BASKING RIDGE, N.J. - Lisata Therapeutics, Inc. (NASDAQ:LSTA), a clinical-stage pharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has awarded its lead product candidate, LSTA1, the Rare Pediatric Disease Designation (RPDD) for the treatment of osteosarcoma in children, adolescents, and young adults.
This designation is a significant regulatory milestone for LSTA1, which has shown potential in preclinical and clinical studies for various advanced solid tumors. "Receiving RPDD from the FDA is an important milestone as we plan future clinical expansion for LSTA1," said Kristen K. Buck, M.D., Executive Vice President of R&D and Chief Medical Officer at Lisata.
The FDA grants RPDD to encourage the development of treatments for rare pediatric diseases, defined as those affecting fewer than 200,000 people in the U.S. and primarily impacting individuals under 18. A key benefit of the RPDD is the eligibility to receive a priority review voucher upon FDA approval of the new drug application for the designated indication.
These vouchers can significantly shorten the FDA review process for other drug applications from ten months to six months and can be sold, with historical sales ranging from $75 to $100 million USD.
LSTA1 is designed to enhance the delivery of anti-cancer drugs to solid tumors by activating a novel uptake pathway. It also aims to modify the tumor microenvironment, potentially making tumors more responsive to immunotherapies.
In addition to the RPDD, LSTA1 has been granted orphan drug designation for pancreatic cancer in the U.S. and Europe, as well as for glioblastoma multiforme in the U.S., and has received a Fast Track designation from the FDA for pancreatic cancer.
Lisata Therapeutics focuses on developing innovative therapies for serious diseases, with a number of clinical and business milestones expected in the next two years. The company's current capital is projected to fund its operations into early 2026.
The information in this article is based on a press release statement from Lisata Therapeutics.
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