RARE Daily

FDA Grants Rare Pediatric Disease Designation to Y-mAbs’ Treatment of Patients with Medulloblastoma

October 12, 2021

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to Y-mAbs Therapeutics for its lutetium labelled omburtamab antibody program for the treatment of medulloblastoma.

Photo: Claus Moller, CEO of Y-mAbs

Medulloblastoma is a malignant pediatric brain tumor that arises in the cerebellum and is the most common malignant brain tumor in children, usually occurring between 5 and 9 years of age, and is rare in adults over the age of 30. It is treatable if diagnosed early.

Y-mAbs’ 177Lu-omburtamab-DTPA, a monoclonal B7-H3 antibody that has been radiolabeled with lutetium-177 and is currently in a multicenter phase 1 clinical trial in pediatric patients with refractory medulloblastoma, and in a multicenter phase 1 clinical trial targeting B7-H3 positive CNS/LM tumors in adults. The company believes that both indications address clear unmet medical needs.  

“The RPDD makes us eligible for a Priority Review Voucher (PRV) upon potential approval of the biologics license application for this rare pediatric cancer. Among our leading compounds under development, four now have RPDDs, and this designation for 177Lu-omburtamab-DTPA further increase our chances of ultimately receiving multiple PRVs,” said Thomas Gad, founder, chairman and president.

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 177Lu-omburtamab-DTPA 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, Albireo sold a priority review voucher for $105 million.

“We are dedicated to bring 177Lu-omburtamab-DTPA to patients who desperately need alternative methods of treatment,” said Claus Moller, CEO of Y-mAbs. “We look forward to expanding the ongoing phase 1 studies with 177Lu-omburtamab-DTPA into separate phase 2 arms.”

Researchers at Memorial Sloan Kettering Cancer Center developed omburtamab, which is exclusively licensed by MSK to Y-mAbs. As a result of this licensing arrangement, MSK has institutional financial interests in the product.

Author: Rare Daily Staff

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