Product Approvals and Launches

Inebilizumab-cdon (Uplizna; Amgen) was approved as the first and only treatment for immunoglobulin G4–related disease (IgG4-RD), a chronic and debilitating immune-mediated inflammatory condition.

Astrasentan becomes the first and only endothelin A receptor antagonist to focus on proteinuria reduction in primary immunoglobulin A (IgA) nephropathy.

The topical ocular drug is an investigational new drug candidate that requires more well-controlled studies to establish efficacy, according to the FDA.

The approval follows the December 2024 priority review designation for this perioperative immunotherapy regimen for patients fighting muscle-invasive bladder cancer.

With strong progression-free survival benefits demonstrated in the CABINET trial and updates to National Comprehensive Cancer Network guidelines, this approval reinforces cabozantinib’s role in improving outcomes for patients facing these challenging cancers.

The approval of revakinagene taroretcel (Encelto; Neurotech) addresses a significant unmet need for patients with macular telangiectasia type 2 (MacTel), clinical investigator Charles C. Wykoff, MD, PhD, Retinal Consultants of Texas, said.

Charles C. Wykoff, MD, PhD, Retinal Consultants of Texas, discusses data supporting the FDA approval of revakinagene taroretcel-lwey (Encelto; Neurotech Pharmaceuticals), the first and only therapy indicated for the treatment of macular telangiectasia type 2 (MacTel).

Iptacopan's (Fabhalta; Novartis) approval makes it the first and only treatment approved for C3 glomerulopathy.

Despite resubmitting after an initial complete response letter (CRL) due to manufacturing issues, the combination therapy received a second CRL as a frontline therapy for unresectable hepatocellular carcinoma (HCC).

The FDA has approved guselkumab (Tremfya) as the first IL-23 inhibitor offering both subcutaneous and intravenous induction options for adults with moderate to severe active Crohn disease.

Christopher L. Moertel, MD, University of Minnesota, discusses the trial data that helped get mirdametinib approved and what providers can do to keep measuring quality of life (QOL) improvements.

Revakinagene taroretcel-lwey (Encelto; Neurotech), an allogeneic encapsulated cell-based gene therapy, is the first therapy to be approved for macular telangiectasia type 2.

Furosemide injection was previously approved to treat congestion from fluid buildup in adults with chronic heart failure.

The FDA approved Stoboclo and Osenvelt, denosumab biosimilars that could offer more affordable treatment options for osteoporosis and bone loss in various patient populations.

Tislelizumab plus chemotherapy received FDA approval as a frontline therapy for individuals with unresectable or metastatic esophageal squamous cell carcinoma (ESCC) whose tumors express PD-L1.

Several rare disease patient populations received their first-ever FDA-approved drug since Rare Disease Day last year, signifying progress in closing treatment gaps for rare disease.

The FDA approves BrainSense Adaptive deep brain stimulation and electrode identifier technology for the treatment of Parkinson disease.

With 2 new self-administration methods, patients living with generalized myasthenia gravis can be more empowered to manage their treatment with greater independence and control.

Merilog (insulin-aspart-szjj) is the first rapid-acting insulin biosimilar to Novolog (insulin aspart) that is designated to improve glycemic control in adults and adolescents with type 2 diabetes.

The FDA approved Amneal Pharmaceuticals' generic lenalidomide capsules, referencing Celgene's Revlimid, in multiple strengths.

The FDA approved Ospomyv and Xbryk, denosumab biosimilars that could expand access to treatment for osteoporosis, cancer-related bone loss, and other endocrine diseases.

The 5 mg risdiplam tablet provides the same efficacy and safety for spinal muscular atrophy as the currently available oral solution.

Adult and pediatric patients with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas that are not amenable to complete surgical resection are indicated in the approval.

Interim data from the ECHELON-3 trial previously showed that adding brentuximab vedotin to lenalidomide and rituximab improved overall survival among patients who have relapsed or refractory large B-cell lymphoma (R/R LBCL).

Jeffrey Stark, MD, vice president and head of medical immunology at UCB, discusses tailored dosing of bimekizumab-bkzx (Bimzelx) for various inflammatory conditions, highlighting its 5 FDA approvals and the remaining challenges.

Mikkael A. Sekeres, MD, Sylvester Comprehensive Cancer Center, discusses the potential benefits of treosulfan, a newly approved agent for allogeneic hematopoietic stem cell transplantation (allo-HSCT) conditioning in acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).

Mikkael A. Sekeres, MD, of Sylvester Comprehensive Cancer Center, University of Miami, discussed data supporting the FDA approval of treosulfan as part of conditioning for allogeneic hematopoeitic stem cell transplantation (allo-HSCT).

On October 14, 2024, the FDA approved a 320-mg single dose of bimekizumab-bkzx (Bimzelx; UCB) in a 2-mL prefilled syringe and autoinjector; both forms are now commercially available in the US.

The FDA has approved the first and only subcutaneous apomorphine infusion device, apomorphine hydrochloride injection (Onapgo), for the treatment of adults with advanced Parkinson disease.

The ranibizumab injection delivered through an implant device could be administered in a convenient schedule twice per year for the treatment of diabetic macular edema (DME).