FDA news: Treatments for gastrointestinal, blood cancers receive orphan drug designations
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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 YB-200 (Ymmunobio), a CEACAM1/5 antibody that acts as a checkpoint inhibitor and has a direct immune agonistic effect on immune cells. The indication is for the treatment of hepatocellular carcinoma.
2. The FDA granted orphan drug designation to Temferon (Genenta Science) for the treatment of glioblastoma multiforme. Temferon is a proprietary cell therapy designed to reprogram the tumor microenvironment by delivering immunomodulatory molecules directly to tumors.
3. The FDA granted orphan drug designation to EP0042 (Ellipses Pharma) for the treatment of acute myeloid leukemia. EP0042 is a dual FLT-3 and Aurora kinase inhibitor.
4. The agency granted breakthrough therapy designation to ripretinib (Qinlock, Deciphera Pharmaceuticals) for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor. The indication is for patients harboring a KIT exon 11 mutation and co-occurring KIT exon 17 and/or 18 mutations.
5. The FDA granted orphan drug designation to ISB 1442 (Ichnos Sciences) for the treatment of relapsed/refractory multiple myeloma. ISB 1442 is a first-in-class biparatopic 2+1 BEAT bispecific antibody that targets CD38 and CD47.
6. The agency granted orphan drug designation to tirabrutinib (Ono Pharma USA) for the treatment of patients with primary central nervous system lymphoma. Tirabrutinib is a selective Bruton tyrosine kinase inhibitor that signals through the B-cell receptor to regulate cellular proliferation and activation.
References:
- FDA grants orphan drug designation to Ymmunobio’s CEACAM antibody for the treatment of Llver cancer (press release). Available at: www.ymmunobio.com/post/fda-grants-orphan-drug-designation-to-ymmunobio-s-ceacam-antibody-for-the-treatment-of-liver-cancer. Published Feb. 28, 2023. Accessed March 23, 2023.
- FDA grants orphan drug designation to Temferon for treatment of glioblastoma multiforme (press release). Available at: ir.genenta.com/news-releases/news-release-details/fda-grants-orphan-drug-designation-temferon-treatment. Published March 2, 2023. Accessed March 23, 2023.
- Ellipses Pharma: EP0042 receives orphan drug designation from the US Food and Drug Administration (press release). Available at: www.businesswire.com/news/home/20230314005101/en/Ellipses-Pharma-EP0042-Receives-Orphan-Drug-Designation-from-the-US-Food-and-Drug-Administration. Published March 14, 2023. Accessed March 23, 2023.
- Deciphera Pharmaceuticals announces QINLOCK included in NCCN guidelines for the treatment of second-line GIST patients and FDA grants breakthrough therapy designation for QINLOCK in second-line GIST patients with mutations in KIT Exon 11 and 17/18. Available at: investors.deciphera.com/news-releases/news-release-details/deciphera-pharmaceuticals-announces-qinlockr-included-nccn. Published March 14, 2023. Accessed March 23, 2023.
- Ichnos Sciences receives orphan drug designation for first-in-class bispecific [CD38 x CD47] antibody innate cell modulator, ISB 1442 (press release). Available at: ichnossciences.com/ichnos-sciences-receives-orphan-drug-designation-for-first-in-class-bispecific-cd38-x-cd47-antibody-innate-cell-modulator-isb-1442/#. Published March 21, 2023. Accessed March 23, 2023.
- Ono Pharma USA’s tirabrutinib receives orphan drug designation from the FDA for the treatment of primary central nervous system lymphoma (press release). Available at: www.prnewswire.com/news-releases/ono-pharma-usas-tirabrutinib-receives-orphan-drug-designation-from-the-fda-for-the-treatment-of-primary-central-nervous-system-lymphoma-301779681.html. Published March 23, 2023. Accessed March 23, 2023.
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