FDA news: Fast track for glioblastoma drug, breakthrough designation for leukemia therapy
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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 accepted a biologics license application for denileukin diftitox (I/ONTAK, Citius Pharmaceuticals) for treatment of persistent or recurrent cutaneous T-cell lymphoma. Denileukin diftitox is an engineered IL-2-diphtheria toxin fusion protein. The FDA’s target action date is Sept. 28.
2. The FDA declined to approve a BLA for 131I-omburtamab (Omblastys, Y-mAbs Therapeutics) for treatment of central nervous system/leptomeningeal metastasis from neuroblastoma. The agent targets tumors that express B7-H3. A complete response letter included a recommendation for a meeting between the company and FDA to discuss “an adequate and well-controlled trial design” that could demonstrate sufficient evidence of effectiveness and a favorable risk-benefit profile, according to a Y-mAbs Therapeutics-issued press release.
3. The agency granted breakthrough therapy designation to revumenib (Syndax Pharmaceuticals) for treatment of adults and children with relapsed or refractory acute leukemia harboring a KMT2A rearrangement. Revumenib is an oral menin inhibitor.
4. The FDA granted fast track designation to WP1122 (Moleculin Biotech) for treatment of glioblastoma multiforme. WP1122 has been developed as a 2-DG prodrug to provide a more favorable pharmacological profile with a higher potency than 2-DG alone. The FDA previously granted orphan drug designation to WP1122.
5. The agency granted orphan drug designation to VAL-083 (Kintara Therapeutics) for treatment of diffuse intrinsic pontine glioma. VAL-083 is a small-molecule chemotherapeutic.
6. The FDA granted orphan drug designation to ilginatinib (NS-018, NS Pharma) for treatment of myelofibrosis. NS-018 is a highly selective and potent inhibitor of the Janus kinase 2 (JAK2) gene. JAK2 mutations are the most common in myelofibrosis.
7. The FDA granted breakthrough therapy designation to adagrasib (Krazati, Mirati Therapeutics) in combination with cetuximab (Erbitux, Eli Lilly) for treatment of patients with KRASG12C-mutated advanced colorectal cancer whose disease progressed after treatment with chemotherapy and an anti-VEGF therapy. Adagrasib is a RAS GTPase family inhibitor.
References:
- Citius Pharmaceuticals Inc. announces U.S. Food and Drug Administration acceptance of biologics license application of denileukin diftitox for the treatment of patients with persistent or recurrent cutaneous T-cell lymphoma. Available at: https://citiuspharma.com/investors/news-media/news/release-details/2022/Citius-Pharmaceuticals-Inc.-Announces-U.S.-Food-and-Drug-Administration-Acceptance-of-Biologics-License-Application-of-Denileukin-Diftitox-for-the-Treatment-of-Patients-with-Persistent-or-Recurrent-Cutaneous-T-Cell-Lymphoma/default.aspx. Published Dec. 1, 2022. Accessed Dec. 27, 2022.
- Y-mAbs announces complete response letter for omburtamab biologics license application. Available at: https://ir.ymabs.com/news-releases/news-release-details/y-mabs-announces-complete-response-letter-omburtamab-biologics. Published Dec. 1, 2022. Accessed Dec. 27, 2022.
- Syndax announces U.S. FDA breakthrough therapy designation granted for revumenib for the treatment of adult and pediatric patients with relapsed or refractory KMT2A-rearranged (MLLr) acute leukemia. Available at: https://ir.syndax.com/news-releases/news-release-details/syndax-announces-us-fda-breakthrough-therapy-designation-granted. Published Dec. 5, 2022. Accessed Dec. 27, 2022.
- Moleculin granted FDA fast track designation of WP1122 for the treatment of glioblastoma multiforme. Available at: https://moleculin.com/moleculin-granted-fda-fast-track-designation-of-wp1122-for-the-treatment-of-glioblastoma-multiforme/. Published on Dec. 7, 2022. Accessed on Dec. 27, 2022.
- Kintara Therapeutics receives orphan drug designation for VAL-083 for treatment of diffuse intrinsic pontine glioma (DIPG). Available at: https://www.kintara.com/news-media/press-releases/detail/1011/kintara-therapeutics-receives-orphan-drug-designation-for. Published on Dec. 15, 2022. Accessed on Dec. 27, 2022.
- NS Pharma announces receipt of orphan drug designation from the U.S. FDA for NS-018, an investigational treatment for myelofibrosis. Available at: https://www.nspharma.com/pdfs/NS-018_Orphan_Drug_Designation_Press_Release_final.pdf. Published on Dec. 20, 2022. Accessed on Dec. 27, 2022.
- Mirati announces adagrasib (KRAZATI) receives breakthrough therapy designation from FDA for patients with advanced, KRAS-mutated colorectal cancer and NEJM publishes phase 1b/2 data from adagrasib with or without cetuximab in colorectal cancer. Available at: https://ir.mirati.com/press-releases/press-release-details/2022/Mirati-announces-Adagrasib-KRAZATI-Receives-Breakthrough-Therapy-Designation-from-FDA-for-Patients-with-Advanced-KRAS-Mutated-Colorectal-Cancer-and-NEJM-Publishes-Phase-1b2-Data-from-Adagrasib-With-or-Without-Cetuximab-in-Colorectal-Cancer/default.aspx. Published on Dec. 21, 2022. Accessed on Dec. 27, 2022.
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