Questions relating to alternatives to eculizumab for aHUS ,and where those developments were up to, featured within many questions about eculizumab itself in the Rare Disease Day Video.
Urijah of Pueblo West California USA asks “I currently receive infusions every three weeks .I heard that there are trials being done with sub cutaneous injections and eight week infusions. When will I know the results?
The alliance’s Linda Burke undertook one the most comprehensive searches of the horizon for complement therapies in development and the results can be seen in the website info centre by clicking here.
Future therapies for aHUS are frequently to be found in developments going on for the treatment of PNH; as was the case for eculizumab. Generally the PNH pharma development cycle is about two years ahead of that for aHUS; but aHUS can still benefit.
Within Linda’s search the drug developments which appear to emerge of particular interest to the aHUS Community include:
Alexion’s new generation eculizumab, which also blocks Complement C5 , and is called ALXN 1210 . Phase 3 trials are beginning on aHUS patients over 12 year old, and are currently recruiting in USA, Australia, France, Korea and UK. The trial is expected to end in December 2019. ALXN 1210 will still be an infusion , albeit at eight week intervals, Urijah, but unlikely to be available to all until the early 2020s.
Molly of Kingston Ohio USA asks “Will I ever be able to get my medicine like a vaccine, a shot to the arm?”
Ryu of Japan asks “Can we expect any medicine for aHUS other than eculizumab?”
Coversin , produced by Akari Therapeuticals, is another complement inhibitor which blocks C5; but which is delivered by subcutaneous injection( into the skin) , Molly. It is currently being trialed ( Phase 2) for use on PNH patients to test doses at intervals of up to 140 days. The trial end date is estimated for December of next year.
If successful, Phase 3 trials, which would hopefully include use for aHUS patients , could begin in 2019 and conclude in the early 2020s. If it works well, Coversin could be available in 5/6 years, or more, from now. So Ryu an alternative to eculizumab could be available in time , and also not be delivered by infusion but by injection, Phoenix.
Phoenix of Massachusetts USA asks “Will eculizumab ever be available in a form other than infusion?”
Anna of Salem Oregon USA asks “When will new treatment be available?”
Possibly available sooner will be Omeros Corporation’s OMS 271, which is at Phase 2 trial stage for TMAs and still recruiting ,including aHUS patients 18 or older, and is due to be completed by June 2018. Another drug delivered by subcutaneous injection, but acting on a different part of Complement System – the Lectin pathway- on a component called MASP2. If the trial is successful , again it will be the early to mid 2020s before it is available, Anna.
Another drug with an alternative approach to complement inhibition is Alnyam’s ALN-CC5 . ALN-CC5 works by “knocking-down” production of the protein C5 in the liver and with spared use of eculizumab. Sub cutaneous injections at fourteen week dosing intervals have been tested in trials which were concluded at the end of 2016.
Isabella of Sothern California asks “Is there a pill form” and also Samuel of Jacksonville North Carolina USA asks “Will he be able to take a pill?”
The alliance has found no developments of a pill as yet Isabella and Samuel but will try to find out why.
Larkin of Zanesville Ohio USA asks “As for drugs in clinical trials when will they become reality and will they be affordable?”
However, clearly there are some alternatives to eculizumab in progress and are now at different stages of study of their clinical effectiveness. If they work, then licences have to be sought so that marketing can begin, and this is where the price comes in, and at present nobody knows what the cost of these drugs will be, nor how cost effective they will be, Larkin.
Laura of Nazareth Pennsylvania USA ” How long will we have to wait for some other form of treatment instead of an infusion every two weeks”
So for the rest of this decade, Laura , except for those recruited to trials, eculizumab remains the most clinically effective ,but not always affordable, treatment for aHUS.
Having said that for all these children who have asked questions, the next decade shows much promise for having a range of different effective treatments for aHUS if they still need them.