FDA Grants Three Rare Pediatric Disease Designations as Program Readies to End

Rare Daily Staff

The U.S. Food and Drug Administration granted Rare Pediatric Disease designations to Applied Therapeutics, Innova Therapeutics, and Mereo BioPharma as the program is set to expire at the end of this month.

The agency granted the designation to Applied Therapeutics’ experimental therapy AT-007 for the treatment of PMM2-CDG, a debilitating rare disease caused by deficiency in the critical enzyme phosphomannomutase-2. The enzyme is required for systemic glycosylation of proteins. PMM2-CDG causes multiple organ failure and severe disability, resulting in approximately 20 percent mortality in the first four years of life.  There are currently no drugs approved to treat PMM2-CDG.

The FDA provided Innova Therapeutics the designation for the company’s experimental therapy IVT-8086 for the treatment of osteosarcoma. Osteosarcoma is a malignant bone tumor with histological findings of osteoid or bone production. It is a highly metastatic cancer that predominantly affects children, teenagers, and young adults. IVT-8086 is a humanized monoclonal antibody with high affinity to a novel anticancer target, secreted frizzled-related protein 2.

And finally, the agency awarded Mereo BioPharma Rare Pediatric Disease designation for setrusumab for the treatment of osteogenesis imperfecta, a rare genetic disorder that is characterized by fragile bones and reduced bone mass resulting in bones that break easily, loose joints and weakened teeth. Setrusumab is a fully humanized monoclonal antibody that inhibits sclerostin, a protein that inhibits the activity of bone-forming cells.

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 these drugs eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA. There are no approved therapies for these conditions.

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.

Though the program is due to expire at the end of the month, a drug that has been granted RPD designation prior to this date is still eligible to receive the voucher if it receives final FDA approval before September 30, 2022. There is also legislation that has been introduced to extend the program.

Editor’s note: This story was updated to correct the most recent sales of a priority review voucher.