Oncology Landscape Continues to Evolve With Expanded Indications, First-in-Class Therapies

Experts concluded the Academy of Managed Care Pharmacy (AMCP) Nexus 2025 meeting in National Harbor, Maryland, by emphasizing how new indications within existing drug classes, along with the introduction of novel agents, are driving more targeted, accessible, and value-driven cancer care.¹

Before adjourning last Thursday, the final keynote session, “Oncology Pipeline 2025: Emerging Breakthroughs and Trends,” featured experts Kaelyn C. Boss, PharmD, and Sita Bhatt, PharmD, BCOP, who highlighted recent and upcoming FDA approvals in oncology.

Evolving Oncology Spending Trends, Drug Development Landscape

Boss opened the session with an overview of recent trends in oncology pharmaceutical spending and drug development. She noted that global oncology spending reached $252 billion in 2024 and is projected to climb to $441 billion by 2029.

“This increase in spending is driven by the increase in access but also by the use of branded and novel medications,” Boss said.

She added that the cost of novel therapies remains high, with the median price of new oncology agents launched in 2024 being $411,855.

“Uptake of biosimilars and generic medication should provide some opportunities for savings within the class, but it’s unlikely to really offset those rising costs,” Boss said.

As of September 15, 2025, there have been 30 oncology FDA approvals this year, including 7 accelerated approvals and 1 biosimilar, down from 45 approvals in 2024. Despite this decrease, she noted that the oncology pipeline shows no signs of slowing down, with about 1600 treatments and vaccines currently in development.

Boss also highlighted that the initiation of new oncology trials increased last year and is up 12% from 2019, with 74% evaluating medicines for rare cancers and 79% focused on solid tumors. Antibody-drug conjugates (ADCs) are the fastest-growing modality in solid tumors, she added, with a 32% annual increase in the number of related clinical trials initiated.

Boss and Bhatt then reviewed FDA approvals in the oncology space since the AMCP Nexus meeting in Las Vegas last October, underscoring the expansion of existing drug classes and the introduction of novel agents.

Recent FDA Approvals Expand ADCs, Bispecific Antibodies Across Cancer Types

Recent FDA approvals have expanded several existing drug classes, including ADCs, which combine monoclonal antibodies with chemotherapy drugs to directly target cancer cells while minimizing damage to healthy tissues.

Among the latest ADC approvals is datopotamab deruxtecan-dlnk (Datroway; Daiichi Sankyo, Inc), granted on January 17, 2025, for adult patients with HR-positive, HER2-negative breast cancer who have previously received endocrine-based therapy and chemotherapy.² Later that year, on June 23, the FDA approved datopotamab deruxtecan for adults with locally advanced or metastatic EGFR-mutated non–small cell lung cancer (NSCLC) who have previously received EGFR-directed therapy and platinum-based chemotherapy.³

Another ADC approved in the NSCLC space is telisotuzumab vedotin-tllv (Emrelis; AbbVie), also known as Teliso-V. On May 14, 2025, it was approved for adult patients with locally advanced or metastatic nonsquamous NSCLC with high c-Met protein overexpression who have received a prior systemic treatment.⁴

Bispecific antibodies have also expanded their indications. For example, linvoseltamab-gcpt (Lynozyfic; Regeneron), a bispecific B-cell maturation antigen–directed CD3 T-cell engager, received FDA approval on July 2, 2025, to treat adults with relapsed or refractory multiple myeloma who have undergone at least 4 prior lines of therapy.⁵

Other bispecific antibody expansions include zanidatamab-hrii (Ziihera; Jazz Pharmaceuticals), a HER2-directed bispecific antibody approved in November 2024 for HER2-positive biliary tract cancer, and zenocutuzumab-zbco (Bizengri; Merus), a HER2- and HER3-directed bispecific antibody approved in December 2024 for NSCLC or pancreatic adenocarcinoma with an NRG1 gene fusion.^6,7

Emerging First-in-Class Oncology Therapies

In contrast, the experts noted that several recent FDA approvals introduced first-in-class therapies to the oncology landscape.¹ Among these are menin inhibitors, which block the interaction between menin and other proteins to prevent activation of genes that drive cell growth, ultimately inhibiting cancer cell proliferation.

Bhatt highlighted that the first menin inhibitor, revumenib (Revuforj; Syndax Pharmaceuticals), received FDA approval in November 2024 to treat relapsed or refractory acute leukemia with a KMT2A translocation in patients 1 year and older.⁸

Last week, it gained an additional indication for relapsed or refractory acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation if there are no other satisfactory treatment options.⁹ Bhatt added that the FDA is currently reviewing another menin inhibitor, ziftomenib, for the same patient population, with a Prescription Drug User Fee Act (PDUFA) date of November 30, 2025.¹

Other menin inhibitors currently in clinical development include bleximenib and enzomenib, both in phase 2/3 trials, which are being investigated for KMT2A-rearranged, NPM1-mutated acute leukemia. Additionally, there are several early-stage agents, namely BN104, balamenib, and icovamenib, that are in phase 1 trials for various hematologic malignancies.

Bhatt further highlighted the approval of novel posttransplant cellular therapies, representing a “new frontier” in extending advanced cellular therapy beyond cancer remission to address severe posttransplant complications that were previously untreatable.

Approved in December 2024, remestemcel-L-rknd (Ryoncil; Mesoblast, Inc.) was indicated for steroid-refractory acute graft-vs-host disease in pediatric patients aged 2 months or older.¹⁰ Bhatt noted that it is the first approved mesenchymal stromal cell therapy derived from allogeneic bone marrow and the first treatment approved for patients younger than 12 years affected by this disease.¹ However, she underscored that costs remain significant, estimated at $194,000 per infusion, which could limit access for this population.

Another posttransplant cellular therapy under review, tabelecleucel (Ebvallo; Atara Biotherapeutics), has a PDUFA date of January 10, 2026. If approved, it would become the first allogeneic, non-genetically modified T-cell therapy for Epstein-Barr virus–positive posttransplant lymphoproliferative disease. As an “off-the-shelf” therapy, Bhatt underscored that tabelcleucel could offer rapid treatment for highly immunosuppressed patients requiring urgent treatment.

The Times They Are A-Changin’

Bhatt concluded the session by emphasizing that the oncology pipeline continues to accelerate, driven by expanded indications and first-in-class therapies. She noted that drug development now increasingly prioritizes convenience and access, with subcutaneous, oral, and off-the-shelf options challenging traditional infusion-based models.

Additionally, Bhatt underscored the importance of smarter benefit design, site-of-care strategies, and outcomes-based contracts as high-cost therapies target smaller patient populations. Therefore, she noted that collaboration among clinicians, payers, and manufacturers is needed to not only manage spending but also to ensure timely patient access to these “transformative therapies.”

“…Innovation is accelerating, but so is our approach to policy, contracting, and care integration,” Bhatt concluded. “As this 2025 pipeline shows, what’s emerging today will redefine oncology access and value discussions for the next decade.”

References

1. Pamlowski P, Bhatt S, Boss K. Oncology pipeline 2025: emerging breakthroughs and trends. Presented at: AMCP Nexus 2025; October 27-30, 2025; National Harbor, MD.
2. Steinzor P. FDA approves datopotamab deruxtecan for HR-positive/HER2-negative metastatic breast cancer. AJMC^®. January 17, 2025. Accessed November 5, 2025. https://www.ajmc.com/view/fda-approves-datopotamab-deruxtecan-for-hr-positive-her2-negative-metastatic-breast-cancer
3. Steinzor P. FDA approves datopotamab deruxtecan for EGFR-mutated NSCLC. AJMC. June 23, 2025. Accessed November 5, 2025. https://www.ajmc.com/view/fda-approves-datopotamab-deruxtecan-for-egfr-mutated-nsclc
4. Klein HE. Teliso-V approved to treat advanced NSCLC with high c-Met protein overexpression. AJMC. May 14, 2025. Accessed November 5, 2025. https://www.ajmc.com/view/teliso-v-approved-to-treat-advanced-nsclc-with-high-c-met-protein-overexpression
5. Klein HE. FDA approves linvoseltamab to treat R/R multiple myeloma. AJMC. July 2, 2025. Accessed November 5, 2025. https://www.ajmc.com/view/fda-approves-linvoseltamab-to-treat-r-r-multiple-myeloma
6. Santoro C. FDA approves zanidatamab-hrii for HER2+ biliary tract cancer. AJMC. November 21, 2024. Accessed November 5, 2025. https://www.ajmc.com/view/fda-approves-zanidatamab-hrii-for-her2-biliary-tract-cancer
7. FDA grants accelerated approval to zenocutuzumab-zbco for non-small cell lung cancer and pancreatic adenocarcinoma. FDA. News release. December 4, 2024. Accessed November 5, 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-zenocutuzumab-zbco-non-small-cell-lung-cancer-and-pancreatic
8. Rosa K. FDA approves revumenib for R/R acute leukemia with a KMT2A translocation. AJMC. November 15, 2024. Accessed November 5, 2025. https://www.ajmc.com/view/fda-approves-revumenib-for-r-r-acute-leukemia-with-a-kmt2a-translocation
9. Joszt L. Revumenib granted FDA approval for R/R NPM1-mutated AML. AJMC. October 24, 2025. Accessed November 5, 2025. https://www.ajmc.com/view/revumenib-granted-fda-approval-for-r-r-npm1-mutated-aml
10. FDA approves remestemcel-L-rknd for steroid-refractory acute graft versus host disease in pediatric patients. FDA. News release. December 18, 2024. Accessed November 5, 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-remestemcel-l-rknd-steroid-refractory-acute-graft-versus-host-disease-pediatric