1. Scrip
  2. >>New Products

PFIC Space Hots Up With Albireo and Mirum Filings

Promising Therapies For Rare Liver Disease

 
• By Alexandra Shimmings

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.

Mirum just pipped Albireo, announcing on 30 November that its marketing authorization application for maralixibat for the treatment of PFIC...

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
Start Free Trial

Already a subscriber?

More from New Products

PTC To Challenge BioMarin In PKU With Oral Sephience

 
• By Joseph Haas

PTC plans to compete with BioMarin’s two phenylketonuria drugs with efficacy data showing strong reduction of phenylalanine and ability for patients to liberalize their diets.

Apellis’ Empaveli Expansion Underway In Rare Kidney Disease

 
• By Jessica Merrill

The US FDA approved the complement C3 inhibitor for new indications in C3G and primary IC-MPGN, providing a new growth opportunity for Apellis.

Analysts Not Impressed With Adaptimmune’s Survival Attempts

 
• By Kevin Grogan

The company is getting just $55m from the sale of Tecelra and a couple of late-stage T-cell therapies to US WorldMeds.

Bayer’s Elinzanetant Is Latest Drug Hit By FDA Review Delays

 
• By Kevin Grogan

Comes Days After PDUFA Pushback For GSK's Blenrep.

More from Scrip

Quotables: Pharma Leaders On The Month’s Hot Topics

 
• By Anabel Costa-Ferreira

The highlights of recent comments and insights from industry executives on the key issues covered in Scrip.

Cipla Revs Up Biosimilars Engine Amid Winds Of Regulatory Change

 
• By Anju Ghangurde

Cipla’s partnered filgrastim biosimilar is expected to debut in the US in Q2 FY26 and the Indian firm expects an investment return ratio for its biosimilar engine “not too far” from that of a complex generic product amid an enabling regulatory environment.

With Planned US Facility Acquisition, Celltrion Looks To Avoid US Pharma Tariffs

 
• By Jung Won Shin

Celltrion is set to acquire a US biologics manufacturing facility that will "eliminate" its US tariff risks and provide a ready-made production base for future expansion.