Rx Insights from November '25

This month's edition provides a focused review of newly approved and recently launched therapies. Expion Health monitors these developments closely to assess clinical relevance, therapeutic positioning, and potential implications for payers, providers, and patients.

KYGEVVI

Indication: First and only approved therapy for thymidine kinase 2 deficiency (TK2d), an ultra-rare mitochondrial disorder.

Clinical Data: 82-patient program showed ~86% reduction in mortality vs untreated individuals; average survival at 10 years was 9.6 years vs 5.7 years in controls.

POHERDY (pertuzumab-dpzb)

Indication: Interchangeable biosimilar to Perjeta for HER2-positive breast cancer, used with trastuzumab across first-line, neoadjuvant, and adjuvant settings.

KOMZIFTI (ziftomenib)

Indication: Menin inhibitor for relapsed/refractory NPM1-mutated acute myeloid leukemia (AML) with no satisfactory alternative.

Clinical Data: KOMET-001 trial (n=112): composite complete remission rate of 21.4%.

REDEMPLO

Indication: ApoC-III-directed siRNA therapy reducing triglycerides in familial chylomicronemia syndrome (FCS).

Clinical Data: Phase 3 PALISADE trial: sustained triglyceride reductions of ~60% or more, with fewer pancreatitis episodes.

HYRNUO

Indication: Selective HER2 tyrosine kinase inhibitor for HER2-mutated non-small cell lung cancer (NSCLC), accelerated approval.

Clinical Data: ORR of 71% in TKI/ADC-naive patients and 38% in patients previously treated with a HER2-targeted ADC.

VOYXACT

Indication: First FDA-approved anti-APRIL antibody, reducing proteinuria in primary IgA nephropathy (IgAN).

Clinical Data: Phase 3 VISIONARY trial: 50% reduction in proteinuria vs 2% increase with placebo after 9 months.

ITVISMA

Indication: Gene-replacement therapy for spinal muscular atrophy (SMA) in patients 2 years and older.

Clinical Data: Phase 3 STEER trial: 1.88-point improvement in HFMSE motor function score vs placebo over 52 weeks.