RARE Daily

FDA Grants Rare Pediatric Disease Designations to Moleculin and Aeglea

December 1, 2020

Rare Daily Staff

The U.S. Food and Drug Administration granted Moleculin Biotech’s experimental candidate WP1066 three separate Rare Pediatric Disease designations for three rare cancers: diffuse intrinsic pontine glioma (DIPG), medulloblastoma, and atypical teratoid rhabdoid tumor.

WP1066 is an immune/transcription modulator capable of inhibiting p-STAT3 and other oncogenic transcription factors while also stimulating a natural immune response, targeting brain tumors, pancreatic cancer, and hematologic malignancies.

“The early activity we are seeing in WP1066 is both unexpected and encouraging,” said Walter Klemp, chairman and CEO of Moleculin. “The approval of these three Rare Pediatric Disease designations is a reminder of just how important our efforts are to potentially help children with brain tumors.”

The FDA also granted Rare Pediatric Disease designation to Aeglea BioTherapeutics’ ACN00177 for the treatment of rare metabolic disorder homocystinuria.

Homocystinuria is a serious metabolic disorder characterized by elevated plasma homocysteine, which leads to a wide range of life-altering complications and reduced life expectancy. ACN00177 is a novel engineered human enzyme therapy designed to lower the total level of homocysteine in the plasma. Aeglea initiated a phase 1/2 clinical trial in the second quarter of 2020 to investigate ACN00177 for the treatment of homocystinuria.

“There is a significant need for new and better therapies for homocystinuria patients given the lack of effective treatment options for many patients,” said Anthony Quinn, president and CEO of Aeglea. “The Rare Pediatric Disease Designation speaks to the significant impact of this disease beginning in childhood and is an important incentive in promoting drug development to improve outcomes in this patient population.”

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 therapies 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, Eiger BioPharmaceuticals sold its priority review voucher for $95 million in November 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, submitted requests for rare pediatric disease designation will be reviewed in a timely manner but the FDA’s previous 60-day response time no longer applies.

Photo: Walter Klemp, chairman and CEO of Moleculin

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