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    Home > Medical News > Latest Medical News > The $100 billion rare disease drug market is "attractive", and pharmaceutical companies have stepped up to enter the "enclosure"

    The $100 billion rare disease drug market is "attractive", and pharmaceutical companies have stepped up to enter the "enclosure"

    • Last Update: 2022-05-17
    • Source: Internet
    • Author: User
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    Recently, Be Biopharma, an engineered B cell therapy startup, announced the completion of a $130 million Series B financing.
    The funds raised will be mainly used to build autologous and allogeneic engineered B cell drug technology platforms, as well as IND work for pipelines under development
    .
    It is reported that Be Biopharma has a rich pipeline of research and development, the main indications are rare diseases and tumors, and the company is currently promoting the research projects to clinical research
    .
    In early January this year, AstraZeneca announced that its rare disease company, Alexion, and Neurimmune have reached a cooperation to obtain the latter's treatment of transthyretin amyloid cardiomyopathy (ATTR-CM) with an initial payment of US$30 million and a milestone payment of up to US$730 million.
    ) global exclusive rights to the monoclonal antibody drug NI006
    .
    ATTR-CM is a systemic, progressive and fatal rare disease with an unmet clinical therapeutic need, and NI006 has the potential to treat patients with advanced ATTR-CM
    .
    On January 4, Shufang Pharmaceutical and Swiss Santhera Pharmaceuticals announced that they had reached an exclusive licensing agreement for the new rare disease drug Vamorolone.
    exclusive rights
    .
    Vamorolone targets Duchenne muscular dystrophy (DMD), a rare X-chromosome recessive disorder in which there is a huge clinical need
    .
    There are currently about 70,000 DMD patients in China, and as the diagnosis rate increases, the number of patients may continue to increase
    .
    There are currently no approved drugs for the treatment of DMD in China
    .
    Behind the deployment of many companies in the rare disease drug market, the market in this field is huge
    .
    According to Frost & Sullivan, the global rare disease drug market will increase from US$135.
    1 billion in 2020 to US$383.
    3 billion in 2030, with a compound annual growth rate of 11%
    .
    Up to now, almost all large multinational pharmaceutical companies with top revenue, including Johnson & Johnson, Pfizer, Roche, AbbVie, Novartis, Merck, Bristol-Myers Squibb, GSK, Sanofi, AstraZeneca, etc.
    , as well as many domestic pharmaceutical companies All have active deployments in the field of rare diseases
    .
    Judging from the layout actions of major pharmaceutical companies, they mainly focus on the acquisition, expansion of the rare disease drug R&D pipeline, and authorization to introduce the rare disease drug market
    .
    Acquisition is a relatively quick way to enter the market, and it is also a measure taken by many multinational pharmaceutical companies
    .
    For example, in 2019, Pfizer completed the acquisition of the rare disease company Therachon Holding AG and obtained the latter's rare disease drug TA-46; in February 2019, Roche acquired the gene therapy pioneer Spark Therapeutics for US$4.
    3 billion and obtained the latter's FDA orphan Luxturna, a gene drug qualified for drug identification, expands its presence in the field of rare diseases by deploying gene therapy; in December 2020, Novartis announced the acquisition of neuroscience drug developer Cadent Therapeutics for $770 million, and obtained the latter for schizophrenia The rare disease product CAD-9303 and the product CAD-1883 for movement disorders (such as cerebellar ataxia); in September 2021, Merck announced the acquisition of Accelron for $11.
    5 billion to enter the field of rare diseases, and announced the acquisition 3 months later Chord Therapeutics, a drug research and development company focusing on rare neuroinflammatory diseases, has further strengthened its layout in the field of rare diseases
    .
    From the perspective of the industry, there is a huge unmet clinical need in the rare disease market, both domestically and overseas
    .
    In recent years, a series of favorable policies have encouraged and supported the research and development of rare disease drugs, and the pace of pharmaceutical companies’ deployment has become more vigorous.
    It is believed that the rare disease market will usher in rapid development, bringing more treatment options to patients
    .
    Disclaimer: Under no circumstances does the information or opinions expressed in this article constitute investment advice to anyone
    .
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