Novartis is currently recruiting adult patients in India in clinical trials for its twice-a-day oral drug Iptacopan. This is a very good opportunity for clinicians in India to enrol their patients into this trial. Here are the locations where they are recruiting patients:
This trial is only for those who have not progressed to End Stage Kidney Disease requiring dialysis. They need patients who have been diagnosed recently and those have an active disease. The listing of this trial in the Clinical Trials website states that eligible patients need to have "evidence of thrombotic microangiopathy (TMA), including thrombocytopenia, evidence of hemolysis, and acute kidney injury". This is a fantastic opportunity for doctors who encounter aHUS patients who have not yet gone into kidney failure. Please give your patients a chance at a completely normal life by enrolling them in this Phase 3 trial after examining the eligibility criteria and ensuring that this trial could help your patient.
We get several emails from newly diagnosed patients who do not know what to do when they get diagnosed with this disease. This is a golden opportunity for them.
Unfortunately, when most of us with aHUS in India got diagnosed, there was no such trial going on in India and no drug was available in India. The only drug currently approved for sale across the world is Soliris (drug name: Eculizumab) from Alexion Pharmaceuticals. Soliris is not being sold in India. It is also not possible to buy the drug and import it to India as the company does not sell it to individuals just like that.
Some companies claim to sell it to individuals but we are not sure of the authenticity or the reliability of such channels.
The drug availability for aHUS in India is slowly changing. More companies are likely to bring their drugs to the country. For clinical trials and even for sale after approvals. The way they will price their drugs is still unclear. The challenge of recovering their research costs from a very minute size of the population still remains. India is attractive for two reasons - the potentially large number of patients and the non-availability of any other drug for the disease.
So, those with aHUS - don't lose hope just yet! A solution may be coming soon.