Product Approvals and Launches

Gerard Criner, MD, FACP, FACCP, MATINEE investigator, highlights trial results that showed reduced exacerbations and delayed disease progression in patients with eosinophilic chronic obstructive pulmonary disease (COPD).

Once-daily roflumilast provides skin clearance and rapid itch relief with no limitation on duration of use.

FDA approves mepolizumab (Nucala; GSK) as the first monthly biologic for chronic obstructive pulmonary disease (COPD), significantly reducing exacerbations in patients with an eosinophilic phenotype.

The FDA previously approved Jivi in 2018 to treat adults aged 12 years and older with hemophilia A.

The FDA approved retifanlimab (Zynyz; Incyte) as the first and only first-line treatment for advanced anal cancer, supported by data from the POD1UM-303/InterAACT2 and POD1UM-202 trials.

The approval marks the second international approval for rozanolixizumab (Rystiggo; UCB Pharma) for generalized myasthenia gravis (gMG), behind the February EU approval of 2 self-administration approvals for the neonatal Fc receptor monoclonal antibody: an infusion pump and manual push with a syringe.

Telisotuzumab vedotin (Teliso-V) gained FDA approval as a targeted therapy for patients with advanced non–small cell lung cancer (NSCLC) with high c-Met overexpression, offering new hope.

"The newer medications that are coming out, and a lot of them are approved, are much more focused on the problem," explains Jonathan Strober, MD, pediatric neurologist with UCSF Benioff Children's Hospital.

FDA approves the first treatment for KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC), offering new hope for patients with this condition.

Nipocalimab (Imaavy; Johnson & Johnson), an FcRn-blocking monoclonal antibody, was approved for patients 12 years and older with generalized myasthenia gravis based on data from the ongoing Vivacity-MG3 study.

Prademagene zamikeracel (Zevaskyn) is the first and only cell-based gene therapy for wound treatment in patients with recessive dystrophic epidermolysis bullosa.

The complete response letter (CRL) specifies that the current dosage duration of aflibercept is still safe but extending the duration is not approved.

Marking the first approval in over a decade for this challenging condition, the FDA has approved dupilumab (Dupixent) to treat chronic spontaneous urticaria in patients 12 years and older whose hives and itching remain uncontrolled by antihistamines.

The combination therapy can be used as first-line treatment for unresectable or metastatic forms of the cancer.

Richard Lafayette, MD, FACP, Stanford University Medical Center, explains the significance of this second approval in a month for proteinuria reduction.

ECHELON-3 (NCT04404283) principal investigator Craig A. Portell, MD, speaks to the significance of this triplet approval from February as an alternative to more complex and time-consuming regimens in the setting of relapsed/refractory large B-cell lymphoma (R/R LBCL).

Carla Nester, MD, MSA, FASN, is coinvestigator for the ongoing APPEAR-C3G trial (NCT04817618), data from which were used to support the FDA’s recent approval of iptacopan (Fabhalta; Novartis) in complement 3 glomerulopathy (C3G).

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.

Data from the ATLAS research comprises a series of trials that have yielded years of long-term, comprehensive data on the safety and efficacy of fitusiran (Qfitlia; Sanofi).

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.