Over 30 million people in India, who are affected by thalassaemia, may soon get a first-ever drug that treats the genetic blood disorder.
Paris-based multinational pharmaceutical company, Sanofi, is working on a drug which aims to boost production of red blood cells and reduces the dependency of patient on blood transfusions and may involve India in the drug’s clinical trials, which are tests done on patients to establish a drug’s efficacy. These trials can take a few years to show results.
“Sanofi Genzyme is working on thalassaemia in alliance with another company close to our headquarters in Cambridge in Massachusetts, and together we are studying new therapeutics,” Carlo Incerti, managing director, senior vice-president and head of global medical affairs, Genzyme, an arm of Sanofi, told HT.
“We probably could initiate clinical trials in the next couple of years and when this will happen, India will be a potential country to be involved in the clinical trials because of the number of the patients and the knowledge of the disease,” Incerti said.
The company plans to study the effect of drug depending on the intensity of disease — from major to intermediate to minor. “In the thalassaemia study what we are going to study is the reduction in the transfusion demand by boosting the life or production of red blood cells,” Incerti said.
If such a drug is invented it could be a major breakthrough, said experts. “It will be a revolution in the treatment of thalassaemia,” said JS Arora, general secretary, National Thalassaemia Welfare Society.
“In India, 10,000 to 12,000 thalassaemia major children are born every year and there are over 100,000 thalassaemia major children who require regular blood transfusion.”