FDA grants Mirum selective ASBT inhibitor maralixibat breakthrough therapy
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Last Update: 2020-06-07
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Source: Internet
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Author: User
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today, Mirum Pharmaceuticals(http://announced that the U.SFDA(http://granted its selective ASBT inhibitor maralixibat breakthrough therapy to treat itching symptoms in children 1 years and older with Alagille syndrome (ALGS)the treatment significantly reduced the patient's serum bile acid (sBA) levels and improved itching symptoms in theof phase 2b clinicaltrials (http://) for the treatment of alGS children, the FDA has granted Maralixibat Breakthrough Therapy a proven use to treat patients with type 2 aggressive family-based hepatic biliic siltation (PFIC)Maralixibat is an oral selective top sodium-dependent bile acid transport protein (ASBT) inhibitorASBT is present in the small intestine, which mediates the absorption of bile acid in the intestine and helps it circulate back to the liverMaralixibat can cause more bile acid to drain with the feces, prevent excessive bile acid accumulation, and control extreme itching associated with bile siltliver liver diseaseCurrently, maralixibat is treating ALGS and PFIC in clinical trialsThe treatment was determined to be based on a phase 2b clinical trial in a placebo-controlled group called ICONICThe results of the trial showed a significantly lower level of SBA in algs children treated with maralixibat than in children treated with a placebo compared to a placebo in addition , indicators such as the itchING RO score and itching scratch index (CSS) were improved
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