Ipsen’s Bylvay approved by FDA for severe itching due to Alagille syndromeAlagille syndrome

14 Jun 2023
www.pmlive.com
Clinical ResultDrug ApprovalAcquisitionPhase 3
Ipsen’s Bylvay approved by FDA for severe itching due to Alagille syndrome
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Source: PMLiVE
Ipsen’s Bylvay (odevixibat) has been approved by the US Food and Drug Administration (FDA) to treat cholestatic pruritus (intense itching) in patients aged 12 months and older with Alagille syndromeAlagille syndrome (ALGS).
The approval marks the second rare disease indication for Bylvay in the US, after the drug was approved in 2021 to treat cholestatic pruritus from progressive familial intrahepatic cholestasis.
ALGS is an inherited rare, genetic disorder that can affect multiple organ systems in the body, including the liver, heart, skeleton, eyes and kidneys.
Liver damage results from having fewer than normal, narrowed or malformed bile ducts, which leads to toxic bile acid build-up.
Approximately 95% of patients with the condition present with chronic cholestasis, usually within the first few months of life, and as many as 88% also present with severe pruritus.
Bylvay is a once-daily, non-systemic bile acid transport inhibitor that Ipsen gained access to after it acquired rare disease specialist Albireo earlier this year for $952m.
The new approval is supported by positive results from the phase 3 ASSERT study, which showed the drug provided "highly statistically significant and clinically meaningful sustained improvements in pruritus, starting early after initiation of treatment,” the company said.
More than 90% of patients were deemed to have responded with reduction in pruritus symptoms, and Bylvay was also associated with significant reductions in serum bile acid concentration – a biomarker of the disease.
Ipsen said the drug will be immediately available via prescription for eligible ALGS patients in the US. The company is also expecting a decision from the European Medicines Agency for Bylvay in this indication in the second half of this year.
The drug is also in late-stage development for paediatric liver disorder biliary atresia, the most common of three main paediatric liver diseases and the leading cause of liver transplants for children.
Howard Mayer, executive vice president and head of research and development for Ipsen, said: “[The] approval of Bylvay in a second indication allows patients and physicians to access an additional treatment option that has the potential to improve the management of pruritus, or intense itch, in this distressing condition that tends to affect young children.
“We are proud to have achieved FDA approval for Bylvay as a treatment for ALGS in the US and we are committed to making it available to many more eligible patients across the world.”
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