Clinical trials for cutting edge, game-changer heart drug set to begin in India

Pune: India is set to launch a crucial clinical trials to evaluate Mavacamten, a cutting-edge drug for symptomatic obstructive hypertrophic cardiomyopathy (HCM).
The heart muscle becomes abnormally thick in this genetic heart condition, making it harder for the heart to pump blood efficiently. In some patients, thickened heart muscles may severely obstruct blood flow, leading to breathlessness, chest pain and may even cause heart failure.
"Unlike traditional medications such as beta-blockers or calcium channel blockers, which only manage symptoms, Mavacamten directly targets the underlying cause of obstruction in HCM. The US FDA has approved it under the brand name Camzyos and has shown significant success in improving heart function and quality of life in global clinical trials," said senior cardiologist Dr Suhas Hardas.
The Phase 4 open-label trials would be conducted at Amrita Institute of Medical Sciences and Research in Kochi and a few other sites (currently in process of shortlisting). It will enroll a total of 50 Indian patients with obstructive HCM, a disease which affects one in 500 people, similar to global estimates, but remains vastly underdiagnosed and untreated in India.
After the necessary drug regulatory clearances, the trials will begin within next two months.
"Currently, Mavacamten is not available in India. If this trials confirms the drug's safety and effectiveness, it could pave the way for Mavacamten's availability in India by late 2025, offering a new hope to thousands of patients struggling with this heart condition," Dr Hisham Ahamed, the principal investigator of the Mavacamten trials in India and also the project lead at Amrita Hypertrophic Cardiomyopathy Centre at Amrita Institute of Medical Sciences and Research in Kochi.
The trials aim to confirm that Mavacamten reduces heart obstruction, relieves symptoms and improves overall quality of life in Indian patients. "Since Indian HCM patients may have distinct disease patterns, this study will provide important real-world data to guide treatment decisions," Dr Ahamed said.
Dr Aftab Khan, senior interventional cardiologist of Apollo Hospitals, Kolkata, said, "Mavacamten works by inhibiting the cardiac myosin and thus helps in reducing the cardiac muscle contractility, a beneficial effect in hypertrophic hearts . Its only serious side-effect is heart failure caused by reduction in ejection fraction, but it can be avoided by regular echocardiogram and stopping the medication if the heart ejection fraction drop below 50 %."
Mavacamten has shown promising results in global trials, but there is little data on how it works in South Asian patients. "The pivotal EXPLORER-HCM trial, which led to Mavacamten's approval in the US, had a predominantly white participant pool, with Asians making up only 2.4% of the total. Since genetics and disease patterns differ across populations, this Indian study will help determine how well the drug works for Indian patients," Dr Ahamed said.
"If Mavacamten proves effective in Indian patients, it could reshape treatment guidelines and ensure equitable access to this breakthrough therapy," Dr Hardas said.