It is used for the treatment of PNH and atypical hemolytic uremic syndrome
10. Soliris (for the treatment of PNH and atypical hemolytic uremic syndrome): worth US$290,000 (about 2.07 million RMB) per year
Soliris is a monoclonal antibody drug used for the treatment of PNH (acquired aplastic anemia) and atypical hemolytic uremic syndrome (aHUS). It costs up to $290,000 a year to treat, making it one of the top 10 most expensive drugs in the United States.
PNH is a very rare autoimmune disease characterized by a lack of coverage of the protective proteins CD55 and CD59 in some red blood cells, which are easily recognized and destroyed by the complement system, leading to hemolytic anemia and complications such as thrombosis. Soliris specifically binds to complement component C5 and blocks the complement activation pathway, thereby preventing complement-mediated lysis of red blood cells.
aHUS is also a rare genetic disorder caused by aberrant complement regulation caused by a series of genetic mutations. This can lead to excessive lysis of red blood cells and platelets, which can eventually impair kidney function. As a highly selective complement C5 inhibitor, Soliris prevents this lysis process.
Soliris has been approved by the FDA for the treatment of PNH and aHUS, and clinical trials have shown that it significantly reduces the frequency and severity of hemolytic episodes, reduces transfusion dependence, and helps avoid kidney failure. Despite its high price, there are currently no alternative treatment options. Considering the rarity of the two diseases mentioned above and the unique efficacy of Soliris, its high price is also somewhat understandable.
So, despite its costing of $290,000 per year, Soliris offers important and unique treatment options for two rare and life-threatening diseases, PNH and aHUS.
Enzyme replacement therapy for the treatment of Pompe disease
9. Myozyme (enzyme replacement therapy for Pompe disease): worth US$300,000 (about 2.14 million RMB) per year
Myozyme, an enzyme replacement therapy drug used to treat Pompe disease, costs up to $300,000 a year and ranks among the top 10 most expensive drugs in the United States.
Pompe disease is a rare inherited metabolic defect caused by the loss or loss of activity of the acidic α-1,4-glucosidase enzyme. The main function of this enzyme is to break down glycogen, and its absence causes glycogen to build up within the body's muscles and cellular tissues, causing progressive muscle weakness and cardiomyopathy.
Myozyme is a recombinant acidic α-1,4-glucosidase enzyme preparation. With regular infusions, it is able to replace the missing enzymes in the patient's body, thereby degrading and removing glycogen build-up. Clinical studies have shown that Myozyme treatment can improve the structure and function of the heart muscle, delay the progression of the disease, and reduce the mortality rate.
Although Myozyme is quite expensive to treat each year, it is currently the only enzyme approved by the FDA for the treatment of Pompe disease due to its rarity and severity of the condition.
Drugs used to treat peripheral T-cell lymphoma
8. Folotyn (drug for the treatment of peripheral T-cell lymphoma): worth US$320,000 (about 2.28 million RMB) per year
Folotyn, a drug used to treat relapsed or refractory peripheral T-cell lymphoma (PTCL), has an annual cost of up to $320,000 and is among the highest in the U.S. drug price list.
PTCL is a relatively rare group of non-Hodgkin lymphomas that arise from mature T lymphocytes. It has an aggressive disease process and a poor prognosis, with a low response rate to initial chemotherapy and a susceptibility to relapse. Folotyn (pangulastatin) is an antineoplastic antibiotic used primarily to treat relapsed or refractory PTCL.
Clinical trials have shown that Folotyn treatment significantly improves the overall response rate and complete response rate of patients with PTCL and prolongs progression-free survival compared with traditional chemotherapy. This opens up new treatment options for patients with PTCL with refractory disease.
Considering the rarity of PTCL and Folotyn's unique clinical benefits, its high annual treatment costs are understandable. Therefore, Folotyn has undoubtedly broadened the treatment options for PTCL and brought hope to patients.
Drugs used to prevent perioperative angioedema
7. Cinryze (a drug used to prevent perioperative angioedema): worth US$350,000 (about 2.49 million RMB) per year
Cinryze is a drug used to prevent and treat episodes of angioedema, mainly for hereditary angioedema. Its annual treatment costs are as high as $350,000, and it is at the top of the list of drug prices in the United States.
Angioedema is a fluid-filled swelling that appears in the deep tissues of the body, usually on the face and extremities. Hereditary angioedema is a rare autoimmune disorder characterized by recurrent swelling of body tissues due to a deficiency of complement C1 enzyme inhibitors (C1-INH).
Cinryze contains a concentrate of C1-INH protein, which is effective in preventing and treating acute attacks of hereditary angioedema. Clinical trials have shown that Cinryze significantly reduces the frequency and severity of attacks compared to placebo. It provides an important treatment option for the prevention and control of the disease.
Considering the rarity of the disease and the risk of flare-up, the high price of Cinryze is also expected. Therefore, Cinryze has undoubtedly greatly improved the quality of life of patients with angioedema.
Enzyme replacement therapy for the treatment of macavia
6. Naglazyme (enzyme replacement therapy for macavia): worth US$365,000 (about 2.6 million RMB) per year
Naglazyme, an enzyme replacement therapy drug used to treat macave's disease (MPS VI), costs up to $365,000 per year, ranking among the top 10 drug prices in the United States.
Makavid disease is a rarer form of inherited mucopolysaccharidosis. The disease is caused by a deficiency in a lysosomal enzyme called N-acetylgalactonine sulfatase, which causes the body to accumulate mucopolysaccharides that cannot be broken down properly, which in turn impairs cell, tissue, and organ function.
Naglazyme is an injectable formulation of recombinant human N-acetylgalactonine sulfarase. It can be administered intramuscularly or intravenously into the patient's cells to replace the defective lysosomal enzymes, thereby effectively degrading the mucopolysaccharide accumulation in the cells. Clinical trials have found that compared with placebo, Naglazyme treatment can significantly improve mobility, growth and development, and effectively control disease progression.
Given the rarity of macaveta and the unique efficacy of Naglazyme, the high annual cost of treatment is understandable. Overall, it provides an important treatment option for this rare disease, saving many young lives.
Enzyme replacement therapy for the treatment of Hunter's syndrome
5. Elaprase (enzyme replacement therapy for Hunter's syndrome): worth US$375,000 (about 2.67 million RMB) per year
Elaprase, an enzyme replacement therapy drug used to treat Hunter's syndrome, ranks among the top 10 most expensive drugs in the United States with an annual cost of $375,000.
Hunter's syndrome is a rare inherited lysosomal storage disorder. The disease is caused by a defect in the gene for the enzyme sulfazine (IDS), which prevents the body from effectively breaking down proteoglycans and glycopeptides, allowing them to accumulate within cells and disrupt normal structure and function. The main clinical manifestations include facial deformity, mental retardation, hepatosplenomegaly, and skeletal deformities.
The active ingredient in Elaprase is recombinant human ildomine sulfatase. Through intravenous infusion, it can enter the cells and organelles in the patient's body to replace the enzyme deficiency and cannot perform normal functioning of the ulosine sulfatase, effectively degrading the proteoglycan and glycopeptide accumulation in the body, and avoiding further damage to cells and tissues.
Given the rarity of the disease and the unique efficacy of Elaprase, the high annual cost of treatment is understandable. As a result, it presents an important treatment option for patients with Hunter's syndrome.
Drugs used to treat chronic myeloid leukemia
4. Actimmune (drug for the treatment of chronic myeloid leukemia): worth US$520,000 (about 3.71 million RMB) per year
Actimmune is a recombinant interferon γ-1b formulation used to treat patients with chronic myeloid leukemia (CML) who are unable to tolerate other therapies. Its annual cost of treatment is as high as $520,000, and it ranks among the top drug prices in the United States.
CML is a myeloid leukemia that originates from hematopoietic stem cells, and is characterized by the continuous proliferation of leukemia cells, crowding out normal blood cells. Interferon γ-1b in Actimmune inhibits the abnormal proliferation of CML cells by activating the patient's immune system.
Clinical trials have shown that for CML patients who have failed treatment or are intolerant to other therapies, Actimmune treatment can improve the hematological and cytogenetic response to a certain extent, control the growth of leukemia cells, and prolong the survival of patients. Given the higher incidence of CML and the unique benefits of Actimmune, its higher annual cost of treatment is also reasonable.
Medications used to treat spinal muscular atrophy
3. Spinraza (a drug used to treat spinal muscular atrophy): worth US$750,000 (about 5.35 million RMB) per year
Spinraza, a drug used to treat type 5 spinal muscular atrophy (SMA), costs up to $750,000 per year and is among the highest drug prices in the United States.
SMA is a group of neurological disorders caused by deletion or mutation of the SMN1 gene, which are characterized by the gradual loss of motor neurons and muscle weakness. The active ingredient in Spinraza is the ribonucleic acid analogue nusinersen, which can specifically bind to the SMN2 gene and increase the expression of SMN protein, thereby protecting motor neurons and improving muscle function.
Clinical trials have shown that Spinraza treatment can significantly improve motor function in children with SMA, helping them to achieve significant benefits in survival and development. Given the severity of the disease and the unique efficacy of Spinraza, the high annual cost of treatment is also acceptable.
Gene therapy for the treatment of an inherited retinal degenerative disease
2. Luxturna (gene therapy for the treatment of an inherited retinal degenerative disease): US$850,000 (about 6.07 million RMB) per dose
Luxturna is a gene therapy drug targeting the RPE65 gene for the treatment of rare secondary retinitis pigmentosa, costing up to $850,000 per course of treatment, ranking second in the U.S. drug price rankings.
The disease is caused by the gradual degeneration of retinal pigment epithelial cells caused by mutations in the RPE65 gene, and its main clinical manifestations are night blindness and gradual loss of peripheral vision. Luxturna contains recombinant RPE65 gene vector biomaterial that can be surgically injected into the vitreous cavity of the affected eye and delivered into retinal cells to achieve alternative expression of the normal RPE65 gene, thereby halting disease progression and partially restoring visual function.
Clinical trials have shown significant improvements in visual function and light perception at night and in dim light in patients treated with Luxturna compared to the control group. Considering the rarity of the disease and the revolutionary efficacy of Luxturna, its high cost per treatment is understandable. Therefore, this gene therapy brings great efficacy and hope to patients with secondary retinitis pigmentosa.
Gene therapy drugs used to treat spinal muscular atrophy
1. Zolgensma (gene therapy drug for the treatment of spinal muscular atrophy): US$2.25 million (about 16.06 million RMB) per dose
Zolgensma, a gene therapy drug used to treat spinal muscular atrophy type 5q (SMA), costs up to $2.25 million per course of treatment, making it currently the most expensive drug in the United States.
SMA is a rare genetic disorder caused by the deletion or inactivation of the SMN1 gene, characterized by the progressive loss of motor neurons and muscle dysfunction. Zolgensma, as a gene therapy targeting the SMN1 gene, contains an in vitro transcribed adenoviral vector that normally expresses the SMN protein. Through one-time intramuscular administration, it can effectively compensate for the functional defects of the SMN1 gene in patients, thereby significantly improving muscle function and survival.
Clinical trials have shown that most infants with SMA can sit and walk independently within 14 months of age after receiving a single injection of Zolgensma, which is significantly higher than that of the untreated group. Considering the severity of the disease and the huge potential of gene therapy, Zolgensma's high cost can be considered reasonable. Therefore, Zolgensma has undoubtedly opened a new era of SMA treatment and saved generations of children.