Therapy Areas: Cardiovascular
Moderna Receives FDA Fast Track Designation for Propionic Acidemia Programme mRNA-3927 is Moderna's Second Rare Disease Programme to Receive Fast Track Designation
25 October 2019 - - The US Food and Drug Administration has granted Fast Track designation for mRNA-3927, US-based biotechnology company Moderna, Inc's (NASDAQ: MRNA) investigational mRNA therapeutic for propionic acidemia. The company announced the open IND for mRNA-3927 on September 30, 2019, the company said.

Fast Track is designed to facilitate the development and expedite the review of therapies and vaccines for serious conditions and fill an unmet medical need.

Programmes with Fast Track designation may benefit from early and frequent communication with the FDA, in addition to a rolling submission of the marketing application.

The company previously received Fast Track designation for its investigational Zika vaccine (mRNA-1893) and methylmalonic acidemia (mRNA-3704) programmes.

Moderna plans to initiate an open-label, multi-center, dose escalation Phase 1/2 study of multiple ascending doses of mRNA-3927 in primarily pediatric patients with PA in the United States and Europe.

The objectives of this study are to evaluate the safety and tolerability of mRNA-3927 administered via IV infusion, characterize the pharmacokinetic profile of mRNA-3927 and assess the pharmacodynamic response as assessed by changes in plasma biomarkers.

PA and MMA are rare diseases that share similar disease pathology and are both typically treated by metabolic specialists.

In order to characterize and describe the natural history of these disorders and identify potential clinical and biomarker endpoints, Moderna is conducting a global, multi-centre, non-interventional observational study for patients with confirmed diagnosis of PA or MMA.

Moderna is currently recruiting patients with MMA for a Phase 1/2 study of mRNA-3704.

mRNA-3927 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, encapsulated within Moderna's proprietary lipid nanoparticle.

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

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.

Propionic Acidemia is a rare, life-threatening, inherited metabolic disorder that is the result of a deficiency in PCC that is 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 is advancing messenger RNA science to create a new class of transformative medicines for patients.

mRNA medicines are designed to direct the body's cells to produce intracellular, membrane or secreted proteins that have a therapeutic or preventive benefit with the potential to address a broad spectrum of diseases.

Moderna's platform builds on continuous advances in basic and applied mRNA science, delivery technology and manufacturing, providing the company the capability to pursue in parallel a robust pipeline of new development candidates.

Moderna is developing therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases and cardiovascular diseases, independently and with strategic collaborators.

Headquartered in Cambridge, Mass., Moderna currently has strategic alliances for development programs with AstraZeneca, Plc. and Merck, Inc., as well as the Defense Advanced Research Projects Agency (DARPA), an agency of the US Department of Defense and the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant secretary for Preparedness and Response within the US Department of Health and Human Services. Moderna has been ranked in the top ten of Science's list of top biopharma industry employers for the past four years.
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