The FDA and the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) have approved or recommended a wave of new drugs that are set to transform cancer treatment. These approvals span across various oncological areas, including innovative therapies for solid tumors and hematological cancers. Let’s dive into these newly approved or recommended drugs below.
Approvals by the FDA
On May 29, 2024, selpercatinib was approved for pediatric patients aged two years and older, with the following:
- Advanced or metastatic medullary thyroid cancer (MTC) with a RET mutation, as detected by an FDA-approved test, requiring systemic therapy.
- Advanced or metastatic thyroid cancer with a RET gene fusion, as detected by an FDA-approved test, requiring systemic therapy and refractory to radioactive iodine (if appropriate).
- Locally advanced or metastatic solid tumors with a RET gene fusion, as detected by an FDA-approved test, that have progressed following prior systemic treatment or have no satisfactory alternative treatment options.
The efficacy was measured in the LIBRETTO-121 study, an international, single-arm, multi-cohort trial which demonstrated an overall response rate (ORR) of 48%.
On May 30, 2024, lisocabtagene maraleucel was approved for adult patients with relapsed or refractory mantle cell lymphoma (MCL) who have received at least two prior lines of therapy, including a Bruton tyrosine kinase inhibitor. The approval came after evaluating the efficacy of lisocabtagene maraleucel in the TRANSCEND-MCL trial.
The trial showed promising results, with an ORR of 85.3% and a complete response rate of 67.6%.
Recommendations by the CHMP
Osimertinib with the addition of pemetrexed and platinum-based chemotherapy has been recommended for approval in the European Union (EU) for 1st-line treatment of adult patients with locally advanced or metastatic epidermal growth factor receptor-mutated (EGFRm) non-small cell lung cancer (NSCLC) whose tumors have exon 19 deletions or exon 21 (L858R) mutations.
Recommendation is based upon the results of the FLAURA2 Phase III trial, which demonstrated a longer progression-free survival with osimertinib-chemotherapy versus osimertinib alone.
Sugemalimab has been recommended for the treatment of adults with metastatic non-small-cell lung cancer. This recommendation is based on the results of a Phase 3 GEMSTONE-302 trial, which demonstrated significant PFS and OS benefits of sugemalimab in combination with chemotherapy as a first-line treatment for NSCLC.
References:
https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-selpercatinib-pediatric-patients-two-years-and-older-ret-altered
https://ascopubs.org/doi/10.1200/JCO.2024.42.16_suppl.10022
https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-lisocabtagene-maraleucel-relapsed-or-refractory-mantle-cell-lymphoma
https://ascopubs.org/doi/10.1200/JCO.23.02214
https://www.astrazeneca.com/media-centre/press-releases/2024/tagrisso-plus-chemo-recommended-for-approval-in-eu.html#!
https://www.nejm.org/doi/full/10.1056/NEJMoa2306434
https://www.cstonepharma.com/en/html/news/3702.html
Viktor Grünwald
