The new rare disease drug ultragenyx monoclonal antibody was approved

Source: there are new drugs for rare and chronic progressive musculoskeletal diseases in jianzhiihealth2018-02-26 The monoclonal antibody burosumab, which is jointly developed by Japan Pharmaceutical Enterprise Association, Kirin and ultragenyx, has recently been approved conditionally by the European Union This is the first new drug approved for the treatment of X-linked hypophosphatemia (XLH) Burosumab can solve the activity of fibroblast growth factor 23 (FGF23) in XLH and is approved for use in children aged 1 and over This McAb is marketed under the name crysvita Xhl affects not only children but also adults XLH is a rare chronic progressive musculoskeletal disease The cause of the disease is the excessive production of FGF23, which leads to the consumption of renal phosphate, leading to X-linked dominant characteristics in men and women XLH first appears in infants and affects adults In children, XLH causes skeletal lesions, resulting in lower extremity deformities and short stature XLH greatly affects the life of patients Routine treatment includes multiple daily doses of phosphate and active vitamin D to counteract the excess effect of FGF23, but does not treat the underlying cause: excessive production of FGF23 This EU approval provides the first treatment option for XLH in children The company said it would strive for the approval to benefit more patients "Through this mandate and our efforts elsewhere in the world, we are committed to bringing crysvita to all XLH patients to benefit from their treatment." The EU approval will be valid in 28 member states The new drug is expected to take the lead in Germany in April this year Then, it is listed in other countries "We will now focus on working with the health sector to ensure that patients in European countries have access to the drug We will also continue to work with healthcare and patient communities to further increase our understanding of real data for people affected by XLH This will help to identify and diagnose correctly and improve the standard of treatment for this rare disease " --Tom Stratford, President and CEO of union Kirin international in the United States, and the U.S Food and Drug Administration (FDA) are also reviewing crysvita's biopharmaceutical license application for the treatment of children and adults with XLH It is expected that the review results will be on April 17 Although the FDA is generally ahead of Europe in new drug approval, the EU is often ahead of the United States in approving new drugs for rare diseases and biological analogues In addition, for another disease, the clinical trial of TiO is also in progress TiO is a benign tumor characterized by excessive FGF23, resulting in severe osteomalacia, fractures, bone and muscle pain, and muscle weakness Ultragenyx focuses on the research and development of new drugs for rare diseases The company was founded in 2010 The NASDAQ listed company's code is rare But in the past few months, the company's share price has been low After the latest EU approval, the share price climbed slightly Company press release: https://