Posted by Michael Wonder on 02 Sep 2024
CAMP4 Therapeutics secures rare paediatric disease designation for CMP-CPS-001 for the treatment of urea cycle disorders
27 August 2024 - CAMP4 Therapeutics today announced the US FDA has granted rare paediatric disease designation to the Company’s lead investigational drug candidate CMP-CPS-001 for the treatment of urea cycle disorders.
The safety, tolerability, and pharmacokinetics of CMP-CPS-001 are currently being evaluated in a randomised, placebo-controlled Phase 1 trial in Australia.
