BMN 333 - (ASPEN)

August 6, 2026: BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported Q2 2026 results and multiple pipeline/regulatory catalysts. Total revenue rose 20% Y/Y to $990 million; 2026 guidance was raised to $3.875-$3.925 billion total revenue, $1.0-$1.05 billion VOXZOGO revenue, and non-GAAP diluted EPS of $4.90-$5.10.

VOXZOGO: BioMarin recently submitted an sNDA to FDA for hypochondroplasia and expects an application-status update with Q3 earnings; if approved, launch could occur in 2027. In May, Phase 3 CANOPY-HCH-3 in children with hypochondroplasia met its primary endpoint, improving annualized growth velocity at week 52 vs placebo (LS mean difference +2.33 cm/year; p<0.0001), with significant gains in standing height, height Z-score, and arm span. Full Phase 3 data will be presented in a late-breaking oral session at the European Society for Paediatric Endocrinology meeting in September. In July, FDA accepted the sNDA for full approval of VOXZOGO in children with achondroplasia, setting a PDUFA date of February 28, 2027.

BMN 333: Phase 1 achondroplasia program data presented at ENDO 2026 supported weekly dosing; Phase 2/3 is enrolling, with a data update expected in 2027.

PALYNZIQ: European Commission approved use in adolescents aged 12+ with PKU.

BMN 820: Added to portfolio for FSGS; Phase 3 ACTION 3 is ongoing, with pivotal data expected in 2028.

BMN 351: Duchenne muscular dystrophy Phase 1/2 update expected by year-end.

BMN 401: Development discontinued across all indications after missing one of two co-primary endpoints in the pivotal ENERGY 3 trial for ENPP1 deficiency.

Business development: In July, BioMarin and the n-Lorem Foundation signed a global exclusive license/collaboration to develop an antisense oligonucleotide for ReNU syndrome.