FDA news: Treatments for pancreatic cancer, Ewing sarcoma 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 agency granted orphan drug designation to osemitamab (TST001, Transcenta), a high affinity humanized anti-CLDN18.2 monoclonal antibody, for the treatment of patients with pancreatic cancer. The agent had previously been granted orphan drug designation for patients with gastric cancer and gastroesophageal junction cancer.
2. The FDA granted fast track designation to RRx-001 (EpicentRx) for the prevention or attenuation of severe oral mucositis in patients with head and neck cancers treated with chemotherapy and radiation. RRx-001 is a direct NLRP3 inhibitor and Nrf2 upregulator with anti-inflammatory and antioxidant properties.
3. The agency granted fast track designation to SynKIR-110 (Verismo Therapeutics) for treatment of patients with mesothelioma. The agent is an investigational drug for the treatment of mesothelin-expressing mesothelioma, cholangiocarcinoma and ovarian cancer.
4. The FDA granted a biologics license application to HLX02 (Accord BioPharma) — a proposed trastuzumab (Herceptin, Genentech) biosimilar — for the adjuvant treatment of HER2-overexpressing breast cancer, metastatic breast cancer and metastatic gastric or gastroesophageal junction adenocarcinoma.
5. The agency granted fast track designation to CB-011 (Caribou Biosciences) for the treatment of patients with relapsed or refractory multiple myeloma. The allogeneic anti-BCMA chimeric antigen receptor T-cell therapy is currently being evaluated in the ongoing phase 1 CaMMouflage trial.
6. The FDA granted orphan drug designation to TP-1287 (Sumitomo Pharma Oncology) for the treatment of patients with Ewing sarcoma. TP-1287 is an investigational oral CDK9 inhibitor.
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.
FDA grants fast track designation to facilitate development and expedite review of drugs that fill an unmet medical need in a serious condition, thus enabling the agents to reach patients sooner.
References:
- Transcenta’s osemitamab (TST001) targeting Claudin18.2 granted orphan drug designation for treatment of pancreatic cancer (press release). Available at: www.transcenta.com/newsDet/id-121. Published March 29, 2023. Accessed April 12, 2023.
- EpicentRx receives fast track designation from the U.S. FDA for lead asset, RRx-001, to prevent/attenuate chemotherapy and radiation treatment induced severe oral mucositis (press release). Available at: www.epicentrx.com/2023/epicentrx-receives-fast-track-designation-from-the-u-s-fda-for-lead-asset-rrx-001-to-prevent-attenuate-chemotherapy-and-radiation-treatment-induced-severe-oral-mucositis/. Published March 29, 2023. Accessed April 12, 2023.
- Verismo Therapeutics secures fast drack designation from the U.S. Food and Drug Administration (FDA) for SynKIR-110 (press release). Available at: www.prnewswire.com/news-releases/verismo-therapeutics-secures-fast-track-designation-from-the-us-food-and-drug-administration-fda-for-synkir-110-301789884.html. Published April 5, 2023. Accessed April 12, 2023.
- Accord BioPharma announces U.S. FDA acceptance of biologics license application for proposed biosimilar trastuzumab HLX02 (press release). Available at: www.prnewswire.com/news-releases/accord-biopharma-announces-us-fda-acceptance-of-biologics-license-application-for-proposed-biosimilar-trastuzumab-hlx02-301790459.html?tc=eml_cleartime. Published April 5, 2023. Accessed April 12, 2023.
- Caribou Biosciences announces FDA granted fast track designation to CB-011, an allogeneic CAR-T cell therapy for relapsed or refractory multiple myeloma (press release). Available at: investor.cariboubio.com/news-releases/news-release-details/caribou-biosciences-announces-fda-granted-fast-track-designation. Published April 4, 2023. Accessed April 12, 2023.
- Sumitomo Pharma Oncology receives orphan drug designation for TP-1287, an investigational oral CDK9 inhibitor for the treatment of Ewing sarcoma (press release). Available at: oncology.sumitomo-pharma.com/news-media/20230410/. Published April 10, 2023. Accessed April 12, 2023.
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