Ipsen's Kayfanda® Receives EU Approval for Alagille Syndrome Treatment
Kayfanda® Gets the Green Light for Treatment in the EU
Ipsen has made significant strides in pediatric healthcare with the recent approval of Kayfanda® (odevixibat) in the European Union (EU). This new treatment targets cholestatic pruritus, a distressing symptom associated with Alagille Syndrome, a rare liver condition affecting children. The approval marks a vital advancement in the therapeutic options available for this challenging disease.
Understanding Alagille Syndrome
Alagille Syndrome (ALGS) is an inherited genetic disorder characterized by developmental defects in the liver, heart, and other vital organs. For patients living with ALGS, chronic cholestasis frequently leads to serious complications, including progressive liver damage and severe itching, known as pruritus. This symptom can severely impact the quality of life for both patients and their caregivers.
The Role of Kayfanda®
Kayfanda is a non-systemic ileal bile acid transport (IBAT) inhibitor, which is administered once daily. The active ingredient, odevixibat, works by blocking the ileal bile acid transporter, leading to decreased serum bile acid levels. This decrease is crucial in alleviating the discomfort associated with cholestasis and its accompanying pruritus.
Research and Approval Process
The approval of Kayfanda came after extensive research, particularly the ASSERT trial. This Phase III clinical trial is the first of its kind, focused on ALGS patients, demonstrating that Kayfanda significantly reduces scratching severity and improves overall well-being compared to placebo.
Dr. Christelle Huguet on the Impact
Dr. Christelle Huguet, Executive Vice President at Ipsen, emphasized the importance of this approval. She noted the often intolerable itch faced by patients and mentioned that this new treatment option will significantly enhance their quality of life. The introduction of Kayfanda is a welcome development for patients in the EU.
Further Implications for Ipsen's Portfolio
The release of Kayfanda is aligned with Ipsen's commitment to addressing rare diseases, especially among children. Additionally, Ipsen has expanded its portfolio of treatments for cholestatic liver diseases to include Iqirvo® (elafibranor) for Primary Biliary Cholangitis. This strategic expansion ensures that Ipsen remains at the forefront of innovative solutions for rare liver disorders.
The Future of Kayfanda
Kayfanda is also recognized as Bylvay® in markets outside the EU, where it has already been approved for treating patients with PFIC. Additionally, future studies, including the ongoing BOLD trial, aim to explore further applications of odevixibat in Biliary Atresia, with results expected around 2026.
Ongoing Support from Ipsen
For patients and families navigating the challenges of ALGS, Ipsen remains dedicated to providing necessary support and resources. By focusing on innovative treatments that improve life quality, Ipsen aims to be a partner in patient care.
Contact Information
For more information regarding Kayfanda and Ipsen's initiatives, interested parties can contact:
Craig Marks | + 44 (0)7584 34 91 93 | craig.marks@ipsen.com
Nicolas Bogler | + 33 6 52 19 98 92 | nicolas.bogler@ipsen.com
Frequently Asked Questions
What is Kayfanda® used to treat?
Kayfanda® (odevixibat) is used to treat cholestatic pruritus in patients with Alagille Syndrome.
How does Kayfanda® work?
Kayfanda works as an ileal bile acid transport inhibitor, reducing serum bile acids and alleviating itching.
Who is eligible for Kayfanda® treatment?
Kayfanda is approved for children aged 6 months and older diagnosed with Alagille Syndrome.
What were the results from the ASSERT trial?
The ASSERT trial showed significant reductions in pruritus symptoms and improvements in sleep quality for patients using Kayfanda.
Is Kayfanda® available in markets outside the EU?
Yes, Kayfanda is known as Bylvay® in markets outside the EU and has been approved for treating Progressive Familial Intrahepatic Cholestasis (PFIC).
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