Approval applications for the first potential treatments for the rare liver disease progressive familial intrahepatic cholestasis (PFIC) from Mirum Pharmaceuticals, Inc. and Albireo Pharma, Inc. are now under review in the US and EU.
PFIC Space Hots Up With Albireo and Mirum Filings
Promising Therapies For Rare Liver Disease
Patients with the rare condition progressive familial intrahepatic cholestasis (PFIC) look set to have two possible therapeutic choices if recent filings for two IBAT inhibitors are successful.
More from New Products
More from Scrip
• By
With new Phase II data, Edgewise asserted that EDG-7500, a sarcomere modulator, could offer better efficacy and safety than cardiac myosin inhibitors in hypertrophic cardiomyopathy.
• By
The company closed a $150m series C funding round shortly after opening the second part of its Phase I/II study of ATSN-201 and partnering with a Nippon Shinyaku on ATSN-101.
• By
Investing heavily in antibody-drug conjugates, AbbVie is pioneering a dual STEAP1xPSMA-targeting approach in prostate cancer, as other candidates advance.