The company Albireo Pharma specializes in rare diseases, which develops new bile acid modulators for the treatment of liver disease in children and adults, today announced the positive results of the Phase 3 Assert study evaluating the safety and efficacy of odevixibat (Bylvay *) in affected patients from Alagille syndrome (Algs) from birth to early adulthood. Following a comparison of the Phase 3 study protocol with the American (Fda, Food and Drug Administration) and European (Ema, European Medicine Agency) drug agencies – both agree that a single study with positive results would be sufficient to approval of the new indication – the company intends to immediately submit the application documents to regulatory authorities in the United States and the European Union, explains a statement.
The Assert study – global, double-blind, randomized, placebo-controlled – met its primary endpoint of improvement in pruritus and its key secondary endpoint of reduction in serum bile acid (sBA), Allbireo reports. Furthermore, the intake of odevixibat was not stopped by the patients. The drug was well tolerated, with low rates of diarrhea (11.4% versus 5.9% for placebo).
Alagille syndrome is a rare and multisystemic genetic disease that affects 25,000 people globally, the note recalls. It can affect the liver, heart, skeleton, eyes, central nervous system, kidneys and facial features. Liver damage is caused by a shortage of the bile ducts in preventing the flow of bile from the liver to the small intestine. About 95% of patients with this condition have chronic cholestasis, usually within the first 3 months of life, and 88% also have severe, intractable itching. “Alagille syndrome is a devastating diagnosis and the families of children with this condition need more treatment options – says Roberta Smith, president of the Alagille Syndrome Alliance – As the mother of a child living with Alagille syndrome, I know in first person the very serious ailment caused by itching and the terrible impact on the quality of life of the child and on his ability to sleep and grow. Knowing that my doctor may have another treatment option is very important. “
A potent non-systemic inhibitor of ileal bile acid transport (IBATi), to be taken once daily, Bylvay has minimal systemic exposure and acts locally in the small intestine. The drug is already approved in the United States for the treatment of pruritus in patients 3 months of age and older in all types of familial progressive intrahepatic cholestasis (Pfic) and, in Europe, for all types of Pfic in patients aged equal to or greater than 6 months.
“The solid results from the Phase 3 Assert study are important because doctors urgently need more options in the treatment of Alagille syndrome. Bylvay has reduced extreme itching, so common in this patient population, and is critical in helping us improve. sleep, among other negative effects of the disease – underlines Nadia Ovchinsky, doctor, gastroenterologist and pediatric hepatologist at the Children’s Hospital of Montefiore and principal investigator of Assert – The study also showed that Bylvay reduced the levels of bile acids in serum, the which could indicate that the severity of liver disease may decrease over time, an important consideration for me as a treating physician. ”
“Our Phase 3 Assert study – says Ron Cooper, President and Chief Executive Officer of Albireo – has shown early, rapid and sustained effects on itching and bile acid reduction in Alagille syndrome, as in the Phase 3 study on Pfic. . We have successfully completed two of the three gold-standard studies in pediatric liver disease, await the imminent completion of the enrollment of a third phase 3 with our Bold Biliary Atresia study and, with over $ 270 million worth of liquidity, we have sufficient resources to carry out our plans. At the same time, we are seeing an acceleration in revenues from Pfic and we expect Bylvay’s third quarter sales to exceed $ 7 million. “
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