the multi-year collaboration. For Translate Bio, the deal could be worth up to nearly $1 billion if all milestones are hit. When the deal was struck, Sanofi’s John Shiver, senior vice president of R&D, said, “mRNA technology has significant potential for rapid and versatile manufacturing, reduced industrialization costs for multiple vaccines, and the improved breadth of immune response for infectious disease vaccines.”

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