Lisata Therapeutics gains U.S. FDA Orphan Drug Designation for osteosarcoma treatment

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to LSTA1, Lisata Therapeutics’ lead product candidate for the treatment of osteosarcoma, a rare cancer that can develop in children, adolescents and young adults.

LSTA1 recently received Rare Pediatric Disease Designation for osteosarcoma.

“We are thrilled to have received another favorable regulatory designation from the FDA. This underscores the significant unmet medical need and demand for better treatments for patients diagnosed with osteosarcoma,” said Kristen K. Buck, M.D., Executive Vice President of R&D and Chief Medical Officer of Lisata.

“Osteosarcoma, while rare, is a type of bone cancer that is often associated with high morbidity, early metastasis, rapid progression, and poor prognosis. Receiving an ODD from the FDA is an important milestone as we plan for future clinical expansion of LSTA1, and we believe it reflects the broad clinical utility of LSTA1 for the treatment of a wide array of solid tumors.”

Orphan Drug Designation is granted by the FDA to drugs or biologics intended to treat a rare disease or condition, defined as one that affects fewer than 200,000 people in the United States.

The designation can provide for a seven-year window of exclusive marketing rights post-approval as well as exemption from user fees and eligibility for tax credits for qualified clinical trials. In addition to the financial benefits, it also may potentially shorten clinical development due to closer collaboration with the FDA.

LSTA1 is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered (i.e., 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 resulting in tumors which are more susceptible to immunotherapies.

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