Intellia Therapeutics' NTLA-2001 to treat transthyretin amyloidosis granted orphan drug status by USFDA
Cambridge: Intellia Therapeutics, Inc., has recently announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis.
This investigational therapy is the first CRISPR therapy to be administered systemically to edit a disease-causing gene inside the human body. NTLA-2001 has the potential to be the first single-dose treatment for ATTR amyloidosis as it may be able to halt and reverse the devastating complications of this disease. ATTR amyloidosis is a rare condition that can impact a number of organs and tissues within the body through the accumulation of misfolded transthyretin (TTR) protein deposits.
"Orphan drug designation underscores the FDA's recognition of NTLA-2001's potential promise as a single-dose, novel therapy for the treatment of ATTR amyloidosis," said Intellia President and Chief Executive Officer John Leonard, M.D. "At Intellia, we are committed to advancing our modular genome editing platform to develop potentially curative treatment options for life-threatening diseases, and we look forward to working with the ATTR amyloidosis community and the FDA to bring a much-needed treatment option to patients."
NTLA-2001 is currently being studied in a Phase 1 trial in adults with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). In June 2021, Intellia and its collaborator Regeneron announced positive interim clinical results from the first two cohorts of this study. These results, which were published in the New England Journal of Medicine, represented the first-ever clinical data supporting the safety and efficacy of in vivo CRISPR genome editing in humans.
The FDA's Orphan Drug Designation program provides orphan status to drugs defined as those intended for the treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States. Orphan drug designation qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions and seven-year marketing exclusivity upon FDA approval. The decision by the FDA follows a March 2021 decision by the European Commission (EC) to also grant NTLA-2001 orphan drug designation for the treatment of ATTR amyloidosis.
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