Amplify your voice with the new Wilson’s disease advocacy toolkit 

Following the Policy brief on Wilson’s disease in 2023, the Health Partnership has officially published their Wilson’s disease advocacy toolkit that offers a wealth of information on how to make your voice heard, drive research, and influence policy changes that can significantly impact the rare liver disease community. 

Jody Tate, the secretariat to the Wilson’s Disease Policy Network, shares that “The Health Policy Partnership (HPP) is proud to have supported the development of a new toolkit, which will help the Wilson’s disease advocates drive the policy changes needed to improve care. The Wilson’s disease community plays a crucial role in supporting the needs of people living with the condition. And the toolkit, developed by members of the multi-stakeholder Network, equips advocates around the world with information, templates and other resources to help them communicate those needs to policymakers.” 

We endorse this toolkit as an essential advocacy tool to share personal journeys with changemakers.

Two new products for cholestatic rare liver diseases may be available in EU soon 

Ipsen Pharmaceuticals has just shared exciting news for rare liver disease patients with cholestasis: They’ve received positive opinions on not one, but two new medicines. Iqirvo® (elafibranor) is designed for adults with primary biliary cholangitis (PBC) who haven’t had success with the usual treatment, ursodeoxycholic acid (also known as urso or UDCA), or can’t tolerate it; Kayfanda® (odevixibat) is focused on treating cholestatic pruritus in children with Alagille syndrome aged six months and older. This development expands the treatment options available to patients allowing them to have a greater chance of finding a therapy that works.

FDA’s new Rare Disease Innovation Hub may promote rare liver disease treatment 

The FDA has responded to the unified voice of the rare disease community by announcing a Rare Disease Innovation Hub to harness cross-agency expertise to accelerate the development of safe and effective treatments for over 10,000 rare diseases affecting millions in the U.S., many of which lack approved therapies. This initiative aims to change the landscape of rare disease care by prioritizing patient insights, integrating rigorous natural history studies, and emphasizing comprehensive endpoint assessments.

For patient advocacy groups, specifically in rare liver diseases, it is crucial that we integrate our perspectives into this program. Kristin Hatcher, GLI’s Program Director of pediatric and rare liver diseases,  shares, “This Hub will back up the need that the rare disease community has expressed for change in the system. As patients, we are desperate to have a so-called ‘one stop shop’ for collaboration.” Patients and advocacy groups will have the opportunity to actively contribute their lived experiences to the creation of therapies tailored to their specific diseases and conditions. Join our council to learn more about how to become involved in the Hub together.

Mirum’s Livmarli is now available in the EU 

Mirum Pharmaceuticals’ Livmarli® (maralixibat) has garnered its second indication in the EU. Following its initial approval for Alagille syndrome in 2022, approval has been extended to Progressive family intrahepatic cholestasis (PFIC) patients as young as 3 months old. Early intervention with Livmarli holds the potential to significantly impact the PFIC landscape by addressing symptoms early in childhood, which may improve long-term outcomes and reduce the need for liver transplants. This is a milestone in global access to Livmarli®, particularly since it is only approved for ages 5 and up in some countries, including the US.

Screen4Care in the EU needs your support for genetic screening in newborns 

Screen4Care, an EU-developed genetic screening approach, is garnering attention from patient organizations for its potential to revolutionize early detection and treatment of rare liver diseases. Dr. Alessandra Ferlini, Associate Professor of Medical Genetics at the University of Ferrara in Italy, advocates for increased medical support to ensure the project demonstrates effective genetic screening for newborns by 2026. This trial offers parents the opportunity to voluntarily screen their newborns for rare diseases, with the opportunity for early treatment initiation if a condition is detected.

You may be able to access OCALIVA (obeticholic acid) for free

Intercept Pharmaceuticals offers the Ocaliva Copay Assistance Program for PBC patients aged 18 and older with commercial or private insurance in the US. This program ensures that patients facing financial challenges can access Ocaliva without worrying about affordability. Learn more and connect with a care coordinator.