Alagille Syndrome
Conference Roundup

Influence of advocacy groups, ALGS families on drug development highlighted at conference

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During presentations at the 10th International Symposium & Scientific Meeting on Alagille Syndrome, Pam Vig, PhD, Head of Research & Development at Mirum Pharmaceuticals, Inc and Christine Clemson, PhD, Senior Vice President, Global Head Medical Affairs at Albireo Pharma, Inc highlighted the dedication their respective companies have to developing treatment options for patients with Alagille syndrome (ALGS) and the impact patients and families living with this rare disease have on their work.

“When you work in rare diseases and get to get close to advocacy groups and hear the stories and talk to the families and see the children, it really resonates with you when you go back to your office. You think ‘I need to work harder, I need to work faster, I need to think of something different’,” Dr Vig told the attendees.

Mirum’s maralixibat (Livmarli) was approved last year for cholestatic pruritus in ALGS and is the only FDA-approved medication for ALGS.

Dr Vig said that the feedback from patients and their families has guided many of Mirum’s decisions, ranging from writing protocol to creating marketing material.

Mirum Access Plus is an end-to-end support system that helps lessen the burden of starting and continuing treatment with maralixibat, created with real feedback from patients. For instance, Dr Vig noted that some ALGS families said it was difficult to get to the pharmacy to pick up their prescription, so through the Mirum Access Plus program they can get it shipped to their home as well as have access to 24-hour pharmacy support.

Dr Clemson also underscored the impact the ALGS community has had on Albireo’s quest to get odevixibat (Bylvay) approved for the treatment of pruritus in patients with ALGS.

“Without the participation of patients and families, we’d never be where we are, which is on the cusp of hopefully getting approval in 2023,” she said.

The ASSERT Phase 3 Global Trial investigating odevixibat for ALGS is now fully enrolled, but patients with ALGS who were unable to enroll in ASSERT and have no other treatment options can receive treatment through Albireo’s Extended Access Program. Similarly, Albiero’s Charitable Access Program allows patients in socioeconomically disadvantaged countries with no other treatment options to receive our treatment. Both programs require physician recommendation.

“Our mission is to provide hope for families living with rare cholestatic liver disease and a big part of that mission is making sure our therapies reach the patients who will benefit from them most,” said Dr Clemson.


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