CDSCO approval granted for Eculizumab for rare disease

Eculizumab is a drug that targets a key part of the immune system called the complement system and inhibits the same.

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CDSCO approval granted for Eculizumab for rare disease
CDSCO approval for Eculizuma

Last Updated on January 17, 2025 by The Health Master

Eculizumab

The Central Drugs Standard Control Organisation approval (CDSCO approval) has been granted to AstraZeneca Pharma India to import, sell, and distribute Eculizumab, a crucial anti-complement therapy.

This CDSCO approval marks an important milestone for patients in India who are suffering from two rare and life-threatening following conditions:

  • Paroxysmal Nocturnal Hemoglobinuria (PNH) and
  • Atypical Hemolytic Uremic Syndrome (aHUS).

What is Eculizumab ?

Eculizumab is a drug that targets a key part of the immune system called the complement system and inhibits the same.

The complement system becomes overactive In both PNH and aHUS which lead to severe and potentially life-threatening issues.

How Does Eculizumab Help ?

By blocking the above overactivity, Eculizumab helps to:

  • Reducing the blood clots: Both PNH and aHUS can cause a high quantity of blood clots, and the same can cause damage to vital organs like the kidneys, heart, and brain.
  • Protecting red blood cells: The overactive complement system destroys red blood cells in PNH, which leads to anemia, fatigue, and other serious issues.
  • Improving quality of life: Eculizumab can significantly improve the quality of life for the patients by controlling the symptoms of these diseases, which allow them to live longer lives.

The Impact of PNH and aHUS

Paroxysmal Nocturnal Hemoglobinuria (PNH):

This type of rare blood disorder actively affects the red blood cells, which can be destroyed by the immune system.

PNH can cause:

  • Chronic anemia: Constant destruction of red blood cells results in weakness, fatigue, and shortness of breath.
  • Blood clots: These can block blood vessels, causing strokes, heart attacks, and kidney damage.
  • Severe pain: sudden, severe pain in the abdomen or back may occur.

Atypical Hemolytic Uremic Syndrome (aHUS):

This type of rare condition damages small blood vessels in the body, which may primarily affect the kidneys.

Key characteristics include:

  • Kidney failure: An acute kidney injury can lead to chronic kidney disease, and the same may require dialysis or even transpalnt of the kidney.
  • Neurological issues: vision problems, strokes, seizures can occur.
  • Gastrointestinal problems: vomiting, diarrhea, and abdominal pain are common.

The importance of the CDSCO Approval for Indian Patients

The CDSCO approval of Eculizumab brings a great hope to patients in India living with PNH and aHUS.

  • Access to life-saving treatment: Previously, access to this vital therapy might have been very difficult and limited for many patients in India.
  • Enhancing quality of life: With Eculizumab, patients can get significant improvements in their health and well-being.
  • Reducing risk of complications: Timely treatment can prevent serious problems, e.g., kidney failure, stroke, and other life-threatening issues.

1. What is Eculizumab ?

Eculizumab is a drug that targets a key part of the immune system called the complement system and inhibits the same.
The complement system becomes overactive In both PNH and aHUS which lead to severe and potentially life-threatening issues.

2. What are the side effects of Eculizumab?

Like any other drug, Eculizumab can have side effects. Common side effects may include headache, dizziness, and injection site reactions.

3. How is Eculizumab administered?

Eculizumab is typically administered intravenously (through a vein) by a healthcare professional.

Disclaimer: This article contains information obtained from the source mentioned below. Our team made changes in the format to rewrite and present the news or article in a unique format.

Disclaimer: The information we have provided is for general knowledge and for informational purpose only and it cannot be treated as medical advice. Always consult your doctor for any health issues and / or for the treatment of the same. The Health Master does not claim responsibility for this information.

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