The US Food and Drug Administration (FDA) has granted rare paediatric disease (RPD) designation for Tyra Biosciences’ TYRA-300, an oral fibroblast growth factor receptor 3 (FGFR3) selective inhibitor, to treat achondroplasia.

Achondroplasia is the most common restricted growth condition and currently has fewer therapeutic options.

It is caused by a specific DNA mutation in the FGFR3 gene, accounting for 99% of cases.

Patients with this condition often face severe skeletal issues including foramen magnum and spinal stenosis, hydrocephalus and sleep apnea.

Tyra Biosciences is also preparing to file an investigational new drug (IND) application to the regulator in the second half of 2024.

The IND will initiate a randomised Phase II clinical trial to assess multiple dose cohorts of TYRA-300 in children with achondroplasia.

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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.

TYRA-300 is the company’s lead programme developed using its SNÅP platform. It is being developed not only for skeletal dysplasias such as achondroplasia but also for cancer treatment.

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.

SURF301 is enrolling adult subjects with advanced urothelial carcinoma and other solid tumours with FGFR3 gene alterations.

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.”