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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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:

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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

,

,

colorectal neoplasms

,

endometrial neoplasms

,

blood coagulation disorders

,

hemostatic disorders

,

type 2 diabetes mellitus

,

diabetes mellitus

prostatic hyperplasia

,

healthy volunteers/patients

,

respiratory tract diseases

,

respiration disorders

,

,

kidney diseases

,

,

,

,

,

respiratory tract infections

,

,

premature obstetric labor

,

,

pulmonary edema

,

,

bacterial infections

,

,

hepatic insufficiency

,

,

non-alcoholic fatty liver disease

,

,

multiple myeloma

,

,

,

,

breast neoplasms

,

,

pulmonary tuberculosis

,

atherosclerosis

,

obstructive lung diseases

,

plasma cell neoplasms

,

,

pancreatic neoplasms

,

,

follicular lymphoma

,

allergic rhinitis

,

coronary disease

,

coronary artery disease

,

myocardial ischemia

,

,

,

hepatocellular carcinoma

,

liver neoplasms

,

precursor cell lymphoblastic leukemia-lymphoma

,

precursor t-cell lymphoblastic leukemia-lymphoma

,

sjogren's syndrome

,

,

,

,

,

systemic lupus erythematosus

,

,

,

acquired immunodeficiency syndrome

,

,

type 2 diabetes mellitus

,

human influenza

,

communicable diseases

,

,

,

parkinson disease

,

,

migraine disorders

,

,

major depressive disorder

,

depressive disorder

,

churg-strauss syndrome

,

granulomatosis with polyangiitis

,

systemic vasculitis

,

eosinophilic granuloma

,

neisseria meningitidis

,

haemophilus influenzae type b

,

restless legs syndrome

,

psychomotor agitation

,

,

hypereosinophilic syndrome

,

,

,

ovarian neoplasms

,

ovarian epithelial carcinoma

,

,

urinary tract infections

,

,

respiratory syncytial virus infections

,

,

chronic obstructive pulmonary disease

,

,

diabetes mellitus

,

,

,

,

pulmonary eosinophilia

,

prostatic neoplasms

,

autoimmune diseases

,

,

growth and development

,

,

,

postherpetic neuralgia

,

healthy volunteers/patients

,

factor vii deficiency

,

,

hidradenitis suppurativa

,

chronic kidney failure

,

diabetes mellitus

,

diffuse scleroderma

,

,

systemic scleroderma

,

rheumatoid arthritis

,

chronic obstructive pulmonary disease

,

soft tissue infections

,

necrotizing fasciitis

,

fournier gangrene

,

,

raynaud disease

,

wounds and injuries

,

myasthenia gravis

,

cardiac surgical procedures

,

,

eosinophilic esophagitis

,

postpartum hemorrhage

,

acute kidney injury

,

sickle cell anemia

,

acute chest syndrome

,

vaso-occlusive crises

,

,

agammaglobulinemia

,

common variable immunodeficiency

,

graft vs host disease

,

hereditary angioedemas

,

afibrinogenemia

,

blood coagulation disorders

,

ischemic stroke

,

,

coronary artery disease

,

,

atherosclerosis

,

heart valve diseases

,

chronic inflammatory demyelinating polyradiculoneuropathy

,

kidney transplantation

,

,

von willebrand diseases

,

hemostatic disorders

,

postoperative hemorrhage

,

dermatomyositis

,

atrial fibrillation

,

,

,

cardiovascular diseases

,

acute coronary syndrome

,

hemorrhagic stroke

,

peripheral arterial disease

,

central nervous system diseases

,

central nervous system infections

,

encephalomyelitis

,

,

meningoencephalitis

,

acquired immunodeficiency syndrome

,

,

factor xiii deficiency

,

thrombocytopenia

,

hematologic neoplasms

,

multiple myeloma

,

b-cell chronic lymphocytic leukemia

,

burkitt lymphoma

,

,

primary immunodeficiency diseases

,

,

hereditary angioedema types i and ii

,

alpha 1-antitrypsin deficiency

,

surgical blood loss

,

immunologic deficiency syndromes

,

thrombocytopenic purpura idiopathic

,

,

cardiovascular surgical procedures

,

tropical spastic paraparesis

,

thromboembolism

,

,

pneumococcal infections

,

,

streptococcal infections

,

bacterial pneumonia

,

bacterial meningitis

,

haemophilus influenzae

,

hereditary complement deficiency diseases

,

neisseriaceae infections

,

,

,

,

communicable diseases

,

ataxia telangiectasia

,

,

x-linked combined immunodeficiency diseases

,

cerebellar ataxia

ovarian neoplasms

,

ovarian epithelial carcinoma

,

,

diffuse intrinsic pontine glioma

,

,

Bos taurus adrenal cortex corticosteroids

colorectal neoplasms

,

endometrial neoplasms

1

2

>

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