An Ipsen drug developed to treat the severe itching that accompanies certain liver disorders is now FDA approved for treating this complication in patients with Alagille syndrome. It’s the second approved indication for Bylvay, a drug that the French pharmaceutical company acquired earlier this year as part of a $952 million deal.
The FDA approval announced Tuesday covers the treatment of the itching symptom in patients age 12 months and older. Ipsen said Bylvay is immediately available via prescription for eligible Alagille syndrome patients who have the rare liver disease.
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Alagille syndrome is an inherited disorder affecting the bile ducts. Patients with the disease have fewer bile ducts or they have bile ducts that are narrow or malformed. The result is a toxic buildup of bile acid, which in turn damages the liver and causes progressive liver disease. Cholestatic pruritus, an intense itching sensation that does not go away with scratching, is a common complication of the disease.
Bile acid levels are regulated by transport proteins. Bylvay is a small molecule that works locally in the small intestine to block ileal bile acid transporter, reducing the organ’s reabsorption of bile acids. While the way that the drug works to reduce itching is not completely understood, it’s thought that blocking bile acid transporters in the small intestine in turn reduces levels of bile salts in the blood that are associated with the severe itching symptom.
Bylvay is an oral capsule taken once daily. The specific dose is determined by the patient’s weight. FDA approval of Bylvay for Alagille syndrome patients is based on the results of a placebo-controlled Phase 3 study that enrolled patients ranging in age from infancy to 17 years old. Results showed statistically significant improvement in pruritus after 24 weeks of treatment according to a scale used to measure itching symptoms. In addition, the study met a secondary goal of showing a statistically significant reduction in bile acid levels in the blood.
The most common adverse reactions reported in the Alagille syndrome study included diarrhea, abdominal pain, hematomas, and weight loss. No patients discontinued the study drug and about 96% of clinical trial participants rolled over into the open-label extension study.
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Ipsen has become an active dealmaker as the Paris-based company looks to expand its drug pipeline and portfolio. Cancer has been one area of focus. Nearly a year ago, Ipsen struck a deal to acquire Epizyme and its follicular lymphoma drug Tazverik. The company also has a drug R&D partnership with Marengo Therapeutics, a preclinical biotech developing antibody-based cancer immunotherapies.
Rare disease is another area of focus. Ipsen added Bylvay to its portfolio via the acquisition of Albireo Pharma earlier this year for $42 per share. That company had secured the drug’s first approvals in 2021, in the U.S. and Europe, for the treatment of pruritus caused by progressive inherited familial intrahepatic cholestasis (PFIC), a rare inherited liver disease. FDA approval of Bylvay in Alagille syndrome approval follows the 2021 approval of Mirum Pharmaceuticals’ Livmarli, the first approved therapy for pruritus in this disease and the biotech’s first and, so far, only approved product. Mirum reported $75 million in product sales in 2022.
“Today’s 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,” Howard Mayer, Ipsen’s executive vice president and head of research and development, said in a prepared statement. “We are proud to have achieved FDA approval for Bylvay as a treatment for [Alagille syndrome] in the U.S. and we are committed to making it available to many more eligible patients across the world.”
Ipsen has submitted an application seeking European Medicines Agency approval of Bylvay in Alagille syndrome. An opinion from the Committee for Medicinal Products for Human Use is expected by the end of this month; a final EMA regulatory decision is expected in the second half of this year.
Bylvay is in Phase 3 testing for yet another rare pediatric liver disease, biliary atresia. The Albireo acquisition agreement includes a contingent value right that will pay $10 per share if the drug receives FDA approval in this indication.
Photo by Albireo Pharma
