Courier FDA approves the first drug therapy for fatal pediatric liver disease to avoid liver transplantation and surgery

▎Albireo Pharma, editor of WuXi AppTec's content team, announced today that the US FDA has approved the listing of Bylvay (odevixibat)
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The press release states that it is the first drug approved for the treatment of pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) of all subtypes
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Bylvay is a potent, non-systemic ileal bile acid transport inhibitor (IBATi) that does not require refrigeration and can be taken in capsule form once a day
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PFIC is a rare childhood disease that causes progressive, life-threatening liver disease
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In many cases, PFIC causes cirrhosis and liver failure within 10 years of the patient’s birth
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The most prominent persistent symptom of PFIC is intense itching, which usually leads to a severe decline in the quality of life
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Previously, PFIC had no approved drugs
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Only surgical options including biliary diversion surgery and liver transplantation are available.
Without them, most PFIC patients cannot live beyond 30 years of age
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Bylvay's approval is supported by data from two global phase 3 clinical trials, PEDFIC 1 and PEDFIC 2
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In the randomized, double-blind, placebo-controlled PEDFIC 1 study, Bylvay reached the primary endpoints of reducing pruritus (p=0.
004) and serum bile acid (p=0.
003), and was well tolerated, with frequent occurrence of diarrhea/defecation The rate is very low
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PEDFIC 2 is a long-term, open-label, phase 3 extension study that once again confirms that Bylvay can continuously reduce serum bile acids in patients treated for up to 48 weeks, and improve pruritus assessment, growth, and other liver function markers
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"Bylvay gave us a non-surgical option and will change how we treat PFIC
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" said Dr.
Richard Thompson, professor of molecular liver disease at King's College London University.
"We are hopeful that these children will have a better prognosis
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" References: [1] Albireo Announces FDA Approval of Bylvay™ (odevixibat), the First Drug Treatment for Patients With Progressive Familial Intrahepatic Cholestasis (PFIC).
Retrieved July 20, 2021, from https:// 2021/07/20/2266030/0/en/Albireo-Announces-FDA-Approval-of-Bylvay-odevixibat-the-First-Drug-Treatment-for-Patients-With-Progressive-Familial-Intrahepatic-Cholestasis-PFIC.
html Note: This article aims to introduce the progress of medical and health research, not a treatment plan recommendation
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If you need guidance on treatment plans, please go to a regular hospital for treatment
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