Last Updated: December 2024
Unsurprisingly, health care is a big-money industry in the U.S. Prescription medications and associated costs can be expensive. Many times, these are a necessity but are difficult to afford.
In 2021, it was estimated that 18 million Americans (7%) could not pay for at least one medicine prescribed to their household. That figure increased to 20% for low-income households earning less than $24,000 a year. Ten percent of adults confessed to skipping doses of medication to save money on their prescriptions.
For some, there may be no hope at all. Especially if they need one of the ten most expensive medications in the U.S. They can be filled at a pharmacy or administered in the hospital.
- Zolgensma: $2,125,000
- Zokinvy: $1,032,480
- Danyelza: $977,664
- Myalept: $889,904
- Luxturna: $850,000
- Folotyn: $817,865
- Brineura: $730,340
- Blincyto: $712,672
- Ravicti: $695,970
- Soliris: $678,392
The prices are not the customer price but the list price of the medications. They are based on how much it costs for the usual course of therapy in one year.
1. Zolgensma
Zolgensma, manufactured by Novartis Gene Therapies, Inc., is a gene therapy medication. It is given to children under two years old to treat spinal muscular atrophy (SMA). It is infused into the vein as a one-time treatment.
Spinal Muscular Atrophy is a genetic disease. It steadily ruins motor neurons. These are nerve cells in the spinal cord and the brainstem. They control skeletal muscle activity. Muscle weakness and atrophy result when these cells get ruined.
Zolgensma is the first FDA-approved gene therapy to treat SMA. It has not been tested for its effectiveness in treating advanced SMA. No studies show that more than one dose is effective.
Zolgensma is a biologic drug. This means it is made from living cells and can’t be exactly copied. Because of this, there isn’t a generic or biosimilar version available.
Other drugs can treat your child’s SMA. Two of those are:
2. Zokinvy
Zokinvy is a medication used to treat Hutchinson-Gilford progeria syndrome. It treats processing-deficient Progeroid Laminopathies. They can occur when the body produces progerin and progerin-like proteins.
Hutchinson-Gilford progeria syndrome (HGPS), or progeria, is a rare genetic disease. It is often called premature aging syndrome because of its features. A child with progeria appears normal when they are an infant. Between 9-24 months, they will start showing signs of the disease. That can include the following:
- Delays in growth
- Low weight and short height
- A small face compared to their head
- Their jaw will not be developed fully, causing malformed and crowded teeth
- Abnormally prominent eyes
- Small nose
- Blue around the mouth (subtle)
The characteristics progress from there as the child ages. On average, children with progeria die by age 15 from heart disease.
Zokinvy is the first treatment approved by the FDA to treat HGPS. It treats some harmful proteins and helps reduce the risk of death due to the disease. This treatment is for children who are aged one and older. It is an oral medication that helps prevent progerin or progerin-like proteins from getting built up.
3. Danyelza
Danyelza is a medication that treats children (1 and older) and adults with high-risk neuroblastoma. Neuroblastomas are a type of cancer usually found in the adrenal glands, which rest on top of the kidneys. Danyelza is approved to treat a neuroblastoma that has:
- Relapsed (come back) or is refractory (unresponsive to previous treatment) and
- Shown some response or stabilized to prior therapy
Neuroblastoma is a type of cancer that starts in nerve cells. Neuro refers to nerves, and blastoma refers to cancer in developing cells. These cells are very early in their development and are often in an embryo or fetus. Therefore, this cancer is commonly found in infants and children. Most neuroblastomas start in the abdomen.
Danyelza is a type of immunotherapy. It is used in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF). This combination targets the tumor cells and helps the immune system fight off cancer.
4. Myalept
Myalept is a hormone replacement medication for people with generalized lipodystrophy (GL). Leptin is a naturally occurring hormone that helps your body control some of the metabolic processes. Myalept is the only leptin replacement therapy approved by the FDA.
Generalized lipodystrophy (GL) is a rare metabolic disease. It is a serious and progressive condition. Someone with GL has a permanent lack or loss of adipose (used to store fat) tissue in their whole body. The result is a leptin deficiency. They may experience metabolic abnormalities that can put them at risk for complications that could be fatal.
Myalept helps lower triglyceride, blood sugar, and A1C levels, usually above normal in someone with GL. When used along with a diet recommended by a doctor, Myalept replaces leptin. This can treat the problems caused by leptin deficiency.
5. Luxturna
Luxturna is a type of gene therapy medication. It is FDA-approved to treat vision loss from retinal dystrophy associated with the biallelic RPE65 gene mutation.
Inherited retinal dystrophies are a group of disorders of the eye. They cause different parts of the retina to degenerate. Different genes may be responsible for degeneration, but Luxturna is for use with specific mutations. That is the mutations to both of the RPE65 genes.
Luxturna is available as a subretinal injection to anyone 12 months and older with confirmed mutations in both genes. This medication gives the retinal cells a working copy of the RPE65 gene. From here, those cells can make the protein needed. The visual cycle can continue and light can be changed into signals for the brain to interpret.
6. Folotyn
Folotyn is used for patients who have relapsed or refractory peripheral T-cell lymphoma. This means that with other types of cancer treatment, it has not gone into remission, worsened, or returned.
Peripheral T-cell lymphomas (PTCLs) are a group of diseases that are uncommon and aggressive. These cancers are part of non-Hodgkin lymphomas. The T-cells (mature white blood cells) in the body turn cancerous. There are three categories of PTCLs. Those are nodal, extranodal, and leukemia. These cancers can occur at any point in life but usually affect those older than 60.
Folotyn is an FDA-approved chemotherapy drug given by IV injection. It is used alongside folic acid and vitamin B12 supplements. Folotyn attacks cells in specific phases of the cell cycle. It works by stopping cancer cell division, causing them to die.
7. Brineura
Brineura is an injectable medication used in children aged 3 and older with late infantile neuronal ceroid lipofuscinosis type 2. It slows the loss of ambulation in children with this disease.
Neuronal ceroid lipofuscinosis 2 (CLN2) is a nervous system disorder. The symptoms usually start to appear between ages 2 and 4. It typically starts with recurring seizures and difficulty controlling body movements. Vision loss and muscle twitches can soon follow. CLN2 causes the following:
- An effect on motor skills: walking, sitting, speech
- Developmental regression: loss of skills that were previously learned
- An intellectual disability that gets worse over time
- Behavioral problems
Most children with CLN2 have to use a wheelchair by late childhood and do not live past their teen years.
Brineura is an enzyme replacement therapy infused into the cerebrospinal fluid (CSF) through a port. The active ingredient, cerliponase alfa, is a form of human TPP1. This enzyme is found to be deficient in CLN2 patients. The infusion is administered once every two weeks and is followed by electrolytes. The whole process lasts about four and a half hours.
8. Blincyto
Blincyto is an immunotherapy drug. It is used to treat CD19-positive B-cell precursor acute lymphoblastic leukemia. To receive this medicine, the patient will be in the first or second remission and have a minimal residual disease equal to or greater than 0.1%. This is for adults and children.
Acute lymphoblastic leukemia (ALL) is a blood and bone marrow cancer. It is the most common cancer in children and adults. ALL causes too many B-cell lymphoblasts (immature white blood cells). When your body makes these white blood cells instead of the mature ones it needs, they do not do their job, like fighting infections. They get built up and leave no room for the mature ones to come in. It is a serious type of cancer that progresses quickly, but with the right treatment, remission is a possibility.
Blincyto targets malignant and benign B-cells through the CD19 cell surface antigen. While doing this, it engages the T-cells of the patient through the CD3 antigen. The newly activated T-cells form a synapse between themselves and the malignant B-cells. This allows the T-cells to fight the B-cells and then persist in the bloodstream, fighting multiple cancer cells.
9. Ravicti
Ravicti is a liquid oral medication used for the chronic management of urea cycle disorders. People with this disorder have high levels of ammonia in their blood. Managing this through a low-protein diet or protein supplements alone can be difficult. Ravicti is used with dietary protein restriction and sometimes dietary supplements.
A urea cycle disorder (UCD) is a rare genetic disease. The body is supposed to break proteins that you eat down into amino acids that help keep your body healthy. Amino acids are turned into ammonia by enzymes in the liver and then turned into urea. This is then passed out of the body in the urine. Someone with UCD is either missing those enzymes or isn’t working right. This causes ammonia buildup in the blood, which harms the body. Someone with UCD is either missing those enzymes or isn’t working right. Someone with UCD is either missing those enzymes or isn’t working right.
There are 8 subtypes of UCD. Ravicti treats most types. It is called an ammonia scavenger medicine. Its goal is to help keep the ammonia levels under control. It can be given to children and adults. It cannot treat extremely high levels of ammonia.
10. Soliris
Soliris is a medication given to adults to treat generalized myasthenia gravis and is positive for anti-acetylcholine receptor (AChR) antibodies. It is not known if Soliris is safe for use in children.
Myasthenia gravis (MG) is a progressive autoimmune disorder. It causes weak muscles in the eyes and eyelids. This disease affects more women before 40 and more men after 60.
Generalized myasthenia gravis (gMG) is a more severe disease. This affects the neck, head, limbs, trunk, and respiratory muscles. MG often progresses into gMG.
In people with myasthenia gravis, self-reactive antibodies attack and damage acetylcholine receptors. These are necessary for muscles to contract and move. About 15% of gMG patients do not have these antibodies targeting the receptors. They have self-reactive antibodies that attack vital molecules involved in communication between the nerves and muscles.
Soliris is administered by IV. It takes about 35 minutes for the infusion to be complete. Soliris will only be administered if the patient has had a meningitis vaccine. They also must be under a Soliris Risk Evaluation and Mitigation Strategy (REMS) program because of the risk of meningitis.
Do You Need Help With High-Cost Medications?
Did you know that the U.S. generally has higher-cost medications than other countries? A study done in 2019 showed that Americans pay 24% to 30% more than other countries (in this study).
That’s difficult, especially in today’s economy. Some people have to choose between getting their medicine and feeding their families.
It shouldn’t have to be that way.
We want to help. You may not have to use any of these 10 medications discussed in this article. But that doesn’t mean your prescriptions aren’t costly. Here at The Rx Advocates, we can help most people with those. Call us to find out how.
