Innovations in Biotechnology and Medical Sciences

Indigenous Drug for Sickle Cell Disease developed

Note4Students

From UPSC perspective, the following things are important :

Prelims level: Sickle Cell Disease (SCD), Anemia

Mains level: NA

sickle cell

What is the news-

  • Based in Delhi, Akmus Drugs and Pharmaceutical Limited unveiled a groundbreaking drug for sickle cell disease on March 16.
  • This drug marks India’s first indigenous, room-temperature stable solution for sickle cell disease, available at a mere 1% of the global price.

What is Sickle Cell Disease (SCD)?

 

  • Sickle Cell Disease (SCD) is a genetically inherited red blood cell disorder.
  • It causes red blood cells to become hard, sticky, and sickle-shaped due to abnormal hemoglobin.
  • This abnormality leads to complications such as pain, infections, acute chest syndrome, and stroke.
  • Different types of SCD include HbSS, HbSC, and HbS beta thalassemia, each varying in severity.
  • SCD is diagnosed through a simple blood test, often identified at birth through new-born screening.
  • Treatment focuses on managing complications with options like bone marrow or stem cell transplantation, gene therapies, and medications like hydroxyurea to reduce symptoms and prevent complications.
  • The recent approval of Casgevy, a groundbreaking gene therapy utilizing Crispr-Cas9 technology,  represents a monumental achievement SCD Treatment.

About Akmus Sickle Cell Drug

  • The oral suspension of Hydroxyurea is the basic component of the drug.
  • Priced at less than ₹600, the drug is poised to revolutionize access to treatment for sickle cell disease patients nationwide.
  • It is tailored for patients across all age groups.
  • It offers convenience and precision in dosage administration through provided oral syringes.

What makes it a revolutionary drug?

  • Room Temperature Stability: Unlike imported hydroxyurea solutions requiring storage at 2-8 degrees Celsius, Akmus Pharmaceuticals’ formulation ensures accessibility without stringent storage conditions.
  • Cost-Efficiency: With the global brand priced at approximately ₹77,000, Akmus’ solution underscores a paradigm shift towards affordable medication.

Sickle Cell Disease Menace in India

 

  • Prevalence among STs: Sickle cell disease is a genetic disorder mostly prevalent in districts with high tribal populations. About one in 86 births among STs have sickle cell disease that affects haemoglobin in red blood cells, resulting in morbidity and mortality (as per WHO).
  • Health Priority: Recognizing sickle cell disease as a significant health challenge, India has identified it among the top 10 issues disproportionately affecting tribal communities.
  • National Mission:  The National Sickle Cell Anemia Elimination Mission launched in 2023, aims to eliminate sickle cell anemia from India by 2047.

 


PYQ:

Q. Consider the following statements in the context interventions being undertaken under Anaemia Mukt Bharat Strategy: (2023)

  1. It provides prophylactic calcium supplementation for pre-school children, adolescents and pregnant women.
  2. It runs a campaign for delayed cord clamping at the time of child-birth.
  3. It provides for periodic deworming to children and adolescents.
  4. It addresses non-nutritional causes of anaemia in endemic pockets with special focus on malaria, hemoglobinopathies and fluorosis.

How many of the statements given above are correct?

  1. Only one
  2. Only two
  3. Only three
  4. All four

Practice MCQ:

Q. Regarding the prevalence of Sickle Cell Disease (SCD) in India, consider the following statements:

  1. Sickle cell disease is a hereditary disorder affecting affects haemoglobin in red blood cells.
  2. About one in 86 births among STs have sickle cell disease.
  3. India has identified it among the top 10 issues disproportionately affecting tribal communities.
  4. The National Sickle Cell Anemia Elimination Mission, aims to eliminate sickle cell anemia from India by 2030.

How many of the given statements is/are correct?

  1. One
  2. Two
  3. Three
  4. Four

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