FDA grants orphan drug designation to Intellia's NTLA-2001

By The Science Advisory Board staff writers

October 21, 2021 -- The U.S. Food and Drug Administration has granted an orphan drug designation to Intellia Therapeutics' NTLA-2001 therapy for the treatment of transthyretin (ATTR) amyloidosis.

ATTR amyloidosis is a rare condition that can affect several organs and tissues within the body through the accumulation of misfolded transthyretin protein deposits. NTLA-2001 is the first CRISPR therapy to be administered systemically to edit a disease-causing gene inside the human body, Intellia said.

In June 2021, Intellia and its collaborator Regeneron released positive interim clinical results from the first two cohorts of a phase I study of MTLA-2001 in adults with hereditary ATTR amyloidosis with polyneuropathy. The results were the first from a clinical trial evaluating the safety and efficacy of CRISPR genome editing in humans, the company said.

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