BridgeBio’s Strategy Shift Tests the Economics of Rare Disease

BridgeBio leans on its ATTR-CM launch to push late-stage assets while facing competition, pricing pressure, and questions about demand durability

30 Oct 2025

BridgeBio Pharma is adjusting its growth strategy in rare diseases, using the early launch of its treatment for transthyretin amyloid cardiomyopathy, or ATTR-CM, to support a more diversified late-stage pipeline as competition in the market increases.

The US biotech group has reported thousands of prescriptions and rising net revenue from its ATTR-CM therapy, marking its first meaningful commercial foothold. Adoption, however, remains at an early stage, and investors are watching closely to see whether demand proves durable in a category facing closer scrutiny over long-term growth and pricing.

Rather than relying heavily on a single product, BridgeBio is advancing two late-stage programmes it views as core to its next phase. Encaleret, a treatment for autosomal dominant hypocalcaemia type 1, and BBP-418, being developed for limb girdle muscular dystrophy type 2I, both target rare conditions with few or no approved therapies.

The company is positioning these assets as focused opportunities where smaller patient populations and clearer clinical endpoints may support regulatory approval and reimbursement. Together, they signal a strategy centred on selective expansion rather than broad exposure to one therapeutic area.

The approach reflects a wider shift across the biotech sector, as companies seek to balance innovation with the financial and regulatory risks of drug development. The environment remains demanding. Competition in ATTR-CM, including from larger groups such as Pfizer, is intensifying, while any slowdown in market growth could limit the cash available to fund research.

At the same time, drug pricing debates and tighter payer oversight continue to weigh on companies developing high-cost medicines for small patient groups. These pressures have become more pronounced as funding conditions across biotech remain uneven.

Analysts describe BridgeBio’s strategy as measured, with an emphasis on scientific differentiation and capital discipline. The company is seeking to advance programmes where it believes clinical data can support long-term value, while avoiding overextension.

As BridgeBio approaches further clinical results and regulatory milestones, attention will focus on the stability of demand for its ATTR-CM therapy and the strength of data from Encaleret and BBP-418. Those outcomes are likely to shape whether its revised strategy delivers sustainable growth in rare diseases.