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    Home > Active Ingredient News > Drugs Articles > Those who are at the forefront of orphan drug development: shire, alexion, Baijian

    Those who are at the forefront of orphan drug development: shire, alexion, Baijian

    • Last Update: 2018-09-26
    • Source: Internet
    • Author: User
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    In 1983, the orphan drug act of the United States was implemented, and in 2000, the incentive policy for orphan drugs was launched in Europe Since then, the development of orphan drugs has entered the fast lane, and the global orphan drug market is expanding rapidly From 2008 to 2017, the annual average growth rate is 7.1%; however, according to the latest forecast, the annual average growth rate of orphan drug sales in 2017-2022 will be as high as 11.1%, which is more than twice the average growth rate of prescription drug sales (5.3%) in the same period In 2008, the global sales volume of orphan drugs was 60 billion US dollars, accounting for 10.1% of the total sales volume of prescription drugs It is predicted that this number will reach 209 billion US dollars in 2022, and the proportion of the total sales volume of prescription drugs will increase to 21.4% In recent years, China has also issued a number of policies and regulations to encourage the development of orphan drugs The State Council and the State Food and drug administration have successively issued regulations and Implementation Measures on special examination and approval, special channels, priority evaluation and acceleration of listing of drugs for rare diseases On the one hand, there is a huge market with rapid growth, and on the other hand, there is an intensive release of new policies to encourage the development of orphan drugs in China Therefore, it can be predicted that orphan drugs will usher in a period of rapid development in China in the future Some domestic pharmaceutical companies with sensitive sense of smell have begun to try to develop orphan drugs, but the common problem is how to take the "first step" In the past, the development of orphan drugs in China was a blank field, and there was no experience to learn from But for those who are determined to take root in orphan drugs, looking abroad may be a better choice Next, Xiaobian will introduce some leading orphan drug developers 1 Shire was founded in 1986, originated in the United Kingdom, and is now headquartered in Ireland In the early days of the company, we mainly did research and development of calcium tablets, and the field of treatment was only focused on osteoporosis After the company's operation slowly got on track, the company began to focus on research and development of neurodegenerative diseases, including Alzheimer's disease, renal failure and so on Table 1 the rise of shire in M & A since 2005, shire has made more than 20 acquisitions After the merger and acquisition, shire focuses on the treatment of blood diseases, immune system diseases, genetic diseases, nervous system diseases, gastrointestinal and internal diseases, tumor diseases, eye diseases and other core areas, and now has 40 A variety of products are on the market in the world, and nearly 40 clinical product projects are in progress, more than half of which have been in the phase III clinical or registration stage, and 70% of clinical projects are aimed at rare diseases Figure 1 Distribution of shire in the stage of product pipeline research At present, shire has more than 24000 employees in more than 100 countries and regions around the world With its expanding global influence and first-class products, it helps more patients with rare diseases to meet their medical needs At present, shire has brought three products into the Chinese market, including the only non blood derived third-generation full-length gene recombinant coagulation factor VIII in China, the world's first fully closed infusion system soft bag albumin flexbumin, and a new generation of calcium free phosphorus reducing drug foslino Figure 2 the global distribution of hemophilia in shire, as an important field of shire, has maintained steady growth However, because hemlibra of Genentech has been approved for marketing, this heavyweight drug for hemophilia treatment will strongly impact the hemophilia market, which will have a great impact on shire Another main product of shire, lialda, which is used to treat ulcerative colitis, has been approved by FDA In order to cope with many challenges, shire recently split the business department into two departments, rare diseases and neuroscience department, which operates independently Among them, neuroscience department is based on adderall, expanding to join intuniv, vyvanse, mydayis and other new products The management also lowered the revenue target for 2020 from the original $20 billion to $17-18 billion 2 Alexion pharmaceuticals was founded in 1992 Due to lack of investment in the early stage, alexion pharmaceuticals was on the verge of bankruptcy in 1995 The emergence of soliris, a unique monoclonal antibody fragment, finally saved alexion In 2007, soliris was approved by FDA for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) The disease leads to severe anemia, there is no cure, about one third of patients died within five years Clinical research data have shown that soliris can significantly increase the life expectancy of PNH patients In addition, soliris was approved for the treatment of atypical hemolytic uremic syndrome (aHUS), a severe rare form of kidney disease Soliris's annual sales reach billions of dollars, becoming the main revenue source of alexion pharmaceutical In addition to soliris, alexion has two drugs on the market, namely strensiq ® (asfotase alfa) and kanuma ® (sebelipase alfa) Figure 3 strensiq ® (asfotase alfa), the "three swordsmen" of alexion pharmaceutical, was used to treat hypophosphatase (HPP) and was approved by FDA in 2015 HPP is a kind of hereditary progressive metabolic disease Many systems of patients will be seriously affected, which will eventually lead to debilitating or life-threatening complications It's an extremely rare disease, with fewer than 20 patients in every million people in the general population HPP is characterized by low alkaline phosphatase (ALP) activity and defective bone mineralization, which can lead to bone deformity and other bone abnormalities, as well as systemic complications such as severe myasthenia, spasm, pain, respiratory failure leading to premature death Kanuma ® (sebelipase alfa) is a highly innovative enzyme replacement therapy (ERT) for the treatment of lysosomal acid lipase deficiency (lal-d) Lal-d is a kind of hereditary and progressive metabolic disease, which is caused by gene mutation, resulting in the decrease or loss of LAL enzyme activity in many human tissue lysosomes, resulting in the long-term accumulation of cholesterol esters and triglycerides in the liver, blood vessel wall and other tissues, inducing organ damage, and even early death The incidence rate is below 1/50000 Kanuma ® (sebelipase alfa) showed a significant survival benefit in infants with lal-d (67%, 6 out of 9) over 12 months, compared with 0 in 21 untreated patients In May 2015, alexion pharmaceutical purchased kanuma of synageva biopharma for us $8.4 billion EMA approved kanuma to be listed on August 28, 2015 under accelerated approval; in addition, FDA granted kanuma breakthrough treatment drug qualification, orphan drug qualification, and biological preparation license application priority review qualification, which was approved by FDA in December 2015 In the R & D end, alexion pharmaceutical just develops on previously approved drugs and indications, and does not conduct large-scale R & D layout For example, in several projects in the later stage of R & D, soliris expanded the indications, and the new indications were neuromyelitis optica spectrum disorders (nmosds) In the research and development project codenamed alxn1210 IV, the indications are PNH and aHUS Table 2 R & D layout of alexion pharmaceutical 3 Genzyme Corporation, founded in 1981 and headquartered in Boston, Massachusetts, is one of the top 10 bio pharmaceutical companies established earlier in the world and also the top 20 NASDAQ listed companies It has grown into a diversified enterprise with annual revenue of more than 3 billion US dollars and more than 8000 employees around the world Its products mainly focus on rare genetic diseases, nephropathy, osteoarthritis, immune system diseases and diagnostic tests Jianzan company is still innovating, and continues to research and develop genetic, immune system diseases, heart disease, cancer and other diseases In the middle of February 2011, jianzan company was acquired by Sanofi - Aventis with cash of $74 per share, totaling about $20.1 billion This is the second largest M & a transaction in the history of the global biological industry, with a price of 20.1 billion US dollars equivalent to 4.7 times of jianzan's sales In the early stage of entrepreneurship, the research of jianzan focused on the development of drugs for Gaucher's disease In 1991, the first heavy-duty product of jianzan, Cerezyme, was successfully launched and became the only effective drug for the treatment of Gaucher's disease Cerezyme has brought huge financial resources to jianzan, and new product research and development has entered a virtuous circle, which has also strengthened the research and development strategy of jianzan's orphan drugs Since then, several new drugs have been certified by FDA orphan drugs, such as fabrazyme for treating fabrizyme's disease and Myozyme for treating Pompeii's disease At present, jianzan has as many as 18 drugs on the market, which strongly supports its huge sales In addition, jianzan is actively engaged in R & D layout, covering immune diseases, tumors and rare diseases In the field of rare diseases, jianzan focuses on the distribution of hemophilia, Fabry and other metabolic diseases, Parkinson's and other neurological diseases As shown in Table 3 Table 3 R & D layout of jianzan in the field of rare diseases 4 The full name of Biogen is Biogen idea company, which was formed by the merger of Biogen, a biotechnology company, and ideoc, a pharmaceutical company, in November 2003 Its headquarter is located in Massachusetts, U.S.A Research and development of cancer, psychiatry, dermatology and rheumatism related drugs More than 4000 employees worldwide The current market value is about 22.2 billion US dollars Biogen is known for its innovations in the treatment of neurodegenerative diseases In 2017, it was rated as the most innovative pharmaceutical company in the world As a global leader in the development, production and marketing of innovative therapies, Biogen idea has set new standards for care in oncology, neurology and immunology George scangos, chief executive of Baijian, took office in 2010 At present, he is leading the company to challenge several refractory neurodegenerative diseases, such as Alzheimer's disease (AD), ALS and spinal muscular dystrophy Baijian's leading market product is Biogen's tecfidera ® (dimethyl fumarate), with an annual sales peak of US $3 billion estimated by analysts, occupying a leading position in the market of multiple sclerosis drugs In the future, BJJ will not only be a multiple sclerosis (MS) company, but it is currently conducting phase 1 and phase 2 trials of 13 drugs The clinical trials of the drugs in research are mostly concentrated in phase II, and there is still a certain time to go on the market At present, Baijian has carried out three phase III clinical trials, focusing on Alzheimer's disease, multiple sclerosis and other diseases As shown in Figure 4 Fig 4 R & D pipeline of Baijian in the late clinical stage 5 Conclusion the above four are just "glimpses" of orphan drug enterprises At this glance, we can see the rise of shire in M & A, alexion pharmaceutical, which relies on "unique and unique skills" to win the world, and jianzan and Baijian, which take advantage of R & D to win the world They pursue different development concepts and have different development paths However, they all succeed in "opening up territory" in their respective advantageous fields and rank in the forefront of orphan drug market It is worth noting that most of them were founded in the period of 1980s-2000s, which can be called "young heroes" Chinese pharmaceutical companies have a chance to make a difference in the field of orphan medicine.
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