FDA grants fast track designation to CD33-directed CAR-T for acute myeloid leukemia
The FDA granted fast track designation to PRGN-3006 for the treatment of adults with relapsed or refractory acute myeloid leukemia.
PRGN-3006 (Precigen Inc.) is an autologous, gene-edited chimeric antigen receptor T-cell therapy that targets the CD33 protein on the surface of cancer cells.
The investigational therapy — designed to reduce manufacturing time down to 1 day — is based on Precigen’s UltraCAR-T therapeutic platform.
The therapy uses the nonviral Sleeping Beauty gene-editing system to concurrently express the CD33-targeted CAR, membrane-bound interleukin-15, and a therapeutic “kill switch” that conditionally eliminates CAR T cells for enhanced safety.
The FDA's fast track designation helps to expedite development, review and potential approval of treatments for serious or life-threatening diseases. The agency previously granted the agent orphan drug
designation for the treatment of adults with relapsed or refractory AML.
“AML is a rapidly progressing disease with a very poor prognosis,” Helen Sabzevari, PhD, president and CEO of Precigen, said in a company-issued press release. “The fast-track designation will help facilitate the timely development of this program, and we look forward to working more closely with the FDA to potentially bring this new and highly differentiated overnight UltraCAR-T therapy to patients.”
PRGN-3006 is being evaluated as part of a single-center phase 1/phase 1B dose-escalation/dose-expansion study to determine the safety and recommended phase 2 dose for adults with relapsed or refractory AML, high-risk myelodysplastic syndrome or chronic myelomonocytic leukemia. Study participants will receive a single IV dose of the therapy with or without prior lymphodepletion.
Interim results from the study presented at last year’s ASH Annual Meeting & Exposition showed a 50% overall response rate among the first six patients in the lymphodepletion cohort. Safety results showed no dose-limiting toxicities. One of 15 patients experienced grade 3 cytokine release syndrome.
https://www.healio.com/news/hematology-oncology/20220405/fda-grants-fast-track-designation-to-cd33directed-cart-for-acute-myeloid-leukemia
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