PTC Therapeutics

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Coordinates: 40°33′03″N 74°25′23″W / 40.550722°N 74.423071°W / 40.550722; -74.423071

PTC Therapeutics, Inc.
Russell 2000 Component
United States

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 (PTC) mechanisms in orphan diseases.[1][2]

In September 2009, PTC has entered into an agreement with Roche for the development of orally bioavailable small molecules for central nervous system diseases.[3]


In 2017, PTC acquired Emflaza[4] (deflazacort) from Marathon Pharmaceuticals. PTC also owns Translarna, (Ataluren) marketed for Duchenne muscular dystrophy.[5] Together, the two products generated revenues of 174 million dollars and 260 million dollars in 2017 and 2018 respectively.[6]

Since 2003, PTC has been working on a drug for Duchenne muscular dystrophy.[7] As of May 2016, PTC portfolio includes small-molecule compounds for treatment of spinal muscular atrophy (RG7916) and Duchenne muscular dystrophy (Ataluren and another exon skipping compound).[8]


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.[9]


  1. ^ Bhutta, Sunya (December 2013). "Biopharma Blossoming in the Garden State". Biobusiness. Gen. Eng. Biotechnol. News. 33 (21). p. 15.
  2. ^ Bain, Lisa J (2006). "Drug development in critical times". NeuroRx. 3 (4): 540–3. doi:10.1016/j.nurx.2006.08.004. PMC 3593415. PMID 17044149.
  3. ^ Bratulic, Anna (2009-09-02). "Roche, PTC Therapeutics to partner on CNS drug discovery - FirstWord Pharma". FirstWord Pharma. Retrieved 2016-05-14.
  4. ^ House, SA Editor Douglas W. (2017-03-16). "PTC acquires DMD med Emflaza from Marathon Pharma for up to $190M". Seeking Alpha. Retrieved 2018-07-03.
  5. ^ "In The Spotlight: PTC Therapeutics". 2018-07-23. Retrieved 2018-07-24.
  6. ^ "In The Spotlight: PTC Therapeutics". 2018-07-23. Retrieved 2018-07-24.
  7. ^ Hamed SA (2006). "Drug evaluation: PTC-124--a potential treatment of cystic fibrosis and Duchenne muscular dystrophy". IDrugs : the investigational drugs journal. 9 (11): 783–9. PMID 17096300.
  8. ^ "PTC Therapeutics | Pipeline". Retrieved 2016-05-14.
  9. ^ "In The Spotlight: PTC Therapeutics". 2018-07-23. Retrieved 2018-07-25.

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