PFIC Scientific Conference: Wrap-Up and Next Steps

The PFIC Scientific Conference Held Many Great Presentations and Roundtables That Informed Both The Scientific And Patient Communities.

By: PFIC Network

June 1, 2022 – 2 min read

Thanks to Our Amazing Community, the PFIC Network Has Been Able to Follow Through with Scientific and Research Priorities to Start the PFIC Network Research Grant Program by the End of the Year.

By: PFIC Network

December 20, 2021 – 3 min read

Cincinnati Children’s Hospital is investing in research of cholestatic liver diseases such as PFIC.

Gene therapy is a realistic option to treat rare diseases like PFIC, though these therapies are new and require very thorough scientific research and development to minimize risk and get approved. The NIH, FDA, and FIH have unveiled a public-private partnership aimed at accelerating gene therapies for the roughly 30 million Americans living with a rare genetic disease. There are several gene therapies already available with many more in clinical trial, though there is a lack of standards, access to tools, and economic incentive. This funding and partnership aims to fix this issue to speed up research on gene therapy and bring it to the masses sooner than the projected time period for beneficial therapies.

To read more on the public-private partnership and what it aims to accomplish you can go to the article. In the article there is also more conversation about gene therapies and the clinical trials that may also provide a better understanding on what is happening in the field.

pfic gene therapy

We are excited to hear that the FDA has approved Bylvay (Odevixibat) for the treatment of Progressive Familial Intrahepatic Cholestasis. This medication has helped many patients with symptoms of pruritus in clinical trials, and has been approved for use in all subtypes. According to their press release, “Bylvay is expected to be packaged and shipped within the coming days. With the immediate Bylvay launch, Albireo is ready with a focus on access and reimbursement, sales promotion, and patient support…Once Bylvay is prescribed, HCPs and families will have the option to use Albireo Assist™, which is a customized patient support program built with input from patient advocates. The program features dedicated, US-based regional Care Coordinators employed by Albireo who will investigate benefits and review financial assistance options to help ensure optimal patient access. They will also proactively assist with facilitating dosing changes, lab work, refill reminders, reauthorization and other activities.”
Congratulations to Albireo Pharmaceuticals! For the full press release, click here

Albireo has prepared and FAQ for patients and families regarding the approval of Bylvay in USA. For more information, please click the button below