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.
Pfizer's Hympavzi (marstacimab-hncq) receives US FDA approval for Hemophilia A or B treatment
Capricor begins BLA submission for deramiocel in Duchenne muscular dystrophy
GE HealthCare expands AI-enabled enterprise imaging with Blackford integration
Biogen receives FDA Breakthrough Therapy Designation for felzartamab
Inspira Technologies develops new disposable kit for perfusion market
Ananda Developments PLC signs contract for Phase 1 PK study on MRX1
Teva's Prolia biosimilar candidate accepted for review by FDA and EMA
Boehringer receives Breakthrough Therapy designation for survodutide
GBI Biomanufacturing agrees manufacturing collaboration with Allterum Therapeutics
Genprex research collaborators to present positive data on Reqorsa Gene Therapy at SITC 2024
ALK secures U.S. FDA approval for AccuTest Allergy Skin Testing Devices