Clinical Trials
114
96 active
Approvals
21
Total approvals
Agencies
4
Regulatory bodies
Founded
N/A
Active, not recruiting
95
83.3%
Completed
4
3.5%
Not yet recruiting
1
0.9%
Recruiting
11
9.7%
Withdrawn
3
2.6%
- BioMarin and Ascendis Pharma resolved all pending patent disputes, with Ascendis agreeing to pay royalties on sales of its achondroplasia drug Yuviwel (navepegritide). - Ascendis will pay BioMarin 20% of Yuviwel net sales in the U.S. and 18% in the EU, Brazil, and South Korea through May 2030. - The license covers all current and potential indications, including achondroplasia and hypochondroplasia, and use in combination with other medicines. - The settlement recognizes BioMarin's pioneering C-type natriuretic peptide (CNP) technology, including its development of VOXZOGO (vosoritide).
- BioMarin Pharmaceutical Inc. has entered a definitive agreement to acquire Alesta Therapeutics, gaining the clinical-stage asset ALE1 for a $275 million upfront payment plus up to $215 million in milestone payments. - ALE1 is an orally active small molecule with the potential to be the first oral therapy for hypophosphatasia (HPP), a rare genetic bone disease caused by ALPL gene mutations. - ALE1 targets inorganic pyrophosphate (PPi), the central disease metabolite, and is currently in a Phase 1/2a trial assessing safety, tolerability, and pharmacokinetics/pharmacodynamics. - The transaction is expected to close this quarter, with Alesta spinning out all non-ALE1 assets and transferring its employees to a new entity prior to close.
- BioMarin has completed its acquisition of Amicus Therapeutics at the end of April, integrating Fabry and Pompe disease communities into its rare disease portfolio. - Chief Commercial Officer Cristin Hubbard emphasized the company's goal to maintain momentum while leveraging BioMarin's global footprint across approximately 80 countries to expand patient access. - The company plans to apply its expertise in genomics, diagnostics, and AI-driven patient identification to help reach more people living with these rare genetic conditions. - The integration aims to deliver therapies without disruption while adding BioMarin's specialized capabilities in identifying underdiagnosed and misdiagnosed patient populations worldwide.
- The average biopharmaceutical employee earned $116,648 in 2025, roughly 30% more than the average S&P 500 worker at $89,744, according to an Endpoints News study. - Gilead topped Big Pharma with a median salary of $239,000, while biotech firms like Alnylam ($312,000) and Ionis ($305,000) far exceeded even the most generous large drugmakers. - Johnson & Johnson, the industry's perennial revenue leader, ranked only 10th in median salary at $91,000, demonstrating that company size and profitability do not directly correlate with employee pay. - The 32 large biotech companies studied collectively employ over one million people with a combined market capitalization of $3.88 trillion.
- BioMarin's Phase 3 ENERGY 3 trial of BMN 401 met one of two co-primary endpoints, showing statistically significant increases in plasma inorganic pyrophosphate levels in children with ENPP1 deficiency. - The study failed to demonstrate improvement in Radiographic Global Impression of Change scores, a critical clinical measure of rickets severity, despite biochemical improvements. - No positive trends were observed across secondary endpoints including Rickets Severity Score and growth measurements, prompting BioMarin to evaluate next steps for the program. - ENPP1 deficiency affects approximately 50% of infants who do not survive beyond six months, highlighting the urgent need for effective treatments in this devastating rare disease.
- Intellia Therapeutics' CRISPR-based treatment lonvo-z demonstrated an 87% reduction in swelling attacks compared to placebo in an 80-patient Phase 3 trial for hereditary angioedema. - The single-dose, in vivo gene editing therapy resulted in 62% of patients being completely attack-free six months after treatment, compared to 11% in the placebo group. - If approved, lonvo-z would become the second FDA-approved CRISPR medicine and the first to edit DNA directly inside the body, with Intellia planning to complete regulatory filing in the second half of 2026.
- BioMarin Pharmaceutical and Veeva Systems announced a long-term enterprise agreement to enhance the biotechnology company's digital capabilities and operational efficiency. - The partnership aims to accelerate BioMarin's development and commercialization of transformative medicines for rare genetic diseases through advanced software and data solutions. - BioMarin CEO Alexander Hardy emphasized the collaboration will combine the company's scientific expertise with Veeva's technological leadership to bring medicines to patients more quickly. - The expanded agreement builds upon an existing relationship between the two companies, with Veeva serving over 1,500 life sciences customers globally.
- Galafold (migalastat) generated $371.5 million in sales during the first nine months of 2025, representing a 12% year-over-year increase and contributing over 80% of Amicus Therapeutics' net product revenues. - The company secured patent protection for Galafold through 2038 and reached a settlement with Teva Pharmaceuticals preventing generic competition until January 2037. - Amicus' combination therapy Pombiliti + Opfolda for late-onset Pompe disease showed robust growth with $77.5 million in sales, up 61% year-over-year. - BioMarin has agreed to acquire Amicus Therapeutics for $4.8 billion, with the transaction expected to close in the second quarter of 2026.
- The FDA is set to make five significant regulatory decisions in November 2025, including approvals for treatments targeting rare genetic disorders, multiple cancer types, and kidney disease. - Arrowhead Pharmaceuticals' plozasiran for familial chylomicronemia syndrome demonstrated an 80% median reduction in triglyceride levels and 83% lower risk of acute pancreatitis in Phase III trials. - Bayer's sevabertinib showed promising efficacy in HER2-mutant non-small cell lung cancer with overall response rates ranging from 38% to 71% depending on prior treatment history. - Multiple bispecific antibodies and targeted therapies are seeking expanded indications, potentially reshaping treatment landscapes in oncology and rare diseases.
- Ascendis Pharma's TransCon CNP awaits FDA approval on November 30 as a weekly treatment for achondroplasia, competing directly with BioMarin's daily Voxzogo injections. - Phase II data showed TransCon CNP achieved 5.42 cm per year growth velocity versus 4.35 cm for placebo, while Voxzogo demonstrated 1.57 cm improvement over placebo in Phase III trials. - The global achondroplasia market represents over $5 billion opportunity with 24,000 eligible patients, though Voxzogo currently captures only 17% market penetration. - Alternative approaches targeting the root cause include BridgeBio's oral infigratinib, which uniquely improves body proportionality unlike CNP-based therapies.
