Clinically in urgent need of the new drug "Broshu monoanti" was approved for listing in China today

On January 15, the official website of the State Drug Administration showed that Concorde Fermentation Kirin Co., Ltd. Burosumab (acceptance number: JXSS1900056, JXSS1900057, JXSS1900058) has been approved for listing by NMPA.
used to treat X-series explicit genetic hyphosphorus rickets (XLH).
monoantigen is a recombinant fully humanized monoclonal IgG1 antibody targeting FGF23, designed to bind to the excess FGF23 in XLH patients, thereby increasing the re-absorption of urinary phosphorus and normalizing blood phosphorus levels.
the production of 1,25 (OH)2D3 to improve bone mineralization and improve symptoms of bone softening in children and adults.
Burosumab was approved by the FDA in April 2018 to become the world's first approved XLH drug for children and adults 1 year of age and older, and in October 2019, expanded to children over 6 months of age.
was approved in October 2020 to treat tumor osteoporosis.
previously, the FDA had awarded the drug breakthrough therapy and orphan drug eligibility.
in positive controlled trials (NCT 02915705) in children aged 1 to 12, subjects were treated with burosumab (0.8 mg/kg, once every 2 weeks) and VD3 plus oral phosphate, respectively.
40 weeks, patients in the burosumab group were treated with VD3 plus oral phosphate.
results showed that the average blood phosphorus value of the burosumab group increased by 0.9 mg/dL over the baseline at week 40 and remained unchanged after 64 weeks.
blood phosphorus in the control group increased by only 0.2 mg/dL over the baseline.
absorption effect of urine phosphorus heavy in the burosumab group was also significantly better than that of the control group.
, the Thacher rickets severity score (RSS) in the burosumab group decreased by 2.2 compared to the baseline, while the control group decreased by only 1.0.
a randomized, double-blind, placebo-controlled Phase III clinical study (NCT 02526160) in adult XLH patients showed that 94 percent of patients in the Burosumab group had normal blood phosphorus levels after 24 weeks of treatment, compared with 8 percent in the placebo group.
September 2017, the Chinese clinical application for the treatment of XLH by burosumab was included in the CDE priority review.
2019, Burosumab was added to the list of second batch of clinically urgently needed new drugs from abroad, and then submitted a listing application in August 2019.
XLH is one of the hereditary low blood phosphorus rickets, which belongs to the rare disease category in our country.
patients due to PHEX, FGF23, DMP1 and other genetic mutations, resulting in excessive or degradation disorders in the body phosphorus factor fibrous growth factor 23 (FGF23), so that the circulation of FGF23 levels increased, resulting in near-end renal tube epithal palate Na-Pi co-transporter on the urine phosphorus heavy absorption reduction, renal phosphorus threshold decreased.
At the same time, because FGF23 can inhibit the activity of the kidneys 1 alpha-hydroxylase, reduce the production of 1,25 (OH)2D3, reduce the absorption of calcium and phosphorus in the intestines, further aggravate hypophosphateemia, leading to bone mineralization disorders, causing rickets or bone softening symptoms.
occurs in childhood called rickets, mainly manifested in square skull, chicken breast, rib beads, limb bending deformity (O-type or X-type legs), growth retardation and so on.
adults with the disease are called bone softening, which manifests itself as weakness, body shape change, shorter body shape, multiple fractures, bone pain, and even disability.
reported abroad that the incidence of hymasphed phosphorus rickets was about 3.9/100,000, and the prevalence rate was about 1/21,000.
there is no relevant epidemiological data in our country.
the disease currently has no effective treatment other than neutral phosphorus and active vitamin D.