FDA news: Breast cancer, melanoma agents win fast track, orphan drug status
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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 fast track designation to CFI-402257 (Treadwell Therapeutics), a threonine kinase inhibitor, for treatment of adults with ER-positive/HER2-negative advanced breast cancer after disease progression on prior CDK 4/6 inhibitors and endocrine therapy, both as a monotherapy and in combination with fulvestrant.
2. The FDA granted orphan drug designation to LNS8801 (Linnaeus Therapeutics), an orally bioavailable and highly specific G protein-coupled estrogen receptor agonist, for treatment of patients with metastatic cutaneous melanoma.
3. The agency granted fast track designation to EVX-01 (Evaxion Biotech), in combination with pembrolizumab (Keytruda, Merck), for patients with metastatic melanoma. Evaxion received FDA approval in December to conduct a phase 2b clinical trial of EVX-01, a peptide-based cancer therapy.
4. The FDA granted fast track designation to tamibarotene (Syros Pharmaceuticals) for the treatment of high-risk myelodysplastic syndrome. Tamibarotene is an oral selective retinoic acid receptor alpha agonist.
5. The FDA granted rare pediatric disease designation to FLAG-003 (FLAG Therapeutics) for treatment of children with diffuse intrinsic pontine glioma. FLAG-003 — a novel multi-specific molecule therapeutic designated to target, bind and kill cancer cells — previously received orphan drug designation for treatment of all gliomas.
6. The agency granted orphan drug designation to BEA-17 (Beactica Therapeutics) for treatment of glioblastoma. BEA-17 is a small molecule targeted degrader of the epigenetic enzyme LSD1 and its co-factor CoREST.
7. The FDA granted orphan drug designation to ezurpimtrostat (GNS561, Genoscience Pharma) for treatment of hepatocellular carcinoma. Ezurpimtrostat is an autophagy inhibitor whose anticancer activity is linked to palmitoyl protein thioesterase-1 (PPT-1) inhibition.
References:
- Treadwell Therapeutics announces fast track designation granted by the FDA to CFI-402257 for the treatment of ER+/HER2- breast cancer (press release). Available at: treadwelltx.com/news/2023/treadwell-therapeutics-announces-fast-track-designation-granted-by-the-fda-to-cfi-402257-for-the-treatment-of-er-her2-breast-cancer/. Published Jan. 10, 2023. Accessed Feb. 1, 2023.
- Linnaeus Therapeutics granted orphan drug designation for LNS8801 for the treatment of patients with metastatic cutaneous melanoma (press release). Available at: linnaeustx.com/linnaeus-therapeutics-granted-orphan-drug-designation-for-lns8801-for-the-treatment-of-patients-with-metastatic-cutaneous-melanoma/. Published Jan. 19, 2023. Accessed Feb. 1, 2023.
- Evaxion receives FDA fast-track designation for personalized cancer immunotherapy (press release). Available at: investors.evaxion-biotech.com/news-releases/news-release-details/evaxion-receives-fda-fast-track-designation-personalized-cancer. Published Jan. 19, 2023. Accessed Feb. 1, 2023.
- Syros receives fast track designation from the FDA for tamibarotene for the treatment of higher-risk myelodysplastic syndrome (press release). Available at: ir.syros.com/press-releases/detail/272/syros-receives-fast-track-designation-from-the-fda-for. Published Jan. 26, 2023. Accessed Feb. 1, 2023.
- FLAG Therapeutics receives rare pediatric disease (RPD) designation for FLAG-003 for the treatment of children with diffuse intrinsic pontine glioma (DIPG) (press release). Available at: www.globenewswire.com/news-release/2023/01/31/2598877/0/en/FLAG-Therapeutics-Receives-Rare-Pediatric-Disease-RPD-Designation-for-FLAG-003-for-the-Treatment-of-Children-with-Diffuse-Intrinsic-Pontine-Glioma-DIPG.html. Published Jan. 31, 2023. Accessed Feb. 1, 2023.
- Beactica Therapeutics receives FDA orphan drug designation for BEA-17 for the treatment of glioblastoma (press release). Available at: www.beactica.com/press/beactica-therapeutics-receives-fda-orphan-drug-designation-for-bea-17-for-the-treatment-of-glioblast. Published Feb. 1, 2023. Accessed Feb. 1, 2023.
- Genoscience Pharma receives FDA orphan drug designation for ezurpimtrostat to treat hepatocellular carcinoma (HCC) (press release). Available at: www.genosciencepharma.com/2023/02/01/odd-obtained-by-genoscience-pharma-from-fda-to-treat-hcc/. Published Feb. 1, 2023. Accessed Feb. 1, 2023.
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