Tyra Biosciences has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to TYRA-300 for the treatment of achondroplasia. This designation highlights the potential of TYRA-300, an oral FGFR3 selective inhibitor, to address significant unmet needs in this rare genetic condition.
Achondroplasia, a prevalent form of dwarfism, affects fewer than 200,000 people in the U.S. and is characterized by severe skeletal complications, including cranial and spinal stenosis, hydrocephalus, and sleep apnea. In over 97% of cases, achondroplasia is caused by a specific mutation in the Fibroblast Growth Factor Receptor 3 (FGFR3) gene. Current therapeutic options are limited, leaving a substantial gap in patient care.
TYRA-300: A Precision Medicine Approach
TYRA-300 is designed as a selective inhibitor of FGFR3, holding promise for significantly impacting achondroplasia and other skeletal dysplasias. Hiroomi Tada, M.D. Ph.D., Chief Medical Officer of TYRA, stated, "People living with achondroplasia can have significant health complications that are not adequately addressed with currently available therapies. Our goals with TYRA-300 in achondroplasia are to address not only height, but the long-term health complications associated with this condition."
The FDA's Orphan Drug Designation provides TYRA Biosciences with several benefits, including tax credits for qualified clinical trials, exemption from certain user fees, and the potential for up to seven years of market exclusivity in the U.S. upon regulatory approval.
Clinical Development Plans
TYRA Biosciences is advancing the clinical development of TYRA-300. The company anticipates submitting an Investigational New Drug (IND) application to the FDA, paving the way for a Phase 2 clinical study in pediatric achondroplasia in 2024. TYRA-300 is also being evaluated in the SURF301 Phase 1/2 clinical study (NCT05544552) for advanced urothelial carcinoma and other solid tumors with FGFR3 gene alterations.
Leadership Appointment
To spearhead the skeletal dysplasia program, TYRA Biosciences has appointed Michael Bober, M.D. Ph.D., as Vice President, Clinical Development and Medical Affairs. Dr. Bober, a recognized expert in skeletal dysplasia, brings extensive experience from his previous role as Medical Director of the Skeletal Dysplasia Program at Nemours Children's Hospital, Delaware. "I believe TYRA-300 has the potential to improve function and quality of life in achondroplasia," said Dr. Bober.
About TYRA-300 and SNÅP Platform
TYRA-300 is the lead program from TYRA Biosciences' SNÅP platform, designed for rapid and precise drug development. It is an investigational oral FGFR3-selective inhibitor. The SURF301 study is a multi-center, open-label trial designed to determine the optimal and maximum tolerated doses of TYRA-300 and evaluate its preliminary antitumor activity.
