Rx Insights from March '26

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.

YUVIWEL (navepegritide)

Indication: FDA approved to increase linear growth in pediatric patients aged 2 years and older with achondroplasia and open epiphyses.

Clinical Data: Phase 3 APPROACH trial. Annualized growth velocity: YUVIWEL 5.89 cm/year vs Placebo 4.41 cm/year (P < 0.001).

ICOTYDE (icotrokinra)

Indication: Treatment of moderate-to-severe plaque psoriasis in adults and pediatric patients ≥12 years weighing at least 40 kg. First oral IL-23R antagonist.

Clinical Data: ICONIC-LEAD trial (N=684): ~74% achieved IGA 0/1 at Week 24. Long-term data showed 67% sustained clear/almost clear skin through Week 52.

LYNAVOY (linerixibat)

Indication: Treatment of cholestatic pruritus associated with primary biliary cholangitis (PBC) in adults. IBAT inhibitor.

Clinical Data: Phase 3 GLISTEN trial (n=237) showed rapid, sustained reductions in pruritus vs placebo.

AVLAYAH (tividenofusp alfa-eknm)

Indication: Enzyme replacement therapy for neurologic manifestations of Hunter syndrome (MPS II) in pediatric patients. Carries boxed warning for hypersensitivity reactions.

Clinical Data: Phase 1/2 study in 47 pediatric patients: 91% average reduction in CSF heparan sulfate at Week 24.

LIFYORLI (relacorilant)

Indication: Treatment of platinum-resistant epithelial ovarian, fallopian tube, and primary peritoneal cancer in combination with nab-paclitaxel.

Clinical Data: Phase 3 ROSELLA trial (n=381): 35% reduction in risk of death vs nab-paclitaxel alone; median OS 16.0 vs 11.9 months.

KRESLADI

Indication: Gene therapy for pediatric patients with severe leukocyte adhesion deficiency type I (LAD-I) lacking an HLA-matched sibling donor.

Clinical Data: Single-arm study of nine pediatric patients; median CD18 expression sustained through at least 42 months.