Health Canada Greenlights Bylvay by Medison and Ipsen: A Breakthrough Treatment for ALGS

Health Canada has approved Bylvay (odevixibat), developed by Medison Pharma and Ipsen, to treat cholestatic pruritus in patients aged 12 months and older with Alagille syndrome (ALGS). This follows its earlier approval in 2023 for patients aged six months and older with progressive familial intrahepatic cholestasis (PFIC), a serious liver disease.

Alagille syndrome is a rare genetic disorder affecting about 1 in 30,000 live births. It impacts various organs, including the liver, heart, and kidneys. The recent approval came after the global Phase III ASSERT study, which highlighted Bylvay’s ability to significantly reduce itching in ALGS patients within weeks of starting treatment.

The ASSERT study was the first comprehensive trial for this condition, showing not just a reduction in itching but also improved sleep quality and lower bile acid levels. The treatment has a favorable safety profile, making it a promising option for patients and their families.

Bylvay works by targeting the small intestine and is administered once daily. It’s unique because it minimizes how much the drug spreads through the body, addressing the itching and bile acid issues ALGS patients face. The medication comes in capsule form, which can be taken as is or mixed with food or drinks.

Launched in the U.S. in 2021 for PFIC, Bylvay was approved in Europe the same year. In June 2023, it also received approval in the U.S. for treating ALGS patients aged 12 months and older.

The ASSERT trial included a diverse group of participants from 32 sites across several regions, such as North America and Europe. The study clearly demonstrated Bylvay’s effectiveness in reducing itching, marked by improvements in what is known as the PRUCISION score, a measure of itching severity.

Pamela Minden, the Canadian country manager for Medison, expressed pride in bringing Bylvay to Canada, stating it represents a commitment to providing innovative treatment for rare diseases.

Understanding the broader landscape of rare disease treatments reveals a growing trend in developing targeted therapies. The market is fueled by advancements in genetic research and a better understanding of disease mechanisms, leading to more effective and personalized treatments.

With increasing awareness of rare diseases and advancements in medical research, treatments like Bylvay provide hope to families dealing with these challenging conditions.

For more detailed information on Bylvay, check out the original announcement from Pharmaceutical Technology.

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Health Canada, ALGS, PFIC, Ipsen, Medison, patients, cholestatic pruritus