Clinically in urgent need of new drugs from abroad! Broshu single anti-listing application in China enters "in approval"

China's State Drug Administration (NMPA) drug registration progress inquiry results of the latest public announcement, Concorde Fermentation Kirin (China) Pharmaceuticals in China submitted a rare disease of the new drug Burosumab three listing application status has been updated to: in the approval.
means the new drug is a step closer to approval in China.
Broschu monotherapy is the first FDA-approved drug approved for the treatment of X-series hypophosphate (XLH) in children and adults 1 year of age and older, and has been recognized by the FDA as a breakthrough therapy and orphan drug.
in China, the drug has been included in the priority review on the grounds of the treatment of rare diseases for FGF23-related hypophosphateemia, and it is also a member of the second batch of clinically urgent need for new drugs from abroad.
screenshot source: NMPA website XLH is a serious rare genetic disease, but also the most common hyalphthalmic phosphate rickets.
most children with XLH experience bending legs, short stature, bone pain and severe toothache.
XLH patients may also experience persistent discomfort or complications such as joint pain, impaired activity, tooth abscesses, and hearing loss.
patients with XLH, effective treatment is urgently needed to alleviate the disease and improve the quality of life.
Broschu single resistance was discovered by Kyowa Hakko Kirin, a Japanese pharmaceutical company, as a fully humanized monoclonal IgG1 antibody in the study of fibroblast growth factor 23 (FGF23).
and its subsidiaries work with Ultragenyx Pharmaceutical to develop and commercialize the product worldwide.
FGF23 reduces phosphate and active vitamin D levels in the serum by regulating phosphate excretion and the production of active vitamin D in the kidneys.
is designed to normalize phosphorus levels by combining excess FGF23 in XLH patients to improve bone mineralization, as well as cartilage disease in children and fractures in adults.
in a placebo-controlled clinical trial, 94 percent of adults who received broshu monoantin (KRN23) were able to achieve normal phosphorus levels (vs.8%) once a month in a placebo-controlled clinical trial.
in children, 94% to 100% of patients who received broshu monothesis every two weeks achieved normal phosphorus levels.
in children and adults, X-ray (XLH-related) results improved in patients receiving broshu monotherapy.
The results of another active Phase 3 clinical trial in adult XLH patients showed that after 48 weeks of treatment with Broshu monotherapy, the study observed normal and maintained serum phosphorus levels in patients, as well as further improvements in body stiffness, body function and pain.
, the stiffness score changed from an average of 7.42 out of 24 weeks to 16.03 out of 48 weeks.
based on data on the safety and efficacy of Brosshu's monoantion in clinical trials, the FDA approved the product in April 2018 for the treatment of XLH in children and adults 1 year of age and older.
an earlier FDA press release, XLH differs from other forms of cartilage disease because vitamin D treatment is ineffective.
is the first FDA-approved drug to treat XLH, a real breakthrough for people with this serious disease.
Screenshot Source: In an earlier press release from Concorde, Dr. Tom Carpenter, professor of pediatric endocrinology at Yale University School of Medicine and director of the X Chain Low Phosphorus Blood Center, said Crysvita's approval was indeed a watershed moment for patients with X-series hypophosphatemia because it was the first treatment designed to correct renal phosphate consumption.
this mechanism, Crysvita can lead to a continuous improvement in phosphate metabolism, while repairing bones, even if traditional methods have been used for treatment.
, Crysvita's dosing options are much smaller than the treatments currently available and should be easily accepted by families.
promises to revolutionized treatments for XLH patients.
a reference to the fda approves first therapy for rare inherited form of rickets, x-linked hypophosphatemia. Retrieved April 17, 2018, from [2] Ultragenyx and Kyowa Kirin Announce FDA Approval of Crysvita® (burosumab-twza) for the Treatment of Children and Adults with X–Linked Hypophosphatemia (XLH). Retrieved April 18, 2018, from the National Drug Administration of China (NMPA) drug registration progress query.From