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!
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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:
|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|
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: