FDA news: Breast cancer, sickle cell disease therapies receive priority review
Click Here to Manage Email Alerts
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 AVB-001 (Avenge Bio Inc.) — an encapsulated cell product engineered to produce native human interleukin-2 — for treatment of mesothelioma.
2. The FDA granted full approval to blinatumomab (Blincyto, Amgen) for treatment of adults or children with CD19-positive B-cell precursor acute lymphoblastic leukemia in first or second complete remission with minimal residual disease of at least 0.1%. Blinatumomab — a bispecific T-cell engager molecule — previously received accelerated approval for this indication.
3. ERAS-801 (Erasca Inc.) — a central nervous system-penetrant EGFR inhibitor — received orphan drug designation for treatment of malignant glioma.
4. The FDA granted priority review to lovotibeglogene autotemcel (Lovo-cel, bluebird bio) — an investigational one-time gene therapy — for treatment of patients aged 12 years or older with sickle cell disease and a history of vaso-occlusive events.
5. The agency approved Foundation One Liquid CDx (FoundationOne) as a companion diagnostic to identify adults with metastatic colorectal cancer who have BRAF V6ooE alterations and may benefit from treatment with encorafenib (Braftovi, Pfizer) plus cetuximab (Erbitux, Eli Lilly & Co.).
6. The FDA granted priority review to capivasertib (AZD5363, AstraZeneca) for use with fulvestrant (Faslodex, AstraZeneca) for treatment of adults with hormone receptor-positive, HER2-negative locally advanced or metastatic breast cancer whose disease progressed or recurred on or after an endocrine-based regimen.
7. The FDA approved Pemrydi RTU (Amneal Pharmaceuticals), a ready-to-use oncology injectable that does not require reconstitution, dilution or refrigeration. The agent is approved for use in combination with pembrolizumab (Keytruda, Merck) and platinum chemotherapy for initial treatment of patients with metastatic nonsquamous non-small cell lung cancer with no EGFR or ALK tumor aberrations, or in combination with cisplatin for patients with malignant pleural mesothelioma.
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:
- Avenge Bio receives FDA orphan drug designation for AVB-001 for the treatment of mesothelioma (press release). Available at: https://avengebio.com/wp-content/uploads/2023/06/Avenge-Bio-Receives-FDA-Orphan-Drug-Designation-for-AVB-001-for-the-Treatment-of-Mesothelioma.pdf. Published June 15, 2023. Accessed June 29, 2023.
- FDA grants full approval for Blincyto (blinatumomab) to treat minimal residual disease-positive B-cell precursor acute lymphoblastic leukemia (press release). Available at: https://www.amgen.com/newsroom/press-releases/2023/06/fda-grants-full-approval-for-blincyto-blinatumomab-to-treat-minimal-residual-diseasepositive-bcell-precursor-acute-lymphoblastic-leukemia. Published June 21, 2023. Accessed June 21, 2023.
- Erasca granted FDA orphan drug designation for CNS-penetrant EGFR inhibitor ERAS-801 for the treatment of malignant glioma (press release). Available at: https://investors.erasca.com/news-releases/news-release-details/erasca-granted-fda-orphan-drug-designation-cns-penetrant-egfr. Published June 22, 2023. Accessed June 23, 2023.
- Bluebird bio announces FDA priority review of the biologics license application for lovotibeglogene autotemcel (love-cel) for patients with sickle cell disease (SCD) 12 years and older with a history of vaso-occlusive events (press release). Available at: https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-announces-fda-priority-review-biologics-license-1. Published June 21, 2023. Accessed June 27, 2023.
- U.S. Food and Drug Administration (FDA) approves FoundationOne LiquidCDx as a companion diagnostic for Pfizer’s Braftovi (encorafenib) in combination with cetuximab to identify patients with BRAF V600E alterations in metastatic colorectal cancer (press release). Available at: https://www.foundationmedicine.com/press-releases/f9b285eb-db6d-4f61-856c-3f1edb803937. Published June 9, 2023. Accessed June 27, 2023.
- Capivasertib in combination with Faslodex granted priority review in the US for patients with advanced HR-positive breast cancer (press release). Available at: https://www.astrazeneca.com/media-centre/press-releases/2023/capivasertib-in-combination-with-faslodex-granted-priority-review-in-the-us.html. Published June 12, 2023. Accessed June 27, 2023.
- Amneal receives 505(b)(2) NDA approval from FDA for Pemrydi RTU, a ready-to-use oncology injectable (press release). Available at: https://investors.amneal.com/news/press-releases/press-release-details/2023/Amneal-Receives-505b2-NDA-Approval-from-FDA-for-PEMRYDI-RTU-a-Ready-to-Use-Oncology-Injectable/default.aspx. Published June 14, 2023. Accessed June 27, 2023.
Collapse
Press Release
Click Here to Manage Email Alerts