PTC has successfully completed acquisition of Censa Pharmaceuticals, Inc., a biopharmaceutical company focused on the development of CNSA-001 (sepiapterin), a clinical-stage investigational therapy for orphan metabolic rare diseases, including phenylketonuria (PKU) and other rare diseases associated with defects in the tetrahydrobiopterin (BH4) biochemical pathways diagnosed at birth.
“This acquisition adds a well-established late-stage PKU clinical program to our growing rare disease portfolio. Given PTC’s established development and commercial capabilities, we’re well-positioned to bring this therapy to patients with unmet medical need expeditiously.” – CEO, Stuart Peltz, Ph.D.
PKU is an inborn error of metabolism caused predominantly by mutations in the phenylalanine hydroxylase (PAH) gene resulting in toxic buildup of the amino acid phenylalanine (Phe) in the brain. It is estimated that there are 16,500 patients in the United States with PKU. If left untreated, severe and irreversible disability can occur to include permanent intellectual disability, seizures, delayed development, behavioral problems, and possibly psychiatric disorders.