The light and dark world of aHUS

Article No. 453

25 August 2021

Recently Astra Zeneca concluded its purchase of the  rare disease business unit, Alexion.

$39 billion dollars was paid.

Those stakeholders who risked their investment which made aHUS and other disease treatments possible have now  been well rewarded. Recipients, including Alexion employees with shares and Board Members, will have benefitted too from being on the light side of the world surrounding the drugs eculizumab/ravulizumab.

There are many patients who have benefitted from complement inhibitors for our  rare diseases but many more who have not. The corporate rhetoric in the glossy pages of annual reports and website mission statements say little to young aHUS  children like Aadyan from India on the dark side of the same drugs.

It has been estimated that there would have been 60,000 more people with aHUS  living today if access to treatment was permitted to them ( Article 372) . Preventing the loss of another 60,000  lives  in the future will not be down to one solution.

Pharma are occasionally asked to show compassion to save just one life , but it cannot even afford to do so and yet be fully aware of what is going to happen.

Below is an article from four years ago. Little has changed for children like Aadyan with no access to existing, nor developing, drug therapies.


A question which featured a number of times in the Rare Disease Day video 2017  was about access to complement inhibitors for everyone with aHUS who needs them.

This is a big issue with many aspects. Among those questions was one by  Aadyan from India was one who asked specifically :

 Is there any way out for patients who can come out from the dark world of aHUS in India where we can get access to a low cost drug?



The alliance has featured several news items about aHUS in India and the issues faced there  in getting a treatment for aHUS

The story about Annanya which featured in an article last year ( read here) illustrates the grave problems patients face.

An alliance associate from aHUS India also spoke at the UN about accessing drugs for rare diseases in countries like India – South Africa faces similar issues.

There are hurdles to overcome even if  research has found found a clinically effective treatment which there is for aHUS even if it is not a cure.

Firstly the manufacturers of the treatment must want to sell their products in the country.

For Indian aHUS patients so far no Pharma has sought to market a complement inhibitor in India; albeit that a couple of small sales of eculizumab have happened under very strict importing controls.

Pharma are reluctant to sell their drugs in India because the patents that covers the drug might be abused by companies in that country which make unregulated copies of the drugs.

So access to eculizumab falls at the first hurdle in India.

But what if  the hurdle could be over come.

Successful access to treatment also needs a leading clinician in the country to recommend it. India has many eminent clinicians who would support access to eculizumab, one in particular, who is prominent at aHUS conference around the world, is Prof. Avrind Bagga.

Prof  Bagga has extensive experience of treating Indian aHUS patients in New Dehli , particularly those with Anti Factor H antibody mediated aHUS. His registry of known  aHUS patients has grown to over 500 patients. There could be between 2500 and 7000 aHUS patients in India (Using the prevalence estimates from the answer to Alaina’s question about how many aHUS patients there are ) .

Prof Bagga is holding a Consensus Meeting* about treating aHUS in India at the end of this month , 31 March in New Delhi. He is bringing together dozens of Indian clinicians from around India with an interest in aHUS to develop  treatment guidelines for aHUS in India.

Pharma will no doubt be observing this development and hopefully may be considering ways and means to make something available with a price offering that in itself is not a barrier. We will see.

aHUS awareness day 2021 is on 24 September.

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