RARE Daily

FDA Grants Rare Pediatric Disease Designation for Flag’s Treatment for Children with DIPG

February 1, 2023

Rare Daily Staff

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation for Flag Therapeutics’ FLAG-003, an investigational small molecule therapy for the treatment of diffuse intrinsic pontine glioma, a rare, highly aggressive and difficult to treat brain tumor found in children.

Diffuse intrinsic pontine glioma (DIPG) is a highly aggressive, difficult to treat malignant brain tumor that is usually diagnosed in children between the ages of five and nine. It accounts for nearly 10 percent of all childhood central nervous system tumors. In the United States, approximately 300 children are diagnosed with DIPG each year. Due to its location in the brain stem, surgery is not an option, and results of first line treatment with radiation are typically short lived, lasting on average approximately six to nine months. At present, there are no approved drug therapies for the treatment of DIPG.

“Receiving Rare Pediatric Disease designation for FLAG-003 for children with DIPG underscores the dire need for a treatment option for this devastating, fatal form of brain cancer,” said Frank Sorgi, president and CEO of Flag Therapeutics. “Data from our preclinical studies have shown that FLAG-003 crosses the blood brain barrier, a major hurdle in the treatment of all forms of brain cancer, and selectively targets and kills tumor cells.”

FLAG-003, the company’s lead program for the treatment of all gliomas, is engineered to cross the blood brain barrier and specifically target and kill cancer cells by simultaneously blocking the formation of a tumor vascular system and disrupting cancer cell replication by inhibiting tubulin.

FLAG-003 was previously granted Orphan Drug designation for the treatment of all gliomas, including glioblastoma multiforme and DIPG, by the FDA.

The Rare Pediatric Disease (RPD) designation program is offered by the FDA to encourage the development of new drugs for rare pediatric diseases (diseases affecting children 18 years of age and younger and fewer than 200,000 people in the United States). Under the RPD program, a sponsor who receives approval for a drug or biologic for a rare pediatric disease for which the RPD designation has been granted, may qualify for a priority review voucher (PRV) at the time of market approval. Holders of a PRV can redeem the voucher to obtain priority review, which shortens review from 10-months to 6-months, for any subsequent marketing application, or they can sell or transfer it to other developers. Recently, Bluebird Bio sold two PRVs, one for $95 million and one for $102 million.

Photo: Frank Sorgi, president and CEO of Flag Therapeutics


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