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Company ReportsAkcea 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:

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Classification:

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Status:

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Phases:

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Drug Name

Phase 1

Phase 2

Phase 3

Phase 4

Indication

cardiovascular abnormalities

,

hyperlipoproteinemia type i

,

,

hyperlipidemias

,

hyperlipoproteinemia type v

,

hyperlipoproteinemias

,

,

familial partial lipodystrophy

,

hypertriglyceridemia

,

familial amyloid neuropathies

,

Sodium chloride

,

,

,

knee osteoarthritis

,

aortic valve stenosis

,

pathologic constriction

,

von willebrand diseases

,

,

,

tibial meniscus injuries

renal insufficiency

,

cardiovascular diseases

,

hyperlipidemias

,

hyperlipoproteinemias

hypertriglyceridemia

,

hypercholesterolemia

,

hyperlipoproteinemia type ii

,

,

familial partial lipodystrophy

,

,

type 2 diabetes mellitus

,

diabetes mellitus

,

non-alcoholic fatty liver disease

,

,

hyperlipidemias

,

hyperlipoproteinemia type v

,

,

hyperlipoproteinemia type i

,

hyperlipoproteinemias

,

homozygous familial hypercholesterolemia

,

,

congenital adrenal hyperplasia

,

adrenogenital syndrome

,

,

,

endocrine bone diseases

,

adrenocortical hyperfunction

,

,

hereditary breast and ovarian cancer syndrome

,

alopecia areata

,

male genital neoplasms

,

,

,

,

,

postmenopausal osteoporosis

,

,

malformed nails

,

pachyonychia congenita

,

palmoplantar keratoderma

,

urologic neoplasms

,

cartilage diseases

,

,

soft tissue neoplasms

,

,

fibrous tissue neoplasms

,

adenocarcinoma of lung

,

non-hodgkin lymphoma

,

hematologic neoplasms

,

b-cell chronic lymphocytic leukemia

,

prolymphocytic leukemia t-cell

,

prolymphocytic leukemia

,

,

kidney neoplasms

,

metabolic syndrome

,

multiple endocrine neoplasia

,

pituitary diseases

,

,

neuroendocrine carcinoma

,

brain neoplasms

,

pheochromocytoma

,

,

,

,

,

cognitive dysfunction

,

endometrial neoplasms

,

hypersensitivity

,

kidney diseases

,

papillary thyroid cancer

,

stomach neoplasms

,

bariatric surgery

,

hyperinsulinism

,

adrenocortical carcinoma

,

colorectal neoplasms

,

hyperandrogenism

,

,

,

turner syndrome

,

,

castration-resistant prostatic neoplasms

,

female genital neoplasms

,

gastrointestinal neoplasms

,

spinal cord injuries

,

,

cardiomyopathies

,

non-alcoholic fatty liver disease

,

,

,

second primary neoplasms

,

post-traumatic stress disorders

,

traumatic stress disorders

,

,

,

chronic pancreatitis

,

chronic renal insufficiency

,

,

gonadal dysgenesis

,

,

renal cell carcinoma

,

hepatocellular carcinoma

,

,

,

medullary carcinoma

,

colonic neoplasms

,

islet cell carcinoma

,

,

,

hypertrophic cardiomyopathy

,

head and neck neoplasms

,

multiple endocrine neoplasia type 2a

,

multiple endocrine neoplasia type 2b

,

,

,

sleep wake disorders

,

,

anaplastic thyroid carcinoma

,

hemangiosarcoma

,

,

,

,

,

,

,

wolfram syndrome

,

paraneoplastic syndromes

,

animal mammary neoplasms

,

combination drug therapy

,

neoplasm micrometastasis

,

uterine cervical neoplasms

,

,

myofibromatosis

,

adrenal cortex neoplasms

,

central nervous system neoplasms

,

malignant mesothelioma

,

nervous system neoplasms

,

postpartum depression

,

hypertriglyceridemia

,

acanthosis nigricans

,

,

pseudohypoaldosteronism

,

biliary tract diseases

,

hypoaldosteronism

,

,

hemorrhagic shock

,

wasting syndrome

,

,

inborn genetic diseases

,

neoadjuvant therapy

,

,

molecular targeted therapy

,

suicidal ideation

,

serous cystadenocarcinoma

,

noonan syndrome

,

,

prostatic neoplasms

,

,

,

,

ovarian neoplasms

,

,

cushing syndrome

,

hyperaldosteronism

,

,

pituitary acth hypersecretion

,

intraductal carcinoma noninfiltrating

,

non-small-cell lung carcinoma

,

ductal carcinoma breast

,

adrenal insufficiency

,

carcinoma in situ

,

,

liver neoplasms

,

lobular carcinoma

,

ductal carcinoma

,

sleep initiation and maintenance disorders

,

metabolic bone diseases

,

adrenal gland neoplasms

,

,

breast diseases

,

ovarian epithelial carcinoma

,

drug-related side effects and adverse reactions

,

craniopharyngioma

,

,

circulating neoplastic cells

,

,

,

addison disease

,

,

musculoskeletal pain

,

hormone-dependent neoplasms

,

male infertility

,

,

fallopian tube neoplasms

,

inflammatory breast neoplasms

,

,

,

,

pancreatic ductal carcinoma

,

peritoneal neoplasms

,

mucinous adenocarcinoma

,

restless legs syndrome

,

psychomotor agitation

,

psychological distress

,

,

breast neoplasms

,

diabetes mellitus

,

type 2 diabetes mellitus

,

,

,

,

type 1 diabetes mellitus

,

endocrine gland neoplasms

,

male breast neoplasms

,

triple negative breast neoplasms

,

polycystic ovary syndrome

,

endocrine system diseases

,

neuroendocrine tumors

,

,

thyroid diseases

,

insulin resistance

,

healthy volunteers/patients

,

neoplasm metastasis

,

pancreatic neoplasms

,

thyroid neoplasms

,

,

,

pituitary neoplasms

,

,

invasive hydatidiform mole

,

,

hyperparathyroidism

,

vitamin d deficiency

,

,

islet cell adenoma

,

carcinoid tumor

,

,

,

,

,

,

coronary artery disease

,

hypopituitarism

,

,

female infertility

,

wounds and injuries

,

pituitary dwarfism

,

malignant carcinoid syndrome

,

traumatic brain injuries

,

,

primary hyperparathyroidism

,

parathyroid neoplasms

,

myocardial ischemia

,

parathyroid diseases

,

breast carcinoma in situ

,

,

spontaneous abortion

,

,

serotonin syndrome

,

coronary disease

,

,

,

,

,

,

atrial fibrillation

,

hormone replacement therapy

,

,

sickle cell anemia

,

alzheimer disease

,

knee osteoarthritis

,

diastolic heart failure

,

,

embryo transfer

,

,

,

osteogenesis imperfecta

,

myelodysplastic syndromes

,

myeloid leukemia acute

,

precursor cell lymphoblastic leukemia-lymphoma

,

blood coagulation disorders

,

aplastic anemia

,

lymphoid leukemia

,

,

optic nerve hypoplasia

,

septo-optic dysplasia

,

atrophic vaginitis

,

pediatric obesity

,

metabolic diseases

,

,

prediabetic state

,

cardiovascular diseases

,

,

multiple endocrine neoplasia type 1

,

,

,

precocious puberty

,

inflammatory bowel diseases

,

,

klinefelter syndrome

,

pregnancy complications

,

gestational diabetes

,

congenital abnormalities

,

,

psychotic disorders

,

mental disorders

,

,

medication adherence

,

growth disorders

,

,

communicable diseases

,

,

premature birth

,

peripheral nervous system diseases

,

diabetes insipidus

,

,

acupuncture therapy

,

chronic disease

,

treatment adherence and compliance

,

disease progression

,

muscle weakness

,

,

subarachnoid hemorrhage

,

postoperative pain

,

psychological stress

,

exocrine pancreatic insufficiency

,

,

diabetic ketoacidosis

,

,

prader-willi syndrome

,

,

cystic fibrosis

,

,

hypoparathyroidism

,

,

,

primary ovarian insufficiency

,

premature menopause

,

,

,

retinal diseases

,

,

,

,

bipolar disorder

,

,

multiple sclerosis

,

attention deficit disorder with hyperactivity

,

,

intestinal neoplasms

,

psychological burnout

,

intestinal diseases

,

,

gastrointestinal microbiome

,

,

,

menstruation disturbances

,

esophageal neoplasms

,

glucose metabolism disorders

,

pancreatic diseases

,

sleep apnea syndromes

,

,

pseudopseudohypoparathyroidism

,

pseudohypoparathyroidism

,

congenital heart defects

,

multiple myeloma

,

feeding behavior

,

,

connective tissue diseases

,

gastrointestinal diseases

,

sjogren's syndrome

,

,

digestive system diseases

,

intellectual disability

,

,

,

,

hirschsprung disease

,

neurodevelopmental disorders

,

body weight changes

,

anorexia nervosa

,

bone marrow diseases

,

adrenocortical adenoma

,

adrenal rest tumor

,

,

glucose intolerance

,

,

nutritional and metabolic diseases

,

,

,

fetal growth retardation

,

,

,

migraine disorders

,

chronic obstructive pulmonary disease

,

,

feeding and eating disorders

,

somatoform disorders

,

depressive disorder

,

diabetic neuropathies

,

,

critical illness

,

,

major depressive disorder

,

graves ophthalmopathy

,

,

,

,

,

acute coronary syndrome

,

,

,

diabetic retinopathy

,

,

,

,

hashimoto disease

,

,

,

beta-thalassemia

,

pregnancy-induced hypertension

,

,

fertilization in vitro

,

left ventricular dysfunction

,

ventricular dysfunction

,

,

hyperthyroidism

,

nasopharyngeal carcinoma

,

nasopharyngeal neoplasms

,

and serous mucinous cystic neoplasms

,

,

disease susceptibility

,

inborn errors metabolism

,

,

,

nutritional status

,

bacterial vaginosis

,

,

post-acute covid-19 syndrome

,

,

professional burnout

,

essential hypertension

,

sedentary behavior

,

habitual abortion

,

segmental mastectomy

,

motivational interviewing

,

musculoskeletal diseases

,

disorders of sex development

,

local anesthesia

,

,

calcium metabolism disorders

,

,

,

,

intracranial aneurysm

,

,

pelvic exenteration

,

,

,

,

congenital hyperinsulinism

,

nesidioblastosis

,

enteral nutrition

,

ideal body weight

,

physiological phenomena

,

appetite regulation

,

chronic kidney failure

,

,

,

peutz-jeghers syndrome

,

,

dermatofibrosarcoma

,

neurotoxicity syndromes

,

gestational weight gain

,

obstructive sleep apnea

,

,

opioid-related disorders

,

endocrine surgical procedures

,

hereditary neoplastic syndromes

,

adrenal gland diseases

,

thymus neoplasms

,

,

,

tetralogy of fallot

,

pulmonary atresia

,

pulmonary valve stenosis

,

,

multiple trauma

,

,

vascular stiffness

,

rheumatoid arthritis

,

thrombocytopenic purpura idiopathic

,

thrombocytopenia

,

systemic lupus erythematosus

,

acquired immunodeficiency syndrome

,

hiv seropositivity

,

systemic scleroderma

,

behcet syndrome

,

short bowel syndrome

,

,

retinal detachment

,

craniocerebral trauma

,

,

mitochondrial diseases

,

mucocutaneous lymph node syndrome

,

eosinophilic esophagitis

,

large-cell lymphoma anaplastic

,

,

shy-drager syndrome

,

marfan syndrome

,

dilated cardiomyopathy

,

muscular diseases

,

walker-warburg syndrome

,

,

multiple system atrophy

,

biliary liver cirrhosis

,

hemolytic-uremic syndrome

,

,

digeorge syndrome

,

,

muscle spasticity

,

myasthenia gravis

,

ataxia telangiectasia

,

multiple abnormalities

,

esophageal achalasia

,

beckwith-wiedemann syndrome

,

leukoencephalopathies

,

polyneuropathies

,

,

hereditary eye diseases

,

fragile x syndrome

,

retinitis pigmentosa

,

,

,

muscular atrophy

,

,

,

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

,

,

phenylketonurias

,

autosomal dominant optic atrophy

,

cerebellar ataxia

,

,

,

,

x-linked bulbo-spinal atrophy

,

,

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

,

,

,

glycogen storage disease

,

leber congenital amaurosis

,

,

mannosidase deficiency diseases

,

osteochondrodysplasias

,

craniofacial abnormalities

,

,

,

hyperargininemia

,

,

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

,

,

cauda equina syndrome

,

corneal opacity

,

,

,

hereditary corneal dystrophies

,

nervous system lead poisoning adult

,

ectodermal dysplasia

,

pathologic nystagmus

,

,

beta-mannosidosis

,

,

olivopontocerebellar atrophies

,

nonketotic hyperglycinemia

,

coffin-lowry syndrome

,

olivary degeneration

,

uniparental disomy

,

,

,

shwachman-diamond syndrome

,

,

intraoperative complications

,

pregnancy in diabetics

,

neurologic manifestations

,

cerebrovascular disorders

,

hyperlipidemias

,

hyperlipoproteinemias

,

oxidative stress

,

musculoskeletal manipulations

,

,

cardiorespiratory fitness

,

testicular neoplasms

,

,

vocal cord paralysis

,

recurrent laryngeal nerve injuries

,

parkinson disease

,

substance-related disorders

,

arterial occlusive diseases

,

vision disorders

,

diverticular diseases

,

prostatic diseases

,

,

hearing disorders

,

,

trauma and stressor related disorders

,

nutrition disorders

,

,

hemochromatosis

,

,

,

nervous system diseases

,

oropharyngeal neoplasms

,

alcohol drinking

,

,

digestive system neoplasms

,

,

autoimmune hepatitis

,

autoimmune polyendocrinopathies

,

neurogenic diabetes insipidus

,

nephrogenic diabetes insipidus

,

,

social adjustment

,

,

molecular typing

,

,

venous thromboembolism

,

thromboembolism

,

survival analysis

,

conduct disorder

,

attention deficit and disruptive behavior disorders

,

oppositional defiant disorder

,

,

obstructive lung diseases

,

psychophysiologic disorders

,

,

acute kidney injury

,

postural balance

,

pharmacogenomic testing

,

,

intractable pain

,

,

,

fetal development

,

gender dysphoria

,

ultrasonography

,

elasticity imaging techniques

,

,

,

,

urinary incontinence

,

,

thyroid dysgenesis

,

androgen-insensitivity syndrome

,

ulcerative colitis

,

,

,

arachnoid cysts

,

skull base neoplasms

,

,

group psychotherapy

,

acth-secreting pituitary adenoma

,

,

cardiac surgical procedures

,

social psychology

,

postoperative complications

,

,

emergence delirium

,

lower urinary tract symptoms

,

intracranial hemorrhages

,

autonomic nervous system diseases

,

,

,

,

radiation injuries

,

,

,

,

unilateral breast neoplasms

,

,

,

,

vascular diseases

,

hypobetalipoproteinemias

,

smoking cessation

,

,

,

binge-eating disorder

,

diabetic nephropathies

,

,

,

mucopolysaccharidoses

,

magnetic resonance imaging

,

,

cholangiocarcinoma

,

brain chemistry

,

congenital hypothyroidism

,

slipped capital femoral epiphyses

,

hyperprolactinemia

,

cerebral infarction

,

,

gynecologic surgical procedures

,

ovarian reserve

,

sleep deprivation

,

,

,

,

autoimmune diseases

,

gastroesophageal reflux

,

,

deficiency diseases

,

,

hypercholesterolemia

,

inborn errors lipid metabolism

,

,

problem behavior

,

vaginal diseases

,

multiple chronic conditions

,

,

,

ischemic preconditioning

,

,

neuromuscular diseases

,

,

child nutrition disorders

,

severe acute malnutrition

,

atopic dermatitis

,

,

neurobehavioral manifestations

,

,

,

,

health behavior

,

abetalipoproteinemia

,

,

abdominal obesity

,

,

immune system diseases

,

chromosome aberrations

,

chromosome disorders

,

sex chromosome aberrations

,

diabetes complications

,

myofascial pain syndromes

,

,

,

health status indicators

,

child development

,

,

,

,

,

,

menstrual cycle

,

,

fine-needle biopsy

,

vegetarian diet

,

,

,

,

lymphatic metastasis

,

muscle neoplasms

,

healthy lifestyle

,

patient compliance

,

treatment refusal

,

surveys and questionnaires

,

,

,

,

polyostotic fibrous dysplasia

,

fibrous dysplasia of bone

,

hip osteoarthritis

,

,

cryopreservation

,

,

,

,

urinary tract infections

,

ureaplasma infections

,

,

lichen sclerosus et atrophicus

,

,

,

anxiety disorders

,

,

,

cardiovascular abnormalities

,

athletic injuries

,

,

preoperative exercise

,

,

physiological sexual dysfunction

,

cognitive behavioral therapy

,

pulmonary hypertension

,

vascular malformations

,

hereditary angioedemas

,

,

,

von hippel-lindau disease

,

hepatolenticular degeneration

,

,

,

,

demyelinating diseases

,

immunoglobulin g4-related disease

,

gaucher disease

,

neurocutaneous syndromes

,

,

,

liver transplantation

,

schizophrenia spectrum and other psychotic disorders

,

,

prenatal exposure delayed effects

,

prenatal diagnosis

,

bladder exstrophy

,

,

,

mixed gonadal dysgenesis

,

paraproteinemias

,

monoclonal gammopathy of undetermined significance

,

,

,

pancreatic intraductal neoplasms

,

pancreatic cyst

,

biotinidase deficiency

,

stem cell transplantation

,

,

uterine hemorrhage

,

,

caloric restriction

,

nutrition therapy

,

,

,

urogenital neoplasms

,

,

Immunoglubulins

,

familial amyloid neuropathies

,

healthy volunteers/patients

,

hepatic insufficiency

,

healthy volunteers/patients

,

aortic valve stenosis

,

pathologic constriction

,

cardiovascular diseases

,

hyperlipidemias

,

hyperlipoproteinemias

,

atherosclerosis

,

myocardial infarction

,

acute coronary syndrome

,

,

,

,

genetic polymorphism

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