Novel FGFR3-Selective Inhibitor Gets Orphan Drug Status for Achondroplasia

The FDA has granted Orphan Drug designation to TYRA-300 for the treatment of achondroplasia.

The Food and Drug Administration (FDA) has granted Orphan Drug designation to TYRA-300 for the treatment of achondroplasia, a rare genetic disorder that results in abnormal bone growth.

Over 97% of cases of achondroplasia are due to a mutation in the fibroblast growth factor receptor (FGFR) 3 gene. The disorder is characterized by short stature and macrocephaly with frontal bossing. Complications may include cranial and spinal stenosis, hydrocephalus, and sleep apnea.

TYRA-300 is an investigational, oral, FGFR3-selective inhibitor. The designation was based on positive preclinical results that demonstrated increases in growth and bone length with TYRA-300 compared with vehicle in treated mice. Tyra Biosciences is hoping to begin a phase 2 trial in pediatric patients in 2024.

“Our goals with TYRA-300 in achondroplasia are to address not only height, but the long-term health complications associated with this condition,” said Hiroomi Tada, MD PhD, Chief Medical Officer of TYRA.  “The FDA’s decision to grant Orphan Drug designation to TYRA-300 is an important recognition of the potential of our approach to deliver 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 pediatric achondroplasia in 2024.”

The Company is also investigating TYRA-300 in the phase 1/2 SURF301 trial ( Identifier: NCT05544552) as a potential treatment for advanced urothelial carcinoma and other solid tumors with FGFR3 gene alterations.

This article originally appeared on MPR


Tyra Biosciences announces FDA Orphan Drug designation for TYRA-300 for the treatment of achondroplasia. News release. Tyra Biosciences. August 1, 2023.  Accessed August 2, 2023.