Rare Diseases: Breaking Ground in Fibrosis and PAH

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While asthma and COPD represent the "volume," the "value" and innovation are occurring in rare respiratory diseases like Idiopathic Pulmonary Fibrosis (IPF) and Pulmonary Arterial Hypertension (PAH). These conditions have historically had very poor survival rates, but 2025 marks a turning point with the introduction of "Disease-Modifying" therapies.

The Respiratory Drugs Market analysis highlights the success of drugs like Ofev (nintedanib) and the arrival of new competitors like nerandomilast. These antifibrotic medicines slow the scarring of lung tissue, extending life expectancy by several years. In the PAH segment, new "activin signaling inhibitors" are moving from clinical trials to the market, offering a more effective way to reverse the vascular remodeling that leads to heart failure.

Gene therapy is also making its first real impact in Cystic Fibrosis (CF). While CFTR modulators like Trikafta remain the "powerhouse" blockbusters of 2025, the first gene-silencing (RNAi) and gene-editing therapies are entering late-stage trials. These therapies aim to "correct" the genetic defect at the cellular level, potentially offering a one-time cure rather than a lifetime of daily pills and nebulizers.

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