Clinical Trials
109
Trial Phases
5 Phases
Drug Approvals
15
Clinical Trials
Distribution across different clinical trial phases (88 trials with phase data)• Click on a phase to view related trials
News
BioMarin Reports Positive Phase 3 Results for PALYNZIQ in Adolescent PKU Patients
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 ROCTAVIAN Gene Therapy Shows Sustained Efficacy After Five Years in Severe Hemophilia A
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 Promising Gene and Cell Therapies Challenge Aldurazyme's Dominance in MPS I Treatment
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.
Federal Circuit Upholds Pfizer's Victory in Gene Therapy Patent Dispute for Hemophilia Treatment
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.
Major Pharmaceutical Companies Expand U.S. Manufacturing Operations, Creating Thousands of New Jobs
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 Extends Access to Brineura for CLN2 Batten Disease While Seeking Long-term Solution
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 Acquires Inozyme Pharma for $270 Million, Adding Late-Stage Enzyme Therapy for Rare Genetic Disorder
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 Spotlights Three Late-Stage Readouts as Potential "Inflection Point" Amid Vyvanse Generic Competition
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.
Big Pharma M&A Activity Surges as Companies Target Undervalued Biotechs Amid Market Recovery
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.
FDA Sets Q3 2025 Approval Dates for Three Breakthrough Therapies Targeting Rare Diseases
The FDA has established PDUFA target action dates for three promising therapies in Q3 2025: sepiapterin for phenylketonuria (July 29), apitegromab for spinal muscular atrophy (September 22), and paltusotine for acromegaly (September 25).