IPSEN, OOO

Regulatory snapshots: global drug submissions, approvals, clinical trial approvals, and other decisions involving Amplia, Apellis, Biocon, Biotech, Ipsen, Janssen, Johnson & Johnson, Merck, Novo Nordisk, Pfizer, Samsung Bioepis, Sanofi.

Ipsen's Kayfanda® (odevixibat) approved in the European Union for treating cholestatic pruritus in Alagille Syndrome, a rare liver disease, based on ASSERT Phase III trial data showing significant itch reduction and improved sleep.

The European Commission has conditionally approved Ipsen’s Iqirvo (elafibranor) for primary biliary cholangitis (PBC), the first drug for the liver disease in nearly a decade. The approval is for Iqirvo in combination with ursodeoxycholic acid in adults who do not respond adequately to ursodeoxycholic acid alone, or as a monotherapy for those unable to take ursodeoxycholic acid. The decision follows an accelerated approval by the FDA in June, based on Phase III ELATIVE trial data showing greater treatment benefits with Iqirvo plus ursodeoxycholic acid compared to a placebo.

Kayfanda® (odevixibat) approved for treating cholestatic pruritus in children with Alagille Syndrome, based on ASSERT Phase III trial data showing significant itch reduction and improved sleep. Kayfanda is a non-systemic IBAT inhibitor, expanding Ipsen's rare cholestatic liver disease portfolio.

Ipsen's Kayfanda® (odevixibat) approved by the European Commission for treating cholestatic pruritus in Alagille Syndrome patients aged 6 months or older, based on ASSERT Phase III trial data showing significant itch reduction and improved sleep parameters.

Ipsen’s Kayfanda® (odevixibat) approved in the E.U. for treating cholestatic pruritus in Alagille Syndrome, based on ASSERT Phase III trial data showing significant itch reduction and improved sleep parameters.

GENFIT announces European Commission's conditional approval of Iqirvo® (elafibranor) 80mg tablets for Primary Biliary Cholangitis (PBC) treatment, following positive CHMP opinion and U.S. FDA Accelerated Approval.

Glucocorticoid-induced hypercoagulopathy in endogenous Cushing syndrome (CS) increases thromboembolic risk, with CS patients having an 18-fold higher risk of venous thromboembolism (VTE) compared to the general population. VTE risk is highest postoperatively, with up to 5.6% incidence. Hypercoagulopathy persists post-surgery and improves within 6 months. Relacorilant, a selective glucocorticoid receptor modulator, showed improvements in coagulation markers without undesirable effects, suggesting potential benefits for CS patients, particularly those at high VTE risk.

European Commission grants conditional marketing authorization for Iqirvo® (elafibranor), a first-in-class treatment for primary biliary cholangitis (PBC), following positive CHMP opinion based on ELATIVE phase III trial data. Iqirvo demonstrated significant efficacy over placebo and was well-tolerated, reinforcing Ipsen’s commitment to advancing medical innovations for rare cholestatic liver diseases.

Zevra Therapeutics wins FDA approval for Miplyffa, the first drug for Niemann-Pick disease type C (NPC), in combination with miglustat for neurological symptoms in patients aged two and older. NPC is a rare neurodegenerative disorder with a life expectancy of about 13 years. Zevra acquired the arimoclomol program from Denmark's Orphazyme, which initially failed to gain FDA approval in 2021. The FDA previously granted rare-pediatric-disease designation to arimoclomol, potentially qualifying Zevra for a priority-review voucher.