Therapy Areas: Oncology
Tyra Biosciences TYRA-300 receives US FDA Rare Pediatric Disease Designation
2 February 2024 -

Tyra Biosciences, Inc. (Nasdaq: TYRA), a Califonia-based clinical-stage biotechnology company, announced on Thursday that it has received Rare Pediatric Disease (RPD) Designation from the US Food and Drug Administration (FDA) for its TYRA-300, an oral FGFR3 selective inhibitor, intended for the treatment of achondroplasia.

The company is planning to submit an Investigational New Drug application (IND) to the FDA in the second half of 2024 for the commencement of a randomised Phase two clinical trial assessing multiple dose cohorts of TYRA-300 for children with achondroplasia, the most common form of dwarfism with limited therapeutic options.

TYRA-300 is the company's lead precision medicine program stemming from its in-house SNÅP platform. TYRA-300 is an investigational, oral, FGFR3-selective inhibitor currently in development for the treatment of cancer and skeletal dysplasias, including achondroplasia. In oncology, TYRA-300 is being evaluated in a multi-centre, open label Phase 1/2 clinical study, SURF301 (Study in Untreated and Resistant FGFR3+ Advanced Solid Tumours).

Rare Pediatric Disease (RPD) Designation is granted by the FDA to investigational drugs and biologics designed to address serious or life-threatening diseases which affect fewer than 200,000 people in the United States, or for which there is no reasonable expectation that the cost of developing and making the drug or biologic available in the US for the applicable disease or condition will be recovered from sales in the US, and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age.