Retrophin is collaborating with Censa Pharmaceuticals to advance CNSA-001 for the potential treatment of phenylketonuria (PKU) . As part of Retrophin’s agreement with Censa, Retrophin will fund the development of CNSA-001 and Censa will run the development program, conducted with the oversight of a joint development committee. Retrophin holds the exclusive option to acquire Censa upon conclusion of a specified option period, pending clinical proof-of-concept and evaluation of product development requirements.


PKU is a rare, genetic metabolic condition in which the body cannot effectively metabolize an amino acid called phenylalanine (Phe) due to the missing or defective activity of an enzyme called phenylalanine hydroxylase (PAH). High levels of phenylalanine can lead to neurological and behavioral impairment. The primary treatment is a low-phenylalanine diet. Learn more about PKU .


We are working with Censa Pharmaceuticals to advance CNSA-001, an orally available form of sepiapterin, a precursor of BH4that may lower levels of Phe in the blood. An increase in BH4 is believed to result in a decrease in Phe in BH4-responsive patients. The investigational approach of the collaboration is to attempt to increase BH4 with CNSA-001 in patients with PKU.


Censa is conducting a Phase 2 proof-of-concept study in patients with PKU.