Every story we have to tell brings us one step closer to finding more treatment options for PFIC. Whether we are raising disease awareness, advocating for our families, inspiring hope or coming together at an event, our families are working tirelessly to make ourselves known.
What story would you like to tell?
By: PFIC Network
December 20, 2021 – 3 min read
Cincinnati Children’s Hospital is investing in research of cholestatic liver diseases such as PFIC.
We have been granted the wonderful opportunity to join the Rare As One Network in part with the Chan Zuckerberg Initiative. We have partnered as a patient led community to work as a patient led collaborative research network, being experts on our own disease, to facilitate research programs for PFIC.
The CZI and RAO initiative align with our values as an organization of inclusivity, patient choice, science and research, and collaboration. We are thrilled to be working with such a well established initiative and organization to better provide support to the PFIC patient community.
Read more on the CZI website. Link below.
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.
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 https://ir.albireopharma.com/news-releases/news-release-details/albireo-receives-european-marketing-authorization-bylvaytm
Albireo has prepared and FAQ for patients and families regarding the approval of Bylvay in USA. For more information, please click the button below
We are excited to hear that the EMA 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, “Albireo plans to directly commercialize Bylvay in the European Union (EU) and is prepared for a global launch with commercial, market access and medical affairs personnel already on the ground. This includes Germany, which has the largest EU market potential, with launch planned for September 2021, following price listing.” Congratulations to Albireo Pharmaceuticals! For the full press release, click here https://ir.albireopharma.com/news-releases/news-release-details/albireo-receives-european-marketing-authorization-bylvaytm
Albireo has prepared and FAQ for patients and families regarding the approval of Bylvay in Europe. For more information, please click the button below
Some great PFIC news! In a rare and complex surgery at HCMCT Manipal Hospitals Delhi, a team of Indian surgeons performed one of the most complex life-saving liver transplant on a nine-month-old boy Ali Hamad who suffered from liver failure due to a PFIC 2.
Every story we have to tell brings us one step closer to finding more treatment options for PFIC. Whether we are raising disease awareness, advocating for our families, inspiring hope, or coming together at an event, our families are working tirelessly to make ourselves known. What story would you like to tell?
How rare disease organization & advocacy networks can work as a team to bring patient communities together.
PFIC Network Family conference.
Rare genetic disease, PFIC.
Boy’s rare disease causes constant itching.