5 Expanded FDA Approvals From October

Throughout October, The American Journal of Managed Care^® (AJMC^®) covered 11 approvals by the FDA. These developments were for medication-induced pregnancy termination, pediatric atopic dermatitis, idiopathic pulmonary fibrosis, cutaneous squamous cell carcinoma, inflammatory bowel disease (IBD), chronic rhinosinusitis with nasal polyps, type 2 diabetes (T2D), multiple myeloma, menopause, acute myeloid leukemia (AML), and pulmonary arterial hypertension. The timeline below highlights 5 of these approvals that were expanded indications.

October 6: FDA Expands Roflumilast Use for Atopic Dermatitis to Children Aged 2 to 5 Years

This approval came on the heels of 2 recent approvals for roflumilast (Zoryve; Arcutis Biotherapeutics), in July 2024 for atopic dermatitis in pediatric patients 6 years and older and in May 2025 for plaque psoriasis in adults and adolescents 12 years and older, and it gave roflumilast 6 FDA-approved indications overall.¹ Based on results from 2 phase 3 trials, INTEGUMENT-PED (NCT04845620) and INTEGUMENT-OLE (NCT04804605), and a phase 1 pharmacokinetic study, promising data showed key factors to the drug’s success are its ability to lead to rapid skin clearance and that it can safely be administered and tolerated by patients. In INTEGUMENT-PED, skin improvements were seen as soon as week 1, and 71.9% of the patients who rolled over to the extension analysis achieved the Eczema Area and Severity Index–75 benchmark by week 56 compared with baseline measures; this improvement was both maintained and continuous.

In an interview with AJMC about this approval, INTEGUMENT study investigator Lawrence F. Eichenfield, MD, professor of dermatology and pediatrics at the University of California San Diego and chief of pediatric dermatology at Rady Children's Hospital San Diego, said,“There’s been a clinical need for effective topical nonsteroids that can be anti-inflammatory and also help with the itch of atopic dermatitis and can be used in different regimens of care, but potentially not just for short periods of time and then coming off of them.”²

October 14: FDA Expands Upadacitinib Label for Treating Inflammatory Bowel Disease

This oral selective Janus kinase inhibitor already had a solid evidence base supporting its use in IBD when the FDA voted to allow its use in adults with moderate to severe active ulcerative colitis or Crohn disease.³ Patients could now be eligible to receive upadacitinib (Rinvoq; AbbVie) if they had received and not failed on at least 1 approved systemic therapy if tumor necrosis factor (TNF)–blocking agents were contraindicated. Previously, patients had to demonstrate an inadequate response to intolerance to at least 1 TNF blocker in order to receive upadacitinib.

Data from the U-ACHIEVE (NCT02819635) and U-ACCOMPLISH (NCT03653026) trials supported the new indication for ulcerative colitis, while data from the U-EXCEL (NCT03345849), U-EXCEED (NCT03345836), and U-ENDURE (NCT03345823) trials supported the new indication for Crohn disease. Together, these trials’ results demonstrated higher and durable clinical remission and endoscopic response. The medication was also shown to be able to maintain its efficacy and safety over maintenance treatment.

“This label update gives health care providers the option to prescribe [upadacitinib] for patients with moderately to severely active inflammatory bowel disease after the use of 1 approved systemic therapy if TNF blockers are deemed clinically inadvisable by the prescribing physician,” said Kori Wallace, MD, PhD, vice president and global head of immunology clinical development at AbbVie.³

October 20: FDA Expands Semaglutide Label to Reduce Cardiovascular Risk in Adults With Type 2 Diabetes

As an oral medication and the first and only glucagon-like peptide 1 (GLP-1) receptor agonist approved at the time to improve glycemic control in T2D alongside diet and exercise, oral semaglutide (Rybelsus; Novo Nordisk) received its first approval in 2019.⁴ This newest approval expands its indications to now include 7-mg and 14-mg doses for use in adults who do not have a history of heart attack or stroke. The expanded approval is also another first for the oral therapeutic: it is now the first and only GLP-1 with an approved cardiovascular risk indication. Data from the phase 3b SOUL trial (NCT03914326) supported Novo Nordisk’s FDA application.

In SOUL, treated patients exhibited a 14% relative risk reduction (HR, 0.86; 95% CI, 0.77-0.96; P = .006) in major adverse cardiovascular events (MACE), with 12% of this group experiencing MACE compared with 13.8% of the placebo cohort. Drilling down, the rate of reduced nonfatal myocardial infarction was considered statistically significant.

“The incidence of serious adverse events was lower among participants receiving oral semaglutide than among those receiving placebo, a difference that was mostly due to the higher incidence of cardiac disorders and infections or infestations in the placebo group,” the researchers said.

October 24: Revumenib Granted FDA Approval for R/R NPM1-Mutated AML

This approval expanded revumenib (Revuforj; Syndax Pharmaceuticals) indications from only being able to be administered in patients 1 year and older with relapsed/refractory (R/R) AML with a KMT2A translocation to now being available in the same age group who have a susceptible NPM1 mutation.⁵

“For the first time, a targeted, oral therapy that is well tolerated and efficacious is approved for R/R NPM1-mutated AML and R/R KMT2A-translocated acute leukemia,” said Joshua F. Zeidner, MD, chief, Leukemia Research, University of North Carolina Lineberger Comprehensive Cancer Center. “The compelling clinical activity observed with [revumenib] in clinical trials and clinical practice paves the way for a new standard of care for these 2 aggressive and difficult-to-treat blood cancers.”

Phase 2 AUGMENT-101 (NCT04065399) trial data, which supported this approval, demonstrated a 23.4% (95% CI, 13.8%-35.7%; P = .0014) rate of complete remission (CR) plus CR with partial hematological recovery. The overall response rate was 48.1%, according to updated results presented at the 2025 European Hematology Association meeting. Responses were also seen across patient subgroups: adults younger than 65 years, adults 65 years and older, and patients with and without a history of venetoclax exposure. Only 11.9% of patients required a dose reduction because of treatment-related adverse events.

October 27: FDA Expands Sotatercept Indication for PAH

As an injectable therapy available at doses of 45 mg and 60 mg, sotatercept (Winrevair; Merck) indications now include improving exercise capacity and World Health Organization functional class, as well as reducing the risk of clinical worsening events in adults living with PAH.⁶ An activin signaling inhibitor, the medication previously received a priority review and breakthrough and orphan drug designation from the FDA; its first approval came in March 2024.

For the new indications, Merck used data from the phase 3 ZENITH trial (NCT04896008) to support its application. Patients were randomized 1:1 to receive either sotatercept plus background PAH therapy or placebo plus background PAH therapy, and treatment was administered every 3 weeks. Most background therapy was triplet therapy (72% of patients). Sotatercept showed an HR of 0.24, meaning that it reduced risks of major morbidity and mortality outcomes by 76% (95% CI, 0.13-0.43; P < .0001) vs placebo, and just 17% of treated patients experienced events of all-cause death, lung transplantation, or PAH-worsening hospitalization of 24 hours or more, the trial’s primary efficacy end point of time to first occurrence.

“Results from the pivotal ZENITH trial add to the growing body of data and support the potential for [sotatercept] as standard of care,” said Vallerie McLaughlin, MD, the Kim A. Eagle MD Endowed Professor of Cardiovascular Medicine, University of Michigan at Ann Arbor; ZENITH study investigator; and member of the adult sotatercept steering committee.

References

1. Shaw M. FDA expands roflumilast use for atopic dermatitis to children aged 2 to 5 years. AJMC. October 6, 2025. Accessed November 21, 2025. https://www.ajmc.com/view/fda-expands-roflumilast-use-for-atopic-dermatitis-to-children-aged-2-to-5-years
2. Shaw M, Eichenfield L. Roflumilast delivers speedy improvement, significant itch reduction in younger pediatric patients: Lawrence F. Eichenfield, MD. AJMC. October 19, 2025. Accessed November 21, 2025. https://www.ajmc.com/view/roflumilast-delivers-speedy-improvement-significant-itch-reduction-in-younger-pediatric-patients-lawrence-f-eichenfield-md
3. Klein H. FDA expands upadacitinib label for treating inflammatory bowel disease. AJMC. October 14, 2025. Accessed November 21, 2025. https://www.ajmc.com/view/fda-expands-upadacitinib-label-for-treating-inflammatory-bowel-disease
4. Klein H. FDA expands semaglutide label to reduce cardiovascular risk in adults with type 2 diabetes. AJMC. October 20, 2025. Accessed November 21, 2025. https://www.ajmc.com/view/fda-expands-semaglutide-label-to-reduce-cardiovascular-risk-in-adults-with-type-2-diabetes
5. Joszt L. Revumenib granted FDA approval for R/R NPM1-mutated AML. AJMC. October 24, 2025. Accessed November 21, 2025. https://www.ajmc.com/view/revumenib-granted-fda-approval-for-r-r-npm1-mutated-aml
6. Shaw M. FDA expands sotatercept indication for PAH. AJMC. October 27, 2025. Accessed November 21, 2025. https://www.ajmc.com/view/fda-expands-sotatercept-indication-for-pah