The Food and Drug Administration (FDA) has granted Fast Track designation to mRNA-3927 (Moderna), an investigational mRNA therapeutic for propionic acidemia (PA).
Propionic acidemia is an inherited metabolic disorder caused by a deficiency in the enzyme propionyl-CoA carboxylase (PCC). Symptoms of the disorder include weak muscle tone, loss of appetite, vomiting, and lethargy; serious complications such as heart abnormalities, seizures, and coma can also occur in patients whose condition has progressed without treatment.
“The disease is characterized by life-threatening illnesses in response to minor stressors, neurological dysfunction and cardiomyopathy,” said George Diaz, MD, PhD, chief, division of medical genetics, Icahn School of Medicine at Mount Sinai Hospital. “Currently there are no approved therapies available that treat the underlying cause of this debilitating disease.”
Moderna’s investigational treatment contains two mRNAs that encode for the alpha and beta subunits of PCC, thereby restoring the missing dysfunctional proteins; mRNA-3927 is intended to treat propionic acidemia regardless of whether patients are missing the alpha or beta subunits.
The Company plans to begin a dose escalation phase 1/2 study of mRNA-3927 primarily in pediatric PA patients. The study will evaluate the safety and tolerability of the treatment administered via intravenous infusion, and will assess the pharmacodynamic response as assessed by changes in plasma biomarkers.
Fast Track designation can aid in expediting the review process, allowing for frequent communication with the FDA.
For more information visit modernatx.com.