The IND will initiate a randomised Phase II clinical trial to assess multiple dose cohorts of TYRA-300 in children with achondroplasia.
RPD designation is given to drugs and biologics that target serious or life-threatening ailments impacting fewer than 200,000 people in the US, particularly those diseases where the cost of drug development is not expected to be recovered from US sales.
The company could receive a priority review voucher for the therapy if a new application is approved. This can expedite the review process for another marketing application or be sold or transferred.
In the oncology field, it is being assessed in the Phase I/II trial SURF301 to identify the maximum tolerated dose and the recommended Phase II dose and to assess its initial anti-tumour activity.
The therapy has shown positive preclinical results for skeletal dysplasias. As well as RPD status, it has also received orphan drug designation from the FDA for achondroplasia.
TYRA chief medical officer Hiroomi Tada stated: “The rare paediatric disease designation recognises the severity of complications associated with achondroplasia in childhood and underscores our opportunity with TYRA-300 to develop a highly selective, oral medication that benefits the achondroplasia community.
“Children with achondroplasia face a significant unmet need – and currently, there are no approved treatment options that address the immediate and long-term health complications associated with this condition. We look forward to advancing TYRA-300 to the clinic and pursuing our goal to bring a much-needed therapy to these children.”
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