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Alagille Syndrome
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Drug Pipeline Insights Report highlights rare liver diseases

Posted on August 31, 2021
Marginal zone lymphoma treatment granted FDA fast track designation

Two new treatments for rare liver diseases currently in the pipeline include Bylvay (odevixibat), which was approved July 20, 2021 and maralixibat, which is expected to have an FDA decision on September 29, 2021. Bylvay treats pruritus associated with progressive familial intrahepatic cholestasis (PFIC) while maralixibat is in development for the treatment of Alagille Syndrome (ALGS).

According to the report, there are about 600 people with PFIC and 1500 people with ALGS in the United States who may benefit from these treatments. Bylvay is the first approved treatment for PFIC and if approved, maralixibat would be the first for ALGS.

Read the full report here.

 

–Kelsey Moroz

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