A majority of rare diseases are genetic in nature, with an estimated 50-75% of these conditions manifesting in childhood. The Rare Pediatric Disease designation was established to encourage and streamline drug development efforts in the rare disease domain. To be eligible for this designation, a therapy must aim to prevent or treat conditions affecting fewer than 200,000 individuals aged 18 or younger in the United States, supported by evidence of its effectiveness. According to Pharmabid, the FDA recently granted Rare Pediatric Disease designation to TYRA-300 for achondroplasia. In the future, if the New Drug Application (NDA) TYRA-300 is approved, the drug’s developer – in this case, Tyra Therapeutics – would receive a Priority Review voucher.
The Potential of TYRA-300 in Treating Achondroplasia
Achondroplasia, a bone growth disorder, results from FGFR3 mutations. Normally, the FGFR3 gene tells the body to make a certain protein that plays a role in bone growth and maintenance. When this gene mutates, the protein becomes overactive. Normal skeletal development is impaired. As a result, people with achondroplasia have a short stature with short arms and legs, a disproportionately large head compared to the body, and underdeveloped facial area between the forehead and upper jaw. People with achondroplasia may also experience complications and symptoms such as low muscle tone, sleep apnea, hydrocephalus, spinal stenosis, abnormal spinal curvature, and chronic ear infections.
Treatment for achondroplasia is usually symptomatic. Although vosoritide was approved in 2021 to address height issues in achondroplasia, there are few interventions to manage long-term health issues. This is where Tyra Therapeutics believes TYRA-300 could make a difference.
TYRA-300 is an investigational, orally administered selective FGFR3 inhibitor. It is being evaluated for skeletal dysplasia as well as oncologic indications such as non-muscle invasive bladder cancer and muscle-invasive bladder cancer. In terms of skeletal dysplasias, preclinical studies have shown positive safety and efficacy data. Tyra Therapeutics hopes to launch a Phase 2 human study in achondroplasia later this year.
