PTC Therapeutics


PTC Therapeutics is a US pharmaceutical company focused on the development of orally administered small molecule drugs that regulate gene expression by targeting post-translational control mechanisms in orphan diseases.
In September 2009, PTC has entered into an agreement with Roche for the development of orally bioavailable small molecules for central nervous system diseases.

Products

In 2017, PTC acquired Emflaza from Marathon Pharmaceuticals. PTC also owns Translarna, marketed for Duchenne muscular dystrophy. Together, the two products generated revenues of 174 million dollars and 260 million dollars in 2017 and 2018 respectively.
Since 2003, PTC has been working on a drug for Duchenne muscular dystrophy., PTC portfolio includes small-molecule compounds for treatment of spinal muscular atrophy and Duchenne muscular dystrophy.

Pipeline

In efforts to expand therapeutic offerings into other indications, PTC acquired Agilis Biotherapeutics in July 2018, a biotech company using DNA therapeutics to treat genetic disorders related to the central nervous system.