Five Major FDA Decisions Expected in November 2025 Across Cancer, Rare Disease, and Genetic Disorders
核心洞察
The FDA is set to make five significant regulatory decisions in November 2025, including approvals for treatments targeting rare genetic disorders, multiple cancer types, and kidney disease.
Arrowhead Pharmaceuticals' plozasiran for familial chylomicronemia syndrome (搜索) demonstrated an 80% median reduction in triglyceride levels and 83% lower risk of acute pancreatitis in Phase III trials.
Bayer's sevabertinib (搜索) showed promising efficacy in HER2 (搜索)-mutant non-small cell lung cancer (搜索) with overall response rates ranging from 38% to 71% depending on prior treatment history.
The FDA faces a packed November 2025 schedule with five major regulatory decisions that could significantly impact treatment options across cancer, rare genetic disorders, and kidney disease. These decisions span innovative therapeutic approaches including RNA interference, bispecific antibodies, and targeted kinase inhibitors.
RNA Interference Breakthrough for Rare Genetic Disorder
Arrowhead Pharmaceuticals awaits FDA approval for plozasiran by November 18, marking a potential milestone for RNA interference therapy in treating familial chylomicronemia syndrome (搜索) (FCS). This rare genetic disorder affects 1 to 2 patients per million people and is characterized by the inability to break down fats, resulting in extremely high triglyceride levels often exceeding 1,000 mg/dL compared to normal concentrations below 150 mg/dL.
Plozasiran works by suppressing production of apolipoprotein C-III (APOC3), a crucial component of triglyceride-rich lipoproteins that prevents clearance of fatty molecules and raises blood triglyceride concentrations. In the Phase III PALISADE study, plozasiran treatment achieved an 80% median reduction in triglyceride levels and lowered the risk of acute pancreatitis by 83% compared with placebo.
Common side effects observed after initiating plozasiran treatment included COVID-19, abdominal pain, nasopharyngitis and nausea, though it remains unclear if these were definitively caused by the RNAi therapy. If approved, plozasiran would join Ionis Pharma (搜索)'s Tryngolza as treatment options for FCS, with Tryngolza generating $26 million in net product sales during the first half of 2025.
Targeted Therapy for HER2-Mutant Lung Cancer
Bayer's oral tyrosine kinase inhibitor sevabertinib (搜索) faces an FDA decision by November 28 for treating patients with non-small cell lung cancer (搜索) whose tumors are positive for HER2 (搜索)/ERBB2 (搜索) mutations. The FDA granted priority designation to the new drug application submitted on May 28.
Data from the ongoing Phase I/II SOHO-01 study, which administered 20-mg sevabertinib (搜索) twice-daily, showed varying efficacy based on prior treatment history. In patients who had previously undergone systemic therapy but not with HER2 (搜索) exon 20 insertion-targeting agents, sevabertinib demonstrated a 64% overall response rate with median progression-free survival of 8.3 months.
Patients who had previously received HER2 (搜索)-directed antibody-drug conjugates showed an overall response rate of 38% and median progression-free survival of 5.5 months. Treatment-naïve patients achieved the highest response rate of 71% and had not reached median progression-free survival at the time of data readout.
Competition in Achondroplasia Treatment
Ascendis Pharma's TransCon CNP (navepegritide) awaits FDA approval by November 30 for achondroplasia (搜索), the most common cause of dwarfism. This genetic disorder disrupts bone development, and Ascendis' solution supplies patients with C-type natriuretic peptide (CNP), a hormone that counteracts the disease's underlying genetic mutation and promotes bone development.
The Phase II ACcomplisH trial demonstrated that TransCon CNP improved annual growth velocity by 5.42 cm per year versus 4.35 cm per year in placebo counterparts, achieving statistical significance. Currently, BioMarin's Voxzogo represents the only approved therapy for achondroplasia (搜索), also working via the CNP pathway and generating $221 million in Q2 2025 revenue.
Industry analysts suggest that if approved, TransCon CNP will prove to be a "major threat" to Voxzogo's market position in treating the more than 250,000 people worldwide affected by achondroplasia (搜索).
Bispecific Antibody Expansion in Lymphoma
Genmab and AbbVie (搜索) seek to expand their bispecific antibody Epkinly into relapsed or refractory follicular lymphoma (搜索), with an FDA decision expected by November 30. Initially approved in 2023 for diffuse large B-cell lymphoma (搜索), Epkinly received additional approval for relapsed and refractory follicular lymphoma in 2024.
The Phase III EPCORE study supporting the expansion demonstrated a 95.7% objective response rate in patients treated with a regimen consisting of Epkinly, rituximab and lenalidomide. The combination also resulted in a 79% reduction in the risk of disease progression or death, with both treatment effects achieving high statistical significance (p-values less than 0.0001).
Administered via subcutaneous injection, Epkinly functions as a bispecific T cell engager targeting the CD3 (搜索) receptor on T cells and the CD20 (搜索) protein on malignant B cells. This mechanism activates the immune system's anti-cancer activity while physically bringing cancer-killing cells closer to their targets.
Novel Approach to Acute Myeloid Leukemia
Kura Oncology and Kyowa Kirin await FDA approval for ziftomenib by November 30, an investigational menin (搜索) blocker proposed for patients with relapsed or refractory acute myeloid leukemia (搜索) (AML) carrying NPM1 (搜索) gene mutations. These mutations, found in approximately 30% of AML cases, encode a protein crucial for regulating cell growth and division.
The Phase II KOMET-001 study found that 23% of patients treated with ziftomenib achieved complete remission or complete remission with partial hematological recovery, with median duration of these endpoints reaching 3.7 months. The treatment demonstrated favorable safety, with only 3% treatment-related dropouts.
AML represents the most common form of acute leukemia in adults, and patients with NPM1 (搜索) mutations suffer from high rates of relapse and poor survival. Currently, no FDA-approved therapies specifically target NPM1-mutant AML. Kura estimates a market opportunity of $350 million to $400 million per year in the U.S. for ziftomenib in relapsed or refractory NPM1-mutant AML if approved.