Tyra Biosciences, Inc.a clinical-stage biotechnology company focused on developing next-generation precision medicines that target large opportunities in Fibroblast Growth Factor Receptor (FGFR) biology, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead precision medicine program, TYRA-300, for the treatment of achondroplasia.

Achondroplasia is the most common form of dwarfism with limited therapeutic options.  People living with achondroplasia may experience severe skeletal complications including cranial and spinal stenosis, hydrocephalus and sleep apnea. A specific mutation in FGFR3 causes over 97% of achondroplasia. TYRA-300 is an oral FGFR3 selective inhibitor whose design may have a meaningful impact on achondroplasia and other skeletal dysplasias.

"People living with achondroplasia can have significant health complications that are not adequately addressed with currently available therapies.  Our goals with TYRA-300 in achondroplasia are to address not only height, but the long-term health complications associated with this condition," said Hiroomi Tada, M.D. Ph.D., Chief Medical Officer of TYRA.  "The FDA's decision to grant Orphan Drug Designation to TYRA-300 is an important recognition of the potential of our approach to deliver benefit to the achondroplasia community.  We remain on track to submit an IND to the FDA to enable a Phase 2 study of TYRA-300 in pediatric achondroplasia in 2024."

The FDA's Office of Orphan Products Development grants orphan designation status to drugs and biologics that are intended for treatment, diagnosis or prevention of rare diseases and conditions that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain benefits, including tax credits for qualified clinical trials and exemption from certain user fees to support clinical development and the potential for up to seven years of market exclusivity in the U.S. upon regulatory approval.

TYRA also announced today the appointment of Michael Bober, M.D. Ph.D., as Vice President, Clinical Development and Medical Affairs, to lead the skeletal dysplasia program.  Dr. Bober is a leader in the diagnosis and management of skeletal dysplasia. He served on numerous scientific and medical advisory boards within the skeletal dysplasia community.  Dr. Bober joins TYRA following a distinguished career as the Medical Director of the Skeletal Dysplasia Program, Nemours Children's Hospital, Delaware.

Dr. Bober added, "I am excited to join TYRA and contribute to the team working to develop TYRA-300 into a therapy for the patient community which I care so much about. I believe TYRA-300 has the potential to improve function and quality of life in achondroplasia."

 

Source:prnewswire.com

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