5 September FDA Approvals That Are Transforming Treatment This Fall

Last month, the FDA approved several treatments for various cancer types, dermatologic conditions, and rare diseases.

The timeline below highlights 5 key dates in September when new treatment options became available for various patient populations.

September 10 - FDA Approves Selumetinib for Children With NF1 and Inoperable Tumors

Early in the month, the FDA approved selumetinib (Koselugo; AstraZeneca) in both granule and capsule formulations for pediatric patients as young as 1 year with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas.¹ This expands the drug’s previous approval for children aged 2 and older, broadening access for younger patients with this rare condition.

Selumetinib directly targets the MEK pathway to slow or halt tumor progression in NF1-associated plexiform neurofibromas, enabling earlier intervention to reduce tumor burden, manage symptoms, and improve long-term outcomes and quality of life for children living with NF1. The initial approval on April 10, 2020, was based on the SPRINT trial (NCT01362803), which demonstrated a 66% overall response rate (n = 33; 95% CI, 51%-79%), with most responses lasting at least 12 months.

The latest approval was supported by additional bioavailability data in healthy adults, allowing efficacy data from older patients to be extrapolated to younger children. Exposure matching across the SPRINT and SPRINKLE (NCT05309668) trials confirmed comparable drug exposure between age groups.

The recommended dose remains 25 mg/m² twice daily until disease progression or unacceptable toxicity. Although no new safety signals were observed, known risks include cardiomyopathy and skin reactions.

September 18 - FDA Approves Ruxolitinib Cream for Patients as Young as 2 Years With Atopic Dermatitis

About a week later, the FDA approved ruxolitinib cream (Opzelura; Incyte), a topical Janus kinase inhibitor, for moderate atopic dermatitis (AD) in nonimmunocompromised patients aged 2 and older.² This expands upon the drug’s previous approvals, first in September 2021 for short-term and noncontinuous use in patients aged 12 and older with mild to moderate nonimmunocompromised AD, and again in July 2022 for the treatment of nonsegmental vitiligo in the same age group.

The latest approval was supported by the phase 3 TRuE-AD3 (NCT04921969) trial, involving children aged 2 to 12 years with AD, which met both its primary and secondary end points. Significantly more patients treated with ruxolitinib achieved Investigator’s Global Assessment–treatment success. Additionally, a greater proportion of patients treated with ruxolitinib cream achieved a 75% improvement in Eczema Area and Severity Index scores at the end of the 8-week vehicle-controlled period.

“With this approval, we now have a new, nonsteroidal topical option that expands how we care for kids with this chronic disease,” Peter Lio, MD, clinical assistant professor of dermatology and pediatrics at Northwestern University Feinberg School of Medicine, said in a news release.³ “This is a meaningful step forward and marks a significant advancement in our ability to better support our pediatric patients.”

September 19 – FDA Approves Use of Subcutaneous Pembrolizumab for NSCLC, Most Solid Tumors

The next day, the FDA approved pembrolizumab (Keytruda; Merck) plus berahyaluronidase alfa-pmph (Keytruda Qlex; Merck) for subcutaneous administration in patients aged 12 and older with solid tumors for which the intravenous (IV) formulation of pembrolizumab is already approved.⁴

Pembrolizumab, a PD-1 inhibitor, prevents T cells from attacking normal cells by blocking the PD-1/PD-L1 interaction, allowing the immune system to target cancer cells more effectively. The previously approved IV formulation is used to treat non–small cell lung cancer (NSCLC) without abnormal EGFR or ALK genes and several other solid tumor types.

The latest approval was based on data from the Study MK-3475A-D77 trial (NCT05722015), which assessed the efficacy of subcutaneous pembrolizumab and berahyaluronidase in treatment-naïve patients with NSCLC lacking EGFR, ALK, or ROS1 mutations. Patients in the trial received the subcutaneous combination plus chemotherapy or IV pembrolizumab plus chemotherapy.

Results demonstrated an overall response rate of 45.4% (95% CI, 39%-52%) with the subcutaneous formulation vs 42.1% (95% CI, 33%-51%) with the IV formulation, demonstrating noninferiority. Median duration of response was 9.1 months (95% CI, 6.9-not reached) in the subcutaneous group compared with 8.0 months (95% CI, 7.4-not reached) in the IV group. However, progression-free and overall survival were similar between groups.

"As a physician, I am thrilled to see these data for subcutaneous pembrolizumab, which, if approved, have the potential to give patients valuable time back in their treatment day with results that are consistent with IV pembrolizumab,” Enriqueta Felip, MS, PhD, head of the Thoracic Tumors Group at Vall d’Hebron Institute of Oncology and coauthor of the phase 3 trial, said in a statement after the study results were reported in March.⁵

September 25 – FDA Approves Imlunestrant for ER+ and HER2-Negative Breast Cancer and Paltusotine for Adults With Acromegaly

Less than a week later, the FDA approved 2 treatments, including imlunestrant (Inluriyo; Eli Lilly and Company) for adults with ER-positive (ER+), HER2-negative, ESR1-mutated advanced, or metastatic breast cancer whose disease progressed after at least 1 line of endocrine therapy.⁶ This approval is particularly significant since ESR1 mutations can drive resistance to hormone therapies, making treatment more challenging.

It was based on the phase 3 EMBER trial (NCT04975308), in which patients were randomized 1:1:1 to receive imlunestrant, standard endocrine monotherapy, or imlunestrant plus abemaciclib. Mean progression-free survival (PFS) was 9.4 months in the combination group, 5.5 months for imlunestrant alone, and 3.8 months for standard therapy.

Notably, the oral formulation of imlunestrant offers a convenient alternative to fulvestrant, which requires monthly intramuscular injections, potentially improving treatment adherence and access.

"This therapy reflects our commitment to developing treatments that improve outcomes for people with breast cancer and represents an important step toward advancing innovative, all-oral treatment approaches," Jacob Van Naarden, executive vice president and president of Lilly Oncology, said in a press release.⁷

That same day, the FDA also approved paltusotine (Palsonify; Crinetics) for the treatment of acromegaly in adults who had an inadequate response to surgery and/or for whom surgery is not an option.⁸This approval marks the first for a once-daily, oral therapy to treat acromegaly and was based on data from the PATHFNDR-1 (NCT04837040) and PATHFNDR-2 (NCT05192382) trials. In both trials, paltusotine demonstrated rapid, sustained efficacy and reliable biochemical control.

PATHFNDR-1 met its primary end point, as 83% of patients maintained an insulin-like growth factor 1 (IGF-1) level of 1.0 or less times the upper limit of normal (xULN) vs 4% of those taking a placebo after switching from the injectable standard of care (P < .0001). The study also met its secondary end points of change from baseline in IGF-1 level, change from baseline in Acromegaly Symptoms Diary total score, and the proportion of participants who maintained a growth hormone level of less than 1.0 ng/mL.

PATHFNDR-2 also met its primary and secondary end points, as 56% of previously pharmacologically treated patients achieved an IGF-1 level of 1.0xULN or less vs 5% of those taking a placebo (P < .0001). The secondary end points aligned with those in the PATHFNDR-1 study, meeting statistical significance.

"The approval of [paltusotine] is a significant advancement for our patients, as there is an unmet need for an easy-to-administer and safe therapeutic option with a rapid action and durable response that can consistently manage acromegaly,” Shlomo Melmed, MBChB, executive vice president of Medicine and Health Sciences and dean of the medical faculty at Cedars-Sinai, said in a statement.⁹

September 30 - FDA Approves Remibrutinib for Chronic Spontaneous Urticaria

The FDA rounded out the month with the approval of remibrutinib (Rhapside; Novartis), a highly selective Bruton tyrosine kinase inhibitor (BTKi), for patients with chronic spontaneous urticaria (CSU) who remain symptomatic despite treatment with H1 antihistamine therapy.¹⁰

This marks the first approval of a BTKi for CSU and is supported by data from the phase 3 REMIX-1 (NCT05030311) and REMIX-2 (NCT05032157) trials. After 12 weeks of treatment, remibrutinib showed more significant changes from baseline in itch, hives, and weekly urticaria activity compared with placebo.

Patients treated with remibrutinib were also more likely to achieve well-controlled disease, defined as a Urticaria Activity Score 7 of 6 or lower, by week 12 vs the placebo group (49.8% vs 24.8% in REMIX-1 [P < .001] and 46.8% vs 19.6% in REMIX-2 [P < .001]), with some achieving control as early as week 2. Efficacy appeared to be maintained through week 24.

“The approval of remibrutinib is an important development in CSU care," Giselle Mosnaim, MD, MS, REMIX trial investigator, said in a news release.¹¹ "It quickly reduces symptoms, offering patients control of the hives and itching that they experience on a daily basis. This is significant because it expands beyond existing injectable treatments and gives patients an oral option that can easily be incorporated into their daily lives.”

References

1. Steinzor P. FDA approves selumetinib for children with NF1 and inoperable tumors. AJMC^®. September 10, 2025. Accessed October 24, 2025. https://www.ajmc.com/view/fda-approves-selumetinib-for-children-with-nf1-and-inoperable-tumors
2. McCrear S. FDA approves ruxolitinib cream for patients as young as 2 years with atopic dermatitis. AJMC. September 19, 2025. Accessed October 24, 2025. https://www.ajmc.com/view/fda-approves-ruxolitinib-cream-for-patients-as-young-as-2-years-with-atopic-dermatitis
3. Incyte announces additional FDA approval of Opzelura (ruxolitinib) cream in children ages 2-11 with atopic dermatitis. News release. Incyte. September 18, 2025. Accessed October 24, 2025. https://investor.incyte.com/news-releases/news-release-details/incyte-announces-additional-fda-approval-opzelurar-ruxolitinib
4. Bonavitacola J. FDA approves use of subcutaneous pembrolizumab for NSCLC, most solid tumors. AJMC. September 19, 2025. Accessed October 24, 2025. https://www.ajmc.com/view/fda-approves-use-of-subcutaneous-pembrolizumab-for-nsclc
5. Merck’s investigational subcutaneous pembrolizumab with berahyaluronidase alfa demonstrates noninferior pharmacokinetics compared to intravenous (IV) Keytruda (pembrolizumab) in pivotal 3475A-D77 trial. News release. Merck. March 27, 2025. Accessed October 24, 2025. https://www.merck.com/news/mercks-investigational-subcutaneous-pembrolizumab-with-berahyaluronidase-alfa-demonstrates-noninferior-pharmacokinetics-compared-to-intravenous-iv-keytruda-pembrolizumab-in-pivotal/
6. McCrear S. FDA approves imlunestrant for ER+ and HER2-negative breast cancer. AJMC. September 25, 2025. Accessed October 24, 2025. https://www.ajmc.com/view/fda-approves-imlunestrant-for-er-and-her2-negative-breast-cancer
7. US FDA approves Inluriyo (imlunestrant) for adults with ER+, HER2-, ESR1-mutated advanced or metastatic breast cancer. News release. Eli Lilly and Company. September 25, 2025. Accessed October 24, 2025. https://investor.lilly.com/news-releases/news-release-details/us-fda-approves-inluriyo-imlunestrant-adults-er-her2-esr1
8. McNulty R. Paltusotine FDA approved for acromegaly in adults. AJMC. September 26, 2025. Accessed October 24, 2025. https://www.ajmc.com/view/paltusotine-fda-approved-for-acromegaly-in-adults
9. Crinetics announces FDA approval of Palsonify (paltusotine) for the treatment of acromegaly in adults. News release. Crinetics. September 25, 2025. Accessed October 24, 2025. https://crinetics.com/crinetics-announces-fda-approval-of-palsonify-paltusotine-for-the-treatment-of-acromegaly-in-adults/
10. McNulty R. FDA approves remibrutinib for chronic spontaneous urticaria. AJMC. September 30, 2025. Accessed October 24, 2025. https://www.ajmc.com/view/fda-approves-remibrutinib-for-chronic-spontaneous-urticaria
11. Novartis receives FDA approval for Rhapsido (remibrutinib), the only oral, targeted BTKi treatment for chronic spontaneous urticaria (CSU). News release. Novartis. September 30, 2025. Accessed October 24, 2025. https://www.novartis.com/news/media-releases/novartis-receives-fda-approval-rhapsido-remibrutinib-only-oral-targeted-btki-treatment-chronic-spontaneous-urticaria-csu