Prediction of the top 10 best selling orphan drugs in the future

According to the National Institutes of Health (NIH), about 7000 kinds of rare diseases have been confirmed, accounting for 10% of the total human diseases In the United States, 33 million people suffer from rare diseases In Europe, about 6% - 8% of people suffer from rare diseases If we classify these rare diseases into one category, people with rare diseases will no longer be "rare" in reality Rare diseases are usually very serious or even fatal Therefore, the development of orphan drugs is of great significance to patients and society But at first, pharmaceutical companies were not willing to develop drugs to treat rare diseases Until 1983, the United States introduced the orphan drug act Later, Europe, Japan and South Korea also introduced laws and regulations to encourage the development of orphan drugs The current development situation of orphan drugs orphan drugs involve a small number of sick people, but due to less competition, favorable policies and other reasons, the performance of orphan drugs is often good, and R & D enterprises have made huge profits At present, due to the increasing difficulty in the research and development of new drugs, the expiration of drug patents, the impact of many generic drugs, profits are gradually declining At the same time, drug regulatory departments are increasingly strict with drug applications, and the cost of new drug research and development is rising The field of rare disease drugs is a new choice for large pharmaceutical companies In 2017, the development of orphan drugs was very good The growth rate of orphan drugs approved by FDA was 11.3%, while that of non orphan drugs was only 5.3% Some organizations predict that from 2018 to 2024, the annual average growth rate of orphan drugs will be 11%, and the market sales will be US $262 billion; during this period, the average growth rate of the whole global drug market will be 6.4% The top 10 best selling orphan drug in the future ❖ top 1 keytruda ® keytruda ® was developed by MSD company in the United States and approved by FDA in 2014 It is the first PD-1 immunosuppressant in the United States As a new type of tumor therapy, keytruda ® has proved to be effective in the treatment of lung cancer, renal cancer, melanoma, head and neck cancer, bladder cancer, breast cancer, liver cancer, gastric cancer, esophageal cancer, brain glioma, colon cancer, Hodgkin lymphoma and other advanced cancer It is expected to improve the survival of patients substantially, and it has been approved by FDA for first-line NSCLC treatment In July 2018, the U.S FDA awarded keytruda ® a priority review qualification for the treatment of HCC In 2017, keytruda's sales volume was US $3.8 billion It is estimated that by 2024, its sales volume will quadruple to US $12.7 billion, with a compound annual growth rate of 19%, becoming the largest orphan drug sales volume ❖TOP 2 Revlimid®
Revlimid ® is an anti-tumor chemical developed by celgene company in the United States It was approved by FDA in 2005 and marketed under the trade name of Revlimid (Revlimid) for the treatment of myelodysplastic syndrome (MDS) In 2006, FDA approved a new indication of Revlimid ®, that is, the combination of dexamethasone to treat multiple myeloma patients who have received at least one treatment In 2013, FDA approved the combination of dexamethasone to treat multiple myeloma patients Lenalidomide is approved for use in the treatment of mantle cell lymphoma (MCL) patients who still relapse or progress after treatment with two drugs, one of which is bortezomib In 2016, lenalidomide achieved remarkable performance, with sales volume reaching 7 billion US dollars and strong growth momentum By 2017, it will have a strong momentum, with sales volume reaching 8.2 billion US dollars, but it may be weak in the future It is estimated that the sales volume will reach 11.9 billion US dollars in 2024, but the drug has a wide range of indications, which will continue to maintain its dominant position in the future ❖ top3 opdivo ® opdivo ® developed by Bristol Myers Squibb is the first PD-1 / PD-L1 inhibitor and listed in Japan In December 2014, the U.S FDA accelerated the approval of opdivo ® for the treatment of advanced melanoma patients who can not be surgically removed or who have metastasis and have no response to other drugs However, a few months later, keysruda ® of MSD was approved by FDA, and there has been fierce competition between the two drugs ever since Although keytruda ® is a stumbling block to opdivo ® sales performance, opdivo ® is still a new generation of tumor immunotherapy drugs with excellent effect In 2017, the sales volume of opdivo ® was US $570 million, and the sales volume is expected to be US $11.2 billion in 2024, with a compound annual growth rate of 10% ❖ top 4 imbruvica ® imbruvica ® was jointly developed by Alberta and Johnson & Johnson, and was approved to be listed by the US FDA in 2013 Imbruvica ® is the first listed Brunton type tyrosine kinase (Btk) inhibitor The drug is an irreversible inhibitor and plays an anti-cancer role by inhibiting the BTK target required for tumor cell replication and metastasis Imbruvica ® has been approved for several indications, including mantle cell lymphoma (MCL), chronic lymphoblastic leukemia (CLL), chronic lymphoblastic leukemia with del 17p deletion mutation, and megaglobulinemia Fahrenheit (WM), chronic graft-versus-host disease (cGVHD); in February 2018, the U.S FDA granted imbruvica ® the orphan drug qualification for the treatment of gastric cancer (including gastric and esophageal junction adenocarcinoma), and other indications are in progress In 2017, the sales volume of the drug was US $4.5 billion It is estimated that in 2024, its annual sales volume will triple to US $13.5 billion, with a compound annual growth rate of 11.42% ❖ in November 2015, darzalex ® injection of Johnson & Johnson was approved by FDA of the United States Darzalex ® is used to treat patients with multiple myeloma who have received at least three previous treatments Darzalex ® is the first monoclonal antibody injection approved for the treatment of multiple myeloma Multiple myeloma is a kind of blood cancer About 10000 people die of multiple myeloma every year in the United States Due to darzalex's outstanding performance, FDA awarded darzalex breakthrough therapy certification, priority approval qualification and orphan drug certification Recently, the US FDA authorized darzalex ® and bortezomib to be used in the treatment of newly diagnosed multiple myeloma In 2017, its sales volume was US $1.2 billion It is estimated that by 2024, the sales volume of darzalex ® injection will be US $6 billion, with a compound annual growth rate of 25% ❖ top 6 soliris ® soliris ® is developed by alexion company in the United States and approved by FDA in 2007 It is used to treat paroxysmal nocturnal hemoglobinuria (PNH), a rare disease Soliris ® is a human monoclonal antibody against complement protein C5 PNH is a refractory hemolytic disease caused by acquired hematopoietic stem cell gene mutation Complement system is an important part of acquired immune system and plays an important role in the disease Soliris ® can significantly, rapidly and continuously reduce the intravascular hemolysis of PNH patients, so as to improve the anemia symptoms of patients About 50% of patients can get rid of blood transfusion and reduce the amount of red blood cell transfusion Soliris ® can also increase the endogenous red blood cells, stabilize the hemoglobin level, and improve the breathing difficulties, fatigue and other symptoms In October 2017, the US FDA approved a new indication of soliris ® - the treatment of myasthenia gravis In 2017, its sales volume was US $31, and its annual sales volume is expected to be US $5.2 billion in 2024, with a compound annual growth rate of 7% ❖ top 7 hemlibra ® hemlibra ® is developed by Roche company and approved by FDA of the United States on November 16, 2017 Hemlibra ® is a double antibody of fxia and FXa, which is used to prevent, prevent or reduce bleeding events in adult and child patients with hemophilia type A with factor VIII inhibitors Hemlibra ® is the first FDA approved new drug for the treatment of hemophilia A type A with factor VIII inhibitors in the body in recent 20 years, and it is also the only drug that can be injected subcutaneously once a week In June 2018, the U.S FDA granted hemlibra ® priority audit rights to treat hemophilia type A patients without VIII factor inhibitors It is estimated that by 2024, its annual sales volume will be 4.4 billion US dollars, with a compound annual growth rate of 183% ❖ top 8 jakafi ® jakafi ® is jointly developed by Incyte and Novartis, and approved by FDA in 2011 Jakafi ® is a Jak1 and JAK2 kinase inhibitor against bone fibrosis, which is suitable for the treatment of moderate or high-risk bone marrow fibrosis, including primary bone marrow fibrosis, post true erythrocytosis bone marrow fibrosis and primary thrombocytosis bone marrow fibrosis In 2017, its sales volume was US $1.9 billion, and it is estimated that in 2024, its sales volume is US $3.9 billion, with a compound annual growth rate of 11% ❖ top 9, developed jointly by abbvie and Roche, was approved by the US FDA in 2016 As a Bcl-2 inhibitor, venclexta is used to treat patients with 17p deletion mutation (del 17p) and chronic lymphoblastic leukemia (CLL) who have received at least one treatment before The sales volume of the drug in 2017 is US $100 million, and it is estimated that in 2024, the annual sales volume is US $2.8 billion, with a compound annual growth rate of 54% ❖ top 10 epidilex ® epidilex ® is a hemp extract developed by GW pharmaceuticals in the UK In June 2018, the US FDA approved epidilex ® oral preparation for the treatment of epileptic seizures (including rare and serious epileptic diseases such as Lennox Gastaut syndrome and Dravet syndrome) in patients over two years old This is the first time that the U.S FDA has approved cannabis extract and its derivatives It is also the first time that FDA has approved drugs for Dravet syndrome It is estimated that in 2024, the annual sales of the drug will be US $2.3 billion Reference: 1.https:// 2.https:// 3.http:// 4.orphan drug report 2018