The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) 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 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,” stated Kristen K. Buck, M.D., 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.”
The FDA defines rare pediatric diseases as rare diseases (those 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, often referred to as a “golden ticket,” once the FDA approves the new drug application (NDA) for the product and indication having received the RPDD. Vouchers are especially valuable as they can be used to compel a priority review of an additional NDA 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 USD historically and, more recently, have sold for $75 to $100 million USD.
