We are delighted to share with you a recent press release from Ipsen about their experimental compound, Elafibrinor. The original press release can be found here:
Whilst we have not seen the full data yet (they haven’t been peer reviewed and published; something that is likely to happen later this year), this is a potentially important step for those affected by PBC. It is good news that we have companies seeking better solutions for PBC patients, both in terms of disease management and in symptom management, and we welcome the interim results.
By the very nature of a press release the information provided is limited. What the results that they have shown us do suggest is that Elafibrinor is really well tolerated by people and is at least as effective as Obeticholic Acid in the people who took part in the trial (all of whom had had an inadequate response to UDCA on its own). The results certainly look good enough for Ipsen to be taking the next steps towards getting the drug licensed for use in PBC (although this on its own does not guarantee that it is made available in any individual country. In the weeks to come, we shall learn more about the data itself and what they mean for PBC patient. There will be decisions to make, not only in terms of licensing but also where this treatment fits in the grand scheme of PBC disease management and care pathways.
In the meantime, we can only share with you what seems to be good news: and that is we have a new drug which seems to make a difference in disease progression in PBC, and we welcome that warmly.
Thank you to everyone who took part in this trial, without whom we woud not be making this progress.
Watch this space…
We have a new survey coming up on the App and we desperately need your voice to guide us. In recent conversations with regulators, they have expressed a “need” for 2 year placebo trial in disease progression in PBC. We find this to be unacceptable, but we need to be absolutely sure this is your view, too. So, we are asking a series of questions on your thoughts to see if we can take data to the regulators in order to fight on your behalf.
Can you please tell us your thoughts so we can include you in the conversation. We would be grateful if you can complete the survey before our International PBC Summit begins on 23rd May.
You can download the App from your own app store or you can use these links.
For International PBC Day 2022, we are launching our new campaign Urso Perfect.
We are drawing new attention to the importance of being on the proper Urso dose with our #UrsoPerfect social media campaign, as we strive to ensure as many patients as possible are on the right dose.
The #UrsoPerfect social media campaign celebrates each and every patient, you included, who are on the right dose. In turn, you can inspire others to ensure they are on the perfect dose too!
For more, please follow our social media channels:
Hannah Fargher was diagnosed with PBC ten years ago.
After her diagnosis, Hannah decided to get back into her childhood sport of Gymnastics. With the support of Buckley Gymnastics Club, Hannah has been able to compete in the Adult British Gymnastics Championships on 28th August.
The PBC Foundation charity has provided excellent support to Hannah and she wanted to give back, by helping to raise funds and awareness.
“It is possible to lead a normal life despite the challenges that this disease can throw at us, and I hope that by competing in this competition I can inspire other people with this condition to be more active and reach their potential.”
Update: In this years championships, Hannah won two silver medals. To read about her success and donate, please click this link: https://www.justgiving.com/fundraising/Hannah-Fargher1
Excellent article this week in The Times ‘Future of Healthcare’ Raconteur supplement highlighting your unmet needs and the research underway to develop new treatments for PBC and its symptoms.
Click here to read the article: Future of Healthcare PBC article
Click here for the full Raconteur Future of Healthcare supplement: Future of Healthcare supplement
Thank you Rare Revolution Magazine for highlighting PBC and the unmet need.
To read the article in full, on pages 10-12, please click HERE
Our webinar on 17 August 2020 included a presentation and Q&A session with Professor David Jones OBE on “The History of PBC (Part 1)”.
Click this link to watch the presentation on our You Tube channel: https://youtu.be/C8uanrwN2U8
A statement from the PBC Foundation Medical Advisory Committee
This advice is intended for those resident in the UK. Covid vaccines are being rolled out in many countries and different countries have adopted different practices. While the principles underlying our advice applies to all, we recommend that those outside the UK familiarise themselves with the recommendations in their country.
At present, the UK has licenced three vaccines to protect people against Covid-19 infection and a vaccination programme is being rolled out across the four nations of the UK. The governments of the UK have identified a list of priorities. Those with advanced liver disease, those on immunosuppressive therapy (including those on corticosteroids) and those who have had a transplant are at greater risk of the effects of Covid and therefore have been given priority.
Vaccination has attracted controversy, much of it ill-informed and inaccurate.
The PBC Foundation Medical Advisory Board wishes to stress their advice to all those offered vaccination:
· Vaccination offers a huge opportunity to help bring the pandemic under control and allow everyone to return to a normal way of life
· We very strongly recommend all those who are offered vaccination to take full advantage of the opportunity
· Vaccination is associated with a low risk of minor complications (such as transient pain, fever and tiredness); we are aware of ongoing concerns about the possibility of the Astra Zeneca vaccine being associated with a risk of thrombosis
· The benefits of the vaccine far outweigh any risk or complication
· Those on immunosuppressive therapy may safely receive the vaccine
The BioNTech/Pfizer and the Moderna vaccines use mRNA technology; this technology is based on a large amount of scientific data and animal work but is new when applied to vaccines for human use. Thus, there is a great deal of background knowledge on this technology, and experience from animal vaccines has shown that this allows for faster development and production of vaccines than classical vaccines. The Oxford/AstraZeneca vaccine uses a modified non-replicating weakened version of the common cold virus.
Evidence to-date shows the side effects of these vaccines are very similar to the transient side-effects we see with most other vaccines, such as local reactions such as pain in the upper arm for a couple of days and in some cases, fatigue, headache and fever lasting up to three days. As with other vaccines, a few cases of allergic reactions have been reported, but they have been confined to patients with a strong allergic predisposition.
These are not live vaccines, so they are safe for immunosuppressed patients. Experience in those with liver disease and on immunosuppression is less than in the general population but there is no evidence that the side-effects are any greater in those with liver disease or on immunosuppression. We are aware of ongoing studies looking at the efficacy of Covid vaccines in preventing disease in transplant recipients but strongly recommend that transplant recipients accept the offer of vaccination.
As with any medical interventions, people need to be aware of potential harm and balance risk and benefit. There has also been a great deal of comment about a possible link between the Astra-Zeneca vaccine and clotting, affecting especially younger people. The possible association between the Astra Zeneca vaccine and clotting events is being closely monitored by the regulatory authorities around the world as well as by the media. The former have access to the actual data and have the expertise to assess causality (that is whether the vaccine does increase the risk of thrombotic episodes or whether this is coincidental). In general, younger people are less at risk of the consequences of Covid and may have a greater risk of thrombosis, so the balance of risks and benefit is different to that in older people or those with liver disease or taking immunosuppressive therapy.
Advice varies between different jurisdictions and will change over time, so we advise readers to familiarise themselves with the guidance in their own country. Even if a direct link between the Astra Zeneca vaccine and clots is shown, the risk from the vaccine is still very small. It has been estimated it is about the same risk as dying in a car accident when driving 250 miles. In the UK, the current advice is to use vaccines other than the Astra Zeneca one for those under 30 years and this is a precautionary measure. As more information becomes available, this advice may change. Overall, considering the huge benefits from the vaccines in saving lives and the number of people dying from Covid or the survivors with long-Covid, the risk-benefit balance seems obvious.
The view of the PBC Medical Advisory Board, in line with other professional and patient-based organisations, remains that the benefits of the vaccine far outweigh the risks and the Board strongly advises all those offered the vaccine to take the opportunity, so protecting their own health and indirectly the health of others and allowing a return to normal life.
As the roll out continues around the world, more vaccines will become available and there will be more information about the benefits and risks. The Medical Advisory Board will endeavour to keep members updated as robust information becomes available.
Finally, people should be aware that some unscrupulous people have lost no time in developing new ways of using the pandemic and roll out of the vaccination programme to scam people. So please be careful: the NHS will not ask you for money or for any financial details. Be vigilant and report any scams.
12th April 2021
Position Statement on Vaccination for COVID-19 in Solid Organ Transplant Recipients (adults, children and young people) – 18th December 2020
Please click the link below to view the above position statement: