another pharma update

[DonnaBoll]

This is in regards to the pharma Ipsen...

They are trialing a drug called Elafibranor. It is being used for treatment in PBC, biliary atresia (in children) , PSC, Alagille Syndrome, and Primary Familial Cholestasis.

With patients taking Urso, often up to 40% do not see improvement. 3-5% of patients can't take it for various reasons. Those on Ocaliva, greater than 50% don't see an improvement.

161 people were trialed. There was a placebo group used for half of them.

They saw decreased alk phos and bilirubin. 51% had decreased alk phos after 1 year. 15% had completely normal lab results. Improvement was seen after 4 weeks in some patients.

Using the Itch Numeric Rating Scale - according to this scale: no itching = 0, mild 1-3 points, moderate 4-6 points, severe 7-8 points, and very severe pruritis (greater than or equal to 9 points.

On this scale a decrease of 1.9 points was seen for those with a

5-10 points score. With a placebo, a decrease of 1.1 points seen.

With those whose itching was in the 0-5 category, there was a decrease of 4.2 points overall. Those with a score 6-10 on the scale, there was a decrease of 1.9, and in the placebo group a decrease of 1.1 points.

The common side effects seen were nausea and vomiting, abdominal pain, and diarrhea.

Ipsen is clearly committed to the disease of PBC. Right now they are working on 2 projects.

1. The patients experience map... and 2. barriers to patients in their health care. They are interested in why certain information isn't being given to patients and also why certain questions aren't being asked. ie. "how is your itch?" "what seems to help with it".....

I was truly impressed with their commitment to making our lives better.

I know for me personally, I have never in the last 3 years, since my PBC has returned, if I was itching or feeling that fatigue again. Have you been asked these questions by your doctor? What would you hope they would ask?!

[Giraffe44]

Hi Donna

Thanks for the updates on new drugs for pbc management. Is there any information or hope that these new drugs might help with fatigue ( they mostly seem to focus on itch symptoms it appears to me as well as improving alk phos levels)?

Fatigue and thus living a half life is my main concern which incidentally is never asked about at my telephone consultations. I guess as it can’t be seen it is mostly ignored. I exercise as best I can , have a sensible diet and keep hydrated but some days the fatigue is overwhelming. Any thoughts?

[DonnaBoll Administrator in reply to Giraffe44]

Yes! I will be posting this information later today. I can remember all too well how the fatigue seemed to just suddenly consume me without warning. Exercising should help but convincing yourself of that on 'those' days can be a real challenge. Someone at a meeting I was at several years ago said for her "it was like trying to walk thru wet cement". I never forgot that... I think one of the most important things to do on fatigue days is just accept it and listen to your body. For me, pushing thru it just didn't work. Our body works very hard for us with PBC. We need to listen when it needs to rest. Great point you made that no one can see it. When we tell someone we are 'tired', can't you almost guarantee that their response is 'I am too today". Ugh!!! the words tired and fatigue just don't do it justice in the least. I know how hard it is to function on those days. Everyone seems to cope differently. I remember all too well when I was working nights as a nurse, I would sleep until around 4 or 5 - get up and go right back to work at 6. On my days off, I would still sleep until 4 and then could hardly get out of bed then... I really believe each of us has to know our own bodies and what we can do - and what we can't. PBC is not easy in any way. Please feel free to post about how you are doing anytime.