RARE Daily

Moderna Receives FDA Fast Track Designation for Propionic Acidemia Program

October 23, 2019

The U.S. Food and Drug Administration granted Moderna Fast Track designation for mRNA-3927, the company’s experimental mRNA therapeutic for propionic acidemia, a rare, life-threatening, metabolic disorder.

Photo: Tal Zaks, chief medical officer at Moderna

Propionic acidemia is the result of a deficiency in PCC, an enzyme critical for metabolism. This deficiency can lead to a toxic buildup of acids in the body. Symptoms of PA typically become apparent during infancy and may include weak muscle tone, poor feeding, vomiting, and lack of energy. More severe health problems can also occur, including heart abnormalities, seizures, and coma.

The only effective treatment for severely affected individuals is liver transplant, which replaces the deficient PCC enzyme. Currently there are no approved therapies to treat the underlying cause of PA, including no enzyme replacement therapy, due to the complexity of the PCC enzyme that requires mitochondrial localization.

Moderna’s experimental mRNA therapeutic is designed to instruct the body to restore the missing or dysfunctional proteins that cause PA. mRNA-3927 contains two mRNAs that encode for the alpha and beta subunits of the mitochondrial enzyme propionyl-CoA carboxylase (PCC), encapsulated within Moderna’s proprietary lipid nanoparticle.

The fast track designation is designed to facilitate the development and expedite the review of drugs that treat serious conditions, potentially accelerating patient access to new therapies.

“Fast Track designation underscores the urgent need for a therapy that treats the underlying cause of propionic acidemia,” said Tal Zaks, chief medical officer at Moderna. “We are preparing to initiate a phase 1/2 clinical study of mRNA-3927 to continue learning about the potential for this investigational therapy to restore enzyme activity in patients with propionic acidemia.”

In addition to Fast Track designation, mRNA-3927 has also been granted Orphan Drug and Rare Pediatric Disease designations from the FDA and Orphan Designation by the European Medicines Agency.

Author: Rare Daily Staff

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