FDA approves odevixibat for cholestatic pruritus in ALGS patients

FDA approves odevixibat for cholestatic pruritus in ALGS patients | Image Credit: © Argus - © Argus - stock.adobe.com.

Odevixibat (Bylvay; Ipsen) has been approved by the FDA to treat cholestatic pruritus in patients with Alagille syndrome (ALGS) 12 months and older, according to a press release from Ipsen.

The company states odevixibat is a “once-daily, nonsystemic ileal bile acid transport inhibitor (IBATi) that acts locally in the small intestine and has minimal systemic exposure.” The approval is the second indication of odevixibat for rare cholestatic liver disease in the United States. In 2021, odevixibat was approved for patients with cholestatic pruritus resulting from progressive familial intrahepatic cholestasis (PFIC). According to Ipsen, odevixibat is “immediately available via prescription for eligible ALGS patients.”

In the phase 3 ASSERT study (NCT04674761), which was presented at the 2022 American Association for Study of Liver Diseases congress, odevixibat demonstrated positive results and met the study’s primary endpoint of, “highly statistically significant improvement in pruritus as measured by the PRUCISION Observer-Reported Outcome scratching score (0-4 point scale), from baseline at month 6 (weeks 21 to 24), compared to the placebo arm (P = 0.002).” The double-blind, placebo-controlled, and randomized study evaluated the safety and efficacy of 120 µg /kg per day for 24 weeks in patients from 32 sites in North America, Europe, Middle East, and Asia Pacific, Ipsen states.

“Physicians urgently need more options to treat patients with Alagille syndrome,” said Nadia Ovchinsky, MD, chief, Division of Gastroenterology and Hepatology, Hassenfeld Children’s Hospital at NYU Langone, ASSERT principal investigator. “The ASSERT study showed that Bylvay reduced pruritus associated with ALGS, which is so common among this patient population and one of the leading indications for a liver transplant.”

Of patients with ALGS–an inherited “genetic disorder that can affect multiple organ systems in the body including the liver, heart, skeleton, eyes and kidneys,”–approximately 95% present with chronic cholestasis. Up to 88% also present with severe, intractable pruritus. In the United States, there are 1,300 estimated patients who could be eligible for IBATi treatment.

“As an advocate for families impacted by Alagille syndrome, it is such a blessing to know physicians now have another drug treatment option for the debilitating pruritus that affects so many Alagille patients,” said Roberta Smith, president, Alagille Syndrome Alliance in the press release. “I know personally the terrible impact of this rare disease on a child; this approval will help to alleviate the pruritus burden for more patients.”

Reference:

US FDA approves Bylvayfor patients living with cholestatic pruritus due to Alagille syndrome. Ipsen innovation for patient care. June 13, 2023. Accessed June 15, 2023. https://www.ipsen.com/press-releases/u-s-fda-approves-bylvay-for-patients-living-with-cholestatic-pruritus-due-to-alagille-syndrome/