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40°33′03″N 74°25′23″W / 40.550722°N 74.423071°W
 | |
| Company type | Public |
|---|---|
|
Nasdaq:
PTCT Russell 2000 Component | |
| Industry | Pharmaceuticals |
| Founded | January 1, 1998 |
| Headquarters | , U.S. |
| Website |
ptcbio |
PTC Therapeutics, Inc. is a US pharmaceutical company focused on the development of orally administered small molecule drugs and gene therapy which regulate gene expression by targeting post-transcriptional control (PTC) mechanisms in orphan diseases. [1] [2]
In September 2009, PTC entered into an agreement with Roche for the development of orally bioavailable small molecules for central nervous system diseases. [3] PTC acquired the Bio-e platform in 2019. [4]
Products
In 2017, PTC acquired Emflaza (deflazacort) from Marathon Pharmaceuticals. [5] PTC also owns Translarna, ( Ataluren) marketed for nonsense mutation Duchenne muscular dystrophy. [6] Together, the two products generated revenues of 174 million dollars and 260 million dollars in 2017 and 2018 respectively. [7]
PTC has the commercialization rights for WAYLIVRA (volanesorsen) in Latin America. [8]
Pipeline
In 2018, PTC acquired Agilis Biotherapeutics and a gene therapy candidate, GT-AADC, with its compelling clinical data in treating aromatic L-amino acid decarboxylase (AADC) deficiency. [9] AADC deficiency is a rare CNS disorder arising from reductions in the enzyme AADC that result from mutations in the dopa decarboxylase (DDC) gene.
In 2020, PTC acquired Censa Pharmaceuticals, Inc., a biopharmaceutical company focused on the development of CNSA-001 (sepiapterin), a clinical-stage investigational therapy for orphan metabolic diseases, including phenylketonuria (PKU) and other diseases associated with defects in the tetrahydrobiopterin (BH4) biochemical pathways diagnosed at birth. [10]
In 2020, PTC announced the FDA approval of Evrysdi (risdiplam) for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months and older. [11]
References
- ^ Bhutta, Sunya (December 2013). "Biopharma Blossoming in the Garden State". Biobusiness. Gen. Eng. Biotechnol. News. Vol. 33, no. 21. p. 15.
- ^ 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.
- ^ Bratulic, Anna (2009-09-02). "Roche, PTC Therapeutics to partner on CNS drug discovery - FirstWord Pharma". FirstWord Pharma. Retrieved 2016-05-14.
- ^ Press Release https://www.ptcbio.com/our-science/expertise/bio-e-electron-transfer/
-
^ 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.
{{ cite news}}:|first=has generic name ( help) - ^ "In The Spotlight: PTC Therapeutics". NASDAQ.com. 2018-07-23. Retrieved 2018-07-24.
- ^ "In The Spotlight: PTC Therapeutics". NASDAQ.com. 2018-07-23. Retrieved 2018-07-24.
- ^ Press Release https://www.ptcbio.com/our-pipeline/approved-medicines/
- ^ PTC Therapeutics website
- ^ PTC Therapeutics website
- ^ Press Release https://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-announces-fda-approval-evrysditm-risdiplam
Further reading
- Schneider, Ilene (2009). "Firm Aims to Regulate RNA in Rare Diseases". Genetic Engineering & Biotechnology News. 29 (8): 14. Retrieved 2009-04-25.
External links
- Official website
- Business data for PTC Therapeutics, Inc.:
 | This pharmacology-related article is a stub. You can help Wikipedia by expanding it. |
| This article has multiple issues. Please help
improve it or discuss these issues on the
talk page. (
Learn how and when to remove these template messages)
|
40°33′03″N 74°25′23″W / 40.550722°N 74.423071°W
|
| |
| Company type | Public |
|---|---|
|
Nasdaq:
PTCT Russell 2000 Component | |
| Industry | Pharmaceuticals |
| Founded | January 1, 1998 |
| Headquarters | , U.S. |
| Website |
ptcbio |
PTC Therapeutics, Inc. is a US pharmaceutical company focused on the development of orally administered small molecule drugs and gene therapy which regulate gene expression by targeting post-transcriptional control (PTC) mechanisms in orphan diseases. [1] [2]
In September 2009, PTC entered into an agreement with Roche for the development of orally bioavailable small molecules for central nervous system diseases. [3] PTC acquired the Bio-e platform in 2019. [4]
Products
In 2017, PTC acquired Emflaza (deflazacort) from Marathon Pharmaceuticals. [5] PTC also owns Translarna, ( Ataluren) marketed for nonsense mutation Duchenne muscular dystrophy. [6] Together, the two products generated revenues of 174 million dollars and 260 million dollars in 2017 and 2018 respectively. [7]
PTC has the commercialization rights for WAYLIVRA (volanesorsen) in Latin America. [8]
Pipeline
In 2018, PTC acquired Agilis Biotherapeutics and a gene therapy candidate, GT-AADC, with its compelling clinical data in treating aromatic L-amino acid decarboxylase (AADC) deficiency. [9] AADC deficiency is a rare CNS disorder arising from reductions in the enzyme AADC that result from mutations in the dopa decarboxylase (DDC) gene.
In 2020, PTC acquired Censa Pharmaceuticals, Inc., a biopharmaceutical company focused on the development of CNSA-001 (sepiapterin), a clinical-stage investigational therapy for orphan metabolic diseases, including phenylketonuria (PKU) and other diseases associated with defects in the tetrahydrobiopterin (BH4) biochemical pathways diagnosed at birth. [10]
In 2020, PTC announced the FDA approval of Evrysdi (risdiplam) for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months and older. [11]
References
- ^ Bhutta, Sunya (December 2013). "Biopharma Blossoming in the Garden State". Biobusiness. Gen. Eng. Biotechnol. News. Vol. 33, no. 21. p. 15.
- ^ 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.
- ^ Bratulic, Anna (2009-09-02). "Roche, PTC Therapeutics to partner on CNS drug discovery - FirstWord Pharma". FirstWord Pharma. Retrieved 2016-05-14.
- ^ Press Release https://www.ptcbio.com/our-science/expertise/bio-e-electron-transfer/
-
^ 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.
{{ cite news}}:|first=has generic name ( help) - ^ "In The Spotlight: PTC Therapeutics". NASDAQ.com. 2018-07-23. Retrieved 2018-07-24.
- ^ "In The Spotlight: PTC Therapeutics". NASDAQ.com. 2018-07-23. Retrieved 2018-07-24.
- ^ Press Release https://www.ptcbio.com/our-pipeline/approved-medicines/
- ^ PTC Therapeutics website
- ^ PTC Therapeutics website
- ^ Press Release https://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-announces-fda-approval-evrysditm-risdiplam
Further reading
- Schneider, Ilene (2009). "Firm Aims to Regulate RNA in Rare Diseases". Genetic Engineering & Biotechnology News. 29 (8): 14. Retrieved 2009-04-25.
External links
- Official website
- Business data for PTC Therapeutics, Inc.:
|
| This pharmacology-related article is a stub. You can help Wikipedia by expanding it. |