Biotechnology company Moderna Inc (Nasdaq:MRNA) reported on Tuesday that it has received US Food and Drug Administration (FDA) Fast Track designation to facilitate the development and expedite the review of its investigational mRNA therapeutic mRNA-3927 for propionic acidemia (PA), a rare, life-threatening, inherited metabolic disorder due to a deficiency in PCC critical for metabolism.

Following the US FDA award of FTD, the company said it plans to launch an open-label, multi-centre, dose escalation Phase 1/2 study of multiple ascending doses of mRNA-3927 in primarily paediatric patients with PA in the US and Europe.

According to the company, mRNA-3927 contains two mRNAs that encode for the alpha and beta subunits of the mitochondrial enzyme propionyl-CoA carboxylase (PCC), encapsulated within its proprietary lipid nanoparticle (LNP).

The objective of the company's study is to evaluate the safety and tolerability of mRNA-3927 administered via IV infusion, characterise the pharmacokinetic profile of mRNA-3927 and assess the pharmacodynamic response as assessed by changes in plasma biomarkers.

Additionally, mRNA-3927 is intended to treat patients with PA regardless of whether they are missing the alpha or beta subunits. It uses the same proprietary LNP formulation used in the company's antibody against chikungunya virus (mRNA-1944) and MMA (mRNA-3704) programme.

Moderna added that it has received US FDA Fast Track designation for its investigational Zika vaccine (mRNA-1893) and methylmalonic acidemia (MMA) (mRNA-3704) programmes.