FDA grants orphan drug designation to gamma-delta T-cell therapy for AML
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The FDA cleared an investigational new drug application for GDX012, a gamma-delta T-cell therapy, for patients with previously treated hematologic malignancies.
The novel cell therapy also received FDA orphan drug designation for patients with previously treated acute myeloid leukemia, according to the manufacturer.
GDX012 (GammaDelta Therapeutics) is an allogeneic, nongene-edited variable delta 1 gamma-delta T-cell therapy. The blood-based therapy comprises gamma-delta T cells that are “purified from blood samples and grown in optimized culture conditions that result in a highly enriched [variable delta 1-positive] T-cell population” for therapeutic use, according to the manufacturer’s website.
GammaDelta’s T-cell platform is being developed as part of a collaborative agreement with Takeda that the two companies formed in 2017.
“Although progress has been made in the treatment of AML, the median overall 5-year survival rate for patients diagnosed with AML remains under 30%,” Michael Koslowski, MD, head of R&D and chief medical officer of GammaDelta Therapeutics, said in a company-issued press release.
“With the development of GDX012 we are aiming to change the treatment paradigm for AML and potentially other hematologic malignancies,” he added. “The unique biological characteristics of [delta 1 gamma-delta] T cells offer a first-in-class [delta 1 gamma-delta] T-cell therapy for AML, where the development of cell therapies has been historically limited due to the lack of specific targets.”
The IND clearance allows GammaDelta to start enrollment in a phase 1, U.S.-based multicenter trial to evaluate the safety, tolerability and antitumor activity of GDX012. The trial will include patients with measurable residual disease-positive AML and is expected to begin later this year, according to the manufacturer.
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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