FDA approves diagnostic for GI stromal tumors, seeks more data for GVHD 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 FDA approved the therascreen PDGFRA RGQ PCR kit (Qiagen) as a companion diagnostic to identify patients with gastrointestinal stromal tumors who may be eligible for treatment with avapritinib (Ayvakit, Blueprint Medicines). The kit is the first platelet-derived growth factor receptor alpha (PDGFRA) assay to receive FDA approval as a companion diagnostic, according to a Qiagen press release.
2. The FDA issued a complete response letter to Mesoblast related to its biologics license application seeking approval of remestemcel-L for treatment of children with steroid-refractory acute graft-versus-host disease. The allogeneic cell therapy — comprised of culture-expanded mesenchymal stem cells derived from the bone marrow of an unrelated donor — is administered to patients in a series of IV infusions. The agency indicated it requires more data to support marketing approval.
3. The agency issued a complete response letter to a new drug application that sought approval of avasopasem manganese (Galera Therapeutics) for treatment of radiotherapy-induced severe oral mucositis among patients receiving standard treatment for head and neck cancer. The agency called for an additional clinical trial, contending results of two trials did not sufficiently establish substantial evidence of avasopasem's safety and effectiveness for reducing severe oral mucositis in this setting. Galera Therapeutics officials intend to request a meeting with FDA representatives to learn more about the rationale for the agency's decision and to discuss next steps.
4. The agency granted orphan drug designation to ABM-1310 (ABM Therapeutics) — a small molecule BRAF inhibitor — for treatment of patients with glioblastoma who harbor BRAF V600 mutations.
5. The FDA granted orphan drug designation to LSTA1 (Lisata Therapeutics) for treatment of malignant glioma. The investigational drug is designed to activate a novel uptake pathway that allows co-administered or tethered anticancer drugs to penetrate solid tumors more effectively, according to a Lisata Therapeutics press release.
6. The agency granted orphan drug designation to quaratusugene ozeplasmid (Reqorsa Immunogene Therapy, Genprex Inc.) for the treatment of small cell lung cancer. Quaratusugene ozeplasmid is a pan-kinase inhibitor that has demonstrated potential to inhibit the EGFR and AKT kinase pathways. The active ingredient is the TUSC2 gene.
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:
- Qiagen receives FDA approval for companion diagnostic to Blueprint Medicines’ Ayvakit (avapritinib) in gastrointestinal stromal tumors (press release). Available at: https://corporate.qiagen.com/English/newsroom/press-releases/press-release-details/2023/QIAGEN-receives-FDA-approval-for-companion-diagnostic-to-Blueprint-Medicines-AYVAKIT-avapritinib-in-gastrointestinal-stromal-tumors/default.aspx. Published Aug. 7, 2023. Accessed Aug. 7, 2023.
- Mesoblast receives complete response from U.S. Food and Drug Administration for biologics license application for steroid-refractory acute graft versus host disease in children. Available at: https://investorsmedia.mesoblast.com/static-files/422cd6da-a0b9-49cf-a177-7fd106f111f2. Published Aug. 4, 2023. Accessed Aug. 7, 2023.
- Galera receives complete response letter from U.S. FDA for avasopasem manganese (press release). Available at: https://investors.galeratx.com/news-releases/news-release-details/galera-receives-complete-response-letter-us-fda-avasopasem. Published Aug. 9, 2023. Accessed Aug. 10, 2023.
- U.S. FDA grants orphan drug designation to ABM-1310 for the treatment of patients with glioblastoma harboring BRAF V600 mutation (press release). Available at: http://www.abmtx.com/site/newsdetails/85. Published Aug. 2, 2023. Accessed Aug. 7, 2023.
- Lisata Therapeutics announces U.S. FDA orphan drug designation granted to LSTA1 for the treatment of malignant glioma (press release). Available at: https://ir.lisata.com/news-releases/news-release-details/lisata-therapeutics-announces-us-fda-orphan-drug-designation. Published Aug. 8, 2023. Accessed Aug. 8, 2023.
- Genprex granted FDA orphan drug designation for REQORSA Immunogene Therapy for the treatment of small cell lung cancer (press release). Available at: https://www.genprex.com/news/genprex-granted-fda-orphan-drug-designation-odd-for-reqorsa-immunogene-therapy-for-the-treatment-of-small-cell-lung-cancer/. Published Aug. 10, 2023. Accessed Aug. 10, 2023.
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