RARE Daily

FDA Grants Receive Rare Pediatric Disease Designation to Three Companies as Extended Deadline for Filing Looms

October 7, 2020

Rare Daily Staff

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to experimental therapies from three companies just ahead of the program’s extended deadline for filing an application.  

The designation was granted to Aegle Therapeutics’ AGLE-102 for the treatment of dystrophic epidermolysis bullosa, a debilitating, genetic skin disease; to Y-mAbs Therapeutics for nivatrotamab for the treatment of the neuroblastoma; and Oncternal Therapeutics for TK216 for treatment of Ewing sarcoma.

Dystrophic epidermolysis bullosa (DEB) is one of the major forms of epidermolysis bullosa, a debilitating, genetic, pediatric disease characterized by skin fragility that can result in widespread blistering, chronic wounds and significant scarring. It is caused by mutations in the COL7A1 gene. There is currently no treatment or cure for DEB.

Aegle’s AGLE-102 is a composite of mesenchymal stem cell-derived extracellular vesicles that deliver proteins, genetic material and regenerative healing factors to diseased and damaged tissue. AGLE-102 will be evaluated in DEB patients in a phase 1/2a trial that will start in 2021. AGLE-102 also has Fast Track designation from the FDA.

“The FDA’s grant of Rare Pediatric Disease designation following its earlier grant of Fast Track Designation to AGLE-102 for DEB underscores the significant unmet medical need of children and adults living with this debilitating disease. AGLE-102 has the potential to be the first multifaceted approach to treat this rare patient population,” said Shelley Hartman, CEO of Aegle Therapeutics.

Y-mAbs’ nivatrotamab is a humanized bispecific anti-GD2 antibody currently in phase 1 clinical development in collaboration with Memorial Sloan Kettering Cancer Center in patients with relapsed/refractory neuroblastoma, a rare pediatric brain cancer, as well as high grade osteosarcoma and other GD2(+) solid tumors, where patients have relapsed or refractory disease that is resistant to standard therapy. The FDA also granted Orphan Drug designation for nivatrotamab.

Ewing sarcoma is the second most common bone tumor among children and adolescents with a median age at diagnosis of 15 and an incidence of about 3 cases per 1 million per year in children under the age of 20 in the United States. Nearly all Ewing sarcoma cases are driven by translocations of ETS family oncogenes. Patients diagnosed with metastatic disease have five-year survival rates between 18 percent and 30 percent.

Oncternal’s TK216 is an experimental, potentially first-in-class, targeted small-molecule inhibitor of the E26 transformation-specific (ETS) family of oncoproteins including fusion proteins. In preclinical models, TK216 was observed to bind to EWS-FLI1, blocking the interaction between this fusion protein and other transcriptome proteins such as RNA helicase A, leading to tumor cell apoptosis and inhibiting tumor growth in animal models. The FDA has also granted Orphan and Fast Track designations to TK216 for the treatment of Ewing sarcoma.

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 the company 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 program was due to expire at the end of September with a drug that had been granted RPD designation prior to that date still eligible to receive the voucher if it receives final FDA approval before September 30, 2022.

On September 30, 2020, Congress provided a short-term extension of the Rare Pediatric Disease Priority Review Voucher 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.

Requests for rare pediatric disease designation submitted within two weeks of a request for fast track designation or orphan drug designation are entitled to a 60-day review. Requests for rare pediatric disease designation not submitted with a request for fast track designation or orphan drug designation are reviewed in a timely manner, however, the 60-day response time does not apply. Friday, October 9, 2020, is the last business day that is not less than 60 days prior to December 11, 2020. The Offices of Orphan Products Development and Pediatric Therapeutics will continue to review all rare pediatric disease designation requests but said it cannot commit to providing a response to requests received after October 9, 2020.


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