Mutations in the fibroblast growth factor receptor 3 (FGFR 3) gene have been linked to achondroplasia or dwarfism. Credit: LANL (Los Alamos National Lab) / commons.wikimedia.org.

Tyra Biosciences has received orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for TYRA-300 to treat achondroplasia, a common form of dwarfism.

Individuals with achondroplasia encounter significant skeletal complications including sleep apnoea, hydrocephalus and cranial and spinal stenosis.

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Originating from the company’s in-house SNÅP platform, TYRA-300 is an investigational, oral, FGFR3-selective inhibitor currently being developed to treat cancer and skeletal dysplasias including achondroplasia.

It is under assessment in an open-label and multi-centre Phase I/II clinical trial for untreated and resistant FGFR3+ advanced solid tumours (SURF301).

The study is enrolling adults with advanced urothelial carcinoma and other solid tumours with FGFR3 gene alterations. 

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TYRA-300 has also shown positive preclinical results in skeletal dysplasias.

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Tyra chief medical officer Hiroomi Tada stated: “The FDA’s decision to grant orphan drug designation to TYRA-300 is an important recognition of the potential of our approach to delivering benefit to the achondroplasia community.

“We remain on track to submit an IND to the FDA to enable a Phase 2 study of TYRA-300 in paediatric achondroplasia in 2024.”

The company has appointed Michael Bober vice-president for clinical development and medical affairs, leading the skeletal dysplasia programme.