FDA Grants Rare Pediatric Disease Designation to Selecta and AskBio for their Gene Therapy for MMA

Rare Daily Staff

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to Selecta Biosciences’ and Asklepios BioPharmaceutical’s gene therapy MMA-101 for the treatment of isolated methylmalonic acidemia due to methylmalonyl-CoA mutase (MMUT) gene mutations.

“MMA is a serious and potentially life-threatening inherited metabolic disorder that presents in patients from newborns to adulthood,” said Sheila Mikhail, CEO and co-founder of AskBio. “AskBio is committed to delivering transformative genetic medicines for rare diseases like this one, and the Rare Pediatric Disease designation helps us continue development of MMA-101.”

Methylmalonic acidemia (MMA) is a rare monogenic disorder in which the body cannot break down certain proteins and fats. This metabolic disease may lead to hyperammonemia and is associated with long-term complications including feeding problems, intellectual disability, chronic kidney disease and inflammation of the pancreas. Symptoms of MMA usually appear in early infancy and vary from mild to life threatening. Without treatment, MMA can lead to coma and in some cases death.

 “This Rare Pediatric Disease designation from the FDA highlights the significant unmet medical need that Selecta and AskBio are seeking to address with MMA-101 for this rare metabolic disorder,” said Carsten Brunn, CEO of Selecta Biosciences. “When used with AAV gene therapy vectors, Selecta’s ImmTOR aims to inhibit the immune response to the AAV vector, potentially allowing re-dosing of gene therapies. Ongoing clinical programs will focus on evaluating product candidate performance in patients who may have been under-dosed or those who may lose transgene expression over time.”

AskBio and Selecta expect to initiate a phase 1 clinical trial of MMA-101 and ImmTOR for patients with MMA in the first half of 2021.

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes VS-01 eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Sarepta sold its priority review voucher to Vifor Pharma $111 million in February 2020.

The voucher program was due to expire at the end of September but Congress provided a short-term extension of the program. According to the current statutory sunset provisions, after December 11, 2020, FDA may only award a voucher for an approved rare pediatric disease product application if the sponsor has rare pediatric disease designation for the drug, and that designation was granted by December 11, 2020. After December 11, 2022, FDA may not award any rare pediatric disease priority review vouchers.

Going forward, requests for rare pediatric disease designation submitted will be reviewed in a timely manner but the FDA’s previous 60-day response time no longer applies.

Photo: Sheila Mikhail, CEO and co-founder of AskBio