A committee of experts constituted by the Central Drug Standard Control Organisation (CDSCO) on Thursday critically evaluated a proposal of the ‘CSIR-Sickle Cell Anaemia (CSIR-SCA) Mission’ and approved the marketing of ‘hydroxyurea’ for SCA treatment, subject to post-marketing surveillance.
Sickle Cell Anaemia (SCA) is a common genetic disorder among Indians affecting the red blood cells. It is transmitted by parents carrying a defective ‘beta globin’ gene. Close to 0.4% of the population suffer from this disease while 10% are carriers who lead to birth of new SCA patients.
The Centre for Cellular and Molecular Biology (CCMB) with the help of pharma major ‘Cipla’, one of the manufacturers of hydroxyurea, and with active support of the Indian Institute Of Integrative Medicine (IIIM), has approached the Drugs Controller General of India for hydroxyurea approval for SCA treatment, said an official release.
The approval currently legalises the drug to be used at standard doses for treatment of SCA. It also sets up the stage for designing various formulations of smaller doses that promise higher compliance rates among children and may even lead to syrup-based formulations.
The disease is well-known in tribal populations as well as prevalent in general populations in Maharashtra, Madhya Pradesh, Chhattisgarh and Odisha. It starts early in life, and the affected children have persistent pain, low amount of haemoglobin (anaemia), low energy, reduced growth plus other abnormalities and multiple episodes of frequent severe pain better known as vaso-occlusive crisis.
Like most genetic disorders, SCA has no cure but has symptomatic treatments for pain, anaemia and vaso-occlusive crisis. Hydroxyurea, largely used as an anti-cancer agent, is also used in SCA treatment without any formal approval. Commercially available formulations are made with its anti-cancer role in mind, and hence, are of big quantity size (minimum 500 mg).
SCA children are typically of low weight, and consequently, their dosage size needs to be much smaller. So it becomes difficult to give the precise doses to such patients. This has led to low compliance and at times unpredictable response.
Under SCA Mission, about six CSIR labs and three government hospitals in Chhattisgarh, Madhya Pradesh and Maharashtra have joined scientists and clinicians trying to identify patients through population-based screening to help families with proper treatment and prevent the disease in the next generation.
