CYSTIC fibrosis (CF) has long been a silent battle in Pakistan that is fought in hospitals and homes. Every chest infection chips away at lung function, and the condition gets worse without proper treatment, leading to many a death.

Luckily, things are taking a turn for the better in the wake of the decision by the Drug Regulatory Authority of Pakistan (Drap) which has recently approved a ground-breaking CF gene modulator therapy as a treatment option.

Even though the therapy is among the costliest in the world, the Indus Hospital in Karachi has taken the initiative to provide it for free to those who meet the specified criterion.

There are 51 patients — children, teenagers and young adults — who have been receiving the therapy for the last three months, and the results have been remarkable in the shape of improved lung function, weight-gain, fewer infections, better sleep, and renewed energy levels.

Approved in over 30 countries since 2019, the therapy has transformed outcomes for up to 90 per cent of CF patients in some populations. Until recently, such treatment was out of reach for anyone in Pakistan.

After the Drap decision, the therapy must not remain confined to one hospital or even a few. We need nationwide access, early screening, more trained specialists, affordability measures, and local data to guide expansion.

The therapy, let us remember, does not just extend life, it transforms it. In the fight against CF, that means everything.

Dr Muhammad Fariduddin
Karachi

Published in Dawn, September 1st, 2025