USA – BridgeBio, a biopharmaceutical company designing medicines for patients with genetic diseases and cancers, has struck a  deal with Kyowa Kirin, marking a pivotal moment in the development of a potential treatment for achondroplasia, the most common form of dwarfism. 

The agreement entails BridgeBio’s affiliate, QED Therapeutics, granting Kyowa Kirin partial rights in Japan to an experimental drug called infigratinib.  

Kyowa Kirin Co., Ltd. is a Japanese pharmaceutical and biotechnology company under the Kirin Holdings and is among the 40 largest in the world by revenue and is headquartered in Chiyoda-ku, Tokyo. 

Infigratinib is currently undergoing Phase 3 clinical trials, with promising prospects as a competitor to BioMarin’s Voxzogo, a marketed medicine for the same condition. 

Under the terms of the deal, BridgeBio stands to receive US$100 million upfront, along with potential milestone payments and royalties from sales if the drug gains approval.  

This infusion of funds provides BridgeBio with the financial stability to continue its research and development efforts. 

Achondroplasia, caused by genetic alterations in the FGFR3 gene, disrupts bone growth, resulting in disproportionately short stature and various health complications.  

Both BridgeBio’s infigratinib and BioMarin’s Voxzogo target the protein encoded by this gene, albeit through different administration methods – Voxzogo is injectable, while infigratinib comes in pill form. 

Early-stage trials have shown promise for infigratinib, suggesting potential benefits for children with achondroplasia, including accelerated growth rates and favorable tolerability.  

The ongoing Phase 3 trial, which includes children aged three to 18, aims to provide more definitive data, with results anticipated next year. 

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BridgeBio’s partnership with Kyowa Kirin expands its reach into the well-developed Japanese market and demonstrates a strategic move to bolster its financial position.  

Additionally, this development comes as BridgeBio prepares for the potential launch of acoramidis, a treatment for transthyretin amyloidosis cardiomyopathy, which could receive U.S. regulatory approval later this year. 

To support its upcoming initiatives, BridgeBio recently secured additional funding through the sale of drug royalties and the establishment of a credit line, totaling up to $1.25 billion underscoring the company’s commitment to advancing novel therapies for rare genetic diseases and addressing unmet medical needs. 

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