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In Boston, Massachusetts, one company is now making significant headway in the development of an innovative treatment for rare pediatric liver diseases.
Led by president and chief executive officer Ron Cooper, Albireo (Nasdaq: ALBO) has since 2021 boasted both US and EU approval for the novel bile acid modulator Bylvay (odevixibat).
The oral medicine addresses an unmet need by providing the first non-surgical treatment option for people with progressive familial intrahepatic cholestasis (PFIC), a rare genetic disorder affecting young children that causes progressive, life threatening liver disease.
Bylvay is approved for the treatment of pruritus in PFIC in the USA, and for PFIC in Europe.
The company has also recently announced positive top-line results from the Phase III ASSERT study, which is testing Bylvay in Alagille syndrome (ALGS), another rare genetic disorder.
Approval in ALGS would open up another source of revenue for the product, and offer hope for families impacted by this serious condition, which affects multiple organ systems including the liver and heart.
Estimates vary for the therapy’s peak revenue potential, but most analysts suggest the product will be able to generate hundreds of millions in annual sales, should all go according to plan.
As well as transforming the treatment of rare conditions with a high disease burden, such an outcome would enable the company to more fully explore the science of IBAT inhibition, an approach which it believes has great potential in a wide range of cholestatic liver diseases.
In this week’s episode of The Pharma Letter Podcast, I’m pleased to be joined by Ron Cooper, for a discussion of his company’s clinical goals and strategic focus.