Thiogenesis Therapeutics (TSXV: TTI; OTCQX: TTIPF) has announced that the FDA has granted Rare Pediatric Disease (RPD) designation to its lead candidate, TTI-0102, for the treatment of Leigh syndrome.
Upon approval of a future New Drug Application, the designation makes Thiogenesis eligible for a Priority Review Voucher, which may be used to obtain priority review of another marketing application or sold to another sponsor.
In a statement, Patrice Rioux, MD, PhD, CEO of Thiogenesis, commented, “Receiving Rare Pediatric Disease designation is an important regulatory milestone for our Leigh syndrome spectrum program and further expands Thiogenesis’ regulatory portfolio. The designation recognizes the significant unmet medical need in Leigh syndrome and reinforces our confidence in the potential of TTI-0102 to address that need.”
Dr. Rioux added, “Since receiving FDA clearance of our IND, we have strengthened our Phase 2 protocol by incorporating important pharmacokinetic learnings from our MELAS clinical program. We are now completing final study start-up activities and look forward to initiating the Phase 2 trial and generating clinical data.”
