Medicine is a constantly evolving field, with discoveries and treatments being developed all the time. However, these new medications come at a cost – both money and time. It can often take years for a new medication to go through all the necessary trials and be approved for use. And even when it is finally released, the price tag can be astronomical. Doctors consider many factors when prescribing a medication, but the cost is often the deciding factor for patients. Here are 10 of the most expensive medications in the world, along with a brief explanation of what they are used for:
10. Soliris® (eculizumab) – $678,392 – Alexion Pharmaceuticals
We start our list with Soliris, a medication used to treat two ultra-rare blood disorders: paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). It was the first medication approved to reduce hemolysis in patients with PNH and is approved for use in PNH patients in nearly 50 countries worldwide. The drug is also approved for the treatment of aHUS in more than 40 countries. It’s been shown to reduce hemolysis, improve renal function in patients with aHUS, and reduce transfusion requirements in PNH patients. Clinical trials’ most common side effects were headache, nasopharyngitis, back pain, and nausea.
9. Ravicti® (glycerol phenylbutyrate) – $695,970 – Horizon Therapeutics
Next up is a somewhat different medication: Ravicti, which treats adults and children (ages two and up) with urea cycle disorders (UCDs). UCDs are a group of rare inherited disorders that affect the liver’s ability to remove ammonia from the blood. Ammonia is a toxic byproduct of protein metabolism that can build up to dangerous levels in the blood if left untreated. Ravicti is a liquid medication taken by mouth, and it works by reducing the amount of ammonia in the blood. It’s FDA-approved for use with other UCD treatments, such as diet and medication adjustments.
8. Blincyto® (blinatumomab) – $712,672 – Amgen
Blincyto is a medication used to treat acute lymphoblastic leukemia (ALL). It is a targeted therapy that works by helping the body’s immune system kill cancer cells. It is approved for use in patients aged 16 and older who have relapsed or are refractory to other ALL treatments. According to the manufacturer’s website, Blincyto effectively induces remission in patients with relapsed or refractory ALL, and it is the only targeted therapy approved for this indication. It’s available as an injection and is typically administered in a hospital setting.
7. Brineura (cerliponase alfa) – $730,340 – BioMarin Pharmaceuticals
Brineura is a medication used to treat Batten disease’s rare and debilitating illness. It is a neurodegenerative disorder typically manifests in childhood and leads to progressive neurological decline and death. Brineura is the first and only treatment approved by the FDA for patients with late infantile Batten disease. It is administered as an infusion into the cerebrospinal fluid and must be given every two weeks. It’s administered through intraventricular injection, infusing it directly into the brain’s fluid-filled ventricles.
6. Folotyn (pralatrexate) – $793,870 – Acrotech Biopharma
Another high-priced medication is Folotyn, a medication used to treat peripheral T-cell lymphoma (PTCL). It is cancer that starts in the white blood cells known as T-cells. Folotyn is the first and only FDA-approved treatment for PTCL and is indicated for patients who have relapsed or are refractory to other treatments. It’s administered as an intravenous infusion and must be given once weekly for six weeks, followed by one week of rest in a seven-week cycle.
5. Luxturna (voretigene neparvovec-rzyl) – $850,000 – Spark Therapeutics
Luxturna is a novel gene therapy used to treat patients with an inherited vision loss called Leber congenital amaurosis (LCA). It is caused by mutations in a gene called RPE65, responsible for producing a protein needed for vision. Luxturna is the first and only FDA-approved gene therapy for LCA and is indicated for patients aged 12 months and older. It is administered as an infusion into the eye and must be given every three to six months. Luxturna is available only through a particular restricted program called the Luxturna Infusion Therapy Program.
4. Myalept (metreleptin) – $889,904 – Aegerion Pharmaceuticals
Next on our list is Myalept, a medication used to treat leptin deficiency in patients. Leptin is a protein hormone that plays a vital role in regulating energy intake and expenditure. In patients with leptin deficiency, Myalept can help to improve energy balance and reduce the risk of obesity and other related health problems. It is estimated that the annual cost of Myalept treatment is around $889,904, which can even go up to $1 million in some cases. The high price of this medication is due to its rarity and that it is only available through specialized clinics. Myalept is administered via subcutaneous injection and is typically given once daily. It is important to note that Myalept is not a cure for leptin deficiency, but it can help to improve the quality of life for patients who are affected by this condition.
3. Danyelza (daratumumab) – $977,664 – Y-mAbs Therapeutics
Danyelza, a monoclonal antibody used to treat patients with multiple myeloma, is the third most expensive medication globally. It is a targeted therapy that works by binding to a protein called CD38, which is found on the surface of myeloma cells. This helps to kill the cancer cells. It’s primarily used in patients who have tried other treatments that haven’t worked. Danyelza is administered as an infusion, and the average cost per infusion is $32,424.
2. Zokinvy (lonafarnib) – $1,032,480 – Eiger BioPharmaceuticals
Zokinvy is a treatment for Hutchinson-Gilford Progeria Syndrome (HGPS), a rare, fatal genetic disorder that causes premature aging in children. A mutation in the LMNA gene causes it. Zokinvy is the first and only FDA-approved treatment for HGPS and is indicated for patients aged two years and older. It is administered as a once-daily oral suspension.
1. Zolgensma (onasemnogene abeparvovec-xioi) – $2,125,000 – AveXis
Zolgensma is a one-time gene therapy for children under two years old who have spinal muscular atrophy (SMA) Type 1, the most severe form of the disease. SMA is a rare motor neuron disease that causes muscle weakness and progressive paralysis. Zolgensma is the most expensive medication globally, with a one-time treatment cost of $2.125 million. According to the manufacturer’s website, it is administered as an infusion and must be given through a vein in the arm. Zolgensma is available only through a particular restricted distribution program.
The world’s most expensive medications are mainly used to treat rare and severe conditions. They are often life-saving or life-changing treatments that can improve the quality of life for patients affected by these conditions. However, the high cost of these medications can be a barrier for many patients who need them. But considering the benefits that these treatments can provide, many patients and families are willing to pay the high costs.