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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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Revenue by Drug

Marketed Drugs

Therapeutic Areas:

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Classification:

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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

,

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

,

pancreatic neoplasms

,

b-cell lymphoma

,

neoplasm metastasis

,

,

large b-cell lymphoma diffuse

,

,

castration-resistant prostatic neoplasms

,

cholangiocarcinoma

,

non-hodgkin lymphoma

,

brain neoplasms

,

colonic neoplasms

,

follicular lymphoma

,

t-cell lymphoma

,

neuroendocrine tumors

,

cutaneous malignant melanoma

,

myeloid leukemia acute

,

kidney neoplasms

,

mouth neoplasms

,

oropharyngeal neoplasms

,

bile duct neoplasms

,

,

,

malignant fibrous histiocytoma

,

pancreatic ductal carcinoma

,

adenocarcinoma of lung

,

,

uterine neoplasms

,

t-cell lymphoma peripheral

,

,

adenoviridae infections

,

gallbladder neoplasms

,

local neoplasm recurrence

,

myeloid leukemia

,

rectal neoplasms

,

laryngeal neoplasms

,

hypopharyngeal neoplasms

,

,

,

,

epstein-barr virus infections

,

,

unknown primary neoplasms

,

,

b-cell chronic lymphocytic leukemia

,

lymphoid leukemia

,

endometrioid carcinoma

,

,

sezary syndrome

,

mycosis fungoides

,

t-cell lymphoma cutaneous

,

colonic diseases

,

histiocytic sarcoma

,

follicular dendritic cell sarcoma

,

interdigitating dendritic cell sarcoma

,

urethral neoplasms

,

ureteral neoplasms

,

pelvic neoplasms

,

environmental biomarkers

,

inflammatory breast neoplasms

,

hemangiosarcoma

,

intestinal neoplasms

,

,

,

nervous system diseases

,

neoplasms by histologic type

,

nerve tissue neoplasms

,

primitive neuroectodermal tumors

,

neuroepithelial neoplasms

,

neuroectodermal tumors

,

salivary gland neoplasms

,

thyroid neoplasms

,

,

carcinoid tumor

,

thyroid diseases

,

vulvar neoplasms

,

reactive arthritis

,

gastrointestinal stromal tumors

,

,

gastrointestinal neoplasms

,

,

mullerian mixed tumor

,

basal cell carcinoma

,

basal cell neoplasms

,

,

male breast neoplasms

,

uveal neoplasms

,

,

,

breast neoplasms

,

colorectal neoplasms

,

triple negative breast neoplasms

,

,

renal cell carcinoma

,

,

head and neck neoplasms

,

ovarian neoplasms

,

esophageal neoplasms

,

liver neoplasms

,

prostatic neoplasms

,

urinary bladder neoplasms

,

hepatocellular carcinoma

,

endometrial neoplasms

,

,

hodgkin disease

,

small cell lung carcinoma

,

stomach neoplasms

,

ovarian epithelial carcinoma

,

uterine cervical neoplasms

,

squamous cell neoplasms

,

transitional cell carcinoma

,

multiple myeloma

,

urologic neoplasms

,

esophageal squamous cell carcinoma

,

,

fallopian tube neoplasms

,

plasma cell neoplasms

,

biliary tract neoplasms

,

,

hypersensitivity

,

nasopharyngeal neoplasms

,

microsatellite instability

,

merkel cell carcinoma

,

malignant mesothelioma

,

nasopharyngeal carcinoma

,

peritoneal neoplasms

,

disease progression

,

non-muscle invasive bladder neoplasms

,

hematologic neoplasms

,

non-small-cell lung carcinoma

,

,

,

squamous cell carcinoma of head and neck

,

squamous cell carcinoma

myotonic dystrophy

,

,

disorders of excessive somnolence

,

idiopathic hypersomnia

hepatic insufficiency

,

renal insufficiency

,

castration-resistant prostatic neoplasms

,

,

,

,

,

unknown primary neoplasms

,

immunomodulation

,

gram-positive bacterial infections

,

t-cell lymphoma peripheral

,

,

merkel cell carcinoma

,

mycosis fungoides

,

clear cell sarcoma

,

myelodysplastic syndromes

,

,

,

pulmonary sarcoidosis

,

lyme neuroborreliosis

,

hypertriglyceridemia

,

,

granuloma annulare

,

mycobacterium avium complex

,

thoracic neoplasms

,

pelvic neoplasms

,

,

,

brain neoplasms

,

stomach neoplasms

,

,

triple negative breast neoplasms

,

multiple myeloma

,

head and neck neoplasms

,

urinary bladder neoplasms

,

ovarian neoplasms

,

thyroid neoplasms

,

plasma cell neoplasms

,

,

small cell lung carcinoma

,

ovarian epithelial carcinoma

,

esophageal neoplasms

,

borrelia infections

,

endometrial neoplasms

,

thyroid diseases

,

keratoconjunctivitis sicca

,

dry eye syndromes

,

uveal neoplasms

,

respiratory syncytial viruses

,

,

,

transitional cell carcinoma

,

kidney diseases

,

small cell carcinoma

,

adenocarcinoma of lung

,

large-cell lymphoma anaplastic

,

,

,

pheochromocytoma

,

,

healthcare-associated pneumonia

,

systemic lupus erythematosus

,

alopecia areata

,

,

,

fallopian tube neoplasms

,

peritoneal neoplasms

,

oligodendroglioma

,

,

hemangioblastoma

,

urogenital neoplasms

,

,

central nervous system neoplasms

,

nervous system neoplasms

,

,

neurofibromatosis 1

,

neurofibromatoses

,

,

germ cell and embryonal neoplasms

,

carcinoid tumor

,

urethral neoplasms

,

ureteral neoplasms

,

alveolar soft part sarcoma

,

,

polycystic kidney diseases

,

autosomal dominant polycystic kidney

,

,

cholangiocarcinoma

,

gene rearrangement

,

lobular carcinoma

,

plasma cell leukemia

,

medulloblastoma

,

,

rhabdomyosarcoma

,

diffuse intrinsic pontine glioma

,

intraductal carcinoma noninfiltrating

,

b-cell lymphoma

,

cutaneous lupus erythematosus

,

keratoconjunctivitis

,

,

respiratory tract infections

,

,

,

,

hidradenitis suppurativa

,

,

meningococcal infections

,

neoadjuvant therapy

,

diabetes mellitus

,

type 1 diabetes mellitus

,

,

multidrug-resistant tuberculosis

,

von hippel-lindau disease

,

,

,

neurodermatitis

,

,

urologic neoplasms

,

urologic diseases

,

neoplasms by site

,

glandular and epithelial neoplasms

,

,

,

benign fibrous histiocytoma

,

,

malignant fibrous histiocytoma

,

myeloid leukemia accelerated phase

,

,

malignant mesothelioma

,

,

urinary bladder diseases

,

adrenal cortex neoplasms

,

adrenocortical carcinoma

,

second primary neoplasms

,

pulmonary tuberculosis

,

hematologic neoplasms

,

amyotrophic lateral sclerosis

,

motor neuron disease

,

mouth neoplasms

,

,

penile neoplasms

,

plexiform neurofibroma

,

,

testicular neoplasms

,

solitary fibrous tumors

,

,

hemangiopericytoma

,

male urogenital diseases

,

urogenital diseases

,

,

adenoid cystic carcinoma

,

,

hemangiosarcoma

,

systemic scleroderma

,

diffuse scleroderma

,

,

,

post-acute covid-19 syndrome

,

,

colorectal neoplasms

,

atopic dermatitis

,

,

,

,

,

kidney neoplasms

,

prostatic neoplasms

,

squamous cell carcinoma

,

hepatocellular carcinoma

,

liver neoplasms

,

pancreatic neoplasms

,

neoplasm metastasis

,

,

psoriatic arthritis

,

squamous cell neoplasms

,

squamous cell carcinoma of head and neck

,

,

male breast neoplasms

,

,

precursor cell lymphoblastic leukemia-lymphoma

,

lymphoid leukemia

,

myeloid leukemia chronic-phase

,

juvenile arthritis

,

,

,

bacterial pneumonia

,

,

mycobacterium avium-intracellulare infection

,

immune system diseases

,

inborn genetic diseases

,

genetic skin diseases

,

eczematous skin diseases

,

hypersensitivity

,

immediate hypersensitivity

,

islet cell carcinoma

,

ankylosing spondylitis

,

bacterial infections

,

pancreatic ductal carcinoma

,

,

,

febrile neutropenia

,

,

mycobacterium infections

,

adenosquamous carcinoma

,

large cell carcinoma

,

nervous system diseases

,

optic nerve diseases

,

disease progression

,

cardiovascular diseases

,

immunologic deficiency syndromes

,

breast neoplasms

,

healthy volunteers/patients

,

,

renal cell carcinoma

,

non-small-cell lung carcinoma

,

,

,

rheumatoid arthritis

,

,

,

,

,

acquired immunodeficiency syndrome

,

communicable diseases

,

bcr-abl positive chronic myelogenous leukemia

,

,

myeloid leukemia

,

neuroendocrine tumors

,

gastrointestinal stromal tumors

,

ulcerative colitis

,

,

,

non-hodgkin lymphoma

,

kidney transplantation

,

philadelphia chromosome

,

islet cell adenoma

,

intraabdominal infections

,

mantle-cell lymphoma

,

,

spondylarthritis

,

,

gastrointestinal neoplasms

,

digestive system neoplasms

,

,

,

,

,

,

ventilator-associated pneumonia

,

chronic hepatitis c

,

,

axial spondyloarthritis

,

,

premature birth

,

bacterial vaginosis

,

vaginal diseases

,

,

infectious skin diseases

,

soft tissue infections

,

,

,

community-acquired infections

,

staphylococcal infections

,

,

hypercholesterolemia

,

coronary disease

,

coronary artery disease

,

myocardial ischemia

,

,

bacterial skin diseases

,

,

asymptomatic infections

,

,

,

respiratory tract diseases

,

noise-induced hearing loss

,

,

parkinson disease

ovarian neoplasms

,

ovarian epithelial carcinoma

,

,

diffuse intrinsic pontine glioma

,

,

Bos taurus adrenal cortex corticosteroids

muscular dystrophies

,

limb-girdle muscular dystrophies

myelodysplastic syndromes

,

thoracic neoplasms

,

glandular and epithelial neoplasms

,

,

,

pharmacokinetics

,

soft tissue neoplasms

,

digestive system neoplasms

,

,

respiratory tract neoplasms

,

bronchogenic carcinoma

,

endocrine gland neoplasms

,

breast diseases

,

bronchial neoplasms

,

intraductal carcinoma noninfiltrating

,

,

oligodendroglioma

,

,

pancreatic neoplasms

,

neoplasm metastasis

,

,

b-cell lymphoma

,

brain neoplasms

,

large b-cell lymphoma diffuse

,

castration-resistant prostatic neoplasms

,

non-hodgkin lymphoma

,

t-cell lymphoma

,

,

cholangiocarcinoma

,

myeloid leukemia acute

,

neuroendocrine tumors

,

follicular lymphoma

,

neuroendocrine carcinoma

,

t-cell lymphoma peripheral

,

kidney neoplasms

,

thyroid neoplasms

,

pancreatic ductal carcinoma

,

uterine neoplasms

,

uveal neoplasms

,

myeloid leukemia

,

invasive hydatidiform mole

,

lymphoid leukemia

,

,

female genital neoplasms

,

small cell carcinoma

,

urethral neoplasms

,

colonic neoplasms

,

vulvar neoplasms

,

b-cell lymphoma marginal zone

,

,

thyroid diseases

,

cutaneous malignant melanoma

,

,

microsatellite repeats

,

gastrointestinal neoplasms

,

mouth neoplasms

,

oropharyngeal neoplasms

,

laryngeal neoplasms

,

ureteral neoplasms

,

salivary gland neoplasms

,

endometrioid carcinoma

,

serous cystadenocarcinoma

,

mantle-cell lymphoma

,

waldenstrom macroglobulinemia

,

penile neoplasms

,

b-cell chronic lymphocytic leukemia

,

hemangiosarcoma

,

,

,

mullerian mixed tumor

,

,

thymus neoplasms

,

precursor cell lymphoblastic leukemia-lymphoma

,

rectal neoplasms

,

hypopharyngeal neoplasms

,

male genital neoplasms

,

bile duct neoplasms

,

respiratory tract infections

,

papillomavirus infections

,

,

adenoid cystic carcinoma

,

,

male breast neoplasms

,

,

malignant fibrous histiocytoma

,

epstein-barr virus infections

,

adenocarcinoma of lung

,

,

neurofibrosarcoma

,

nerve sheath neoplasms

,

,

,

sezary syndrome

,

mycosis fungoides

,

t-cell lymphoma cutaneous

,

,

pelvic neoplasms

,

,

germ cell and embryonal neoplasms

,

neoplasms by histologic type

,

adenoviridae infections

,

basal cell carcinoma

,

anaplastic thyroid carcinoma

,

reactive arthritis

,

gallbladder neoplasms

,

clear cell adenocarcinoma

,

adenosquamous carcinoma

,

gestational trophoblastic disease

,

alveolar soft part sarcoma

,

biphenotypic leukemia acute

,

local neoplasm recurrence

,

,

,

meningeal carcinomatosis

,

papillomaviridae

,

second primary neoplasms

,

immunoblastic lymphadenopathy

,

enteropathy-associated t-cell lymphoma

,

intestinal diseases

,

,

carcinoma in situ

,

cervical intraepithelial neoplasia

,

uterine cervical dysplasia

,

squamous intraepithelial lesions of the cervix

,

central nervous system diseases

,

extranodal nk-t-cell lymphoma

,

,

,

,

unknown primary neoplasms

,

synovial sarcoma

,

,

oral leukoplakia

,

,

lymphoproliferative disorders

,

,

colonic diseases

,

histiocytic sarcoma

,

follicular dendritic cell sarcoma

,

interdigitating dendritic cell sarcoma

,

environmental biomarkers

,

circulating neoplastic cells

,

inflammatory breast neoplasms

,

meningeal neoplasms

,

intestinal neoplasms

,

central nervous system neoplasms

,

nervous system neoplasms

,

nervous system diseases

,

nerve tissue neoplasms

,

primitive neuroectodermal tumors

,

neuroepithelial neoplasms

,

neuroectodermal tumors

,

adrenocortical carcinoma

,

carcinoid tumor

,

mediastinal neoplasms

,

testicular neoplasms

,

gastrointestinal stromal tumors

,

hairy cell leukemia

,

burkitt lymphoma

,

laryngeal diseases

,

vaginal neoplasms

,

acinar cell carcinoma

,

adenomyoepithelioma

,

hereditary breast and ovarian cancer syndrome

,

maxillary sinus neoplasms

,

basal cell neoplasms

,

urogenital neoplasms

,

male urogenital diseases

,

urogenital diseases

,

,

,

breast neoplasms

,

colorectal neoplasms

,

triple negative breast neoplasms

,

,

renal cell carcinoma

,

,

ovarian neoplasms

,

,

head and neck neoplasms

,

urinary bladder neoplasms

,

prostatic neoplasms

,

esophageal neoplasms

,

stomach neoplasms

,

liver neoplasms

,

hepatocellular carcinoma

,

endometrial neoplasms

,

uterine cervical neoplasms

,

ovarian epithelial carcinoma

,

small cell lung carcinoma

,

transitional cell carcinoma

,

hodgkin disease

,

fallopian tube neoplasms

,

urologic neoplasms

,

multiple myeloma

,

,

squamous cell neoplasms

,

,

plasma cell neoplasms

,

,

biliary tract neoplasms

,

esophageal squamous cell carcinoma

,

malignant mesothelioma

,

peritoneal neoplasms

,

microsatellite instability

,

merkel cell carcinoma

,

non-muscle invasive bladder neoplasms

,

hematologic neoplasms

,

hypersensitivity

,

nasopharyngeal neoplasms

,

disease progression

,

nasopharyngeal carcinoma

,

muscle neoplasms

,

,

non-small-cell lung carcinoma

,

,

squamous cell carcinoma of head and neck

,

squamous cell carcinoma

urethral neoplasms

,

transitional cell carcinoma

,

urologic neoplasms

,

small cell carcinoma

,

small cell lung carcinoma

,

neuroendocrine tumors

,

neuroendocrine carcinoma

,

kidney neoplasms

,

ureteral neoplasms

,

colorectal neoplasms

,

,

castration-resistant prostatic neoplasms

,

,

triple negative breast neoplasms

,

urinary bladder neoplasms

,

,

laryngeal neoplasms

,

laryngeal diseases

,

hepatocellular carcinoma

,

liver neoplasms

,

uterine cervical neoplasms

,

cholangiocarcinoma

,

gallbladder neoplasms

,

maxillary sinus neoplasms

,

neoplasm metastasis

,

,

prostatic neoplasms

,

non-small-cell lung carcinoma

,

,

squamous cell carcinoma of head and neck

,

,

esophageal neoplasms

,

ovarian neoplasms

,

squamous cell carcinoma

,

stomach neoplasms

,

endometrial neoplasms

,

ovarian epithelial carcinoma

,

fallopian tube neoplasms

,

nasopharyngeal carcinoma

,

nasopharyngeal neoplasms

,

head and neck neoplasms

,

disease progression

,

peritoneal neoplasms

,

squamous cell neoplasms

,

esophageal squamous cell carcinoma

,

breast neoplasms

,

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