FDA news: Multiple myeloma, pancreatic cancer therapies receive orphan drug designation
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The FDA announced several regulatory actions the past few weeks.
Here is an overview of decisions that may be relevant to your practice.
1. The FDA issued a ”safe to proceed” for an investigational new drug application for NY-ESO-1 TCR/IL-15 NK (Syena) — an engineered cell therapy comprised of cord blood-derived natural killer cells — for treatment of advanced synovial sarcoma and myxoid/round cell liposarcoma.
2. The agency granted orphan drug designation to inobrodib (CellCentric) for treatment of multiple myeloma. Inobrodib binds to a specific part of proteins p300 and bromodomain to reduce the expression of key cancer drivers.
3. The FDA granted breakthrough therapy designation to zenocutuzumab (Zeno, Merus) for treatment of certain patients with pancreatic cancer. The antibody-dependent cell-mediated cytotoxicity-enhanced bispecific antibody utilizes the Merus Dock & Block mechanism to inhibit a pathway in solid tumors with NRG1 fusions.
4. The agency granted orphan drug designation to padeliporfin vascular targeted photodynamic therapy (ImPact Biotech) — a minimally invasive oncology platform for the treatment of solid tumors — for treatment of locally advanced pancreatic cancer.
5. The FDA granted priority review to zolbetuximab (Astellas) — an investigational claudin 18.2-targeted monoclonal antibody — for treatment of certain patients with locally advanced resectable or metastatic HER2-negative gastric or gastroesophageal junction adenocarcinoma.
6. The FDA removed the partial clinical hold on the TakeAim Leukemia phase 1/phase 2 study of emavusertib (Curis), a triple target inhibitor, at a recommended phase 2 dose of 300 mg twice a day as monotherapy in patients with acute myelogenous leukemia or myelodysplastic syndromes.
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.
References:
- Replay and MD Anderson announce FDA clearance of IND application for first-in-class T-Cell Receptor Natural Killer (TCR-NK) cell therapy for sarcoma (press release). Available at: http://replay.bio/news/replay-and-md-anderson-announce-fda-clearance-of-ind-application. Published June 20, 2023. Accessed July 6, 2023.
- CellCentric granted FDA orphan drug designation for inobrodib (CCS1477) for the treatment of multiple myeloma (press release). Available at: https://www.cellcentric.com/press-release/cellcentric-granted-fda-orphan-drug-designation-for-inobrodib-ccs1477-for-the-treatment-of-multiple-myeloma/. Published June 29, 2023. Accessed July 6, 2023.
- Zenocutuzumab (Zeno) granted breakthrough therapy designation by the U.S. Food & Drug Administration for the treatment of NRG1+ pancreatic cancer (press release). Available at: https://ir.merus.nl/news-releases/news-release-details/zenocutuzumab-zeno-granted-breakthrough-therapy-designation-us. Published June 29, 2023. Accessed July 6, 2023.
- ImPact Biotech receives FDA orphan drug designation for padeliporfin VTP in pancreatic cancer (press release). Available at: https://www.biospace.com/article/impact-biotech-receives-fda-orphan-drug-designation-for-padeliporfin-vtp-in-pancreatic-cancer/. Published July 5, 2023. Accessed July 6, 2023.
- Astellas announces U.S. FDA grants priority review for zolbetuximab biologics license application (press release). Available at: https://www.astellas.com/en/news/27946. Published July 6, 2023. Accessed July 6, 2023.
- FDA removes partial clinical hold on TakeAim Leukemia study RP2D established at 300 mg BID (press release). Available at: https://investors.curis.com/2023-07-06-FDA-Removes-Partial-Clinical-Hold-on-TakeAim-Leukemia-Study-RP2D-Established-at-300-mg-BID. Published July 6, 2023. Accessed July 6, 2023.
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