RARE Daily

FDA Grants GlycoMimetics Rare Pediatric Disease Designation for SCD Drug

October 6, 2020

Rare Daily Staff

The U.S. Food and Drug Administration has granted Rare Pediatric Disease designation to GlycoMimetics’ rivipansel for the treatment of sickle cell disease in patients 18 years old and younger.

Sickle cell disease (SCD) is a lifelong, inherited blood disorder in which red blood cells are abnormally shaped in a crescent, or “sickle” shape, which restricts the flow in blood vessels and limits oxygen delivery to the body’s tissues, leading to severe pain and organ damage. It is also characterized by severe and chronic inflammation that worsens vaso-occlusive crises (VOCs) during which patients experience episodes of extreme pain and organ damage.

Approximately 100,000 people in the United States have sickle cell disease. While the majority of people with SCD are of African descent, the disease can affect all ethnic groups, especially those from areas where malaria is or was endemic, such as the Middle East, India and the Southern Mediterranean.

“The FDA’s designation recognizes the morbidity and mortality burdens of sickle cell disease as well as its significant impact during childhood with life-long implications. With this designation, the agency acknowledges the urgent need for improved treatment of children living with sickle cell disease,” said Helen Thackray, chief medical officer of GlycoMimetics.

Rivipansel is a glycomimetic drug candidate that binds to all three members of the selectin family. After the phase 3 RESET trial conducted by Pfizer, GlycoMimetics’ former collaborator, produced disappointing results in 2019, new clinical outcome data from a post hoc analysis of early treatment with rivipansel were published in June 2020 in advance of a poster shown at the Foundation for Sickle Cell Disease Research Meeting on September 24, 2020.

“With global rights for rivipansel in our hands, we are exploring options to move forward in sickle cell disease, including discussions with the FDA as to whether there is a regulatory path to approval,” said Rachel King, CEO of GlycoMimetics. “We plan to roll out the full data set from the phase 3 RESET program at upcoming medical meetings, based on the acceptance of abstract submissions.”

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 rivipansel 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.

Photo: Rachel King, CEO of GlycoMimetics

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