AVRO AVROBIO Inc

By Chris Wack

AvroBio Inc. said Tuesday that the U.S. Food and Drug Administration has granted rare pediatric disease designation to AVR-RD-04, an investigational gene therapy for the treatment of cystinosis.

The FDA's Rare Pediatric Disease Designation and Voucher Program is intended to help the development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

Companies that receive approval for a new drug application or biologics license application for a rare pediatric disease may be eligible to receive a voucher for a priority review of a subsequent marketing application for a different product. The priority review voucher may be used by the company or sold to a third party.

Cystinosis is a life-threatening disease that causes progressive multiorgan damage that affects about 1,600 people in the U.S., Europe and Japan. AVR-RD-04 is designed to genetically modify patients' own hematopoietic stem cells to express the gene encoding cystinosin, the protein that is critically deficient in people living with cystinosis, AvroBio said.

The company said preliminary data from an ongoing Phase 1/2 clinical trial suggest that this approach is well-tolerated, with no adverse events related to the drug product reported to date.

AvroBio shares were up 11% to 89 cents in premarket trading.

Write to Chris Wack at chris.wack@wsj.com

(END) Dow Jones Newswires

September 20, 2022 07:39 ET (11:39 GMT)

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