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Akcea Therapeutics
Akcea Therapeutics
(AKCA)
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Trade Name
Drug Name
Approved
Patent Expires
Indication
Tegsedi
Inotersen
2018-10-05
2025-2031
Amyloidosis
,
Familial amyloid neuropathies
Waylivra
Volanesorsen
2019-05-03
Hyperlipoproteinemia type i
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
Volanesorsen
cardiovascular abnormalities
,
hyperlipoproteinemia type i
,
syndrome
,
hyperlipidemias
,
hyperlipoproteinemia type v
,
hyperlipoproteinemias
,
lipodystrophy
,
familial partial lipodystrophy
,
hypertriglyceridemia
Inotersen
amyloidosis
,
familial amyloid neuropathies
,
pregnancy
Nepidermin
head and neck neoplasms
,
squamous cell carcinoma of head and neck
,
squamous cell carcinoma
,
glioblastoma
,
neoplasms
,
colorectal neoplasms
,
carcinoma
,
folliculitis
,
pancreatic neoplasms
,
urinary bladder neoplasms
,
cholangiocarcinoma
,
stomach neoplasms
,
ovarian neoplasms
,
ovarian epithelial carcinoma
,
breast neoplasms
,
non-small-cell lung carcinoma
,
lung neoplasms
,
adenocarcinoma
,
adenocarcinoma of lung
,
urologic neoplasms
,
hepatocellular carcinoma
,
liver neoplasms
,
small cell lung carcinoma
Pelacarsen
renal insufficiency
,
cardiovascular diseases
,
hyperlipidemias
,
hyperlipoproteinemias
VUPANORSEN
healthy volunteers/patients
,
hypertriglyceridemia
,
hyperlipidemias
,
hyperlipoproteinemias
,
hypercholesterolemia
,
hyperlipoproteinemia type ii
,
lipodystrophy
,
familial partial lipodystrophy
,
liver diseases
,
type 2 diabetes mellitus
,
diabetes mellitus
,
non-alcoholic fatty liver disease
,
fatty liver
,
hyperlipoproteinemia type v
,
syndrome
,
hyperlipoproteinemia type i
,
dyslipidemias
,
homozygous familial hypercholesterolemia
Endocrine
body weight
,
bone diseases
,
congenital adrenal hyperplasia
,
adrenogenital syndrome
,
alopecia
,
hiv infections
,
endocrine bone diseases
,
adrenocortical hyperfunction
,
bone neoplasms
,
hereditary breast and ovarian cancer syndrome
,
alopecia areata
,
graves disease
,
male genital neoplasms
,
lymphoma
,
narcolepsy
,
cataplexy
,
postmenopausal osteoporosis
,
porokeratosis
,
malformed nails
,
pachyonychia congenita
,
palmoplantar keratoderma
,
urologic neoplasms
,
cartilage diseases
,
lip neoplasms
,
soft tissue neoplasms
,
eye neoplasms
,
fibrous tissue neoplasms
,
adenocarcinoma of lung
,
non-hodgkin lymphoma
,
hematologic neoplasms
,
b-cell chronic lymphocytic leukemia
,
prolymphocytic leukemia t-cell
,
prolymphocytic leukemia
,
osteosarcoma
,
kidney neoplasms
,
metabolic syndrome
,
multiple endocrine neoplasia
,
pituitary diseases
,
acromegaly
,
neuroendocrine carcinoma
,
brain neoplasms
,
pheochromocytoma
,
covid-19
,
liver diseases
,
diabetic foot
,
kidney diseases
,
foot ulcer
,
cognitive dysfunction
,
endometrial neoplasms
,
hypersensitivity
,
papillary thyroid cancer
,
bariatric surgery
,
stomach neoplasms
,
female genital neoplasms
,
hyperinsulinism
,
adrenocortical carcinoma
,
colorectal neoplasms
,
hyperandrogenism
,
depression
,
non-alcoholic fatty liver disease
,
fatty liver
,
chronic pancreatitis
,
ulcer
,
turner syndrome
,
paraganglioma
,
castration-resistant prostatic neoplasms
,
gastrointestinal neoplasms
,
spinal cord injuries
,
sarcoma
,
cardiomyopathies
,
chronic renal insufficiency
,
melanoma
,
second primary neoplasms
,
liposarcoma
,
post-traumatic stress disorders
,
traumatic stress disorders
,
lung neoplasms
,
head and neck neoplasms
,
mood disorders
,
dwarfism
,
gonadal dysgenesis
,
therapeutics
,
renal cell carcinoma
,
hepatocellular carcinoma
,
mesothelioma
,
osteoarthritis
,
medullary carcinoma
,
colonic neoplasms
,
islet cell carcinoma
,
ataxia
,
optic atrophy
,
hypertrophic cardiomyopathy
,
multiple endocrine neoplasia type 2a
,
multiple endocrine neoplasia type 2b
,
suicide
,
dyspepsia
,
sleep wake disorders
,
cachexia
,
contraception
,
anaplastic thyroid carcinoma
,
hemangiosarcoma
,
menopause
,
radiosurgery
,
stomatitis
,
oligospermia
,
azoospermia
,
nephritis
,
wolfram syndrome
,
paraneoplastic syndromes
,
animal mammary neoplasms
,
combination drug therapy
,
neoplasm micrometastasis
,
uterine cervical neoplasms
,
fibrosarcoma
,
myofibromatosis
,
adrenal cortex neoplasms
,
central nervous system neoplasms
,
malignant mesothelioma
,
nervous system neoplasms
,
postpartum depression
,
hypertriglyceridemia
,
acanthosis nigricans
,
sars-cov-2
,
pseudohypoaldosteronism
,
biliary tract diseases
,
hypoaldosteronism
,
shock
,
hemorrhagic shock
,
wasting syndrome
,
inborn genetic diseases
,
neutropenia
,
squamous cell carcinoma of head and neck
,
molecular targeted therapy
,
suicidal ideation
,
serous cystadenocarcinoma
,
noonan syndrome
,
myxoid liposarcoma
,
carcinoma
,
prostatic neoplasms
,
osteoporosis
,
recurrence
,
hypertension
,
overweight
,
ovarian neoplasms
,
hyperaldosteronism
,
cushing syndrome
,
adenocarcinoma
,
pituitary acth hypersecretion
,
intraductal carcinoma noninfiltrating
,
non-small-cell lung carcinoma
,
sleep initiation and maintenance disorders
,
ductal carcinoma breast
,
metabolic bone diseases
,
adrenal insufficiency
,
carcinoma in situ
,
hot flashes
,
liver neoplasms
,
breast diseases
,
lobular carcinoma
,
ductal carcinoma
,
adrenal gland neoplasms
,
arthralgia
,
ovarian epithelial carcinoma
,
drug-related side effects and adverse reactions
,
craniopharyngioma
,
adamantinoma
,
circulating neoplastic cells
,
hypercalcemia
,
disease
,
neoadjuvant therapy
,
drug therapy
,
addison disease
,
amenorrhea
,
musculoskeletal pain
,
hormone-dependent neoplasms
,
male infertility
,
fallopian tube neoplasms
,
inflammatory breast neoplasms
,
eunuchism
,
thyrotoxicosis
,
meningioma
,
pancreatic ductal carcinoma
,
peritoneal neoplasms
,
mucinous adenocarcinoma
,
restless legs syndrome
,
psychomotor agitation
,
psychological distress
,
ependymoma
,
breast neoplasms
,
diabetes mellitus
,
type 2 diabetes mellitus
,
neoplasms
,
obesity
,
syndrome
,
type 1 diabetes mellitus
,
endocrine gland neoplasms
,
triple negative breast neoplasms
,
male breast neoplasms
,
polycystic ovary syndrome
,
endocrine system diseases
,
neuroendocrine tumors
,
thyroid diseases
,
infertility
,
healthy volunteers/patients
,
insulin resistance
,
neoplasm metastasis
,
thyroid neoplasms
,
pancreatic neoplasms
,
hyperparathyroidism
,
adenoma
,
weight loss
,
pituitary neoplasms
,
hypogonadism
,
invasive hydatidiform mole
,
vitamin d deficiency
,
laron syndrome
,
pancreatitis
,
hyperglycemia
,
islet cell adenoma
,
heart failure
,
carcinoid tumor
,
inflammation
,
coronary artery disease
,
thalassemia
,
hyperplasia
,
hypopituitarism
,
hypoglycemia
,
female infertility
,
wounds and injuries
,
pituitary dwarfism
,
primary hyperparathyroidism
,
malignant carcinoid syndrome
,
myocardial ischemia
,
traumatic brain injuries
,
brain injuries
,
parathyroid neoplasms
,
parathyroid diseases
,
breast carcinoma in situ
,
postmenopause
,
spontaneous abortion
,
atrophy
,
serotonin syndrome
,
coronary disease
,
leukemia
,
thyroid nodule
,
anemia
,
pre-eclampsia
,
eclampsia
,
atrial fibrillation
,
hormone replacement therapy
,
goiter
,
sickle cell anemia
,
alzheimer disease
,
knee osteoarthritis
,
diastolic heart failure
,
transgenes
,
embryo transfer
,
macular edema
,
edema
,
osteogenesis imperfecta
,
myelodysplastic syndromes
,
myeloid leukemia acute
,
precursor cell lymphoblastic leukemia-lymphoma
,
blood coagulation disorders
,
aplastic anemia
,
lymphoid leukemia
,
prolactinoma
,
optic nerve hypoplasia
,
septo-optic dysplasia
,
atrophic vaginitis
,
metabolic diseases
,
pediatric obesity
,
malnutrition
,
morbid obesity
,
infections
,
prediabetic state
,
cardiovascular diseases
,
multiple endocrine neoplasia type 1
,
hemorrhage
,
precocious puberty
,
inflammatory bowel diseases
,
schizophrenia
,
communicable diseases
,
klinefelter syndrome
,
pregnancy complications
,
gestational diabetes
,
congenital abnormalities
,
pain
,
peripheral nervous system diseases
,
psychotic disorders
,
mental disorders
,
heart diseases
,
medication adherence
,
growth disorders
,
life style
,
chronic disease
,
iron overload
,
cystic fibrosis
,
hypoparathyroidism
,
premature birth
,
diabetes insipidus
,
hypothyroidism
,
sarcopenia
,
hypospadias
,
acupuncture therapy
,
treatment adherence and compliance
,
disease progression
,
muscle weakness
,
dysmenorrhea
,
subarachnoid hemorrhage
,
postoperative pain
,
psychological stress
,
exocrine pancreatic insufficiency
,
ketosis
,
diabetic ketoacidosis
,
weight gain
,
prader-willi syndrome
,
fibrosis
,
tic disorders
,
sepsis
,
adiposity
,
primary ovarian insufficiency
,
premature menopause
,
dyslipidemias
,
gastroparesis
,
retinal diseases
,
epilepsy
,
hemophilia b
,
anorexia
,
bipolar disorder
,
neuralgia
,
stroke
,
multiple sclerosis
,
diabetic neuropathies
,
thyroidectomy
,
attention deficit disorder with hyperactivity
,
frailty
,
hashimoto disease
,
intestinal neoplasms
,
psychological burnout
,
intestinal diseases
,
endometriosis
,
gastrointestinal microbiome
,
musculoskeletal diseases
,
anesthesia
,
paresis
,
menstruation disturbances
,
esophageal neoplasms
,
glucose metabolism disorders
,
pancreatic diseases
,
sleep apnea syndromes
,
lactation
,
pseudopseudohypoparathyroidism
,
pseudohypoparathyroidism
,
congenital heart defects
,
multiple myeloma
,
feeding behavior
,
hearing loss
,
connective tissue diseases
,
gastrointestinal diseases
,
sjogren's syndrome
,
dementia
,
digestive system diseases
,
intellectual disability
,
rare diseases
,
hypertrophy
,
deafness
,
hirschsprung disease
,
neurodevelopmental disorders
,
body weight changes
,
anorexia nervosa
,
bone marrow diseases
,
adrenocortical adenoma
,
adrenal rest tumor
,
bone fractures
,
glucose intolerance
,
sarcoidosis
,
hyperlipidemias
,
nutritional and metabolic diseases
,
hyperlipoproteinemias
,
menstruation
,
alcoholism
,
fetal growth retardation
,
cardiorespiratory fitness
,
dysphonia
,
migraine disorders
,
chronic obstructive pulmonary disease
,
gastritis
,
feeding and eating disorders
,
somatoform disorders
,
depressive disorder
,
anovulation
,
critical illness
,
microbiota
,
major depressive disorder
,
graves ophthalmopathy
,
eye diseases
,
keratoconus
,
oral pathology
,
lung diseases
,
acute coronary syndrome
,
toxemia
,
diabetic retinopathy
,
chronic pain
,
pregnancy rate
,
thyroiditis
,
cognition
,
ulcerative colitis
,
beta-thalassemia
,
pregnancy-induced hypertension
,
burns
,
fertilization in vitro
,
pregnancy
,
left ventricular dysfunction
,
ventricular dysfunction
,
asthma
,
hyperthyroidism
,
nasopharyngeal carcinoma
,
nasopharyngeal neoplasms
,
and serous mucinous cystic neoplasms
,
hyperprolactinemia
,
disease susceptibility
,
inborn errors metabolism
,
lipodystrophy
,
hyperkinesis
,
nutritional status
,
bacterial vaginosis
,
multiple chronic conditions
,
self efficacy
,
post-acute covid-19 syndrome
,
survivorship
,
aging
,
professional burnout
,
essential hypertension
,
sedentary behavior
,
habitual abortion
,
segmental mastectomy
,
motivational interviewing
,
disorders of sex development
,
local anesthesia
,
asthenia
,
calcium metabolism disorders
,
neck pain
,
back pain
,
myocarditis
,
ruptured aneurysm
,
intracranial aneurysm
,
rupture
,
pelvic exenteration
,
lead poisoning
,
poisoning
,
gastric bypass
,
congenital hyperinsulinism
,
nesidioblastosis
,
enteral nutrition
,
ideal body weight
,
physiological phenomena
,
appetite regulation
,
chronic kidney failure
,
acidosis
,
birth weight
,
peutz-jeghers syndrome
,
carney complex
,
dermatofibrosarcoma
,
neurotoxicity syndromes
,
gestational weight gain
,
obstructive sleep apnea
,
analgesia
,
opioid-related disorders
,
endocrine surgical procedures
,
hereditary neoplastic syndromes
,
adrenal gland diseases
,
thymus neoplasms
,
adrenarche
,
neuroblastoma
,
tetralogy of fallot
,
pulmonary atresia
,
pulmonary valve stenosis
,
immunity
,
multiple trauma
,
transsexualism
,
vascular stiffness
,
rheumatoid arthritis
,
thrombocytopenic purpura idiopathic
,
thrombocytopenia
,
systemic lupus erythematosus
,
acquired immunodeficiency syndrome
,
hiv seropositivity
,
systemic scleroderma
,
behcet syndrome
,
short bowel syndrome
,
cataract
,
retinal detachment
,
craniocerebral trauma
,
arthritis
,
mitochondrial diseases
,
mucocutaneous lymph node syndrome
,
eosinophilic esophagitis
,
large-cell lymphoma anaplastic
,
leiomyosarcoma
,
shy-drager syndrome
,
marfan syndrome
,
dilated cardiomyopathy
,
muscular diseases
,
walker-warburg syndrome
,
osteomyelitis
,
multiple system atrophy
,
biliary liver cirrhosis
,
hemolytic-uremic syndrome
,
hemolysis
,
digeorge syndrome
,
lyme disease
,
muscle spasticity
,
myasthenia gravis
,
ataxia telangiectasia
,
multiple abnormalities
,
esophageal achalasia
,
beckwith-wiedemann syndrome
,
leukoencephalopathies
,
polyneuropathies
,
blindness
,
hereditary eye diseases
,
fragile x syndrome
,
retinitis pigmentosa
,
meningitis
,
myoclonus
,
muscular atrophy
,
rectal fistula
,
telangiectasis
,
metachromatic leukodystrophy
,
intestinal pseudo-obstruction
,
eye abnormalities
,
hemophagocytic lymphohistiocytosis
,
neonatal myasthenia gravis
,
spinocerebellar degenerations
,
fanconi syndrome
,
sensorineural hearing loss
,
vitamin b 12 deficiency
,
frontotemporal dementia
,
williams syndrome
,
spinocerebellar ataxias
,
machado-joseph disease
,
oculocerebrorenal syndrome
,
friedreich ataxia
,
scheuermann disease
,
mucolipidoses
,
phenylketonurias
,
autosomal dominant optic atrophy
,
cerebellar ataxia
,
wagr syndrome
,
dysarthria
,
cystinosis
,
x-linked bulbo-spinal atrophy
,
halitosis
,
idiopathic hypersomnia
,
disorders of excessive somnolence
,
hypophosphatasia
,
gastroenteritis
,
silver-russell syndrome
,
de lange syndrome
,
vitamin e deficiency
,
hereditary sensory and autonomic neuropathies
,
atypical hemolytic uremic syndrome
,
nerve degeneration
,
nijmegen breakage syndrome
,
alstrom syndrome
,
leigh disease
,
retinitis
,
glycogen storage disease
,
leber congenital amaurosis
,
aniridia
,
mannosidase deficiency diseases
,
osteochondrodysplasias
,
craniofacial abnormalities
,
amnesia
,
meningocele
,
hyperargininemia
,
papilloma
,
biliary atresia
,
primary progressive aphasia
,
pick disease of the brain
,
undiagnosed diseases
,
kleine-levin syndrome
,
klippel-feil syndrome
,
frasier syndrome
,
denys-drash syndrome
,
wolf-hirschhorn syndrome
,
alagille syndrome
,
cockayne syndrome
,
viral meningitis
,
channelopathies
,
transient global amnesia
,
refsum disease
,
cauda equina syndrome
,
corneal opacity
,
arthrogryposis
,
hyperacusis
,
hereditary corneal dystrophies
,
nervous system lead poisoning adult
,
ectodermal dysplasia
,
pathologic nystagmus
,
apraxias
,
beta-mannosidosis
,
cogan syndrome
,
olivopontocerebellar atrophies
,
nonketotic hyperglycinemia
,
coffin-lowry syndrome
,
olivary degeneration
,
uniparental disomy
,
brachydactyly
,
cryotherapy
,
shwachman-diamond syndrome
,
lipomatosis
,
intraoperative complications
,
pregnancy in diabetics
,
neurologic manifestations
,
cerebrovascular disorders
,
oxidative stress
,
musculoskeletal manipulations
,
exercise test
,
testicular neoplasms
,
hoarseness
,
vocal cord paralysis
,
recurrent laryngeal nerve injuries
,
parkinson disease
,
substance-related disorders
,
arterial occlusive diseases
,
vision disorders
,
diverticular diseases
,
prostatic diseases
,
gout
,
hearing disorders
,
diverticulum
,
trauma and stressor related disorders
,
nutrition disorders
,
bone density
,
hemochromatosis
,
hemosiderosis
,
hypoxia
,
nervous system diseases
,
oropharyngeal neoplasms
,
alcohol drinking
,
hyperphagia
,
digestive system neoplasms
,
keratitis
,
autoimmune hepatitis
,
autoimmune polyendocrinopathies
,
neurogenic diabetes insipidus
,
nephrogenic diabetes insipidus
,
delusions
,
social adjustment
,
molecular typing
,
body image
,
venous thromboembolism
,
thromboembolism
,
survival analysis
,
conduct disorder
,
attention deficit and disruptive behavior disorders
,
oppositional defiant disorder
,
hemophilia a
,
obstructive lung diseases
,
psychophysiologic disorders
,
gynecomastia
,
acute kidney injury
,
postural balance
,
pharmacogenomic testing
,
dna damage
,
intractable pain
,
acrodynia
,
humans
,
fetal development
,
gender dysphoria
,
ultrasonography
,
elasticity imaging techniques
,
dent disease
,
cryptorchidism
,
tobacco use
,
urinary incontinence
,
enuresis
,
thyroid dysgenesis
,
androgen-insensitivity syndrome
,
crohn disease
,
oncogenes
,
arachnoid cysts
,
skull base neoplasms
,
psychotherapy
,
group psychotherapy
,
acth-secreting pituitary adenoma
,
mutation
,
cardiac surgical procedures
,
social psychology
,
postoperative complications
,
delirium
,
emergence delirium
,
lower urinary tract symptoms
,
intracranial hemorrhages
,
autonomic nervous system diseases
,
ischemia
,
uveitis
,
headache
,
radiation injuries
,
scleritis
,
hyponatremia
,
oogenesis
,
unilateral breast neoplasms
,
anxiety
,
smoking
,
vascular diseases
,
hypobetalipoproteinemias
,
smoking cessation
,
attitude
,
bulimia
,
binge-eating disorder
,
diabetic nephropathies
,
sclerosis
,
hirsutism
,
mucopolysaccharidoses
,
magnetic resonance imaging
,
puberty
,
cholangiocarcinoma
,
brain chemistry
,
congenital hypothyroidism
,
slipped capital femoral epiphyses
,
cerebral infarction
,
infarction
,
gynecologic surgical procedures
,
ovarian reserve
,
sleep deprivation
,
parasomnias
,
gigantism
,
papio
,
autoimmune diseases
,
gastroesophageal reflux
,
mental health
,
deficiency diseases
,
oligomenorrhea
,
hypercholesterolemia
,
inborn errors lipid metabolism
,
leiomyoma
,
problem behavior
,
vaginal diseases
,
self care
,
sudden death
,
ischemic preconditioning
,
exercise
,
neuromuscular diseases
,
child nutrition disorders
,
severe acute malnutrition
,
atopic dermatitis
,
dermatitis
,
neurobehavioral manifestations
,
healthy diet
,
breast feeding
,
herd immunity
,
health behavior
,
abetalipoproteinemia
,
fabry disease
,
abdominal obesity
,
nurse's role
,
immune system diseases
,
chromosome aberrations
,
chromosome disorders
,
sex chromosome aberrations
,
diabetes complications
,
myofascial pain syndromes
,
fibromyalgia
,
bruxism
,
health status indicators
,
child development
,
sinusitis
,
nasal polyps
,
polyps
,
rhinosinusitis
,
polypharmacy
,
menstrual cycle
,
scoliosis
,
fine-needle biopsy
,
vegetarian diet
,
vegan diet
,
metabolome
,
skin ulcer
,
lymphatic metastasis
,
muscle neoplasms
,
healthy lifestyle
,
patient compliance
,
treatment refusal
,
surveys and questionnaires
,
apnea
,
psoriasis
,
perimenopause
,
polyostotic fibrous dysplasia
,
fibrous dysplasia of bone
,
hip osteoarthritis
,
pneumonia
,
cryopreservation
,
candidiasis
,
lichen planus
,
vulvodynia
,
urinary tract infections
,
ureaplasma infections
,
vaginitis
,
lichen sclerosus et atrophicus
,
nerve block
,
ganglion cysts
,
anxiety disorders
,
synovial cyst
,
periodontitis
,
cardiovascular abnormalities
,
athletic injuries
,
thoracica
,
preoperative exercise
,
pneumonectomy
,
physiological sexual dysfunction
,
cognitive behavioral therapy
,
pulmonary hypertension
,
vascular malformations
,
hereditary angioedemas
,
eosinophilia
,
amyloidosis
,
von hippel-lindau disease
,
hepatolenticular degeneration
,
mastocytosis
,
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P2y purinoceptor 12
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acute coronary syndrome
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