Also known as: CNP
BioMarin reported first-quarter 2026 revenue of $766 million and raised full-year guidance to $3.825 billion-$3.925 billion. The company highlighted FDA approval of PALYNZIQ for adolescents and upcoming Phase 3 readouts for BMN 401 and VOXZOGO.
The FDA approved navepegritide, a once-weekly injectable therapy for achondroplasia, while phase 3 data showed infigratinib, an investigational oral medication, achieved the highest growth velocity reported in randomized trials for the condition.
The FDA has granted accelerated approval for YUVIWEL (navepegritide), the first once-weekly treatment for children with achondroplasia. Commercial availability is expected in early Q2 2026, with a Rare Pediatric Disease Priority Review Voucher granted.
BridgeBio Pharma reported positive Phase 3 results for oral infigratinib in children with achondroplasia, meeting primary and secondary endpoints with superior growth velocity and the first statistically significant improvement in body proportionality. The company plans regulatory submissions in the second half of 2026.
