Ravulizumab is the next generation complement inhibitor for aHUS.
It still uses eculizumab as the active ingredient but the drug has been re engineered to be effective for longer in the blood.
Infusions of Ravulizumab are therefore done at eight week intervals instead of every two weeks for eculizumab.
It has had marketing authorisation in the USA for PNH for over a year and for aHUS since October 2019. New on set patients there are receiving it and existing eculizumab treated patients have begun the transition to Ravulizumab treatment.
It has not been authorised by the European Medicine Agency, EMA for aHUS use as yet, but such authorisation is expected soon.
EMA authorised it for PNH last summer and NICE began its process to evaluate Ravulizumab for PNH in November. Starting with the “scoping stage”. It was a surprise however when NICE wrote to stakeholders, including Answers for aHUS, before Christmas asking for views on a draft scoping document for an evaluation of Ravulizumab for aHUS.
A scoping document sets out the basis for an evaluation. What the drug will treat, how many patients, what alternative treatments it should be compared with , what the relative clinical outcomes would be.
A response has been given by Answers to the draft.
NICE will host a “scoping workshop” on 27 January 2020 to consider all comments made by stakeholders including those from Answers.
This is just the beginning of the beginning just as aHUSUK were in October 2011 when a scoping meeting was held for eculizumab for aHUS.
If NICE agree to move to the next stage, the supply of evidence, Answers will again provide what is needed to make a relevant and compelling patient case. There is likely to be a call to action from the aHUS patient community.
More will be known and communicated after the scoping workshop meeting.
Note. The Answers for aHUS website has been “inactive” for several years and has been merely an archive of previous advocacy. It has now been woken up to provide information about the next journey for aHUS in the UK that of gaining Ravulizumab for aHUS.