Ipsen is a global biopharmaceutical company focused on oncology, rare disease, and neuroscience. It brings medicines to patients in more than 80 countries and operates with global development hubs.
GENFIT will receive a $20 million milestone from Ipsen after Iqirvo posted $208 million in 2025 net sales in primary biliary cholangitis. The sales also triggered a €30 million instalment under GENFIT’s royalty-financing deal.
The FDA has accepted Regeneron's biologics license application for garetosmab with priority review, targeting fibrodysplasia ossificans progressiva. A decision is expected by August 2026 based on Phase 3 trial data showing significant reductions in new bone lesions.
The Consolidated Appropriations Act of 2026 extends the FDA's authority to award rare pediatric disease priority review vouchers through September 30, 2029, restoring a key incentive for developing therapies for rare pediatric diseases.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07588880 |
A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in China |
RECRUITING | |
| NCT07585097 |
A Study to Observe the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment |
NOT_YET_RECRUITING | |
| NCT07556913 |
Elafibranor Pregnancy Surveillance Program: A Study to Evaluate the Safety of Elafibranor During Pregnancy |
NOT_YET_RECRUITING | |
| NCT07497724 |
Odevixibat Outcomes in Patients With PFIC Versus an External Control Cohort (OvEC-PFIC) |
ACTIVE_NOT_RECRUITING | |
| NCT07484971 |
Study of the Epidemiological, Clinical, Diagnostic, and Therapeutic Characteristics of Prostate Cancers in Algeria |
RECRUITING | |
| NCT07441707 |
A Study to Assess a Medicine Called Tovorafenib in Japanese Children and Young Adults With Brain Tumours |
RECRUITING | PHASE1 |
| NCT07435428 |
A Study to Assess the Effectiveness and Safety of IPN10200 Over Time in Adults With Moderate to Severe Wrinkle-like Lines Between the Eyebrows |
RECRUITING | PHASE3 |
| NCT07427797 |
A Study to Assess the Effectiveness and Safety of IPN10200 in Adults With Moderate to Severe Wrinkle-like Lines Between the Eyebrows |
RECRUITING | PHASE3 |
| NCT07387549 |
A Study to Assess How Well and Safely Elafibranor Works in Adult Participants With Primary Sclerosing Cholangitis |
RECRUITING | PHASE3 |
| NCT07340151 |
Resection/Synchronous Pulmonary or Hepatic Oligometastatic Pancreatic Ductal Adenocarcinoma |
RECRUITING | NA |
