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    Home > Active Ingredient News > Drugs Articles > Fabojin pamrevlumab was awarded the second fast-track qualification by the FDA for the treatment of Duchenne muscular dystrophy

    Fabojin pamrevlumab was awarded the second fast-track qualification by the FDA for the treatment of Duchenne muscular dystrophy

    • Last Update: 2021-07-14
    • Source: Internet
    • Author: User
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           On April 12, FibroGen, Inc.
    announced that the FDA has granted its connective tissue growth factor (CTGF) antibody drug pamrevlumab fast track qualification for the treatment of Duchenne muscular dystrophy (DMD)

    .

           Duchenne muscular dystrophy (DMD) is a serious, devastating, life-threatening X-linked genetic disease.
    It is caused by changes or mutations in the gene encoding dystrophin.
    Dystrophin maintains the stability of muscle membranes.
    And function required

    .
    With age, the muscle degeneration of DMD patients gradually worsens

    .

           Children with DMD usually begin to show symptoms at the age of 3 to 5 years.
    Muscle weakness can start as early as 3 years old.
    Affected children may experience developmental delays, such as difficulty in walking, climbing stairs, or standing from a sitting position

    .
    As the disease progresses, muscle weakness of the lower limbs spreads to the arms, neck and other parts

    .
    Most patients need to use a wheelchair full-time in their teens, and then gradually lose the ability to carry out activities of daily living independently.
    Eventually, breathing difficulties increased due to respiratory muscle insufficiency require ventilatory support, and cardiac insufficiency can lead to heart failure

    .

           DMD is the most common muscular dystrophy in the world, and patients usually die of the disease in their 20s
    .
    According to statistics, 1 out of 3500-5000 live births of male babies has DMD.
    There are about 140,000 cases of DMD in boys worldwide, and about 30,000 cases in the United States and Europe

    .
    At present, there is no cure for DMD.
    The clinical control of the disease is mainly through drug therapy, physical therapy, gene therapy, and stem cell therapy.
    Among them, gene therapy and stem cell therapy are important directions for DMD treatment in recent years

    .

           Since 2010, 4 DMD small molecule DMD drugs have been approved globally, namely Translarna (Ataluren) of PTC Therepeutics, Exondys 51 (eteplirsen) of AVI BioPharma, Vyondys 53 (Golodirsen) of Sarepta Therapeutics and Viltepso ( viltolarsen)
    .
    Exondys 51, Vyondys 53, and Viltepso are all antisense oligonucleotide therapies.
    Exondys 51 targets DMD patients with a skipping mutation in exon 51 of the dystrophin gene, and Vyondys 53 and Viltepso target a skipping mutation in exon 53.
    DMD patients

    .

           Pamrevlumab is a first-in-class fully humanized monoclonal antibody that targets to inhibit the activity of connective tissue growth factor (CTGF).
    CTGF is the central mediator of tissue remodeling and fibrosis, involving a wide range of fibrosis and proliferation Diseases affect the system of organs throughout the body

    .
    Previously, the drug was granted orphan drug designation for the treatment of idiopathic pulmonary fibrosis (IPF), pancreatic cancer and DMD by the FDA, as well as fast-track designation for the treatment of pancreatic cancer

    .

           This time, Pamrevlumab was awarded the fast track qualification for the treatment of DMD based on the positive results of its Phase II study on the treatment of DMD
    .
    The results of the interim trial published in July 2019 showed that after 52 weeks of treatment, the average left ventricular ejection fraction (LVEF%) of patients in the intent-to-treat group (ITT) increased by 0.
    29% compared to baseline

    .
    In the subgroup of patients with a baseline LVEF% greater than 50%, treatment with pamrevlumab resulted in an average increase of 1.
    79% in this index

    .
    At the same time, pamrevlumab also correspondingly reduced the level of myocardial fibrosis in the patient, and the rate of decline in lung function and the rate of decline in upper arm muscle function was also alleviated

    .

           FibroGen is an advanced American biopharmaceutical company dedicated to using its cutting-edge expertise in the biology and clinical development of hypoxia-inducible factor (HIF) and connective tissue growth factor (CTGF) to discover and develop applications Innovative therapies in the treatment of anemia, fibrosis and cancer
    .
    In addition to pamrevlumab, Fabojin has two drug candidates, FG-5200 and roxadustat.
    FG-5200 is a biosynthetic cornea containing recombinant human collagen developed for the treatment of corneal blindness

    .
    Roxadustat is a pioneering oral small molecule HIF-PH inhibitor.
    It has been approved in China for anemia caused by chronic kidney disease (CKD) that is undergoing dialysis treatment, as well as non-dialysis-dependent chronic Anemia of nephropathy (NDD-CKD)

    .
           

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