One of the FDA’s potential approvals this month could break an existing monopoly in the treatment space for a rare growth disorder.
There are five big regulatory events on the FDA’s docket this month, including one decision for an RNA interference therapy and target action dates for drugs to treat three different types of cancer.
Read below for more.
Arrowhead Proposes RNAi Therapy for Rare Genetic Disorder
By Nov. 18, the FDA is expected to release its decision on Arrowhead Pharmaceuticals’ plozasiran, an investigational RNA interference treatment for familial chylomicronemia syndrome (FCS).
Affecting around 1 to 2 patients per million people, FCS is a rare, genetic disorder characterized by the inability to break down fats, resulting in extremely high triglyceride levels, often exceeding 1,000 mg/dL where normal concentrations are typically lower than 150 mg/dL. Patients with this condition can develop acute pancreatitis, which, if left unchecked, can be fatal.
Plozasiran addresses the unchecked lipid levels in FCS by suppressing the production of the apolipoprotein C-III (APOC3) molecule, a crucial component of triglyceride-rich lipoproteins. APOC3 also prevents the clearance of these fatty molecules, in turn raising blood triglyceride concentrations. In the Phase III PALISADE study, plozasiran treatment led to an 80% median reduction in triglyceride levels and lowered the risk of acute pancreatitis by 83%, as compared with placebo.
Common side effects observed after initiating plozasiran treatment included COVID-19, abdominal pain, nasopharyngitis and nausea, though it is unclear if these were definitively caused by the RNAi therapy.
Bayer Targets Approval in HER2-Mutant NSCLC
Bayer is proposing its oral tyrosine kinase inhibitor sevabertinib for the treatment of patients with non-small cell lung cancer whose tumors are positive for HER2/ERBB2 mutations.
The FDA accepted the pharma’s new drug application (NDA) on May 28 and gave it priority designation, suggesting that a verdict could come on or before Nov. 28, barring delays.
To back its regulatory bid, Bayer submitted data from the Phase I/II SOHO-01 study, an ongoing open-label study that administered 20-mg sevabertinib twice-daily. Data presented last month at the 2025 congress of the European Society for Medical Oncology showed a 64% overall response rate (ORR) in patients who had previously undergone systemic therapy but not with agents that specifically target HER2 exon 20 insertion. Median progression-free survival (PFS) in this subpopulation was 8.3 months.
Meanwhile, in patients who had previously received HER2-directed antibody-drug conjugates, sevabertinib had an ORR of 38% and a median PFS of 5.5 months. Patients who were completely naïve to systemic therapies saw an ORR of 71% and had not reached median PFS at the time of the readout.
Ascendis Approaches Achondroplasia Arena With TransCon CNP
Looking to compete in the currently cornered achondroplasia market is Ascendis Pharma, which is proposing its C-type natriuretic peptide (CNP) analog TransCon CNP for the disorder. The FDA is expected to release its decision by Nov. 30.
Achondroplasia, the most common cause of dwarfism, is a genetic disorder that disrupts bone development. Ascendis’ solution is to supply patients with CNP, a hormone that counteracts the disease’s underpinning genetic mutation and promotes bone development.
There is currently only one approved therapy for achondroplasia: BioMarin’s Voxzogo, which also works via the CNP pathway.
To support its application, Ascendis used data from the Phase II ACcomplisH trial, which found that TransCon CNP improved annual growth velocity by 5.42 cm per year, versus 4.35 cm per year in placebo counterparts—an effect that met statistical significance.
If approved, TransCon CNP will prove to be a “major threat” to Voxzogo, Sadaf Javed, manager of Forecasting and Analytics at DelveInsight, told BioSpace in an email interview last month.
AbbVie, Genmab Eye Epkinly Expansion
After its initial approval in 2023 for diffuse large B-cell lymphoma, Genmab and AbbVie are now working to expand its bispecific antibody Epkinly into relapsed or refractory follicular lymphoma. The application is currently under review with a target action date of Nov. 30.
Supporting the expansion bid are data from the Phase III EPCORE study, which demonstrated a 95.7% objective response rate in patients treated with a regimen consisting of Epkinly, rituximab and lenalidomide. The combo also resulted in a 79% reduction in the risk of disease progression or death. Both treatment effects were highly statistically significant, with p-values less than 0.0001, respectively.
Given via a subcutaneous injection, Epkinly is a bispecific T cell engager that works by targeting the CD3 receptor found on T cells and the CD20 protein expressed on malignant B cells. This mechanism of action not only allows Epkinly to activate the immune system’s anti-cancer activity but also physically brings the cancer-killing cells closer to their targets.
Genmab and AbbVie are collaborating on Epkinly under a 2020 contract. Aside from its initial 2023 approval, the FDA also cleared Epkinly for relapsed and refractory follicular lymphoma in 2024.
Kura Oncology, Kyowa Kirin Await AML Verdict
Completing the FDA’s trio of decisions on Nov. 30 is Kura Oncology and Kyowa Kirin’s ziftomenib, an investigational menin blocker being proposed for use in patients with relapsed or refractory acute myeloid leukemia (AML).
In particular, the partners are looking to zero in on patients carrying mutations in the NPM1 gene, which encodes a protein that plays a crucial function in regulating cell growth and division. According to the companies, AML is the most common form of acute leukemia in adults, of which 30% of cases can be traced back to NPM1 mutations. Patients with this genetic anomaly suffer from high rates of relapse and poor survival.
To support their application, Kura and Kyowa Kirin filed findings from the Phase II KOMET-001 study, which found that 23% of patients treated with ziftomenib achieved complete remission or complete remission with partial hematological recovery. Median duration of these endpoints was 3.7 months. Ziftomenib was also safe overall, with only 3% treatment-related dropouts.
If approved, ziftomenib will follow in the footsteps of Syndax Pharmaceuticals’ Revuforj, which last month won the FDA’s nod for the same indication.
