FDA News

FDA grants orphan drug designation to PRGN-3006 for AML

The FDA granted orphan drug designation to PRGN-3006 for the treatment of relapsed or refractory acute myeloid leukemia, according to the agent’s manufacturer.

PRGN-3006 (Precigen Inc.) uses Precigen’s UltraCAR-T therapeutic platform, which is designed to eliminate ex vivo expansion, reduce manufacturing time and allow for the administration of chimeric antigen receptor T-cell therapy to patients 1 day after nonviral gene transfer.

The multigenic CAR T-cell therapy co-expresses a CAR, membrane-bound interleukin-15, and a kill switch intended to provide greater precision and control for targeting relapsed or refractory AML and higher-risk myelodysplastic syndrome.

A trial is underway to evaluate the therapy for relapsed or refractory AML, and Precigen completed enrollment of the first cohort in the summer. Initial data are expected to be available in the second half of this year.

“This regulatory designation underscores the critical medical need for new therapies to treat [patients with AML],” Helen Sabzevari, PhD, president and CEO of Precigen, said in a company-issued press release. “As the first regulatory designation for our proprietary UltraCAR-T platform, this orphan drug designation helps to advance the PRGN-3006 investigational therapy and provides important incentives and support to deliver this medicine as rapidly as possible for those patients suffering from this disease.”

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.


The FDA granted orphan drug designation to PRGN-3006 for the treatment of relapsed or refractory acute myeloid leukemia, according to the agent’s manufacturer.

PRGN-3006 (Precigen Inc.) uses Precigen’s UltraCAR-T therapeutic platform, which is designed to eliminate ex vivo expansion, reduce manufacturing time and allow for the administration of chimeric antigen receptor T-cell therapy to patients 1 day after nonviral gene transfer.

The multigenic CAR T-cell therapy co-expresses a CAR, membrane-bound interleukin-15, and a kill switch intended to provide greater precision and control for targeting relapsed or refractory AML and higher-risk myelodysplastic syndrome.

A trial is underway to evaluate the therapy for relapsed or refractory AML, and Precigen completed enrollment of the first cohort in the summer. Initial data are expected to be available in the second half of this year.

“This regulatory designation underscores the critical medical need for new therapies to treat [patients with AML],” Helen Sabzevari, PhD, president and CEO of Precigen, said in a company-issued press release. “As the first regulatory designation for our proprietary UltraCAR-T platform, this orphan drug designation helps to advance the PRGN-3006 investigational therapy and provides important incentives and support to deliver this medicine as rapidly as possible for those patients suffering from this disease.”

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.


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