5 Key FDA Approval Dates Mark a Milestone-Filled May

May was a productive month for the FDA, with multiple drug approvals spanning a range of therapeutic areas, including chronic obstructive pulmonary disease (COPD), plaque psoriasis, and hemophilia A.

The timeline below highlights 5 key dates last month when significant advancements were made across these and other disease states.

In May, the FDA granted 5 significant drug approvals across various therapeutic areas, marking notable progress in expanding treatment options for patients. | Image Credit: - Tada Images- stock.adobe.com

May 8 - FDA Approves First Treatment for KRAS-Mutated Recurrent LGSOC

The FDA began the month by granting accelerated approval to the combination of avutometinib and defactinib (Avmapki Fakzynja Co-pack; Verastem Oncology) for adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who received prior systemic therapy.1

This marks the first FDA-approved treatment specifically for KRAS-mutated recurrent LGSOC. The approval was based data from the phase 2 RAMP-201 trial (NCT04625270), an open-label multicenter study of 57 adult patients with measurable KRAS-mutated recurrent LGSOC who had received at least 1 prior systemic therapy.

Patients received oral avutometinib and defactinib for 3 weeks of a 4-week cycle until disease progression or intolerable toxicity. The median treatment duration was 12 months (range, 0.03-40), with a confirmed overall response rate (ORR) of 44% (95% CI, 31%-58%) and duration of response (DOR) ranging from 3.3 to 31.1 months.

“The approval of avutometinib plus defactinib brings a much-needed therapeutic option to patients and establishes this combination as the new standard of care for women with recurrent low-grade serous ovarian cancer harboring a KRAS mutation," Rachel Grisham, MD, global lead principal investigator of GOG-3097/ENGOT-ov81/GTG-UK/RAMP 301, said in a press release.2

May 14 - Teliso-V Approved to Treat Advanced NSCLC With High c-Met Protein Overexpression

One week later, telisotuzumab vedotin-tllv (Emrelis; AbbVie), known as Teliso-V, was approved for adults with locally advanced or metastatic nonsquamous non–small cell lung cancer (NSCLC) exhibiting high c-Met protein overexpression, and who had prior systemic treatment.3

Teliso-V is an antibody-drug conjugate targeting surface c-Met expression and is now the only approved therapy for this patient population. The approval was supported by data from the ongoing phase 2 LUMINOSITY trial (NCT03539536), which reported a 35% ORR (95% CI, 24%-46%) and a median DOR of 7.2 months (95% CI, 4.2-12) among 84 patients.

"This approval is a welcomed targeted therapy for those with high c-Met protein overexpressing late-stage, non–small cell lung cancer who have seen very limited treatment innovation in the last decade," Upal Basu Roy, PhD, MPH, executive director of research at the LUNGevity Foundation, said in a news release.4

May 15 - Retifanlimab Becomes First FDA-Approved Frontline Treatment for Advanced Anal Cancer

One day later, the FDA approved retifanlimab-dlwr (Zynyz; Incyte) in combination with platinum-based chemotherapy for the first-line treatment of adult patients with inoperable locally recurrent or metastatic squamous cell carcinoma of the anal canal (SCAC). It was also approved as a monotherapy for patients with disease progression or intolerance to platinum-based chemotherapy.5

This makes retifanlimab, a humanized monoclonal antibody targeting PD-1, the only approved first-line treatment for advanced anal cancer in the US. The approval was based on data from the phase 3 POD1UM-303/InterAACT2 (NCT04472429) and phase 2 POD1UM-202 (NCT03597295) trials.

In POD1UM-303/InterAACT2, patients receiving the retifanlimab combination achieved a median progression-free survival of 9.3 months (95% CI, 7.5-11.3) compared with 7.4 months (95% CI, 7.1-7.7) for placebo (HR, 0.63; 95% CI, 0.47-0.84; P = .0006). Interim analysis showed median overall survival (OS) of 29.2 months (95% I, 24.2-not evaluable) in the retifanlimab group vs 23 months (95% CI, 15.1-27.9) in controls (HR, 0.70; 95% CI, 0.49-1.01; P = .0273). The ORR in the retifanlimab group was 56% (95% CI, 48-64), with 22% complete and 34% partial response rates.

The POD1UM-202 study supported the monotherapy indication, showing a 49% disease control rate (95% CI, 38.5%-59.5%) and a 13.8% ORR (95% CI, 7.6%-22.5%).

"The FDA approval of Zynyz marks a pivotal moment, bringing effective combination and monotherapy options to patients with advanced anal cancer after decades of limited innovated," Hervé Hoppenot, Incyte's CEO, said in a press release.6

May 19 - FDA Approves Jivi for Pediatric Patients Aged 7 to 12 Years With Hemophilia A

Next, the FDA expanded the indication for Bayer’s Jivi (antihemophilic factor [recombinant], PEGylated-aucl) to include patients between 7 and younger than 12 years with hemophilia A.7

Previously approved for patients aged 12 and older with hemophilia A, Jivi can be used for on-demand treatment, bleeding control, perioperative management, and routine prophylaxis. Jivi's expansion was supported by data from the Alfa-PROTECT (NCT05147662) and PROTECT Kids (NCT01775618) trials, which demonstrated safety and efficacy in pediatric patients with severe hemophilia A.

Of the 35 patients who received prophylactic treatment with Jivi twice weekly (40-60 IU/kg) for at least 50 exposure days over 26 weeks in Alfa-PROTECT, 32 completed the treatment phase and continued in an 18-month extension study. The trial met its objectives, with no unexpected safety concerns and low annualized bleed rates (ABRs).

The PROTECT Kids study further assessed the pharmacokinetics, safety, and efficacy of Jivi in previously treated patients aged under 12. It confirmed Jivi's significant ABR reductions and safety profile.

“The FDA’s approval for pediatric patients 7 to <12 years of age is reflective of Bayer’s commitment to the hemophilia A community and underscores our commitment to supporting families through their treatment journey,” Jessica Charlet, scientific director at Bayer, said in a statement.8

May 22 - FDA Approves Mepolizumab for COPD With Eosinophilic Phenotype, Roflumilast Topical Foam 0.3% for Plaque Psoriasis

Before the month ended, the FDA approved 2 additional treatments: mepolizumab (Nucala; GSK) for COPD and roflumilast foam 0.3% (Zoryve; Arcutis Biotherapeutics) for plaque psoriasis.9,10

Now approved for its fifth indication, mepolizumab, a monoclonal antibody that targets interleukin-5, is indicated as an add-on maintenance treatment for adults with inadequately controlled COPD, becoming the first once-monthly biologic approved for this patient population.9 Approval was supported by positive results from the phase 3 METREX (NCT02105948) and MATINEE (NCT04133909) trials.

In the METREX trial, mepolizumab added to triple inhaled therapy significantly reduced the annualized rate of moderate or severe exacerbations compared with placebo (rate ratio, 0.82; 95% CI, 0.68-0.98; adjusted P = .04).

Similarly, in the MATINEE trial, mepolizumab achieved a 21% reduction in annualized moderate or severe exacerbations (0.80 vs 1.01 events per year; rate ratio, 0.79; 95% CI, 0.66-0.94; P = .01) and prolonged time to first moderate or severe exacerbation (median, 419 vs 321 days; HR, 0.77; 95% CI, 0.64-0.93; P = .009).

“The MATINEE trial uncovers new possibilities in the treatment landscape for COPD patients with type 2 inflammation, as we strive to target drivers of disease and improve the lives of patients suffering with COPD,” Frank Sciurba, lead investigator of the MATINEE trial, said in a press release.11

Roflumilast topical foam 0.3%, a once-daily steroid-free topical treatment for plaque psoriasis, was approved for use on the scalp and body in patients aged 12 and older.10 The approval was based on positive results from the phase 3 ARRECTOR trial (NCT05028582) and phase 2 Trial 204.

In the ARRECTOR trial, 66.4% of patients treated with roflumilast achieved Scalp-Investigator Global Assessment (IGA) Success at week 8 compared with 27.8% using vehicle (P < .0001). Additionally, 45.5% achieved Body-IGA Success compared with 20.1% using vehicle (P < .0001).

Trial 204 demonstrated similar results. At week 8, 56.7% of patients achieved Scalp-IGA Success and 39.0% achieved Body-IGA Success vs 11.0% and 7.4%, respectively, in the vehicle group.

"We are pleased to see new advancements and innovation in treatments for the millions afflicted with this serious skin disease that can be used long-term and anywhere the disease presents," Leah Howard, president and CEO of the National Psoriasis Foundation, said in a press release.12

References

1. McCormick B. FDA approves first treatment for KRAS-mutated recurrent LGSOC. AJMC®. May 9, 2025. Accessed June 4, 2025. https://www.ajmc.com/view/fda-approves-first-treatment-for-kras-mutated-recurrent-lgsoc
2. FDA approves the Avmapki Fakzynja combination therapy as the first-ever treatment for adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer. News release. Verastem Oncology; May 8, 2025. Accessed June 4, 2025. https://investor.verastem.com/news-releases/news-release-details/fda-approves-avmapkitm-fakzynjatm-combination-therapy-first-ever
3. Klein HE. Teliso-V approved to treat advanced NSCLC with high c-Met protein overexpression. AJMC. May 14, 2025. Accessed June 4, 2025. https://www.ajmc.com/view/teliso-v-approved-to-treat-advanced-nsclc-with-high-c-met-protein-overexpression
4. U.S. FDA approves Emrelis (telisotuzumab vedotin-tllv) for adults with previously treated advanced non-small cell lung cancer (NSCLC) with high c-met protein overexpression. AbbVie. News release. May 14, 2025. Accessed June 4, 2025. https://news.abbvie.com/2025-05-14-U-S-FDA-Approves-EMRELIS-TM-telisotuzumab-vedotin-tllv-for-Adults-With-Previously-Treated-Advanced-Non-Small-Cell-Lung-Cancer-NSCLC-With-High-c-Met-Protein-Overexpression
5. McCormick B. Retifanlimab becomes first FDA-approved frontline treatment for advanced anal cancer. AJMC. May 16, 2025. Accessed June 4, 2025. https://www.ajmc.com/view/retifanlimab-becomes-first-fda-approved-frontline-treatment-for-advanced-anal-cancer
6. Incyte announces FDA approval of Zynyz (retifanlimab-dlwr) making it the first and only approved first-line treatment for advancd anal cancer patients in the United States. News release. Incyte; May 15, 2025. Accessed June 4, 2025. https://investor.incyte.com/news-releases/news-release-details/incyte-announces-fda-approval-zynyzr-retifanlimab-dlwr-making-it
7. Jeremias S. FDA approves Jivi for pediatric patients aged 7 to 12 years with hemophilia A. AJMC. May 19, 2025. Accessed June 4, 2025. https://www.ajmc.com/view/fda-approves-jivi-for-pediatric-patients-aged-7-to-12-years-with-hemophilia-a
8. U.S. FDA grants approval for Jivi antihemophilic factor (recombinant), PEGylated-aucl in pediatric patients 7 to under 12 years of age with hemophilia A (congenital factor VIII deficiency). Press release. Bayer; May 19, 2025. Accessed May 19, 2025. https://www.businesswire.com/news/home/20250519742201/en/U.S.-FDA-Grants-Approval-for-Jivi-Antihemophilic-Factor-Recombinant-PEGylated-aucl-in-Pediatric-Patients-7-to-Under-12-Years-of-Age-With-Hemophilia-A-Congenital-Factor-VIII-Deficiency
9. McCormick B. FDA approves mepolizumab as first once-monthly biologic for COPD with eosinophilic phenotype. AJMC. May 22, 2025. Accessed June 4, 2025. https://www.ajmc.com/view/fda-approves-mepolizumab-as-first-once-monthly-biologic-for-copd-with-eosinophilic-phenotype
10. Steinzor P. FDA approves roflumilast topical foam 0.3% for plaque psoriasis. AJMC. May 22, 2025. https://www.ajmc.com/view/fda-approves-roflumilast-topical-foam-0-3-for-plaque-psoriasis
11. Nucala (mepolizumab) delivers clinically meaningful and statistically significant reduction in COPD exacerbations, with positive MATINEE trial results published in New England Journal of Medicine. News release. GSK; April 30, 2025. Accessed May 6, 2025. https://www.gsk.com/en-gb/media/press-releases/nucala-mepolizumab-delivers-clinically-meaningful-and-statistically-significant-reduction-in-copd-exacerbations-with-positive-matinee-trial-results-published-in-nejm/
12. Arcutis’ Zoryve (roflumilast) topical foam 0.3% approved by US FDA for the treatment of plaque psoriasis in adults and adolescents ages 12 and older. Arcutis Biotherapeutics. News release. May 22, 2025. Accessed June 4, 2025. https://www.arcutis.com/arcutis-zoryve-roflumilast-topical-foam-0-3-approved-by-u-s-fda-for-the-treatment-of-plaque-psoriasis-in-adults-and-adolescents-ages-12-and-older/