This leverages established commercial infrastructure and scientific expertise in skeletal growth disorders. The Enzyme Therapies portfolio demonstrated resilience with 8% overall growth despite generic erosion of KUVAN (down 31%). PALYNZIQ's 22% growth and positive Phase 3 data in adolescents support regulatory submissions for label expansion in H2 2025, potentially extending the product's growth runway. The advancement agreement with FDA for BMN 333 (long-acting CNP) creates regulatory clarity for a 2030 approval target. BioMarin's pipeline prioritization is evident in the discontinuation of BMN 370 for von Willebrand disease, allowing resource reallocation to programs with higher probability of technical and commercial success. The company's R&D strategy appears increasingly focused on leveraging established expertise in rare diseases while pruning programs that don't meet internal thresholds for continued investment.