Glossary of Terms

Acute Myeloid Leukemia (AML)

A disorder marked by an abnormal proliferation of immature myeloid cells. Normal hematopoietic function is impaired by unlimitedly proliferation of leukemia cells in the bone marrow, causing a variety of symptoms that include infectious disease and bleeding.

Alcohol dependence syndrome

Alcohol dependence syndrome is a disease that progresses by continuing heavy consumption of alcohol for a long time, gradually causing you unable to do without alcohol use, and involves withdrawal symptoms including, but not limited to, irritability, anxiety, hand tremors, inability to sleep at night, sweating, and vomiting what you have eaten. The number of patients requiring treatment is estimated to be about 1.09 million.

Antisense oligonucleotide

An antisense oligonucleotide is a single-stranded synthetic nucleic acid consisting of approximately 20 to 30 bases complementary to the target sequence of an mRNA or mRNA precursor, and is a type of oligonucleotide therapeutics. Known oligonucleotide therapeutics include antisenses, siRNAs, miRNAs, aptamers, and decoy oligonucleotides.

Arteriosclerosis obliterans (ASO)

ASO is a vascular disease occurring mainly in the arteries of the legs (lower extremities), in which narrowing or clogging of the arteries reduces blood flow through the legs and causes painful gait disturbances. The number of patients is estimated to be about two million.

Chronic pain

Chronic pain is the pain that persists for several months or longer despite the cure of an illness or injury, or resolution of an obvious physical abnormality, or pain that persists for a long time due to a chronic disease that is difficult to cure.

Chronic Thromboembolic Pulmonary Hypertension (CTEPH)

A disorder whereby organized thrombi cause a occlusion in the artery leading from the heart to the lungs (pulmonary artery), leading to abnormally high blood pressure in the pulmonary artery. Clinical symptoms include shortness of breath during exertion.

Dravet syndrome (DS)

Dravet syndrome(DS) is a rare, devastating and life-long form of epilepsy that generally begins in infancy or early childhood and is marked by frequent treatment-resistant seizures, frequent resulting hospitalizations and medical emergencies, significant developmental and motor and behavioral impairments. In Japan, DS has been designated as an intractable disease by MHLW, and there are estimated to be about 3,000 patients living with DS in Japan, based on MHLW Patient Survey

Duchenne Muscular Dystrophy (DMD)

A hereditary muscular disorder whereby dystrophin gene abnormalities cause a loss of dystrophin proteins, which protect muscle cell membranes. It is the most frequently occurring type of muscular dystrophy, occurring in one of every 3,500 newborn boys. It is identified by symptoms such as a tendency to fall and inability to walk quickly in children aged 2 to 5, with muscular atrophy and muscle strength deterioration following. Patients become unable to walk on their own before their early teens, require the use of a wheelchair, and generally die in their 20s or 30s from respiratory failure or heart failure.


Menstrual pain (painful periods) with a degree of pain interfering with your daily life. There are two types of dysmenorrhoea: functional dysmenorrhea without a causative disease, and organic dysmenorrhoea caused by diseases such as endometriosis, adenomyosis uteri, uterine myoma, and chlamydial infection.


Endometriosis is a benign sickness in which endometrial tissue, which should normally be found inside the uterus, grows outside the uterus.

Endothelin Receptor Antagonist (ERA)

Endothelin, a substance in the body that functions to constrict blood vessels, acts by binding to endothelin receptors. Endothelin is ubiquitously distributed in the body of patients with pulmonary hypertension. ERA is a medication that inhibits the binding of endothelins to endothelin receptors. There are two types of endothelin receptors, A and B. Macitentan and bosentan block both A and B receptors, and ambrisentan blocks A receptors.

Erectile dysfunction (ED)

ED refers not only to cases in which no erection occurs, but also to conditions where no erection sufficient for satisfactory sexual intercourse can be obtained, such as insufficient hardness and an inability to maintain an erection.

European Medicines Agency (EMA)

An organization in charge of procedures, applications, and tests required for marketing approval and clinical trials of pharmaceuticals in Europe.

Exon skipping

Restoring the open reading frame of amino acids via medications that use antisense oligonucleotides to remove (skip) certain parts of the mRNA region (exon) that are translated into proteins. This has the effect of generating functional proteins.

Fast track designation

Fast track designation is a system whereby the U.S. Food and Drug Administration (FDA) performs priority review of the new drugs that are expected to be highly effective against diseases that are difficult to cure completely.

Glutamatergic neural activity

Glutamate is one of the major excitatory neurotransmitters in the brain. Stimulation of nerves in the brain promotes the release of glutamate, and the stimulation is transmitted through glutamate receptors. In alcohol dependence syndrome, increased glutamatergic neuronal activity is considered to create an imbalance between excitatory and inhibitory neurotransmission.

Hepatic sinusoidal obstruction syndrome (SOS)

SOS develops when a large amount of anticancer agents and radiation used in the conditioning regimen for hematopoietic cell transplants cause circulatory disturbances in the liver. SOS is a serious disease whose symptoms include, but not limited to, weight gain, painful hepatomegaly, ascites, and jaundice, and in severe cases, multiple organ failure with a mortality rate of 80%. The number of patients is estimated to be about 400. SOS is also known as hepatic central vein obstruction (VOD).

IP receptor (prostacyclin receptor) agonist

IP receptors function for vasodilation and other activities by binding to prostacyclin (PGI2). This function is weakened in patients with pulmonary hypertension. IP receptor agonists, similarly to PGI2, bind to IP receptors and exhibit vasodilatory effects.

Iron deficiency anaemia

Iron deficiency anaemia is caused when the body is unable to produce sufficient hemoglobin due to lack of iron. Symptoms such as fatigue, headache, palpitations, and shortness of breath appear due to insufficient energy production in cells. The causes are varied and include, but not limited to, heavy menstrual bleeding, postpartum bleeding, haemorrhage of digestive tract, and chronic renal disease. The number of patients is estimated to be about 780,000.


A drug discovery method for therapies such as low-molecular weight compounds, peptide (medium molecule weight) drugs, and nuclear acid drugs.

Myelodysplastic Syndromes (MDS)

Intractable diseases that carry a poor prognosis and very often lead to leukemia. The main symptoms are general fatigue caused by anemia, an increased susceptibility to infections due to a decrease in white blood cells, and bleeding tendency as a result of a decrease in platelet count.

Myelofibrosis (MF)

MF is a disease in which fibrosis is observed in a wide area of the bone marrow. The number of patients in Japan is estimated to be about 1,000. Fibrosing of bone marrow is considered to be caused by the production and release of factors that promote fibroblast proliferation from megakaryocytes, the mother cells of platelets proliferating in the bone marrow.

Non-Hodgkin lymphoma (NHL)

NHL is a malignant lymphoma with swollen lymph nodes, and the mass is characterized by painlessness. The number of patients in Japan is estimated to be about 24,000.

Oligonucleotide therapeutics

Medications which have the structure of nucleic acids, which are the components of genes, and target a gene causative of diseases. Oligonucleotide therapeutics show effect by stopping or regulating the production of proteins generated from the target gene. Oligonucleotide therapeutics are expected to enable treatment of diseases that has been difficult with conventional low molecular medications, and are called next-generation medications for their high specificity and excellence in safety.

Opioid receptor agonist action

Activating opioid receptors to suppress pain transmission by sensory neurons in the spinal cord and to suppress the excitation of pain information transduction pathways in the brain, thereby showing analgesic effects. Typical medications include morphine.

Orphan Drugs

Drugs of high medical necessity, for treating intractable diseases and other conditions suffered by small numbers of patients. Pharmaceuticals designated as orphan drugs are eligible for preferential treatment in the form of priority clinical trial consultation, priority reviews, and re-examination period extensions by the Ministry of Health, Labor and Welfare and the Pharmaceuticals and Medical Devices Agency.

Patient Centricity

This is a concept which involves always putting the patient at the center, providing responses that are focused on the patient, and ultimately respecting the individual judgement of the patient to the maximum extent.

PDE5 inhibitors (phosphodiesterase-5 inhibitors)

An enzyme called PDE5 is ubiquitously distributed in pulmonary blood vessels, and breaks down cyclic GMP (cGMP), a substance in the body that dilates blood vessels. PDE5 inhibitors are medications that inhibit this degradation of cGMP by PDE5, strengthen the action of cGMP, and dilate pulmonary blood vessels.

Priority Review Voucher

A voucher issued by the FDA in the U.S. to incentivize new drug development for rare conditions affecting children (including tropical diseases), priority review vouchers grant the right to have a new drug designated for priority review at the time of application approval. These rights can also be traded.

Product life cycle management

An abbreviation of Product Life Cycle Management. With new pharmaceutical development having become increasingly challenging, this is a means of improving the value of existing products by adding additional indications and dosage forms.

Pulmonary Arterial Hypertension (PAH)

A life-threatening disorder characterized by abnormally high blood pressure in the artery leading from the heart to the lungs. PAH has a variety of symptoms that begin with minor shortness of breath and fatigue during everyday activity, then restricted physical activities, eventually leading to death due to right ventricular failure.

“Sakigake” designation system

“Sakigake” designation system is a system to promote domestic development of innovative medicines to lead the world in practical application of such products in Japan for life-threatening diseases for which there are no effective treatments. The purpose of this system is to shorten the approval and review period in Japan by providing priority in consultations and reviews related to regulatory approval.

Unmet medical needs

Medical needs for which no therapy has yet been established.

Urination impaired associated with prostatic hyperplasia

It is a disease in which the prostate enlarges with age and compresses the urethra, thereby causing difficulty in urinating. It mainly appears in men of age 50 or higher, with diverse symptoms including "weak urine stream," "difficulty in urinating," and "repeatedly going to the toilet at night."

U.S. Food and Drug Administration (FDA)

A government agency under the Department of Health and Human Services (HHS) of the United States. The FDA specializes in administration, such as authorization of products and control of violations, concerning foods and pharmaceuticals which consumers may use in their daily lives.