BridgeBio Pharma, Inc. and Kyowa Kirin have announced a partnership wherein BridgeBio’s affiliate, QED Therapeutics, grants Kyowa Kirin an exclusive license to develop and commercialise infigratinib for achondroplasia, hypochondroplasia, and other skeletal dysplasias in Japan. 

In exchange, BridgeBio will receive an upfront payment of $100 million as well as royalties up to the high-twenties percent on sales of infigratinib in Japan, with the potential for additional milestone-based payments.

Infigratinib is an oral small molecule designed to inhibit FGFR3 and thus target FGFR3-driven skeletal dysplasias at their source, including achondroplasia and hypochondroplasia.

Achondroplasia is the most common cause of disproportionate short stature, affecting approximately 55,000 people in the US and EU, and 6,000 in Japan. Achondroplasia impacts overall health and quality of life, leading to medical complications such as obstructive sleep apnea, middle ear dysfunction, kyphosis, and spinal stenosis. The condition is uniformly caused by an activating mutation in FGFR3.

Yasuo Fujii, Chief Strategy Officer, Managing Executive Officer, Vice President, Head of Strategy Division at Kyowa Kirin said, “It is important for us to strengthen our portfolio by introducing pipelines in the fields of bone & mineral disorders, including achondroplasia. Based on the results from the latest clinical trials, we believe BridgeBio’s infigratinib has high potential for treating achondroplasia. We will steadily advance the development in Japan and aim to deliver life-changing value to people with skeletal dysplasias including achondroplasia.”