Infigratinib
Achondroplasia
Key Facts
About BridgeBio Pharma
BridgeBio Pharma is a clinical-stage biotech with a mission to translate genetic insights into life-changing medicines for rare diseases and cancers. The company has achieved significant milestones, including three FDA approvals, a robust late-stage pipeline with six Phase 3 trials conducted, and a market valuation reflecting strong investor confidence. Its core strategy leverages a decentralized hub-and-spoke operational model to pursue multiple high-precision programs simultaneously, targeting conditions with clear genetic etiology and significant unmet need.
View full company profileAbout BridgeBio Pharma
BridgeBio Pharma is a clinical-stage biotech with a mission to translate genetic insights into life-changing medicines for rare diseases and cancers. The company has achieved significant milestones, including three FDA approvals, a robust late-stage pipeline with six Phase 3 trials conducted, and a market valuation reflecting strong investor confidence. Its core strategy leverages a decentralized hub-and-spoke operational model to pursue multiple high-precision programs simultaneously, targeting conditions with clear genetic etiology and significant unmet need.
View full company profileAbout LianBio
LianBio's mission was to accelerate the availability of innovative medicines for patients in China and broader Asian markets by in-licensing clinical-stage assets from global partners. The company built an integrated regional development and commercialization platform to navigate complex regulatory and commercial landscapes. Its primary achievement was advancing a portfolio of promising candidates, including mavacamten for oHCM which reached NDA acceptance in China, before the Board's decision to wind down operations and liquidate the pipeline following a strategic review in November 2023.
View full company profileTherapeutic Areas
Other Achondroplasia Drugs
| Drug | Company | Phase |
|---|---|---|
| Navepegritide (TransCon CNP) | VISEN Pharmaceuticals | Phase 2 |
| VOXZOGO (vosoritide) | BioMarin Pharmaceutical | Approved/Marketed |