Hydroxyurea approved for Sickle Cell Anemia treatment

An expert committee constituted by the Central Drug Standard Control Organisation has approved the marketing of hydroxyurea for the treatment of Sickle Cell Anemia (SCA).

The approval, researchers said, legalizes the drug usage in standard doses for SCA treatment, while allowing the designing of various formulations of smaller dose sizes that promise higher compliance rates in SCA children and may even lead to syrup-based formulations.

SCA is a common genetic disorder among Indians affecting red blood cells. It is transmitted by parents who carry a defective beta-globin gene without suffering themselves. Close to 0.4 per cent of the population suffers from the disease while 10 per cent people act as carriers leading to birth of new SCA patients. The disease is well-known in tribal populations and also prevalent in general populations in states like Maharashtra, Madhya Pradesh, Chhattisgarh and Odisha.

Like most genetic disorders, SCA has no cure but has symptomatic treatments for pain, anemia and Vaso-occlusive crisis. One of the rather inexpensive drugs, hydroxyurea, largely used as an anti-cancer agent, is also used in SCA treatment without any formal approval.

But the commercially available hydroxyurea formulations are made with its anti-cancer role in mind, and so, are of big quantity (minimum 500 mg). Since SCA children are typically of low weight, their dosage size needs to be much smaller.

Given the fixed, larger size of commercially available hydroxyurea capsules, it is difficult to provide the correct dosage to SCA patients. Although there is robust response to hydroxyurea therapy, the complexity and cumbersome nature of disbursing the right dose often leads to low compliance and sometimes unpredictable response.

The Central Scientific and Industrial Research's Sickle Cell Anaemia Mission involving six CSIR labs and three government hospitals located in Chhattisgarh, Madhya Pradesh and Maharashtra is trying to address these lacunae in SCA diagnosis and disease management in the country.

The mission focus is on identifying the patients through population-based screening in states with high disease prevalence and helping the family with proper treatment and preventing the disease in the next generation. One of the mission objectives has been obtaining approval for hydroxyurea use in SCA treatment.

The CSIR-SCA Mission, coordinated by the Hyderabad-based CSIR-Centre for Cellular and Molecular Biology (CSIR-CCMB), with help of Cipla, a Hydroxyurea manufacturer, had approached the Drugs Controller General of India for approval.

On December 9, the expert committee of CDSCO evaluated the proposal and approved the marketing of hydroxyurea for SCA treatment, subject to post-marketing surveillance.

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