FDA grants orphan drug designation to CRISPR-edited cell therapy for AML
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The FDA granted orphan drug designation to NTLA-5001 for treatment of adults with relapsed or refractory acute myeloid leukemia.
NTLA-5001 (Intellia Therapeutics) is an autologous T-cell receptor (TCR)-engineered T-cell therapy. The agent uses ex vivo CRISPR gene editing to insert a TCR on a patient’s T cells that targets the Wilms’ tumor 1 (WT1) antigen within and on the surface of certain cancer cells.
“The FDA’s decision to grant orphan drug designation for NTLA-5001 reflects the serious need for novel treatment options for people living with AML, a disease with notably poor long-term survival,” John Leonard, MD, president and CEO of Intellia, said in a company-issued press release.
“As part of our full-spectrum genome editing strategy, we seek to leverage our proprietary CRISPR/Cas9-based platform to engineer differentiated cell therapies targeting cancers for which there are currently limited or no treatment options,” Leonard added. “We look forward to advancing our investigational TCR T-cell therapy through the clinic in hopes of improving future treatment options for patients in need.”
Intellia is enrolling patients on a phase 1/phase 2A trial to evaluate the safety, tolerability and antitumor activity of a single dose of NTLA-5001 for adults with relapsed or refractory AML who previously received standard first-line therapy. The multicenter dose-expansion/dose-escalation study will include up to 54 patients.
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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