A sense of hope filled 14-year-old Bhavin Khare early Monday as he and his mother, Tanuja, left their home in Murbad, Thane, on a 100-km journey to Wadia Hospital in Parel. Bhavin was about to receive something that could change his life—a year’s supply of a US-made drug worth over Rs 1 crore that could ease the severe symptoms of cystic fibrosis, a rare genetic disorder he has battled since birth.
“Bhavin has been struggling all his life. He manages to attend school only about half the time because of constant coughing and chest infections,” said his mother. With the new medicine, she hopes he can finally experience what she calls a “normal life.”
Bhavin is among five children who received a three-month supply of Trikafta—a breakthrough drug from US-based Vertex Pharmaceuticals—at Wadia Hospital on Monday. Thirty-five more children are expected to receive it soon.
“We’ve signed an MoU with the company, which has agreed, on compassionate grounds, to provide a lifelong supply of Trikafta for 40 children,” said Dr Parmarth Chandane, head of the hospital’s pulmonology department, who spent nearly a year coordinating with the US firm. In total, the hospital had requested the drug for 80 children, but only 40 patients over six years of age met the eligibility criteria for the free-for-life supply.
What is Cystic Fibrosis?
Cystic fibrosis is a genetic condition that mainly affects the lungs and pancreas. It causes thick, sticky mucus to build up, leading to breathing difficulties, recurring lung infections, and digestive issues. In children, it often results in poor growth and difficulty gaining weight. If left untreated, it can lead to permanent lung damage and severe complications.
Diagnosing the Disease
Until recently, the standard diagnostic test—a sweat chloride test—wasn’t widely available in India. Between 1 in 10,000 to 40,000 newborns may be affected, though actual numbers could be higher due to underdiagnosis. Early intervention and proper treatment can significantly improve both the lifespan and quality of life for these children.
Drug Not Available in India
Due to patent restrictions, Trikafta is not available in India and must be imported. Children in wealthier countries have had access to the drug for nearly eight years. Meanwhile, parent groups in Karnataka, South Africa, and Brazil have approached their respective courts, demanding mechanisms such as compulsory licensing to make the drug more accessible.
“The irony is that the active pharmaceutical ingredients for this so-called miracle drug are manufactured in India,” said a doctor familiar with the case, “yet our children remain deprived of the final product.”