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.    

liver
• Source: Shutterstock

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.

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Scrip for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required

More from New Products

Pipeline Watch: Ten Approvals And Eleven Phase III Readouts

Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.

Pipeline Watch: 13 Approvals And 13 Phase III Trial Updates

Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.

Vanrafia Approval Plays Into Novartis’s Multipronged IgAN Approach

 

The approval of another Novartis drug with a different mechanism means the company is further cornering multiple parts of IgAN pathogenesis.

Orphans Cling On To Growth Advantage As Market Share Heads Towards 20%

 

It might be the beginning of the end for the orphan drugs party but there is still sales growth enjoyment to be had for the sector, whose star performers are now looking increasingly like mainstream drugs.

More from Scrip

Camzyos nHCM Failure Could Open Door To Threat From Cytokinetics’ Aficamten

 

BMS’s Phase III ODYSSEY-HCM study did not show statistical significance for either of its two primary endpoints.

Taxes, Not Tariffs, Influence US Manufacturing, J&J CEO Says

 

The big pharma forecast a $400m expense this year related to tariffs, but those estimates only reflect medtech tariffs already announced, not potential pharma tariffs.

Disappointing Asthma Phase II Results Take Wind Out Of Sanofi’s Amlitelimab

 

Sanofi’s OX40L candidate missed a Phase II primary endpoint, but the company believes it can still work in a sub-group of ‘heterogeneous inflammatory asthma’ patients.