10 most expensive drugs in the world

Research and clinical trials can be very time-consuming, sometimes even last for decades before the drug reaches patients, and many substances fail during this process. Adverse and side effects or economic reasons can end the whole research process overnight.

When the number of patients affected by a condition is relatively low, sometimes concerning only hundreds, the financial burden of research reflects on the treatment price: it reaches enormous numbers. This article lists the 10 most expensive treatments per patient in the world.

10. Spinraza

API Nusinersen

The annual cost of treatment is $375,000 ($750,000 in the first year).
Manufacturer: Biogen, an American multinational biotechnology company.

Nusinersen (branded name Spinraza) treats a disease known as Spinal Muscular Atrophy (SMA). SMA occurs due to a mutation on a fifth chromosome (5q) characterized by progressive muscle atrophy that typically begins shortly after birth (although some forms of the disease have late onset). The drug comes in 12mg vials as a solution for injection.

It’s given directly into the spine by specially trained personnel. The first dose is administered after the diagnosis, followed by one vial at 2, 4, and 9 weeks after the first one. After that, a maintenance dose is administered every 4 months for as long as the patient benefits.

9.  Lumizyme

API Alglucosidase alfa

To treat the annual cost of treatment is $520,000 (and can go up to $625,000).
Manufacturer: Sanofi Genzyme, MA, US.

The drug treats Pompe disease (a rare, inherited disorder). The disease is characterized by the buildup of glycogen throughout the body, ultimately leading to minimal impaired function of the affected organs (predominately muscles and liver).

It affects one in every 40,000 persons in the US. The drug is given as an intravenous infusion, 20mg per kilogram of body mass, every two weeks. The drug comes in 50mg vials with a price of $870. 

8. Elaprase

API Idursulfase

The price per vial (6mg) is $4,215, and annual treatment for a child that weighs 35kg is $657,000.
Manufacturer: the Shire, a Biotechnology company from Ireland.

The drug is used for the treatment of a rare inherited disease that affects only males- Hunter syndrome. Due to the inability to breakdown specific metabolic byproducts, they build up throughout the body, which causes walking and breathing difficulties. Without treatment, the symptoms become more severe over time.

The dose depends on the patient’s body weight (0.5mg/kg), and the drug is given weekly through slow intravenous infusion.

7. Brineura

API Cerliponase alfa

The annual cost of treatment reaches $700,000.
Manufacturer: US-based pharmaceutical company BioMarin Pharmaceutical Inc.

Brineura treats CLN2 disease (neuronal ceroid lipofuscinosis type 2 disease), a rare disorder that causes progressive brain damage.

The drug is delivered every two weeks through a special device that runs from outside the skull to the infusion region for treatment (inside the brain). The treatment lasts for as long as it remains beneficial for the patient.

6. Soliris

API Eculizumab

The cost per vial is $6,830, and depending on the indication, the annual cost can reach up to $700,000.
Manufacturer: Alexion Pharmaceuticals Inc. USA

 Eculizumab (brand name Soliris) is a drug used for the treatment of a rare group of diseases that affect red blood cells.

It comes in vials as a solution for intravenous infusion. The dosage depends on the stage of treatment (initial or maintenance) and the patient’s body mass, so it may be administered anywhere from once a week to once every three weeks.

5.  Carbaglu

API Carglumic acid

Manufacturer: Recordati, an Italian pharmaceutical company.

Carbaglu is used to treat patients with elevated ammonia levels in the blood. The indication area comes down to a handful of rare disorders, so the number of patients indicated for this kind of treatment is low. The daily dose ranges from 100 mg to 250 mg per kilogram of body mass.

The price for 5 tablets (each containing 200 mg of active substance) is around $1,040, and depending on the severity of the condition, annual treatment cost can build up to $790,000 (although, typically, it’s closer to half of this price).

4. Ravicti

API Glycerol Phenylbutyrate

Manufacturer: Germany-based pharma company Horizon Pharma GmbH.

The drug treats patients with urea cycle disorders (UCDs). Aside from precisely defined dosage and intake dynamics, patients who suffer UCDs need to adhere to a rigorous dietary regiment.

Ravicti is an oral solution in 25mL vials, costing $5,016 per vial.

The body surface area and the patient’s age determine the dosage, so the annual cost of treatment can reach up to $794,000.

3. Luxturna

API Voretigene neparvovec

It is a one-time treatment with a price tag of $850,000.
Manufacturer: Novartis, Switzerland.

Luxturna treats adults and children with a specific kind of retinal dystrophy (mutations in gene RPE65). The drug is given as an intraocular injection after a few days of preparation (immunosuppressive medicines that reduce the chance of medicine being rejected by the patient’s body). The drug is applied in a highly controllable hospital setting by an experienced eye surgeon.

2. Zokinvy

API Ionafarnib

It is used twice a day with a year’s supply worth more than 1 million dollars – $1,032,480
Manufacturer: Eiger BioPharmaceuticals

Zokinvy is the first and only drug used to decrease mortality risk in Hutchinson-Gilford Progeria Syndrome. Hutchinson-Gilford progeria syndrome (HGPS) is an extremely rare disorder that affects approximately one in 20 million individuals.

It results in young people aging way more quickly than the usual pace, which leads to the death of most patients before the age of 15 years from heart disease. Before the FDA’s approval, supportive care and therapies were the only treatment options. Now, using the drug regularly can help increase patients’ lifespan by up to several years.

1. Zolgensma

API Onasemnogene abeparvovec-xioi

It is a one-time treatment with a price tag of $2,125,000.
Manufacturer: Novartis, Switzerland.

In May 2019, FDA approved new gene therapy for SMA, and with a price tag of $2,125,000, it’s the most expensive drug the world has ever seen. The drug comes in the form of suspension for intravenous infusion and targets children under the age of 2 with SMA, including those not showing the symptoms yet (the diagnosis is made through genetic testing).

It’s a one-time treatment, and this is the argument that justifies such a high price- compared to the only rival on the market- Biogen’s Spinraza, Zolgensma provides a definitive cure for the disease. Considering the clinical research evidence collected so far, it might become a standard treatment for SMA in the future.

References
https://www.drugs.com
http://www.health.gov.on.ca
https://www.ema.europa.eu

I have written this blog to the best of my knowledge and research. Prices and dosages of drugs may differ per each country and are calculated as an average using publicly available data, but may be subject to errors, omissions, or typos. I’m not responsible for any errors and omissions. Any mistake, error, or discrepancy noted may be brought to my attention, and appropriate efforts shall be undertaken to rectify the same to the possible extent.