Intellia Gets FDA Orphan Drug Designation for Acute Myeloid Leukemia Treatment

 Intellia Therapeutics Inc. said the U.S. Food and Drug Administration has granted its acute myeloid leukemia treatment an orphan drug designation.

NTLA-5001 is an autologous T-cell receptor therapy that targets the Wilms' Tumor antigen, which is a common expression in acute myeloid leukemia and other hematologic and solid tumors.

The treatment is currently being reviewed in a Phase 1/2a study in adults who have a detectable sign of the disease and have received standard first-line treatment, the clinical-stage genome editing company said.

The FDA's designation is granted to drugs that are intended for the treatment, diagnosis or prevention of rare diseases that can affect fewer than 200,000 people in the U.S. Drugs that receive the designation can qualify companies for certain tax credits, prescription drug user-fee exemptions and a seven-year marketing exclusivity once the drug is approved by the FDA.

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