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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Company Revenue

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Annual

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*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

healthy volunteers/patients

,

renal insufficiency

,

,

hepatic insufficiency

,

muscular dystrophies

,

duchenne muscular dystrophy

,

limb-girdle muscular dystrophies

ELADOCAGENE EXUPARVOVEC

argininosuccinic aciduria

,

phenylketonurias

,

mitochondrial diseases

,

friedreich ataxia

,

,

,

diffuse cerebral sclerosis of schilder

,

drug resistant epilepsy

,

,

,

status epilepticus

,

nerve degeneration

,

mitochondrial encephalomyopathies

,

breast neoplasms

,

central nervous system neoplasms

,

nervous system neoplasms

,

,

myeloid leukemia acute

,

myeloid leukemia

,

,

,

neurofibromatosis 2

,

neurofibromatosis 1

,

neurofibromatoses

,

,

,

coronavirus infections

,

,

myelodysplastic syndromes

,

pharmacokinetics

,

thoracic neoplasms

,

invasive hydatidiform mole

,

parkinson disease

,

nerve degeneration

,

pancreatic neoplasms

,

,

b-cell lymphoma

,

large b-cell lymphoma diffuse

,

,

castration-resistant prostatic neoplasms

,

,

brain neoplasms

,

,

colonic neoplasms

,

non-hodgkin lymphoma

,

follicular lymphoma

,

neuroendocrine tumors

,

kidney neoplasms

,

hypersensitivity

,

epstein-barr virus infections

,

myeloid leukemia acute

,

mouth neoplasms

,

oropharyngeal neoplasms

,

,

,

microsatellite instability

,

pancreatic ductal carcinoma

,

adenocarcinoma of lung

,

,

t-cell lymphoma

,

t-cell lymphoma peripheral

,

,

urethral neoplasms

,

ureteral neoplasms

,

,

local neoplasm recurrence

,

rectal neoplasms

,

laryngeal neoplasms

,

hypopharyngeal neoplasms

,

,

,

x-linked bulbo-spinal atrophy

,

,

,

malignant fibrous histiocytoma

,

nasopharyngeal carcinoma

,

,

,

unknown primary neoplasms

,

,

hematologic diseases

,

b-cell chronic lymphocytic leukemia

,

bone marrow diseases

,

endometrioid carcinoma

,

,

sezary syndrome

,

mycosis fungoides

,

t-cell lymphoma cutaneous

,

colonic diseases

,

histiocytic sarcoma

,

follicular dendritic cell sarcoma

,

interdigitating dendritic cell sarcoma

,

pelvic neoplasms

,

environmental biomarkers

,

inflammatory breast neoplasms

,

intestinal neoplasms

,

kidney diseases

,

,

,

nervous system diseases

,

neoplasms by histologic type

,

nerve tissue neoplasms

,

neuroepithelial neoplasms

,

neuroectodermal tumors

,

salivary gland neoplasms

,

thyroid neoplasms

,

,

carcinoid tumor

,

vulvar neoplasms

,

parotid neoplasms

,

reactive arthritis

,

gastrointestinal stromal tumors

,

,

central nervous system neoplasms

,

spinal cord neoplasms

,

adenoviridae infections

,

gastrointestinal neoplasms

,

digestive system neoplasms

,

,

uterine neoplasms

,

,

mullerian mixed tumor

,

basal cell carcinoma

,

basal cell neoplasms

,

,

male breast neoplasms

,

uveal neoplasms

,

,

,

,

colorectal neoplasms

,

renal cell carcinoma

,

triple negative breast neoplasms

,

breast neoplasms

,

neoplasm metastasis

,

squamous cell carcinoma

,

head and neck neoplasms

,

ovarian neoplasms

,

stomach neoplasms

,

liver neoplasms

,

urinary bladder neoplasms

,

hepatocellular carcinoma

,

,

endometrial neoplasms

,

prostatic neoplasms

,

small cell lung carcinoma

,

esophageal neoplasms

,

hodgkin disease

,

,

uterine cervical neoplasms

,

squamous cell neoplasms

,

transitional cell carcinoma

,

ovarian epithelial carcinoma

,

multiple myeloma

,

urologic neoplasms

,

esophageal squamous cell carcinoma

,

biliary tract neoplasms

,

fallopian tube neoplasms

,

cholangiocarcinoma

,

nasopharyngeal neoplasms

,

plasma cell neoplasms

,

bile duct neoplasms

,

merkel cell carcinoma

,

,

gallbladder neoplasms

,

malignant mesothelioma

,

cutaneous malignant melanoma

,

peritoneal neoplasms

,

hematologic neoplasms

,

non-small-cell lung carcinoma

,

,

squamous cell carcinoma of head and neck

,

healthy volunteers/patients

,

non-small-cell lung carcinoma

,

,

,

,

family relations

healthy volunteers/patients

,

,

,

,

,

muscle spasticity

,

healthy volunteers/patients

,

type 1 diabetes mellitus

,

insulin resistance

,

,

,

,

squamous cell carcinoma of head and neck

,

multiple myeloma

,

head and neck neoplasms

,

,

t-cell lymphoma peripheral

,

,

protein c deficiency

,

plasma cell neoplasms

,

neoplasm metastasis

,

rheumatoid arthritis

,

sleep initiation and maintenance disorders

,

intrinsic sleep disorders

,

granulosa cell tumor

,

wounds and injuries

,

traumatic brain injuries

,

craniocerebral trauma

,

,

colonic neoplasms

,

subarachnoid hemorrhage

,

colorectal neoplasms

,

chronic renal insufficiency

,

chronic kidney failure

,

,

,

,

von willebrand diseases

,

non-small-cell lung carcinoma

,

diabetes mellitus

,

primary immunodeficiency diseases

,

essential hypertension

,

,

,

,

,

ulcerative colitis

,

type 2 diabetes mellitus

,

thrombotic thrombocytopenic purpura

,

cardiovascular diseases

,

,

viral pneumonia

,

glucose metabolism disorders

,

,

prostatic neoplasms

,

,

,

ischemic stroke

,

large b-cell lymphoma diffuse

,

interdigitating dendritic cell sarcoma

,

chronic inflammatory demyelinating polyradiculoneuropathy

,

hodgkin disease

,

,

large-cell lymphoma anaplastic

,

,

precocious puberty

,

non-hodgkin lymphoma

,

,

alpha 1-antitrypsin deficiency

,

t-cell lymphoma

,

hematologic diseases

,

chronic obstructive pulmonary disease

,

mucopolysaccharidoses

,

mucopolysaccharidosis i

,

mucopolysaccharidosis ii

,

ovarian neoplasms

,

ovarian epithelial carcinoma

,

,

diffuse intrinsic pontine glioma

,

,

uveal neoplasms

,

,

,

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