Tuesday, September 16, 2025

How India's National Program for Rare Diseases can help aHUS patients

Here are some important facts to know about the Indian Government's National Program for Rare Diseases (NPRD) can help aHUS patients.

National Program for Rare Diseases

This program, instituted by Ministry of Health and Family Welfare, offers financial help and access to care for rare disease patients. As of August 2024, 63 rare diseases were covered under this program. Atypical Hemolytic Uremic Syndrome (aHUS) is part of this list of diseases.

Three groups of rare diseases are covered by this program:

  1. Diseases that can be cured with a one-time treatment
  2. Diseases that need lifelong treatment but costs are low
  3. Diseases that need lifelong treatment and costs are very high
aHUS comes under the third group.

Support offered by the government

The government will fund up to Rs. 50 lakh per patient for treatment. The government may offer extra flexibility for Group 3 cases like aHUS, based on the patient’s situation. No GST or customs duty will be levied on rare disease drugs imported for treatment.

Centres of Excellence

The government has identified a list of Centres of Excellence (CoE) which would be the nodal agencies to facilitate treatment under this program. Here is a list of the CoEs in different cities in India.

Things to do to avail the support from the government

Patients must speak to their doctors about registering with the NPRD at a Centre of Excellence. This would involve medical evaluation to confirm the diagnosis, some documentation which would included some tests, medical history etc. There is an application form that would need to be filled in. Once that is submitted, then patients would need to wait for the approval to come.


List of Centres of Excellence under NPRD

Here is a list of the Centres of Excellence listed under the National Program for Rare Diseases (NPRD). These centres are authorised to approve any treatment for rare disease patients under the NPRD where a fund of up to Rs. 50 lakhs is granted.

  • All India Institute of Medical Sciences, New Delhi
  • Maulana Azad Medical College, New Delhi
  • Sanjay Gandhi Post Graduate Institute of Medical Sciences, Lucknow
  • Post Graduate Institute of Medical Education and Research, Chandigarh
  • CDFD with Nizam Institute of Medical Sciences, Hyderabad
  • King Edward Memorial Hospital, Mumbai
  • Institute of Post-Graduate Medical Education and Research, Kolkata
  • Center For Human Genetics (CHG) with Indira Gandhi Hospital, Bengaluru
  • All India Institute of Medical Sciences, Jodhpur
  • Institute Of Child Health and Hospital for Children, Chennai
  • Government Medical College, Thiruvananthapuram
  • All India Institute of Medical Sciences, Bhopal

Monday, September 15, 2025

Eculizumab Launched in India

In a long-awaited welcome development, Eculizumab has been formally launched in India. In a press release issued recently, AstraZeneca, the company that acquired Alexion Pharmaceuticals, the manufacturer of the drug (brand name: Soliris) said, "Until now, limited awareness, diagnosis, and access to targeted therapies have contributed to prolonged hospitalisations and increased complications for those affected. The availability of Eculizumab marks a breakthrough in the standard of care."

For aHUS patients in India, this is a tremendous breakthrough in their collective journey. Until now, the drug was either not available or available only by importing it specifically for their use - a very arduous and complicated process.

Hopefully now, that would be a thing of the past. While there is still some lack of clarity on how the drug can be accessed and the cost at which it is available (which the aHUS India Foundation is working closely with the company on getting more information about), at least the delays in importing the drug would not be there.

The aHUS India Foundation strongly urges patients to speak to their doctors on their treatment plans and how they would change with the availability of the drug.

The Foundation also urges doctors with aHUS patients to be aware that the drug is now available and to please reach out to the company or us (ahusindia@gmail.com) and get access to the drug for your patients.

We, at the aHUS India Foundation are grateful to the company and the regulatory authorities for their efforts in making the drug available in India.

Saturday, September 6, 2025

aHUS India - Patient Journeys

Often beyond all the news and information, real patient journeys get hidden. These journeys are however the real story. Individual stories of pain, suffering and yes - even in the midst of all the despair - hope. We, at the Atypical HUS India Foundation are bringing to you such stories where patients have fought back to take control of their lives.

In the first such story, we bring to you a young male from Mumbai who was diagnosed with this disease out of the blue, with no prior warning. This diagnosis turned his life upside down. And yet, he continues fighting, even today in the hope that one day he will get access to a complement inhibitor which will enable him to lead a normal life.



Thursday, July 3, 2025

aHUS Simplified - Webinar Summary

Here is a summary of what was discussed in the webinar we held on 30 June:


Dr. Arvind Bagga (Pediatric Nephrologist, Indraprastha Apollo & Professor Emeritus, AIIMS Delhi)

Presented a foundational explanation of the immune system and its two parts – innate and adaptive immunity.

Focused on the complement system, especially the alternative pathway which is rapidly activated and can become overactive in aHUS.

Explained how regulatory proteins (Factor H, Factor I, CD46) function as brakes on the complement system; their failure leads to uncontrolled immune activation and damage to the body’s own tissues.

Outlined that aHUS leads to microangiopathic hemolytic anemia, thrombocytopenia, and acute kidney injury – hallmark features of thrombotic microangiopathy (TMA).

Described diagnostic process, stressing it is largely one of exclusion, and explained the limited utility of low C3 as a consistent diagnostic marker.

Detailed genetic causes (autosomal recessive or dominant mutations in Factor H, I, CD46, C3, DGKE) and role of Factor H autoantibodies in Indian children.

Reiterated that plasma exchange is particularly useful in anti-Factor H autoantibody cases, but not a substitute for complement inhibitors like Eculizumab.

Urged greater awareness among pediatricians and hematologists for early diagnosis and referral.

Dr. Valentine Lobo (Senior Nephrologist, KEM Hospital, Pune)

Highlighted the evolving nomenclature, noting that 'aHUS' is now better classified under complement-mediated thrombotic microangiopathies (TMAs).

Shared his extensive clinical experience in adults: common triggers include pregnancy (40% of his cohort), snake bites (16%), transplants, drugs, and autoimmune diseases.

Discussed difficulty in distinguishing primary (genetic/autoimmune) vs secondary aHUS (triggered by known factors). Emphasized that genetic testing is not always required for secondary aHUS.

Provided criteria for diagnosis in adults – anemia, thrombocytopenia, acute kidney injury – and how it's often missed due to overlapping symptoms with more common illnesses.

Outlined findings from his cohort of 121 patients over 12–15 years and the role of biopsy in complex cases (e.g., post-transplant or late presentation).

Warned against unregulated dosing once complement inhibitors become available, emphasizing the importance of making an accurate diagnosis first.

Described current and past clinical trials in India (Crolimab, Iptacopan), with India contributing significantly due to high patient volumes.

Stressed the urgent need for reference labs for anti-Factor H testing, genetic interpretation expertise, and establishment of structured treatment pathways at COEs.

Shiv (Patient Speaker)

Shared a deeply personal account of living with aHUS: first symptoms appeared at age 22 with hematuria, jaundice, and high BP; was quickly placed on dialysis.

Received plasma exchange but sustained irreversible kidney damage; underwent 11 months of dialysis before kidneys partially recovered (~40% function).

Maintained partial function from 2014–2024, after which kidney function again declined and he resumed dialysis.

Only recently diagnosed with a CFI mutation after undergoing genetic testing, unavailable during his initial episode.

Highlighted emotional toll, work-life challenges, and physical exhaustion from dialysis, but also stressed importance of family support and community.

Encouraged others to explore India’s National Rare Disease Policy and register under the scheme through COEs to gain access to future therapies.

Expressed hope for access to eculizumab or ravulizumab in India, required before his planned kidney transplant.

Audience Q&A (with Drs. Bagga & Lobo)

Recurrent disease post-transplant is likely due to a pathogenic complement mutation – full genetic workup recommended (gene sequencing, CNV, haplotype, and therapy prediction).

Plasma exchange is superior to plasma infusion in acute settings due to higher volume clearance of antibodies and regulators.

Withdrawal of eculizumab therapy has been studied: low relapse rates in patients with no detected mutation, but higher in those with pathogenic mutations.

Typical vs Atypical HUS vs TTP: History of diarrhea suggests typical HUS (Shiga toxin); low platelets with less severe kidney failure points to TTP; aHUS is a diagnosis of exclusion.

Doctors called for co-specialty awareness (hematologists, internists) to avoid misdiagnosis and delays in therapy.

Announced development of a point-of-care test (similar to a pregnancy test) for anti-Factor H antibodies in collaboration with Spanish researchers and Indian labs (THSTI, ICMR).

Stressed need for strong guidelines to ensure correct dosing and avoid cost-driven under-treatment once drugs are introduced.

Kamal Shah (Moderator & Host; Founder, aHUS India Foundation)

Introduced the webinar as the first of its kind in India for aHUS patients, highlighting the historical lack of access to treatment.

Expressed hope due to recent advancements and encouraged patients to engage by registering with the foundation, sharing their stories, and subscribing to the newsletter.

Explained that aHUS was previously seen as a double misfortune – genetic and geographic – but now there's hope with new treatments becoming available in India.

Described the Indian government's National Policy for Rare Diseases, which offers financial support of up to ₹50 lakh for patients under Category 3 (treatable but high-cost diseases like aHUS).

Detailed the need for patients to register with designated Centers of Excellence (COEs) through their doctors to access this scheme.

Listed 12 COEs and provided the foundation’s website (www.hus.in) for further information.

International Guests Acknowledged

Linda Burke (USA), Jeff Schmidt (USA), and Margriet Eygenraam (Canada) who had joined the webinar from various parts of the world were acknowledged for their contributions to the global aHUS community. 

Recognized for their leadership and continued support to Indian patients through international collaboration and awareness.

Closing Remarks

Kamal Shah reiterated key calls to action: register with the aHUS India Foundation, subscribe to the newsletter, and submit patient stories.

Appreciated the participation of all speakers and attendees and promised to conduct more such webinars.

Thanked AstraZeneca for supporting the event, and the Kidney Warriors Foundation for its consistent partnership.

Wednesday, July 2, 2025

Webinar Recording: aHUS Simplified for Patients

We had an overwhelming response for the first aHUS webinar we held on Monday 30 June. For those who missed it and wish to watch it or if you would like to share it with someone you know, here is the recording:


You can use this link to share it as well:  https://youtu.be/C6oY0E9OMN4


Saturday, June 21, 2025

Our first webinar for aHUS Patients!

The Atypical HUS India Foundation is hosting its first webinar. This event is for aHUS patients and their families, aiming to simplify the complex disease. There will be sessions for both adult and child patients, as well as their families. Anyone interested in learning about the disease can join too. The event is being done in collaboration with NephroPlus, Asia's largest dialysis provider, Kidney Warriors Foundation, India's leading kidney patient support group and AstraZeneca.

We are excited to have two top nephrologists, Dr. Arvind Bagga from Delhi and Dr. Valentine Lobo from Pune. Both have done significant work on aHUS and are very passionate about it. We will also have a patient who will recount his journey with this disease.

The webinar will be online on Zoom on Monday, June 30th at 4:30 pm. Please mark your calendars and use this link to register.



Wednesday, May 14, 2025

Share your aHUS story

Tell the world your story! The Atypical HUS India Foundation invites patients and their family to record short videos about their story - how did you know you had aHUS? What did you do soon after? What is your current status? What frustrates you about our situation? Share your videos with us by tagging us on social media or sending us an email.

Sunday, May 11, 2025

The importance of a database of aHUS patients in India

If you ask someone what is the number of aHUS patients in India, the only answer you will get are 'guesstimates'. No data exists on the number of aHUS patients in India. Given estimates of the global prevalence of this disease of 2-3 per million people, one can assume that there could be between 2000-3000 patients. That is a huge number.

The trouble with this disease is that is so rare that most doctors rarely see aHUS patients. And surprisingly, while research shows more adults are diagnosed than children, most doctors think of this as a pediatric disease. In India, probably, there are more children diagnosed with aHUS than adults simply because of more awareness of this disease among pediatricians and pediatric nephrologists.

The aHUS India Foundation is now launching a registration for aHUS patients in India. All you need to do is to fill this form and you will be added to the aHUS database. The database is completely confidential and the data in it will not be shared with anyone - including doctors, researchers, pharmaceutical companies, the government etc. No one will have access to your information. No one can identify you after you've filled in this form.

Without your help, however, we will not be able to build this database. Without this database, we cannot lobby with the government to improve access to treatment for aHUS patients. Without this database, we cannot have discussions with pharmaceutical companies on the number of aHUS patients in India which is important for them to bring their drugs to India.

So, please take a minute to fill out the form. It takes less than 2 minutes and many of the questions are optional. Also share this with any other patients you might know. Share this with your doctor and request them to ask their aHUS patients to fill it out.

Here's the link to share with anyone:

https://forms.gle/yBMqTanwwW4e34GD6

Thanks so much for your support!

Saturday, January 18, 2025

Soliris gets approval for sale in India

Soliris (brand name for the drug Eculizumab from Alexion Pharmaceuticals, now acquired by Astra Zeneca) was finally approved by the Indian drug control body, Central Drugs Standard Control Organization (CDSCO) yesterday.

The drug was advised for approval by the Experts Committee in May last year. The approval finally came yesterday. This is very welcome news for patients afflicted by life-changing diseases such as Atypical Hemolytic Uremic Syndrome (aHUS).

This clears the way for Astra Zeneca to start marketing and selling the drug in the country.

What remains to be seen though - and this is critical for a country like India - is what the drug will be priced at by the company. This is what will dictate if anything at all changes for aHUS patients. If the drug is priced even close to what it is priced at in developed countries, this approval does not mean anything at all. At least for most aHUS patients.

A lot of the discussion around this drug often centres around patients who need it short term. While there are patients who need it short term - some cancer patients, patients with certain conditions and even some aHUS patients with a certain set of mutations - the vast majority of aHUS patients are affected by mutations like those in the Complement Factor H (CFH). These patients would need the drug long term, potentially life long.

The Indian Government's Rare Disease Program provides a grant of Rupees Fifty Lakh (5 million) to rare disease patients which, at the current rates in developed countries, will barely fund the drug for a few months. What happens after that?

So, while aHUS patients in India do have a reason to cheer news of this approval, we cannot start celebrating yet. News on pricing of this drug is the next most critical aspect that we now await.