PTC Therapeutics 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
A rare disease is any disease that affects a small percentage of the population. In some parts of the world, an orphan disease is a rare disease whose rarity means there is a lack of a market large enough to gain support and resources for discove ...
.
In September 2009, PTC entered into an agreement with
Roche
F. Hoffmann-La Roche AG, commonly known as Roche, is a Swiss multinational healthcare company that operates worldwide under two divisions: Pharmaceuticals and Diagnostics. Its holding company, Roche Holding AG, has shares listed on the SIX ...
for the development of orally bioavailable
small molecule
Within the fields of molecular biology and pharmacology, a small molecule or micromolecule is a low molecular weight (≤ 1000 daltons) organic compound that may regulate a biological process, with a size on the order of 1 nm. Many drugs ar ...
s for
central nervous system
The central nervous system (CNS) is the part of the nervous system consisting primarily of the brain and spinal cord. The CNS is so named because the brain integrates the received information and coordinates and influences the activity of all par ...
diseases. 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, in partnership with the SMA Foundation and Roche.
PTC acquired the Bio-e platform in 2019
The Bio-e platformutilizes expertise in electron-transfer chemistry to modulate key biological processes beyond the reach of current drug development approaches. The lead compounds from the Bio-e platform, PTC743 and PTC857, target the enzyme 15-lipoxygenase – a key enzymatic hub that regulates the inflammation and oxidative stress that underpin mitochondrial disease and CNS pathology. Two pivotal studies will investigate the safety and efficacy of PTC743: a Phase 2/3 trial in refractory mitochondrial epilepsy and a Phase 3 trial in Friedreich’s ataxia.
Products
In 2017, PTC acquired
Emflaza (deflazacort) from
Marathon
The marathon is a long-distance foot race with a distance of , usually run as a road race, but the distance can be covered on trail routes. The marathon can be completed by running or with a run/walk strategy. There are also wheelchair div ...
Pharmaceuticals. PTC also owns Translarna, (
Ataluren
Ataluren, sold under the brand name Translarna, is a medication for the treatment of Duchenne muscular dystrophy. It was designed by PTC Therapeutics.
Medical use
Ataluren is used in the European Union to treat people with Duchenne muscular dys ...
) marketed for
nonsense mutation
In genetics, a nonsense mutation is a point mutation in a sequence of DNA that results in a premature stop codon, or a ''nonsense codon'' in the transcribed mRNA, and in leading to a truncated, incomplete, and usually nonfunctional protein produc ...
Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is a severe type of muscular dystrophy that primarily affects boys. Muscle weakness usually begins around the age of four, and worsens quickly. Muscle loss typically occurs first in the thighs and pelvis fol ...
. Together, the two products generated revenues of 174 million dollars and 260 million dollars in 2017 and 2018 respectively.
PTC ha
the commercialization rights for WAYLIVRA™(volanesorsen) in Latin America. WAYLIVRA™ is approved in the European Union for the treatment of familial chylomicronemia syndrome (FCS). TEGSEDI® (inotersen) was granted marketing approval from the Brazilian Health Regulatory Agency (ANVISA) for the treatment of stage 1 or 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). PTC has licensed the commercialization rights for TEGSEDI® (inotersen) in Latin America from Akcea Therapeutics.
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. 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.
PTC Therapeutics website
/ref>, 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.
References
Further reading
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External links
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Biotechnology
Pharmaceutical companies based in New Jersey
Companies based in Middlesex County, New Jersey
South Plainfield, New Jersey
Companies listed on the Nasdaq
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