Indigenous Sickle Cell Disease Drug Developed

Facts for Prelims (FFP)

 

Source: TH

 Context: Delhi-based Akmus Drugs and Pharmaceutical Limited has developed a new India’s first room-temperature stable indigenous drug for sickle cell disease.

 

How Akmus Sickle Cell Drug is a Game-Changer?

  • Basic Component: The drug’s primary component is an oral suspension of Hydroxyurea.
  • Affordable Price: Priced at less than ₹600 (at just 1% of the global price)
  • Suitable for All Ages: Tailored for patients of all age groups, ensuring broad applicability.
  • Precise Administration: Provided with oral syringes, it offers convenience and accuracy in dosage.
  • Room Temperature Stability: Eliminates the need for stringent storage conditions, unlike imported solutions requiring refrigeration (at 2-8 degrees Celsius)

 

What is SCD?

Sickle cell disease, a genetic blood disorder, impacts patients throughout their lives. SCD is a group of inherited Red Blood Cell (RBC) disorders or haemoglobin disorders. It changes the shapes of RBCs from round to a C-shaped farm tool called a sickle. Sickle cells clog the blood flow and die early, causing a constant shortage of RBCs.

 

India’s initiatives:

  • National Sickle Cell Anaemia Elimination Mission (launched in 2023, it aims to eliminate sickle cell genetic transmission by 2047)
  • The recent approval of Casgevy, a groundbreaking gene therapy utilizing CRISPR-Cas9 technology
  • India has acknowledged SCD as a top health concern for tribal communities, emphasizing the need for targeted interventions.