FDA Approvals in 2024: Advancing Treatment Paradigms in Solid Tumors and Hematologic Malignancies
核心洞察
The FDA granted over 65 approvals in 2024, significantly impacting treatment paradigms across various cancers, including breast, gynecologic, skin, and genitourinary malignancies.
Several tumor-agnostic approvals, such as fam-trastuzumab deruxtecan-nxki (Enhertu) for HER2 (搜索)-positive solid tumors and repotrectinib (Augtyro) for NTRK (搜索) fusion-positive tumors, marked advancements in precision medicine.
Immunotherapies like nivolumab (Opdivo) and pembrolizumab (Keytruda) received multiple approvals, including combinations with chemotherapy for urothelial and endometrial carcinomas, improving patient outcomes.
The year 2024 witnessed a surge in FDA approvals, bringing forth new treatment options and expanded indications across a spectrum of solid tumors and hematologic malignancies. These approvals signify a notable step forward in cancer care, offering hope for improved patient outcomes and addressing previously unmet medical needs.
Breast Cancer
In breast cancer, several key approvals have refined treatment strategies. Ribociclib (Kisqali) gained approval for adjuvant treatment in HR-positive, HER2 (搜索)-negative, high-risk early breast cancer. According to Dr. Bora Lim from the University of Texas MD Anderson Cancer Center, this approval expands opportunities for patients in the curative setting. Additionally, inavolisib (Itovebi) combined with palbociclib (Ibrance) and fulvestrant (Faslodex) was approved for endocrine-resistant, PIK3CA-mutated, HR-positive, HER2-negative locally advanced or metastatic breast cancer. Dr. Hope S. Rugo from UCSF Helen Diller Family Comprehensive Cancer Center highlighted the rapid translation of phase 3 INAVO120 trial data into clinical practice, offering valuable insights for treating these patients.
Gynecologic Cancers
Significant strides were also made in gynecologic cancers. Pembrolizumab (Keytruda) plus chemoradiotherapy was approved for FIGO 2014 stage III to IVA cervical cancer (搜索). Mirvetuximab soravtansine-gynx (Elahere) received approval for FRα-positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer after 1-3 prior systemic treatments. Dr. Gottfried Konecny from UCLA Health emphasized the unmet need in platinum-resistant ovarian cancer (搜索) and the potential of mirvetuximab soravtansine to become a new standard of care. Tisotumab vedotin-tftv (Tivdak) was approved for recurrent or metastatic cervical cancer progressing after chemotherapy. Furthermore, durvalumab (Imfinzi) and pembrolizumab, both in combination with chemotherapy, and dostarlimab-gxly (Jemperli), were approved or had their indications expanded for primary advanced or recurrent endometrial cancer (搜索). Dr. Krishnansu S. Tewari from the University of California, Irvine School of Medicine, noted the full approval of dostarlimab based on strong survival data from the RUBY trial.
Tumor-Agnostic Approvals
Several tumor-agnostic approvals underscored the growing importance of precision medicine. Fam-trastuzumab deruxtecan-nxki (Enhertu) received accelerated approval for HER2 (搜索)-positive (IHC 3+) solid tumors with no satisfactory alternative options. Dr. Ronan J. Kelly from Baylor Scott & White Charles A. Sammons Cancer Center described this as a "game-changing" approval, validating precision medicine approaches. Repotrectinib (Augtyro) was granted accelerated approval for adult and pediatric patients (12+) with NTRK (搜索) gene fusion-positive solid tumors who have progressed after prior treatment or lack satisfactory alternatives.
Genitourinary Cancers
The genitourinary (GU) cancer space also benefited from key approvals. Nivolumab (Opdivo) combined with cisplatin and gemcitabine was approved as a first-line treatment for unresectable or metastatic urothelial carcinoma (搜索). Erdafitinib (Balversa) received full approval for FGFR3 (搜索)-altered urothelial carcinoma after prior systemic therapy, amending its previous accelerated approval. Additionally, nogapendekin alfa inbakicept-pmln (Anktiva) plus BCG was approved for BCG-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ.
Gastrointestinal Cancers
In gastrointestinal (GI) cancers, zolbetuximab-clzb (搜索) (Vyloy) was approved for HER2 (搜索)-negative gastric or gastroesophageal junction (GEJ) adenocarcinoma with CLDN18.2 (搜索)-positive tumors, marking the first CLDN18.2-targeted therapy. Lutetium Lu 177 dotatate (Lutathera) received expanded approval for pediatric patients (12+) with somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors (搜索) (NETs). Adagrasib (Krazati) plus cetuximab (Erbitux) gained accelerated approval for KRAS G12C (搜索)-mutant locally advanced or metastatic colorectal cancer (搜索) (CRC) after prior chemotherapy. Tislelizumab-jsgr (Tevimbra) also received approval for unresectable or metastatic esophageal squamous cell carcinoma (搜索) following prior systemic chemotherapy that did not include a PD-(L)1 inhibitor.
Other Notable Approvals
Other significant approvals include lifileucel (Amtagvi), the first tumor-infiltrating lymphocyte (TIL) therapy, for melanoma (搜索); tovorafenib (Ojemda) for relapsed or refractory low-grade glioma (搜索) with BRAF (搜索) alterations; selpercatinib (Retevmo) for various RET (搜索)-altered thyroid and solid tumors; afamitresgene autoleucel (Tecelra) for synovial sarcoma (搜索); vorasidenib (Voranigo) for IDH1 (搜索)/2-mutated low-grade glioma; cosibelimab-ipdl (搜索) (Unloxcyt (搜索)) for cutaneous squamous cell carcinoma (搜索); axatilimab-csfr (Niktimvo) for chronic graft-vs-host disease (搜索) (cGVHD); and subcutaneous formulations of atezolizumab (Tecentriq Hybreza) and nivolumab (Opdivo Qvantig) for various indications.
These FDA approvals in 2024 reflect a dynamic and evolving landscape in cancer therapy, characterized by targeted treatments, immunotherapies, and innovative approaches that aim to improve patient outcomes and quality of life.