FDA Approvals and Designations in Oncology: September 2024
核心洞察
The FDA approved a subcutaneous formulation of atezolizumab (Tecentriq Hybreza) for various cancers, offering a more convenient administration option for patients.
Ribociclib (Kisqali) gained approval for early-stage breast cancer (搜索), expanding treatment options for this disease and showing improved invasive disease-free survival.
Pembrolizumab (Keytruda) in combination with chemotherapy was approved for unresectable advanced or metastatic malignant pleural mesothelioma (搜索), improving overall survival.
The FDA made significant strides in oncology during September 2024, approving new treatments and granting designations that promise to improve patient outcomes across a range of cancers. These approvals span new formulations, expanded indications, and novel therapies, reflecting the agency's commitment to advancing cancer care.
Subcutaneous Atezolizumab Approved for Multiple Cancers
Atezolizumab (Tecentriq Hybreza), a PD-L1 inhibitor, received approval as a subcutaneous formulation for all adult indications of the intravenous version. This includes non-small cell lung cancer (NSCLC (搜索)), small cell lung cancer (SCLC (搜索)), hepatocellular carcinoma (搜索) (HCC), melanoma (搜索), and alveolar soft part sarcoma. The approval offers a more convenient administration route for patients, with preliminary data showing that over 70% of patients preferred the subcutaneous formulation to the intravenous infusion.
Ribociclib Approved for Early-Stage Breast Cancer
Ribociclib (Kisqali), a kinase inhibitor, was approved for adjuvant treatment in hormone receptor-positive, HER2-negative stage II and III early breast cancer (搜索) at high risk of recurrence. The approval was supported by data from the phase 3 NATALEE trial (NCT03701334), which demonstrated a significant improvement in invasive disease-free survival (iDFS) with the combination of ribociclib and an aromatase inhibitor. The 36-month iDFS rates were 90.7% in the ribociclib arm and 87.6% in the aromatase inhibitor alone arm (HR, 0.749; 95% CI, 0.628-0.892).
Pembrolizumab Approved for Malignant Pleural Mesothelioma
The FDA approved pembrolizumab (Keytruda) in combination with pemetrexed and platinum chemotherapy as a first-line treatment for unresectable advanced or metastatic malignant pleural mesothelioma (搜索). This approval was based on the phase 2/3 KEYNOTE-483 trial (NCT02784171), which showed a significant overall survival (OS) benefit with the addition of pembrolizumab to chemotherapy (median OS: 17.3 months vs 16.1 months; HR, 0.79; 95% CI, 0.64-0.98; P = .0162).
Amivantamab Approved for NSCLC with EGFR Exon 19 Deletions or Exon 21 L858R Substitution Mutations
Amivantamab-vmjw (Rybrevant) plus carboplatin and pemetrexed were approved for the treatment of locally advanced or metastatic NSCLC (搜索) with EGFR (搜索) exon 19 deletions or exon 21 L858R substitution mutations. The phase 3 MARIPOSA-2 trial (NCT04988295) supported this approval, demonstrating a significant improvement in progression-free survival (PFS) with the amivantamab combination compared to chemotherapy alone (median PFS: 6.3 months vs 4.2 months; HR, 0.48; 95% CI, 0.36-0.64; P < .0001).
Isatuximab Approved for Transplant-Ineligible Multiple Myeloma
Isatuximab (Sarclisa) plus bortezomib, lenalidomide, and dexamethasone (VRd) was approved for the treatment of patients with transplant-ineligible, newly diagnosed multiple myeloma (搜索). Data from the phase 3 IMROZ trial (NCT03319667) showed that Isa-VRd improved PFS compared to VRd alone (HR, 0.596; 98.5% CI, 0.406-0.876; P < .001).
Osimertinib Approved for Stage III EGFR-Mutated NSCLC
Osimertinib (Tagrisso) was approved for the treatment of patients with stage III EGFR (搜索)-mutated NSCLC (搜索) whose disease did not progress during or following concurrent or sequential platinum-based chemoradiation. The approval was supported by the phase 3 LAURA trial (NCT03521154), which demonstrated a significant improvement in PFS with osimertinib compared to placebo (median PFS: 39.1 months vs 5.6 months; HR, 0.16; 95% CI, 0.10-0.24; P < .001).
Selpercatinib Receives Traditional Approval for Medullary Thyroid Cancer
Selpercatinib (Retevmo) received traditional approval for the treatment of adult and pediatric patients aged 2 years and older with advanced or metastatic medullary thyroid cancer (搜索) with a RET (搜索) mutation. The phase 3 LIBRETTO-531 study (NCT04211337) supported this approval, demonstrating improved PFS with selpercatinib compared to physician's choice of cabozantinib or vandetanib (HR, 0.280; 95% CI, 0.165-0.475; P < .0001).
Fast Track Designations
Several agents received fast track designation, including OBX-115 for unresectable or metastatic melanoma (搜索) resistant to immune checkpoint inhibitor therapy, IBI363 for unresectable locally advanced or metastatic melanoma, and 212Pb VMT01 for unresectable or metastatic melanoma with MC1R tumor expression. EO-3021 also received fast track designation in advanced or metastatic gastric and gastroesophageal junction (GEJ) cancer expressing Claudin18.2 that has progressed on or after prior therapy.
Orphan Drug Designations
ABD-147 received orphan drug designation for the treatment of neuroendocrine carcinoma (搜索), certepetide for cholangiocarcinoma (搜索), tebapivat for myelodysplastic syndromes (搜索)-associated anemia, and elraglusib for soft tissue sarcoma (搜索). CF33-hNIS (Vaxinia) also earned orphan drug designation for the treatment of patients with cholangiocarcinoma.
Regenerative Medicine Advanced Therapy (RMAT) Designations
OBX-115, a novel TIL therapy, was granted RMAT designation in unresectable or metastatic melanoma (搜索) resistant to immune checkpoint inhibitor therapy. P-BCMA-ALLO1 also received RMAT designation for the potential treatment of relapsed/refractory (R/R) multiple myeloma (搜索).