Cell Therapy for AML Granted Fast Track Designation

This content originally appeared on our sister site, Pharmacy Times.

The FDA has granted fast track designation to Vor Biopharma’s VOR33, a hematopoietic stem cell therapeutic candidate to treat acute myeloid leukemia (AML).

VOR33 uses CRISPR genome-edited hematopoietic stem and progenitor cells engineered to lack CD33. The therapy is designed to protect patients’ healthy cells from anti-CD33 therapies, such as VCAR33 or gemtuzumab ozogamicin (Mylotarg). Vor intends VOR33 to replace standard of care transplants for patients with AML at high risk of relapse. The company hopes it will be able to seamlessly integrate into current transplant settings.

“Receiving fast track designation is an important milestone for Vor, which signals the FDA’s recognition of the serious and life-threatening medical condition of patients facing acute myeloid leukemia and the potential of VOR33 to address this unmet medical need,” Robert Pietrusko, PharmD, chief regulatory and quality officer at Vor, said in a statement.

VOR33 is Vor's lead program developed using their novel scientific platform, which they have designed to create next-generation, treatment-resistant transplants. Vor is investigating using its genome engineered hematopoietic stem cell platform in combination with multiple therapeutic modalities. A phase 1/2a clinical trial (NCT04849910) evaluating VOR33 is currently enrolling.

READ MORE: New Collaboration to Assess Stem Cell Therapy With Bispecific Antibody in AML

“We will continue to work closely with the FDA to expedite the development of VOR33, which is now actively enrolling in its Phase 1/2a clinical trial for AML patients who currently have limited treatment options,” Pietrusko added. “We continue to remain on-track to report VOR33’s initial clinical data in the first half of 2022.”

Vor designed VOR33 to protect an individual’s healthy cells from anti-CD33 therapies, such as VCAR33 or emtuzumab ozogamicin, and to replace standard care transplants for individuals with AML who are at risk for relapse.

Fast track designation expedites reviews of products that will help treat serious, and life-threating conditions and therapeutic candidates with fast track designation can be eligible for priority review.

REFERENCE

PureTech founded entity Vor Biopharma announces VOR33 granted U.S. Fast Track designation for Acute Myeloid Leukemia. Business Wire. News release. September 10, 2021. Accessed September 10, 2021. https://www.businesswire.com/news/home/20210909006184/en