Rare disease therapeutics market to hit $495.27b by 2033

The sector’s growth is attributed to gene therapies and RNA-based treatments.

The rare disease therapeutics market is expected to reach $495.27b by 2033 at a compound annual growth rate of 13.8%, according to a DataM Intelligence report.

The sector’s expansion is attributed to gene therapies, RNA-based treatments, enzyme replacement therapies, and targeted biologics.

In addition, the pharmaceutical industry’s shift towards precision medicine, orphan drug incentives, and approvals of advanced therapeutics are further driving the market.

By therapy type, enzyme replacement therapy generated over $46b in 2024, driven by established treatments for Gaucher disease, Fabry disease, and Pompe disease.