PTC Therapeutics - PharmaKB

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Company ReportsPTC Therapeutics

PTC Therapeutics(PTCT)

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.

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Marketed Drugs

Clinical Trials

Financial

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*Currency in USD

Company Revenue

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Annual

Annual

Projections

*Quarterly sales USD (in millions)

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Revenue by Drug

Marketed Drugs

Therapeutic Areas:

All

Classification:

All

Include Generics:

Trade Name	Drug Name	Approved	Patent Expires	Indication
Emflaza	Deflazacort	2017-02-09		Inflammation , Duchenne muscular dystrophy
Translarna	Ataluren	2014-07-31		Duchenne muscular dystrophy
Upstaza	Eladocagene exuparvovec	2022-07-18		Inborn errors amino acid metabolism

Clinical Trials

Therapeutic Areas:

All

Classification:

All

Approval:

All

Status:

All

Phases:

All

Drug Name

Phase 1

Phase 2

Phase 3

Phase 4

Indication

ELADOCAGENE EXUPARVOVEC

argininosuccinic aciduria

,

phenylketonurias

,

breast neoplasms

,

central nervous system neoplasms

,

nervous system neoplasms

,

,

myeloid leukemia acute

,

myeloid leukemia

,

,

,

neurofibromatosis 2

,

neurofibromatosis 1

,

neurofibromatoses

,

,

,

coronavirus infections

,

,

prostatic neoplasms

,

small cell carcinoma

,

small cell lung carcinoma

,

,

neuroendocrine tumors

primary ovarian insufficiency

,

female infertility

,

,

ovulation induction

,

,

postmenopausal osteoporosis

,

healthy volunteers/patients

,

alzheimer disease

,

female genital neoplasms

,

,

chronic hepatitis b

,

,

major depressive disorder

,

depressive disorder

,

triple negative breast neoplasms

,

pancreatic neoplasms

,

,

coronary disease

,

coronary artery disease

,

myocardial ischemia

,

,

,

sickle cell anemia

,

,

,

,

precursor cell lymphoblastic leukemia-lymphoma

,

parkinson disease

,

autistic disorder

,

autism spectrum disorder

,

pervasive child development disorders

,

chronic hepatitis

,

rectal neoplasms

,

,

,

b-cell chronic lymphocytic leukemia

,

lymphoid leukemia

,

,

nasopharyngeal carcinoma

,

nasopharyngeal neoplasms

,

stomach neoplasms

,

esophageal neoplasms

,

,

neoplasm metastasis

,

antiphospholipid syndrome

,

,

colorectal neoplasms

,

non-small-cell lung carcinoma

,

lupus nephritis

,

human influenza

,

breast neoplasms

,

,

large b-cell lymphoma diffuse

,

b-cell lymphoma

,

non-hodgkin lymphoma

,

myeloid leukemia acute

,

multiple sclerosis

,

,

,

hepatocellular carcinoma

,

colonic neoplasms

,

thrombocytopenic purpura idiopathic

,

liver cirrhosis

,

neuromyelitis optica

,

membranous glomerulonephritis

,

chronic progressive multiple sclerosis

,

,

nephrotic syndrome

,

ovarian neoplasms

,

ovarian epithelial carcinoma

,

,

hemolytic anemia autoimmune

,

,

chronic hepatitis c

,

,

,

rheumatoid arthritis

,

,

,

juvenile arthritis

,

,

follicular lymphoma

,

,

,

myelodysplastic syndromes

,

,

,

acquired immunodeficiency syndrome

,

kidney transplantation

,

drug-related side effects and adverse reactions

,

heart disease risk factors

,

cognitive dysfunction

complex regional pain syndromes

,

,

,

,

,

,

stress urinary incontinence

,

,

prostatic hyperplasia

,

choroidal neovascularization

,

,

diabetes mellitus

,

magnetic resonance imaging

,

,

,

hypertrophic cicatrix

,

,

major depressive disorder

,

carpal tunnel syndrome

,

premature ejaculation

,

,

rectal neoplasms

,

low anterior resection syndrome

,

,

penile induration

,

thoracic outlet syndrome

,

postherpetic neuralgia

,

motor neuron disease

,

thyroid diseases

,

,

ovarian neoplasms

,

stomach neoplasms

,

head and neck neoplasms

,

esophageal neoplasms

,

,

urinary bladder neoplasms

,

small cell lung carcinoma

,

endophthalmitis

,

lacrimal apparatus diseases

,

,

radiation injuries

,

uveal neoplasms

,

,

pancreatic neoplasms

,

macular degeneration

,

,

breast neoplasms

,

myofascial pain syndromes

,

neurogenic urinary bladder

,

,

,

,

,

non-small-cell lung carcinoma

,

dystonic disorders

,

temporomandibular joint disorders

,

,

,

somatoform disorders

,

systemic scleroderma

,

raynaud disease

,

diffuse scleroderma

,

urinary bladder diseases

,

multiple sclerosis

,

,

postoperative pain

,

breast diseases

,

hereditary breast and ovarian cancer syndrome

,

,

,

,

patellofemoral pain syndrome

,

allergic conjunctivitis

,

,

muscle spasticity

,

overactive urinary bladder

,

urinary incontinence

,

migraine disorders

,

,

,

,

healthy volunteers/patients

,

,

,

dry eye syndromes

,

,

prostatic neoplasms

,

diabetic retinopathy

,

parkinson disease

,

,

,

,

,

chronic kidney failure

,

renal insufficiency

,

iron-deficiency anemia

,

iron deficiencies

,

,

interstitial cystitis

,

graves ophthalmopathy

,

,

,

spinal cord injuries

,

developmental disabilities

,

,

,

left ventricular hypertrophy

,

,

sjogren's syndrome

,

retinal vein occlusion

,

herpes labialis

,

traumatic brain injuries

,

,

,

,

keratoconjunctivitis sicca

,

,

,

muscle strength

,

clostridium botulinum type a

ovarian neoplasms

,

ovarian epithelial carcinoma

,

,

diffuse intrinsic pontine glioma

,

,

Bos taurus adrenal cortex corticosteroids

anti-glomerular basement membrane disease

,

cystic fibrosis

breast neoplasms

,

urinary bladder neoplasms

,

ureteral neoplasms

,

prostatic neoplasms

,

renal cell carcinoma

,

,

,

,

triple negative breast neoplasms

,

hepatocellular carcinoma

,

liver neoplasms

,

uterine cervical neoplasms

,

,

malignant fibrous histiocytoma

,

,

urethral neoplasms

,

transitional cell carcinoma

,

urologic neoplasms

,

pancreatic ductal carcinoma

,

esophageal neoplasms

,

cholangiocarcinoma

,

squamous cell carcinoma

,

colorectal neoplasms

,

,

pancreatic neoplasms

,

ovarian neoplasms

,

squamous cell carcinoma of head and neck

,

endometrial neoplasms

,

biliary tract neoplasms

,

colonic neoplasms

,

rectal neoplasms

,

malignant mesothelioma

,

non-hodgkin lymphoma

,

multiple myeloma

,

head and neck neoplasms

,

non-small-cell lung carcinoma

,

,

,

neoplasm metastasis

,

respiratory tract diseases

,

,

respiratory tract neoplasms

,

bronchogenic carcinoma

,

thoracic neoplasms

,

neoplasms by site

,

bronchial neoplasms

,

neoplastic processes

,

pathologic processes

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