FDA Grants Orphan Drug Designation to Treatment of Malignant Glioma

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The investigational drug was developed to activate a novel uptake pathway, allowing antineoplastic drugs to combat solid tumors more successfully.

In a press release on Aug. 8, 2023, Lisata Therapeutics announced FDA’s Orphan Drug Designation had been granted to LSTA1 for the treatment of malignant glioma. Lisata Therapeutics is a clinical-stage pharmaceutical company developing therapies for advanced solid tumors and other major diseases. The company created LSTA1 to activate a new uptake pathway, allowing co-administered or tethered antineoplastic drugs to combat solid tumors more successfully. The drug can also modify the tumor microenvironment to make tumors more vulnerable to immunotherapies.

LSTA1 is currently undergoing multiple global studies in a range of solid tumor types in combination with a range of antineoplastic regimens. In the near future, Lisata plans to collaborate with researchers from the University of Tartu in Estonia to conduct a clinical study evaluating LSTA1 in previously untreated glioblastoma multiform (GBM). The company plans to study the impact of LSTA1 when added to standard-of-care (SoC) to treat GBM. The study will be conducted at multiple sites in Estonia and Latvia, with an estimate of 30 patients (with a randomization of 2:1 LSTA1 + SoC vs placebo + SoC). Lisata expects the first patient will be treated in the fourth quarter of 2023.

“Malignant glioma is one of the most aggressive and deadly malignancies,” said Kristen K. Buck, M.D., executive vice president of R&D and chief medical officer of Lisata, in a company press release. “This Orphan Drug Designation acknowledges the high unmet medical need of this patient population as well as the potential of LSTA1 to benefit patients in this setting.”


The Orphan Drug Designation is given by FDA to drugs or biologics designed to treat a rare disease or condition that affects fewer than 200,000 individuals in the United States. These designations create fiscal incentives for clinical development and a seven-year period of US market exclusivity if FDA approval is met. A large amount of non-clinical data suggests that LSTA1 enhances a range of antineoplastic therapies; LSTA1 also showed positive safety, tolerability, and activity in clinical trials enhancing standard-of-care chemotherapy for pancreatic cancer. Lisata anticipates LSTA1 will assist with a range of treatments to treat solid tumors more effectively.

Source: Lisata.com