ICMR seeks to provide oral formulation of hydroxyurea to treat sickle cell disease in children

ICMR seeks to provide oral formulation of hydroxyurea to treat sickle cell disease in children

Sub: Science and tech

Sec: Health

ICMR’s Call for Expressions of Interest (EoI):

• Purpose: The Indian Council of Medical Research (ICMR) is seeking Expressions of Interest (EoI) from eligible organisations for the joint development and commercialisation of low-dose or paediatric oral formulation of “hydroxyurea” to treat sickle cell disease (SCD) in India.

Challenges in children’s dosage of hydroxyurea:

• Hydroxyurea is an effective treatment for SCD and thalassemia but currently only available in high doses.
• Most pharmaceutical companies in India offer hydroxyurea in 500 mg capsules or 200 mg tablets.
• The prescribed dose for children is 10-15 mg per kilogram of body weight after two years of age.
• There is a lack of hydroxyurea in suspension form, which is critical for paediatric patients.
• Breaking down high-dose tablets or capsules for children’s use is cumbersome and risks inaccurate dosing, affecting treatment efficacy.
• Due to the lack of paediatric doses and fear of toxicity, healthcare providers only initiate hydroxyurea therapy for symptomatic children as per the National Health Mission’s guidelines.

Proposed Solution:

• The availability of paediatric formulation would facilitate better dose titration and reduce dose-related side effects.
• ICMR is open to exclusive or non-exclusive agreements with manufacturing companies for the development and commercialisation of paediatric oral formulations of hydroxyurea for SCD.

Sickle cell disease (SCD):

• Sickle Cell Disease (SCD) is an inherited haemoglobin disorder characterised by a genetic mutation that causes red blood cells (RBCs) to assume a sickle or crescent shape rather than their normal round shape.
• This abnormality in RBCs results in increased rigidity, impairing their ability to circulate effectively throughout the body. Consequently, individuals with SCD often experience complications such as anaemia, organ damage, recurrent and severe pain episodes, and a shortened lifespan.
• Sickle cell disease is a common monogenic disorder of haemoglobin.
• India has the highest prevalence of SCD in South Asia, with over 20 million affected individuals.
• As per the Ministry of Health and Family Welfare, marginalised tribal populations are most vulnerable to SCD.
• Symptoms: Symptoms of sickle cell disease can vary, but some common symptoms are-
  • Chronic anaemia which leads to fatigue, weakness, and paleness.
  • Painful episodes (also known as sickle cell crisis) cause sudden and intense pain in the bones, chest, back, arms, and legs.
  • Delayed growth and puberty.
• Treatment Processes:
  • Blood Transfusions: These can help relieve anaemia and reduce the risk of pain crises.
  • Hydroxyurea: This medication can help reduce the frequency of painful episodes and prevent some of the disease’s long-term complications.
  • Gene Therapy: It can also be treated by bone marrow or stem cell transplantation by methods like Clustered regularly interspaced short palindromic repeats (CRISPR).

National Mission to Eliminate SCD 2047:

• There is a need for a paediatric formulation of hydroxyurea in line with the National Mission to eliminate SCD by 2047.
• Under the Sickle Cell Anaemia Mission, the Council of Scientific and Industrial Research (CSIR) is developing gene-editing therapies for SCD.

Source: TH