Thursday, January 18, 2024

Crovalimab trial in India in children and adults

Just a few weeks back, we posted this article where we alerted Indian aHUS patients to a trial on Iptacopan, an oral aHUS drug. And now there is another trial for aHUS patients for the drug Crovalimab from Roche.

Crovalimab is a complement inhibitor which can be administered to patients recently diagnosed with aHUS. The Phase III trial is for both adults and children who have not yet progressed to kidney failure (are dialysis dependent). 

You can find more details about the trial including the inclusion and exclusion criteria here: 



While Indian sites are not yet listed on these websites, we have confirmed information that one of the sites is Medanta Hospital, Gurgaon where Dr. Sidharth Sethi, eminent and reputed Pediatric Nephrology is conducting this trial. He can be reached at

This is an earnest request to all nephrologists and other doctors who come across aHUS patients in India. Please take this opportunity to enrol your patients into these trials. Until now, aHUS patients had nowhere to turn to. We no at least have trials happening in the country which should hopefully, pave the way for general availability in due course.

Sunday, October 22, 2023

A drug for aHUS: Currently Recruiting Adult Patients in India

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:

  • Thiruvananthapuram
  • Chennai
  • Vellore
  • Pune
  • Lucknow
  • Nagpur
  • Chandigarh

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.

Tuesday, May 10, 2022

An aHUS Drug may finally come to India!

The Atypical HUS India Foundation recently learnt of a clinical trial involving a complement inhibitor being conducted by Novartis. Iptacopan is an oral drug being manufactured by Novartis. They have also started a clinical trial for adult aHUS patients (read more here: NCT04889430) for which recruitment is ongoing.

This article titled aHUS Trial Watch 15 highlights key information for the currently enrolling Novartis APPELHUS study. With a twice daily oral delivery, and seemingly a small molecule drug rather than a biopharmaceutical, Iptacopan holds potential to become an aHUS therapeutic drug candidate which may finally address the dual issues of cost and access.

The Atypical HUS India Foundation is in touch with Novartis and they are looking for clinicians in India who might be interested in participating in this trial.

If you are a clinician interested in participating in this trial, please let us know by emailing us at and we will forward your email to the right person in Novartis and hopefully connect the dots. 

Also, please let us know of any other doctors who might be interested in this adult trial and we will reach out to them as well. Thanks!

If you are an adult aHUS patient, please share this website link with your doctor and encourage them to participate in this trial.

India has never seen a trial for a complement inhibitor. So, this is a fantastic opportunity for clinicians, patients and the aHUS community in general.

Once the trial completes, hopefully, if the results are as expected, Indians may soon have access to a complement inhibitor at last!

Saturday, September 26, 2020

Reflections on aHUS Awareness Day

 aHUS Awareness Day is observed on 24th September every year. The date was picked by members of the aHUS Alliance, an umbrella group of patient organisations from around the world. It started being observed only 5-6 years ago as that was around when the Alliance was formed. Every year, a flurry of activity is seen around this date. People from all over the world participate in various activities like videos of hope by patients, dissemination of information among the public and a lot of education about the disease.

And yet, with every passing year, while some countries move from one successful drug to another, Indian patients continue to die or be condemned to a life on dialysis. Some do recover after the initial flare but these are only a small proportion of the total.

I started The Atypical HUS India Foundation a few years back with the intent of providing information and support to Indian aHUS patients and their family members. When I was diagnosed way back in 1997, there was hardly anything that was known about this disease. We scoured the internet only to find small bits of information here and there. I thought having a website and social media accounts that provided information and support to Indian patients would provide at least some hope.

I was only partially correct.

When people get diagnosed and come across the aHUS India Foundation, their first question is what is the solution? They somehow cannot get their heads around a diagnosis that has a cure but is not available in India. What are the other options, they ask? My heart sinks when I get this question because I hate to have to tell them that there are no other options. Is there a way to get Eculizumab to India? There are some shady companies that claim to be able to get it to India but the quality is doubtful and the price is beyond the reach of anyone but maybe the top 0.0001% of Indians.

The US is now moving on from Eculizumab to Ravulizumab. The infusion frequency is reduced and patients would need to go only once every 1-2 months, roughly half the number of visits that Eculizumab required. Other developed countries will soon evaluate this new drug as well.

Several other drugs are supposed to be in various stages of clinical trials. The concern however is even if these drugs get approved eventually (several years from now), will they ever be brought to India? How would they be priced?

The curse of this disease is many fold. On the one hand is the severity of the disease. On the other hand is the miniscule number of patients that exist. Any drug that pharmaceutical companies develop has only a few patients to profit from. So each patient is charged a bomb.

I hate to sound negative. But the situation is hopeless. Unfortunately, I just do not see things changing in the near future at all. In India, even basic healthcare problems are far for being resolved. Just to take one example, India's infant mortality stands at 32 per 1000 births compared to the US's 5.7 and the UK's 3.9. This is hardly surprising when you see that India spends about 3.6% of its GDP on healthcare (of this, government spending is a shocking 1.3%) while the US spends about 18% and the UK spends about 10%

So, the problems are aplenty and the resources are few. In this dire state of affairs, how can we expect the Indian government to spend massive sums of money on drugs that will save only a few lives? If the money spent on one patient's cost of Eculizumab is spent on improving infant mortality for example, by strengthening the primary healthcare centre infrastructure in a rural village, several hundred lives can be saved. Which investment offers a better return?

And yet, try explaining that to Ananya's father. He has no clue about GDP figures and healthcare budgets. All he knows is that he lost his beautiful little daughter when there was a drug available that could cure her. But it was simply an accident of geography coupled with a cruel accident of genetics that took her away forever.

Friday, September 18, 2020

New aHUS Factsheet out

The aHUS Alliance Action comes out with a fact sheet every year which is an excellent resource of the latest information available from the aHUS world comprising of new research, status of new drugs for aHUS and so on.

Please click here to see the post on the Alliance Action website that has the factsheet which you can download.

Thursday, January 19, 2017

Address to the United Nations opening of the NGO Committee for Rare Diseases

Founder of Atypical HUS India Foundation, Kamal D. Shah spoke (via video) at the United Nations opening of the NGO Committee of Rare Diseases recently. The committee aims to address issues pertaining to rare disease patients across the globe.

Shah spoke about the issues of access to drugs in developing countries. Shah also said that the work of the committee can help patients from countries like India. Here, patients did not have access to drugs that can cure them even though the drugs were available elsewhere in the world.

This is a picture from the event.

Monday, August 15, 2016

An accident of geography?

I was in the middle of a meeting. My cell phone rang. The number was unknown. I muted the phone. I called back after the meeting was done. It was an unknown male voice. He introduced himself as Abhijit Chatterjee*. He spoke in Hindi with a heavy Bengali accent. I found it difficult to follow what he was saying. But I could figure that he was talking about his daughter, Ananya Chatterjee* who was diagnosed with aHUS. She was currently on Peritoneal Dialysis.

I get roughly one call a month these days. After setting up the Atypical HUS India Foundation, putting up a website and a Facebook and Twitter account and trying to publicise the news that trickles in from time to time, people have been contacting me. My response is usually very standard. After all, nothing much changes in the grim, dark world of aHUS in India. No Eculizumab. No other good options for most of the mutations.

Over the next several weeks, I got introduced to Abhijit Chatterjee through a series of different people. The Atypical HUS Foundation in the US, aHUS UK, other aHUS family members from different parts of the globe. The man was desperately trying every source of information and help he could find on the internet. He contacted every person or organisation he found linked to aHUS. Unfortunately for Abhijit Chatterjee, all his efforts finally ended at my email Inbox or my phone.

My response was standard and offered little hope. “Eculizumab is the only good treatment for this disease. It is not available in India. Until it becomes available (this may take a few years more) or another drug becomes available, we need to do maintenance treatment like plasmapheresis, plasma infusions, dialysis etc.” I introduced him to some leading hospitals and clinicians so that he could talk to them first hand and see what other options might be available. He probably got similar answers from them as well.

On one call, Chatterjee talked about his daughter. I could sense the fondness he had for her, for little Ananya.

“She is so cute and innocent. She has no idea what is wrong with her. She has such a sweet face. I am feeling very helpless. I have spent lakhs of rupees on her treatment. I have borrowed money from everyone I know. I am not sure how I will repay them. I just want her to become well.”

As my eyes swelled, I could feel my gut wrench. What have we done to deserve this? Wouldn’t it have been better to have a disease that had no cure? It was so frustrating to know that there is a cure, there is a drug that can cure us completely. It’s just that we can’t afford it. It’s just that we were born in a country that did not have access to it.

Then, one day, there was suddenly a ray of hope. It came in the form of an email from a company that was working on a new complement inhibitor. They wanted to explore the possibility of conducting a clinical trial in India. As eculizumab was already available in countries like the US and the EU, it made perfect sense for them to do a trial in a country like India where there would be patients without any options and who might be perfect candidates for a trial.

I promised all help. Their Asia representatives were asked to contact me. I put them in touch with all the clinicians I knew who could have such patients. My hopes however came crashing down when I learnt that the company was currently enrolling only adult incident patients. That is adult patients who had just been diagnosed and had not proceeded to kidney failure.

I wrote to the company asking them to include paediatric patients as well as they would be able to enrol many more patients that way. The company responded that it would get back to me after discussing with the management. I haven’t heard back from them yet.

About a month back, I got a call from Abhijit Chatterjee again. They had sent Ananya’s blood samples for genetic testing. The reports showed a Complement Factor I Mutation. She would need Eculizumab for a transplant, I thought to myself. The doctors were recommending a kidney transplant. I asked him what the plan was in case of recurrence. He said he did not know. I asked him to discuss with the doctor that since they did not have eculizumab, what was the point in undergoing a kidney transplant especially if she had a Factor I mutation?

He said he would discuss with the doctor and get back to me. 

A couple of days back, Chatterjee called again. I thought he must be calling to update me about the transplant plan. However, I could immediately sense from his voice that this was something else. He sounded completely distraught. Little Ananya was admitted in the ICU at a very reputed hospital in South India. She had worsening blood counts. Her Platelet Count was dangerously low. She was put on a ventilator. They were monitoring very closely. I did not say much.

That evening I could not stop thinking about the little girl. 

The next afternoon, I got a call from Chatterjee. I was honestly scared to take the call. I feared the worst. I answered the phone after an excruciatingly long wait. Chatterjee broke down. “It’s all over. Last night, Ananya had a cardiac arrest, multiple organ failure and then she died.” I hung up. My heart sank. My throat choked. My mind went blank. Little Ananya was no more.

I muttered a curse to the skies. I asked myself what was wrong with this world? Of what good was all the progress mankind has made when the life of a little girl could not be saved merely due to an accident of geography? Why do people in some countries have access to the drug while others do not? 

Alexion Pharmaceuticals, the company that manufactures the drug has priced it so high that very few people in the world can afford it. Forbes magazine has declared their drug to be the most expensive drug in the world today. They have probably spent millions of dollars on the research for the drug. They have probably spent a lot of time, money and effort in navigating the various patent laws and making their drug available to people. Given that the number of patients in the world is so small, are they not justified in pricing their drug high enough to be able to make profits?

I honestly don’t know the answer to that question. All I know is that what happened to Ananya is just wrong. I don’t know what can be done to change the situation for aHUS patients in India and many other countries in the world where the drug is not available. Something needs to change. How many more such lives are going to be lost fighting this disease? How many more Ananyas are going to die before this situation is remedied? Someone needs to find a way out.

(* Names have been changed to protect identity.)