RARE Daily

FDA Grants Rare Pediatric Disease Designation to Lisata’s LSTA1 for the Treatment of Osteosarcoma

March 21, 2024

Rare Daily Staff

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to Lisata Therapeutics’ lead product candidate LSTA1 for the treatment of osteosarcoma, a rare cancer that can develop in children, adolescents, and young adults.

“LSTA1 has already demonstrated promise in preclinical and clinical settings for the treatment of several advanced solid tumors, and we believe that potential extends to a beneficial impact in osteosarcoma, a rare cancer that usually develops in the osteoblast cells that form bone,” said Kristen Buck, executive vice president of R&D and chief medical officer of Lisata. “Receiving RPDD from the FDA is an important milestone as we plan future clinical expansion for LSTA1, and, more importantly, it underscores the high unmet medical need of this patient population.”

LSTA1 is an investigational drug designed to activate a novel uptake pathway that allows co-administered or covalently bound anti-cancer drugs to penetrate solid tumors more effectively. LSTA1 actuates this active transport system in a tumor-specific manner, resulting in systemically co-administered anti-cancer drugs more efficiently penetrating and accumulating in the tumor. LSTA1 also has the potential to modify the tumor microenvironment, with the objective of making tumors more susceptible to immunotherapies.

Lisata and its collaborators have amassed significant non-clinical data demonstrating enhanced delivery of a range of existing and emerging anti-cancer therapies, including chemotherapeutics, immunotherapies, and RNA-based therapeutics. Additionally, LSTA1 has demonstrated favorable safety, tolerability, and activity in clinical trials to enhance delivery of standard of care chemotherapy for pancreatic cancer. Lisata is exploring the potential of LSTA1 to enable a variety of treatment modalities to treat a range of solid tumors more effectively.

LSTA1 has been granted orphan drug and fast track designations for pancreatic cancer in the United States and Europe as well as for glioblastoma multiforme in the United States.

The FDA defines rare pediatric diseases as those diseases with fewer than 200,000 cases in the United States that are serious or life threatening and primarily affect individuals under 18 years of age. A substantial benefit of a RPDD is receipt of a priority review voucher if the FDA approves the company’s new drug application (NDA). Vouchers can be used to compel a priority review of an additional new drug or biologic license application for another product or indication, reducing the standard review time of approximately ten months to six months. The voucher may be used by the sponsor or sold to another sponsor for their use. Priority review vouchers have sold for as much as $350 million and, more recently, have sold for $75 to $100 million.

Photo: Kristen Buck, executive vice president of R&D and chief medical officer of Lisata

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