Wednesday, 15 July 2015 21:11

Updates from the aHUS UK and aHUS alliance meetings in London

Written by  Kamal Shah
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I attended the aHUS UK patient meeting and the aHUS Alliance meeting in London during the last week of June. I learnt a lot of new things about aHUS. I am going to summarise the learnings here.

The biggest takeaway for me was that there are four new drugs in the pipeline to treat Atypical HUS. Dr. Wynne Weston-Davies of Volution Immuno Pharmaceuticals presented this information:

- ALN-CC5 from Alnylam
- Compstatin from Apellis
- OMS721 from Omeros
- Coversin from Volution

This should ideally result in a reduction of the price of Soliris from Alexion which would mean a better chance of access to it as well. Competition is always a good thing!

For more details on this, please click here

Each of these drugs acts in different ways but the end result would be similar - treatment of aHUS, at initial occurrence, subsequent occurrences, for kidney transplants etc. Another advantage of some of these drugs is that they could be administered by sub-cutaneous injection or even orally rather than the IV infusions that Soliris needs. There was also some talk about Alexion working on a sub-cutaneous version of Soliris.

All these drugs are still in early stages of development and clinical trials and may be years before they will be usable by patients in India. However, at least something is happening!

I also learnt of a new mutation that is implicated in aHUS - DGKE. This mutation unfortunately is not treatable by Soliris since it is not associated with complement activity.

Another important discussion that was had was the availability of complement inhibitors (currently only Soliris) in different countries. Here is a summary of the information:

Ideally, any patient should have access to a complement inhibitor (currently only Soliris)  without having to pay out of pocket:

- At initial diagnosis
- Subsequent episodes
- During a Transplant and after to maintain the transplanted kidney

The following table summarises the situation in different countries with respect to this:
Country Status
France Full access
Italy Full access
UK Full access
Germany Full access
USA Full access
Russia Full access
Belgium Available for those with current aHUS activity, not available for transplants
Spain Available with some restrictions
Canada Available in some provinces and to others on a case by case basis
Netherlands Full access, future uncertain
Australia Available only for flairs and for a maximum of 12 months
India No access
*Full Access means access for all the above three situations

We had a good discussion on how to improve situations in countries where access is not complete.

Another discussion was had on the possibility of withdrawing Soliris and monitoring closely. This would reduce the cost per patient enabling more patients to get access to the drug. Patients were wary of this due to the risks involved.

Prof. Tim Goodship presented the history of aHUS treatment in the UK and said that currently a National Service has been created temporarily being managed by his team at Newcastle upon Tyne. The National Service is responsible to decide which patients should be given Soliris.

Overall, it was a great opportunity to meet so many others from different countries, each fighting the same battle. Here's a picture of the entire team:

image

... http://www.kamaldshah.com/2015/07/updates-from-ahus-uk-and-ahus-alliance.html

Kamal Shah

Kamal Shah

Hello, I'm Kamal from Hyderabad, India. I have been on dialysis for the last 13 years, six of them on PD, the rest on hemo. I have been on daily nocturnal home hemodialysis for the last four and half years. I can do pretty much everything myself. I love to travel and do short weekend trips or longer trips to places which have dialysis centers. Goa in India is a personal favorite. It is a great holiday destination and has two very good dialysis centers.

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