Oncology Drugs Approved by the FDA in June 2024

Throughout the month of June, several cancer therapies have received FDA approval for diseases including thyroid cancer, myelodysplastic syndrome (MDS), colorectal cancer (CRC), endometrial cancer, and follicular lymphoma (FL).

Here is a select list of oncology drugs that have been approved this past month.

Imetelstat Gets FDA Approval for Low- to Intermediate-1-Risk MDS With Transfusion-Dependent Anemia

The FDA approved imetelstat (Rytelo) for treating adults with low- to intermediate-1-risk MDS and transfusion-dependent anemia who need 4 or more red blood cell (RBC) units every 8 weeks and are unresponsive to erythropoiesis-stimulating agents.^1-3 The approval is based on the phase 3 IMerge study (NCT02598661), which demonstrated a significant improvement in RBC transfusion independence (RBC-TI) rates for patients receiving imetelstat compared to placebo. Specifically, 39.8% of patients on imetelstat achieved 8-week RBC-TI vs 15% on placebo (P < .001). Additionally, imetelstat showed notable rates of 16-, 24-, and 52-week RBC-TI at 31.4%, 28.0%, and 13.6%, respectively.

Common adverse events with imetelstat included cytopenias, necessitating interventions like myeloid growth factors and platelet transfusions. The approval of imetelstat, a first-in-class telomerase inhibitor, offers a new therapeutic option for patients with MDS.

FDA Grants Traditional Approval to Selpercatinib for Advanced/Metastatic RET Fusion-Positive Thyroid Cancer

The FDA granted traditional approval to selpercatinib (Retevmo) for treating advanced or metastatic RET fusion-positive thyroid cancer in patients aged 2 years and older who require systemic therapy and whose disease is refractory to radioactive iodine.⁴

This approval is based on the LIBRETTO-001 trial (NCT03157128), which included 65 patients. The overall response rate (ORR) was 85% in previously treated patients and 96% in systemic therapy-naïve patients. The median duration of response (DOR) was 26.7 months and not evaluable, respectively.

Common adverse reactions included diarrhea, edema, and fatigue. The recommended dose varies by age, based on body surface area or weight.

FDA Approves Durvalumab-Chemo Regimen for Advanced or Recurrent dMMR Endometrial Cancer

The FDA approved durvalumab (Imfinzi) with carboplatin and paclitaxel, followed by durvalumab monotherapy, for primary advanced or recurrent endometrial cancer that is mismatch repair deficient (dMMR).⁵

This decision is based on the phase 3 DUO-E trial (NCT04269200), where patients with dMMR endometrial cancer (n = 95) had a median progression-free survival (PFS) that was not reached (95% CI, NR-NR) compared with 7 months (95% CI, 6.7-14.8) for the placebo arm (HR, 0.42; 95% CI, 0.22-0.80).

Common adverse effects included peripheral neuropathy, musculoskeletal pain, nausea, and fatigue. The recommended durvalumab dose varies based on body weight.

FDA Approves Pembrolizumab Plus Chemo for Advanced or Recurrent Endometrial Cancer

The FDA approved pembrolizumab (Keytruda) with carboplatin and paclitaxel, followed by pembrolizumab monotherapy, for adult patients with primary advanced or recurrent endometrial carcinoma.⁶

The approval is based on the KEYNOTE-868/NRG-GY018 trial (NCT03914612), a multicenter, randomized, double-blind, placebo-controlled trial involving 810 patients. Patients were grouped by MMR status: 588 with proficient MMR (pMMR) and 222 with dMMR. The primary efficacy measure was PFS. For the dMMR cohort, median PFS was not reached with pembrolizumab plus chemotherapy, compared to 6.5 months with placebo (HR = 0.30; P < .0001). For the pMMR cohort, median PFS was 11.1 months vs 8.5 months (HR = 0.60; P < .0001).

Common adverse reactions included rash. The recommended pembrolizumab dose is 200 mg every 3 weeks or 400 mg every 6 weeks.

FDA Approves Adagrasib for KRAS G12C-Mutated Metastatic CRC

The FDA granted accelerated approval to adagrasib (Krazati) with cetuximab (Erbitux) for adults with KRAS G12C-mutated locally advanced or metastatic CRC previously treated with oxaliplatin-, fluoropyrimidine-, and irinotecan-based chemotherapy.^7-8

Approval is based on the KRYSTAL-1 trial, which showed a 34% ORR with a median DOR of 5.8 months. Common adverse reactions included nausea, rash, vomiting, and diarrhea. Adagrasib is dosed at 600 mg orally twice daily.

FDA Approves Epcoritamab for Relapsed/Refractory FL

The FDA granted accelerated approval to epcoritamab-bysp (Epkinly) for adults with relapsed or refractory FL after 2 or more lines of systemic therapy.^9-10 Approval is based on the EPCORE NHL-1 trial (Study GCT3013-01; NCT03625037), which showed an 82% ORR and a 60% complete response rate. The median DOR was not reached, with a 12-month DOR rate of 68.4%.

Epcoritamab was administered using a step-up dosing schedule and continued until disease progression or intolerable toxicity. Common adverse events included cytokine release syndrome, injection site reactions, and fatigue. Serious toxicities occurred in 66% of patients, with 9% experiencing fatal adverse events.

References

1. FDA approves imetelstat for low- to intermediate-1 risk myelodysplastic syndromes with transfusion-dependent anemia. FDA. June 6, 2024. Accessed June 6, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-imetelstat-low-intermediate-1-risk-myelodysplastic-syndromes-transfusion-dependent
2. March 14, 2024, Meeting of the Oncologic Drugs Advisory Committee (ODAC). FDA. https://www.youtube.com/watch?v=Om-cKKBsMS4
3. Zeidan AM, Platzbecker U, Santini V, et al. IMerge: results from a phase 3, randomized, double-blind, placebo-controlled study of imetelstat in patients (pts) with heavily transfusion dependent (TD) non-del(5q) lower-risk myelodysplastic syndromes (LR-MDS) relapsed/refractory (R/R) to erythropoiesis stimulating agents (ESA). J Clin Oncol. 2023;41(suppl 16):7004. doi:10.1200/JCO.2023.41.16_suppl.7004
4. FDA approves selpercatinib for RET fusion-positive thyroid cancer. News release. FDA. June 12, 2024. Accessed June 12, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-selpercatinib-ret-fusion-positive-thyroid-cancer
5. FDA approves durvalumab with chemotherapy for mismatch repair deficient primary advanced or recurrent endometrial cancer. FDA. June 14, 2024. Accessed June 14, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-durvalumab-chemotherapy-mismatch-repair-deficient-primary-advanced-or-recurrent
6. FDA approves pembrolizumab with chemotherapy for primary advanced or recurrent endometrial carcinoma. News release. FDA. June 17, 2024. Accessed June 17, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-pembrolizumab-chemotherapy-primary-advanced-or-recurrent-endometrial-carcinoma
7. FDA grants accelerated approval to adagrasib with cetuximab for KRAS G12C-mutated colorectal cancer. News release. FDA. June 21, 2024. Accessed June 21, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-adagrasib-cetuximab-kras-g12c-mutated-colorectal-cancer
8. Adagrasib. Mirati Therapeutics. June 2024. Accessed June 21, 2024. https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/216340s005lbl.pdf
9. FDA grants accelerated approval to epcoritamab-bysp for relapsed or refractory follicular lymphoma. FDA. June 26, 2024. Accessed June 26, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-epcoritamab-bysp-relapsed-or-refractory-follicular-lymphoma
10. Epkinly. Prescribing information. Genmab US, Inc; 2024. Accessed June 26, 2024. https://www.genmab-pi.com/prescribing-information/epkinly-pi.pdf