OncLive’s FDA Approval Report: The Regulatory Rundown for February 2025

Below is your guide to all of the oncologic therapeutic options cleared by the FDA in February 2025. The roundup provides everything you need to know, right at your fingertips—all the topline findings that supported the decisions and expert insights detailing what they mean for clinical practice.

2/11: Mirdametinib in NF1-Associated Plexiform Neurofibromas

The regulatory agency cleared mirdametinib (Gomekli) for the treatment of adult and pediatric patients aged 2 years and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable for complete resection. The agent elicited confirmed overall response rates (ORRs) of 41% (95% CI, 29%-55%) and 52% (95% CI, 38%-65%) in adult (n = 58) and pediatric (n = 56) patients, according to findings from the phase 2 ReNeu study (NCT03962543).

“The approval of mirdametinib is significant because this a highly selective MEK inhibitor that will provide benefit to both adults and children and NF1 who have PN that are not amenable to surgery and are causing significant quality-of-life or pain issues,” Christopher L. Moertel, MD, said in an interview with OncLive. “Therefore, for this population, this is a big win. Adult patients with NF1 have been without an FDA-approved drug for quite some time. It’s good that we’re there.” Moertel serves as a pediatric neuro-oncologist and professor at the University of Minnesota School of Medicine in Minneapolis, where he directs the Pediatric Neuro-Oncology Fellowship Program and co-leads the Pediatric Neuro-Oncology and Neurofibromatosis Programs.

OTHER RELATED COVERAGE

• In a prior interview, Angela Hirbe, MD, PhD, of Siteman Cancer Center, highlighted the significant morbidity and quality of life (QOL) detriment associated with NF1-PN, the efficacy of mirdametinib in reducing tumor size and pain in both pediatric and adult patients as seen in the ReNeu trial, and the potential future roles of MEK inhibitors for post-surgical adjuvant therapy and secondary prophylaxis in this disease setting.
• In another past interview, Timothy Gershon, MD, PhD, of Emory University, discussed data from an exploratory analysis of ReNeu.
• In another interview, Rene Y. McNall-Knapp, MD, of University of Oklahoma Health Sciences Center, shed light on health-related QOL outcomes from the trial.

2/12: Brentuximab Vedotin Plus Lenalidomide and Rituximab in R/R LBCL

The FDA approved brentuximab vedotin (Adcetris) paired with lenalidomide (Revlimid) and rituximab (Rituxan) for use in adult patients with relapsed or refractory large B-cell lymphoma, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, DLBCL arising from indolent lymphoma, or high-grade lymphoma, following at least 2 lines of systemic therapy who are not eligible for autologous hematopoietic stem cell transplantation or CAR T-cell therapy.

The decision was based on findings from the phase 3 ECHELON-3 study (NCT04404283) in which the addition of the antibody-drug conjugate (n = 112) led to a median overall survival (OS) of 13.8 months (95% CI, 10.3-18.8) vs 8.5 months (95% CI, 5.4-11.7) with lenalidomide/rituximab alone (n = 118), translating to a 37% reduction in the risk of death (HR, 0.63; 95% CI, 0.45-0.89; P = .0085). The triplet led to a median progression-free survival of 4.2 months (95% CI, 2.9-7.1) vs 2.6 months (95% CI, 1.4-3.1) with the doublet (HR, 0.53; 95% CI, 0.38-0.73; P < .0001).

“This triplet combination, with its promising OS benefit, has the potential to address the high unmet need of patients with relapsed/refractory DLBCL, particularly those who are not able to receive CAR T-cell therapy or bispecific antibodies or who have relapsed/refractory disease following these treatments,” Christopher A. Yasenchak, MD, of Willamette Valley Cancer Institute and Research Center/US Oncology Research, in Eugene, Oregon, said in a previous presentation of trial data.

2/14: Vimseltinib in Symptomatic Tenosynovial Giant Cell Tumor

The regulatory agency gave the green light to vimseltinib (Romvimza) for use in patients with symptomatic tenosynovial giant cell tumor (TGCT) for whom surgical resection will potentially cause worsening functional limitation or severe morbidity based on data from the phase 3 MOTION trial (NCT05059262).

The agent (n = 83) induced an ORR of 40% (95% CI, 29%-51%) at week 25 by RECIST v1.1 criteria compared with 0% with placebo (n = 40) in the intention-to-treat population (P < .0001). In the vimseltinib arm, 5% had a complete response and 35% had a partial response rate. The median duration of response (DOR) was not reached (range, 2.5+ to 19.4+). Notably, 85% of patients experienced a DOR of at least 6 months and 58% experienced a DOR of at least 9 months.

“We’re very excited to have vimseltinib recently approved,” William Tap, MD, of Memorial Sloan Kettering Cancer Center in New York, New York, said in an interview with OncLive. “TGCT, especially the diffuse type, can be a very devastating disease for people. It’s not something that generally threatens their life, but it can really alter the trajectory of their life. People live with the disease for long periods of time and can have tremendous symptoms and a lot of disability from it, so to have medical options for TGCT is critical for us.”

OTHER RELATED COVERAGE

• In a prior interview, R. Lor Randall, MD, FACS, of University of California Davis Comprehensive Cancer Center in Sacramento, California, discussed efficacy and safety data seen with vimseltinib in patients with TGCT.
• In a concurrent interview, Randall underscored that focusing on patient QOL is the most important aspect of the management of TGCTs, and vimseltinib could represent a safe option with tolerable adverse effects.
• In a past OncLive On Air episode supported by Deciphera, Suzanne George, MD, of Dana-Farber Cancer Institute, and Margaret von Mehren, MD, of Fox Chase Cancer Center, highlighted how vimseltinib could alleviate unmet needs in those with TGCT.

Other Noteworthy Decisions:

A label expansion for the PATHWAY anti-HER2/neu (4B5) Rabbit Monoclonal Primary Antibody has been cleared to identify patients with hormone receptor–positive, HER2-ultralow metastatic breast cancer who may be eligible for fam-trastuzumab deruxtecan-nxki (Enhertu).

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