‘Normal life’ within reach: Mumbai doctor's global outreach brings Rs 1-crore US drug Trikafta to Indian kids; 40 cystic fibrosis patients to benefit

MUMBAI: Early Monday morning, 14-year-old Bhavin Khare and his mother, Tanuja, set off from Thane's Murbad for Wadia Hospital, Parel, around 100km away, with a sense of anticipation. They had to collect a US drug—estimated to cost over Rs 1 crore for a year's supply—that would help him control the debilitating symptoms of cystic fibrosis, a genetic disease he suffers from."Bhavin has been struggling all his life. He manages to go to school only 50% of the days due to repeated cough and chest infections," Tanuja said, hoping the new drug would let him lead a "normal life".He is one of five children with cystic fibrosis who received a three-month supply of Trikafta, manufactured by US-based Vertex Pharmaceuticals, at the hospital Monday. Thirty-five others are scheduled to get it in the coming days."We have signed an MoU with the company which, on compassionate grounds, will provide a lifelong supply of Trikafta to 40 patients," said pulmonology department head Dr Parmarth Chandane, who communicated with the US company for almost a year.Due to patent laws, the drug isn't available in India and can only be imported. Affected children in wealthy nations have had access to the drug for almost eight years, and parents' groups in Karnataka, South Africa and Brazil have moved local courts seeking some mechanism like compulsory licensing to ensure their children get access to the drug, too. "The active ingredients for the three-drug combination (Trikafta—elexacaftor/tezacaftor/ivacaftor) that is now being called a miracle, are made in India, but our children don't have access to the finished product," said a doctor who didn't want to be identified.The Wadia Hospital distribution is the beginning of an alternative access for Indian patients. "The drug has shown promise, with certain research reports claiming patients will be able to enjoy a normal lifespan of 80 years as against the 15-20 years one normally associates with the disease," said Dr Chandane, who communicated with the US company for almost a year, seeking access for the 80 patients registered at Wadia Hospital. The company, however, said it would only provide drugs for patients over six years of age, resulting in only 40 qualifying for the free-for-life supply.Each of the affected children has to take three drugs—two in the morning and one in the evening—daily to prevent the buildup of thick, sticky mucus that usually clogs organs such as the lungs, pancreas and intestines, which could further lead to malnutrition, poor growth, frequent respiratory infections, as well as chronic lung disease.It's learnt that Vertex Pharmaceuticals will provide lifelong supply of the drug to patients registered at CMC Vellore in Tamil Nadu, too. "We are also in talks with the company for our patients," said paediatrician Dr Indu Khosla from SRCC Hospital, Haji Ali."By offering such costly and essential medication free of charge, we're removing financial barriers for families. These children are getting a chance to breathe easier, grow healthy and live longer," said Dr Minnie Bodhanwala, CEO of Bai Jerbai Wadia Hospital for Children.