Clinical Trials
11
0 active
Approvals
0
Total approvals
Agencies
0
Regulatory bodies
Founded
N/A
Completed
10
90.9%
No Longer Available
1
9.1%
No approval data available
- Ipsen received CHMP approval for odevixibat under the new brand name Kayfanda for Alagille syndrome, marking the second approval for the same drug after rebranding due to orphan status complications. - The company simultaneously secured CHMP recommendation for Iqirvo (elafibranor) as a treatment for primary biliary cholangitis, representing a rare dual approval achievement. - Odevixibat demonstrated statistically significant improvements in scratching severity in the ASSERT trial, the world's first phase 3 study completed in Alagille syndrome patients. - Both approvals address significant unmet medical needs in rare cholestatic liver diseases, with analysts estimating the PBC market alone could exceed $1.5 billion annually.
- Madrigal Pharmaceuticals' resmetirom demonstrated significant efficacy in the Phase 3 MAESTRO-NASH trial, achieving both primary endpoints of NASH resolution and liver fibrosis improvement. - The once-daily oral thyroid hormone receptor β-selective agonist showed NASH resolution in 26-30% of treated patients versus 10% on placebo, positioning it as a potential first-approved therapy for this growing health concern. - Resmetirom's success stands in stark contrast to Gilead's selonsertib, which failed its Phase 3 STELLAR-4 trial, highlighting the difficulty in developing effective NASH treatments despite the condition's multi-billion dollar market potential.
