FDA grants breakthrough designation to colorectal cancer agent, fast tracks HNSCC therapy
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The FDA announced several regulatory actions in the past week.
Here is an overview of decisions that may be relevant to your practice.
The FDA announced several regulatory actions in the past week.
1. The FDA granted breakthrough therapy designation to fam-trastuzumab deruxtecan-nxki (Enhertu; Daiichi Sankyo, AstraZeneca) — a HER2-directed antibody-drug conjugate — for treatment of two specific patient populations. One designation applies to treatment of adults with unresectable or metastatic HER2-expressing metastatic solid tumors that progressed after prior treatment. The other applies to patients with HER2-positive metastatic colorectal cancer who received at least two prior regimens.
2. The agency granted fast track designation to ALE.C04 (Alentis Therapeutics) — a monoclonal antibody designed to target claudin-1 (CLDN1) on cancer cells — for treatment of patients with recurrent or metastatic CLDN1-positive head and neck squamous cell carcinoma.
3. The FDA granted orphan drug designation to bexmarilimab (Faron Pharmaceuticals Ltd.) for the treatment of acute myeloid leukemia. Bexmarilimab is a humanized monoclonal antibody that binds to Clever-1, an immunosuppressive receptor found on macrophages that contributes to tumor growth and spread.
4. The agency granted orphan drug designation to the three key ingredients in INT230-6 (Intensity Therapeutics) for treatment of soft tissue sarcoma. The three active moieties are cisplatin, vinblastine sulfate and the diffusion enhancer SHAO-FA (8-((2-hydroxybenzoyl) amino)octanoate).
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:
- Enhertu granted two breakthrough therapy designations in U.S. for patients across multiple HER2 expressing cancers (press release). Available at: https://www.daiichisankyo.com/files/news/pressrelease/pdf/202308/20230831_E2.pdf. Published Aug. 31, 2023. Accessed Sept. 1, 2023.
- Alentis Therapeutics receives FDA fast track designation for ALE.C04 for the treatment of claudin-1 positive HNSCC (press release). Available at: https://alentis.ch/alentis-therapeutics-receives-fda-fast-track-designation-for-ale-c04-for-the-treatment-of-claudin-1-positive-hnscc/. Published Aug. 24, 2023. Accessed Sept. 3, 2023.
- Faron receives FDA orphan drug designation for bexmarilimab in acute myeloid leukemia (press release). Available at: https://otp.tools.investis.com/clients/uk/faron2/rns/regulatory-story.aspx?cid=2223&newsid=1711777. Published Aug. 29, 2023. Accessed Aug. 29, 2023.
- Intensity Therapeutics receives orphan drug designation for the three key ingredients in INT230-6 for the treatment of soft tissue sarcoma (press release). Available at: https://ir.intensitytherapeutics.com/news-events/press-releases/detail/72/intensity-therapeutics-receives-orphan-drug-designation-for. Published Sept. 7, 2023. Accessed Sept. 8, 2023.
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