BIOMARIN PHARMACEUTICAL INC.

RIBOMIC's Phase 2 trial of umedaptanib pegol demonstrated significant height growth improvements in children with achondroplasia, with some patients achieving increases of up to 5.0 cm/year.

BioMarin's Phase 3 PEGASUS trial demonstrated a 49.7% reduction in blood phenylalanine levels in adolescents aged 12-17 with PKU treated with PALYNZIQ compared to diet alone.

BioMarin's Phase 3 GENEr8-1 trial demonstrates that ROCTAVIAN gene therapy maintains durable bleed control and sustained factor VIII expression five years after treatment in severe hemophilia A patients.

Four innovative therapies are advancing through clinical trials to address critical unmet needs in MPS I treatment, particularly targeting central nervous system complications that current therapy Aldurazyme cannot reach.

The Federal Circuit has affirmed the Patent Trial and Appeal Board's decision invalidating two patents covering experimental gene therapy for hemophilia treatment, handing Pfizer a significant legal victory.

Eli Lilly plans to add over 3,000 highly skilled manufacturing positions across four new U.S. facilities to be announced later this year.

NICE has secured continued access to cerliponase alfa (Brineura) for current patients and those starting treatment before December 2025, despite not recommending it for routine NHS use due to cost concerns.

BioMarin Pharmaceutical has entered into a definitive agreement to acquire Inozyme Pharma for $270 million, strengthening its enzyme therapy portfolio with the addition of INZ-701.

Takeda Pharmaceutical is positioning three upcoming late-stage clinical trial readouts as a strategic "inflection point" to offset revenue losses from Vyvanse's generic competition.

Pharmaceutical M&A activity is experiencing a significant rebound with major deals including Novartis's $1.7 billion acquisition of Regulus Therapeutics and Merck KGaA's $3.9 billion purchase of SpringWorks Therapeutics.