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Monthly News Roundup - December 2023

Medically reviewed by Leigh Ann Anderson, PharmD. Last updated on Dec 30, 2023.

Landmark FDA Approval for Casgevy, a CRISPR-Based Therapy for Sickle Cell Disease

This past month the U.S. Food and Drug Administration (FDA) cleared Casgevy (exagamglogene autotemcel), the first approved CRISPR/Cas9 genome-edited cell therapy for the treatment of sickle cell disease (SCD) in patients 12 years and older with frequent vaso-occlusive crises (VOCs). Casgevy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, has been shown to reduce or eliminate vaso-occlusive crises for patients with SCD.

Lyfgenia Approved to Treat Patients with Sickle Cell Disease and a History of Vaso-Occlusive Events

The FDA has approved Bluebird Bio’s Lyfgenia (lovotibeglogene autotemcel), an autologous hematopoietic stem cell-based gene therapy for the treatment of patients 12 years of age or older with sickle cell disease (SCD) and a history of vaso-occlusive crises (VOCs). Lyfgenia is produced individually for each patient using their own blood stem cells. It’s infused back into the patient as part of an autologous hematopoietic stem cell transplant (HSCT) after myeloablative busulfan conditioning.

FDA Grants Approval to Avzivi (bevacizumab-tnjn), the Fifth Biosimilar to Avastin

This past month the FDA approved Avzivi (bevacizumab-tnjn), a vascular endothelial growth factor inhibitor biosimilar to Avastin used for the treatment of colorectal cancer, non-small cell lung cancer, glioblastoma, renal cell carcinoma, cervical cancer, and epithelial ovarian, fallopian tube, or primary peritoneal cancer. Avzivi is from Bio-Thera Solutions, Ltd.

Novartis’ Fabhalta OK’d for Adults with Paroxysmal Nocturnal Hemoglobinuria (PNH)

In December, Fabhalta (iptacopan) received FDA approval as the first oral monotherapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). Fabhalta is a first-in-class, complement factor B inhibitor and works by binding to Factor B of the alternative complement pathway. This prevents destruction of red blood cells (RBCs) within and outside the blood vessels.

Wainua Autoinjector Cleared to Treat Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis

In December the FDA cleared Wainua (eplontersen) from Ionis Pharmaceuticals and AstraZeneca. Wainua is a transthyretin-directed antisense oligonucleotide indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN) in adults. It is the only approved medication for ATTRv-PN that can be self-administered using an auto-injector.

FDA Approves Iwilfin as Oral Maintenance Therapy for High-Risk Neuroblastoma

Iwilfin (eflornithine) from US WorldMeds has been FDA-approved to reduce the risk of relapse in adults and children with high-risk neuroblastoma (HRNB). It is indicated in patients who have demonstrated at least a partial response to prior therapies, including anti-GD2 immunotherapy.

FDA Approves Long-Duration iDose TR Implant to Treat Glaucoma

iDose TR (travoprost intracameral implant), from Glaukos Corporation is now approved for the reduction of intraocular pressure (IOP) in patients with open-angle glaucoma or ocular hypertension. iDose TR continuously delivers therapeutic levels of a proprietary formulation of travoprost inside the eye for extended periods of time.

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