5 Notable Oncology FDA Approvals Granted in May

May was an active month for oncology drug development, with the FDA issuing 5 notable approvals that expand treatment options across breast cancer, acute myeloid leukemia (AML), mantle cell lymphoma (MCL), and non–muscle-invasive bladder cancer (NMIBC).

Several of the approvals represented first-in-class or first-in-indication advances, including the first approved proteolysis-targeting chimera (PROTAC) therapy for breast cancer, the first all-oral regimen for newly diagnosed AML in older adults ineligible for intensive chemotherapy, and the first immunotherapy combination approved for high-risk NMIBC in more than 3 decades.

Together, these decisions highlight the continued shift toward targeted therapies, antibody-drug conjugates, and novel immunotherapy approaches designed to address unmet needs and improve outcomes across a broad range of hematologic and solid tumor malignancies.

Learn more about these 5 oncology FDA approvals below:

1. FDA Approves Vepdegestrant for ESR1-Mutated, ER-Positive, HER2-Negative Advanced Breast Cancer

The FDA kicked off the month by approving vepdegestrant (Veppanu; Arvinas Operations Inc.) for adults with estrogen receptor (ER)–positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer whose disease has progressed following at least 1 prior line of endocrine therapy.¹

The agent is the first heterobifunctional protein degrader, commonly referred to as a PROTAC, to receive this indication for breast cancer. The recommended dose is 200 mg orally once daily with food, continued until disease progression or unacceptable toxicity.

The May 4 approval was supported by the phase 3 VERITAC-2 trial (NCT05654623), which enrolled 624 adults with ER-positive, HER2-negative advanced or metastatic breast cancer previously treated with endocrine therapy and a CDK4/6 inhibitor.

In the ESR1-mutant subgroup, vepdegestrant significantly improved median progression-free survival (PFS) compared with fulvestrant (5.0 vs 2.1 months; HR, 0.57). It also showed higher objective response rates (19% vs 4%). However, overall survival (OS) data remain immature, with only 16% of events recorded at the time of the PFS analysis.

“This milestone demonstrates that targeted protein degradation can translate into meaningful clinical impact,” Randy Teel, PhD, president and CEO at Arvinas, said in a statement.² “It also strengthens our confidence in the breadth and versatility of our exciting clinical pipeline across oncology, neurodegenerative, and neuromuscular diseases.”

2. FDA Approves First All-Oral Regimen for AML

More than a week later, on May 13, the FDA approved decitabine and cedazuridine (Inqovi; Taiho), formerly the investigative AStx727-07, plus venetoclax for patients aged 75 years and older, newly diagnosed with AML, who are not eligible to undergo intensive induction chemotherapy.³ The regimen is now the first all-oral combination therapy for this indication.

The decision was supported by data from the phase 2 ASCERTAIN-V trial (NCT04657081), which included 101 patients. The study met its complete response (CR) end points, with an overall CR rate of 41.6% and a CR plus CR with incomplete hematologic recovery rate of 63.4%. The median time to CR was 2 months, while the median duration of CR and duration of response had not yet been reached at the time of analysis, with over 75% of responders still in CR at 12 months. Median OS was 15.5 months, and no new safety signals were identified.

“We've taken the disease to a place where, yes, it's still a hard conversation, it still has all the same risks, but to be able to offer a therapy [where] you're not compromising on effectiveness, but you can go home, you can take it, and you can manage side effects; it's really pretty awesome,” Karilyn Larkin, MD, physician and associate professor of hematology at The Ohio State University Comprehensive Cancer Center–The James, said in an interview with AJMC.⁴

3. Sonrotoclax Granted Accelerated Approval for R/R Mantle Cell Lymphoma

That same day, the agency granted accelerated approval for sonrotoclax (Beqalzi; BeOne Medicine), a next-generation BCL2 inhibitor for adults with relapsed or refractory MCL who have received at least 2 lines of systemic treatment, including a Bruton tyrosine kinase (BTK) inhibitor.⁵

Sonrotoclax’s efficacy was evaluated in BGB-11417-201 (NCT05471843), a single-arm, multicenter trial involving 103 adults with relapsed or refractory MCL who previously received anti-CD20–based therapy and a BTK inhibitor. The overall response rate (ORR) was 52%, with a median time to response of 1.9 months. In addition, the median duration of response (DOR) was 15.8 months, following an estimated median follow-up of 11.9 months.

Regarding dosing, sonrotoclax begins with a 4-week ramp-up phase to reduce the risk of tumor lysis syndrome, followed by 320 mg taken orally once daily until disease progression or unacceptable toxicity.

“BeOne is leading the advancement and enhancement of BCL2 inhibition to revolutionize how we treat patients living with B-cell malignancies,” Amit Agarwal, MD, PhD, chief medical officer of hematology at BeOne Medicines, said in a news release.⁶ “Today’s approval of Beqalzi represents critical progress for patients with mantle cell lymphoma and reinforces our strategy of building foundational medicines designed to raise the standard of care in B-cell malignancies.”

4. Datopotamab Deruxtecan Wins First-Line FDA Approval for Immunotherapy-Ineligible Triple-Negative Breast Cancer

The week after, the FDA approved datopotamab deruxtecan (Datroway; AstraZeneca/Daiichi Sankyo) as the first trophoblast cell-surface antigen 2 (TROP2)-directed antibody-drug conjugate (ADC) for first-line treatment of adults with unresectable or metastatic triple-negative breast cancer (TNBC) who are not candidates for PD-1 or PD-L1 inhibitor therapy.⁷

The approval, granted on May 26, marks the first time a targeted therapy has demonstrated a significant OS advantage over chemotherapy in this difficult-to-treat population. It was based on results from TROPION-Breast02 (NCT05374512), a global, randomized, open-label phase 3 trial of 644 patients with previously untreated, locally recurrent inoperable or metastatic TNBC who were not candidates for immunotherapy.

Those treated with datopotamab deruxtecan experienced significantly improved outcomes compared with chemotherapy, including a median PFS of 10.8 months vs 5.6 months, corresponding to a 43% reduction in the risk of disease progression or death (HR, 0.57; P < .0001).

Datopotamab deruxtecan also demonstrated a clinically meaningful OS benefit, extending median OS to 23.7 months compared with 18.7 months for chemotherapy. The antibody-drug conjugate more than doubled the ORR (63% vs 29%) and prolonged the median DOR (12.3 vs 7.1 months).

“As the first antibody drug conjugate to demonstrate a median OS of 2 years in the 1st-line metastatic setting of TNBC, Datroway has the potential to redefine the treatment landscape for these patients,” Ken Keller, global head of oncology business and president and CEO of Daiichi Sankyo, Inc., said in a statement.⁸

5. FDA Approves Durvalumab Plus BCG as First Immunotherapy Combo Regimen for High-Risk NMIBC

The FDA wrapped up the month by approving durvalumab (Imfinzi; AstraZeneca) in combination with Bacillus Calmette-Guérin (BCG) induction and maintenance therapy for adult patients with BCG-naive, high-risk NMIBC.⁹

The decision was based on positive results from the phase 3 POTOMAC trial (NCT03528694) and represents the first new treatment for this patient population in more than 30 years and the only approved immunotherapy combination regimen.

The trial evaluated whether adding durvalumab to standard BCG therapy could improve outcomes in patients with BCG-naive, high-risk NMIBC following transurethral resection of bladder tumors. The primary analysis showed that durvalumab combined with BCG induction and maintenance significantly improved disease-free survival. After a median follow-up of 60.7 months, the combination reduced the risk of high-risk disease recurrence or death by 32% compared with BCG alone (HR, 0.68; 95% CI, 0.50-0.93; P = .015).

In an interview with AJMC, Neal D. Shore, MD, a POTOMAC trial investigator, discussed how the regimen could shift the standard of care. He explained that the therapy “takes the brakes off of the immune system,” allowing white blood cells and T cells to better recognize antigens on cancer cells and exert an antineoplastic effect.

References

1. Hohmann E. FDA approves vepdegestrant for ESR1-mutated, ER-positive, HER2-negative advanced breast cancer. AJMC. May 4, 2026. Accessed June 5, 2026. https://www.ajmc.com/view/fda-approves-vepdegestrant-for-esr1-mutated-er-positive-her2-negative-advanced-breast-cancer
2. Arvinas announces FDA approval of VEPPANU (vepdegestrant) for the treatment of ESR1m, ER+/HER2- advanced breast cancer. News release. Arvinas Inc. May 1, 2026. Accessed June 5, 2026. https://ir.arvinas.com/news-releases/news-release-details/arvinas-announces-fda-approval-veppanu-vepdegestrant-treatment
3. Shaw ML. FDA approves first all-oral regimen for AML. AJMC. May 13, 2026. Accessed June 5, 2026. https://www.ajmc.com/view/fda-approves-first-all-oral-regimen-for-aml
4. Shaw ML, Larkin K. The first all-oral regimen for AML is a game changer: Karilyn Larkin, MD. AJMC. May 13, 2026. Accessed June 5, 2026. https://www.ajmc.com/view/the-first-all-oral-regimen-for-aml-is-a-game-changer-karilyn-larkin-md
5. Caffrey M. Sonrotoclax granted accelerated approval for R/R mantle cell lymphoma. AJMC. May 13, 2026. Accessed June 5, 2026. https://www.ajmc.com/view/sonrotoclax-granted-accelerated-approval-for-r-r-mantle-cell-lymphoma
6. BeOne Medicines’ Beqalzi (sonrotoclax) approved by US FDA as first and only BCL2 inhibitor for R/R mantle cell lymphoma. News release. BeOne. May 13, 2026. Accessed June 5, 2026. https://ir.beonemedicines.com/news/beone-medicines-beqalzitm-sonrotoclax-approved-by-us-fda-as-first-and-only-bcl2-inhibitor-for/a57256f1-5e36-427a-94c3-2b6be7f2bfb9
7. Hohmann E. Datopotamab deruxtecan wins first-line FDA approval for immunotherapy-ineligible triple-negative breast cancer. AJMC. May 22, 2026. Accessed May 22, 2026. https://www.ajmc.com/view/datroway-wins-first-line-fda-approval-for-immunotherapy-ineligible-triple-negative-breast-cancer
8. Datroway approved in the US as first TROP2-directed antibody drug conjugate for 1st-line treatment of patients with metastatic triple-negative breast cancer who are not PD-1/PD-L1 inhibitor candidates. News release. AstraZeneca. May 22, 2026. Accessed June 5, 2026. https://www.astrazeneca.com/media-centre/press-releases/2026/datroway-approved-in-us-for-1l-triple-negative-bc.html
9. McCormick B. FDA approves durvalumab plus BCG as first immunotherapy combo regimen for high-risk NMIBC. AJMC. May 29, 2026. Accessed June 5, 2026. https://www.ajmc.com/view/fda-approves-durvalumab-plus-bcg-as-first-immunotherapy-combo-regimen-for-high-risk-nmibc