Top10, the world's most expensive drug, in 2021

，GoodRx。，GoodRx10，。，。

NO.

，。，11,0001。SMA3，Zolgensma，。2020Evrysdi，（5mg，220kg，qd）,30。

Paying for Zolgensma is not easy, and the insurance company may not be able to afford it; or, the insurance company may have certain requirements to make the patient eligible for coverage.

NO.

Although the dosage depends on the surface area of ​​the body, patients usually take about 200 mg of Zokinvy per day.

NO.

Danyelza has been approved for the market through accelerated approval, and Dituximab with the same target and indication has been fully approved.

NO.

The price of Myalept increased from US$71,306 per month to US$74,159 in February of this year, at an annual cost of nearly US$890,000.

NO.

Fortunately, the competition for hereditary retinal dystrophy is accelerating, and there are also many drugs in or near the late stage of clinical research.

NO.

Folotyn is a dihydrofolate reductase (DHFR) inhibitor and is approved for the treatment of peripheral T-cell lymphoma, with an annual cost of nearly US$800,000.
Folotyn was first listed in the US in 2009 and will also be approved in China in 2020.

DHFR is a key enzyme for the synthesis of thymidine (dTMP), an essential precursor of DNA.
It plays an important role in cell proliferation and is an important target for tumor therapy.
However, the competition for the indications of peripheral T-cell lymphoma is accelerating, and HDAC and CD30 drugs have been added one after another; coupled with the gradual validity period of Folotyn's patent, these may accelerate Folotyn's withdrawal from the TOP list of the most expensive drugs.

NO.
7 Brineura

Brineura is a first-in-class enzyme replacement therapy drug.
It was first launched in 2017 for the treatment of late-stage infant neuronal neurolipid fusion protein type 2 (CLN2) Batten disease (CLN2-Batten).
Batten disease is a common name for a rare and fatal genetic disease of the nervous system.
They are also known as neuronalceroid lipofuscinoses (NCLs).
Due to specific gene defects, the ability of cells to remove metabolite molecules decreases.
.
According to the mutated gene, Batten's disease can be divided into 13 types, called CLN1-8, 10-14.

CLN2 disease is a genetic disease that mainly affects the nervous system.
The symptoms and signs of this condition usually begin between 2 to 4 years of age.
The initial features usually include recurrent seizures (seizures) and difficulty in motor coordination (ataxia).
The affected child also has muscle twitches (myoclonus) and decreased vision.
CLN2 disease affects motor skills, such as sitting and walking, and speech development.
This condition can also lead to loss of previously acquired skills (developmental regression), progressive deterioration of intellectual disability, and behavioral problems.
People with this condition usually need to use a wheelchair in the later stages of childhood, and the general life span will not exceed the teenage years.
Brineura is used to treat the slow decline of walking ability in children aged 3 years and older.

Due to technical problems, whether it is targets or disease considerations, Brineura's competition is seriously insufficient.
In January 2021, the 300mg specification of Brineura was raised again by 2.
14% to 28,090 US dollars, and the recommended dose is 300 mg every 2 weeks, which means that the total cost in a year is 730,000 US dollars.

NO.
8 Blincyto

Blincyto is a CD3/CD19 bispecific antibody developed by Amgen.
It was launched in the United States in 2014 for the treatment of relapsed or refractory CD19-positive B cell precursor acute lymphoblastic leukemia.
Blincyto is administered in cycles.
The first cycle is called the induction phase, which aims to reduce the number of cancer cells; the second to fourth cycles are called the consolidation phase, which helps new healthy cells grow.
At each stage, patients need to use different amounts of drugs, but usually about 168 bottles are used each year.
According to the current pricing, it reaches US$4,242 per bottle, and the annual cost is US$712,672.

Blincyto's sales in 2020 were 379 million U.
S.
dollars, a decrease in growth compared to 2019.
Innovative therapies continue to emerge, and the number of therapeutic drugs available in the field of acute lymphoblastic leukemia is increasing.
Coupled with the competition of small molecule drugs and the back-line label of Blincyto, its market performance in 2021 is still uncertain.
Blincyto will also price in January this year.
An increase of 5.
89% is considered to provide some driving force for subsequent growth.

NO.
9 Ravicti

Ravicti (glycerol phenylbutyrate) is used to treat urea cycle diseases.
It is an upgraded therapy developed by Horizon for its drug sodium phenylbutyrate for the same indication.
Patients need to take 132 bottles each year.
In January 2021, the list price of Ravicti increased by 4.
8% to US$5,273 per bottle.
The annual cost based on the list price was US$690,000.

Disturbance of the urea cycle is a genetic condition that causes high levels of ammonia in the blood.
If left untreated, it can lead to confusion, coma and even death.
Phenylbutyric acid mediates the excretion of nitrogenous waste through the active metabolite phenylacetic acid (PAA).
PAA forms phenylacetylglutamine (PAGN) with glutamine in the body.
Similar to urea, PAGN also contains two nitrogens, which can be transported with urine.
discharge.

As an early product of Horizon, sodium phenylbutyrate is in the recommended daily dose, and the intake of sodium salt exceeds the guideline recommendation.
Ravicti (glycerin phenylbutyrate) not only avoids this problem, but also improves the fat solubility.
In addition, glycerin phenylbutyrate is a light yellow, odorless, and tasteless oily liquid, while sodium phenylbutyrate has problems such as poor sensory (salty, smelly), high sodium content, and large amount of tablets.

NO.
10 Soliris

Soliris costs US$678,392 per year for the treatment of paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, myasthenia gravis, and neuromyelitis optica.
The dosage regimen of Soliris may vary according to the age of the patient and the disease being treated, but most patients require a maintenance dose of 1200 mg every 2 weeks; however, Soliris has a high risk of infection, and many patients have breakthroughs Hemolysis.
Therefore, Alexion (AstraZeneca) has developed a long-acting complement C5 inhibitor Ultomiris (ravulizumab).

Ultomiris (ravulizumab) is the first and only long-acting C5 inhibitor, injected every 8 weeks, and has been approved for the treatment of paroxysmal nocturnal hemoglobinuria and hemolytic uremic syndrome in adults.

Soliris achieved US$4 billion in revenue in 2020, but its growth slowed down (+3%), while the improved Ultomiris expanded rapidly, exceeding US$1 billion with an increase of over 200%, becoming a new blockbuster drug.
It also contributed to AstraZeneca's acquisition of Alexion at a price of 39 billion U.
S.
dollars at the end of 2020.

to sum up

In the TOP10 list of the world's most expensive drugs, in addition to some familiar drugs, such as Myalept, Folotyn, and Soliris, we also saw some new members, including Zokinvy and Danyelza, which were just launched at the end of 2020.
The changes in the TOP list of the world's most expensive drugs have shown progress in science and technology to a certain extent, reflecting that the huge development costs of drugs require higher market pricing to recover, and also reflecting some social problems of high-priced drugs due to insufficient competition.
It is gratifying that most pharmaceutical companies are carrying out corresponding drug rescue and subsidy measures to meet the medication needs of patients as much as possible.
It is hoped that the advancement of technology and the emergence of innovative therapies will continue to meet the needs and availability of medications of patients.