New drug for hereditary osteopathy! FGF-23 targeted monoantial Crysvita treatment X-chain hypophosphatemia is significantly more effective than conventional:

Japanese pharmaceutical company Kyowa Hakko Kirin and partner Ultragenyx recently announced positive results from a 64-week clinical study of the 64-week efficacy and safety analysis of Crysvita (burosumab, KRN23) for children with X-chain hypophosphedia (XLH). The new results show that Crysvita is superior to current conventional therapies (oral phosphate plus active vitamin D) in all key secondary therapeutic endpoints, including significant improvements in cartilage severity, lower limb malformation, growth, and bodily performance. The safety of 64 weeks is similar to that observed in 40 weeks and other Crysvita pediatric XLH studies.
XLH is the most common cause of hereditary rickets in children, and early identification is critical for proper pediatric management and monitoring of disease-related complications and treatment.
is the world's first drug to treat XLH and was approved by the U.S. and Europe in 2018 to treat children and adults 1 year of age and older with XLH. Crysvita was developed to treat hypothyrophosphate diseases associated with fibroblast growth factor 23 (FGF-23), such as XLH, tumor-induced osteoporosis (TIO)/peritderysis (ENS).
the crystal vita active drug ingredient burosumab is a monoclonal antibody that targets binding and inhibits the activity of FGF-23. FGF23 reduces phosphate and active vitamin D levels in the serum by regulating phosphate excretion and the production of active biotin D in the kidneys. Phosphate consumption in FGF-23-related hypophosphate cartilage and bone softening is caused by FGF-23 levels and high activity. By blocking FGF-23, burosumab increases the reabsorbation of phosphates by the kidneys and increases the production of active vitamin D, thus also enhancing the absorption of phosphates and calcium in the intestines.
the study was a randomized, positive drug control, open label III study that included 61 children aged 1-12 years with XLH and assessed the efficacy and safety of Crysvita (n-29) relative to conventional therapy (n-32). The main endpoint was changes in rickets at 40 weeks of treatment, assessed by three independent blind paediatric radiologists using the Radiological Overall Change Impression Scale (RGI-C). Secondary endpoints include other rickets assessments, pharmacological assessments, growth rate and height changes, walking ability, patient-reported pain, fatigue, and body function assessment results, and safety using the RGI-C scale and the Thacher cartilage severity score (RSS) system. All endpoints were also evaluated at 64 weeks, such as RGI-C lower limb deformities and elevated Z scores. Prior to the group study, all patients received an average of 4 years of routine treatment. The initial dose in the Crysvita treatment group was 0.8 mg/kg per 2 weeks, and the dose in 8 patients was increased to 1.2 mg/kg. Patients in the regular treatment group receive local standard solutions in accordance with expert guidelines and are continuously optimized by each patient's physician.64-week efficacy and safety analysis results:
Crysvita relative to conventional therapy after 64 weeks of osteopathic results: (1) cartilage score: by 3 independent blind pediatric radiologists using RGI-C overall score evaluation, Crysvita better than conventional therapy (LS average treatment difference: 1.02, p<0.0001) ;(2) cartilage rehabilitation: RGI-C score shows that Crysvita treatment group has 8 6.2% of patients showed significant recovery from cartilage disease, compared with 18.8% in the conventional therapy group (p.0002);(3) in the severity score of cartilage disease (RSS): Crysvita treatment group and routine The therapeutic group showed greater improvement (LS average treatment difference: -1.21, p<0.0001) ;(4) average serum alkaline phosphatase levels as a biotransm to measure cartilage disease, Crysvita treatment group reduced to the normal range in week 60 significantly better than conventional therapy treatment group (p<0.0001);(5) lower limb deformity (bend/limb deformity RGI-C score): Cry The svita treatment group decreased more than the conventional therapy group (LS average treatment difference: 0.97, p<0.0001) ;(6) standing height/bed length Z score: Compared with the conventional treatment group, the growth during Crysvita treatment showed significant statistically significant improvements (LS average treatment difference: :0.14, p=0.0490) ;(7) 6-minute walking test (6MWT) assessed walking ability: Crysvita therapeutic walking ability showed an improvement compared to conventional treatment (LS difference: :45.6 m, p=0.0999).
: The average serum phosphorus levels in the Crysvita treatment group reached the lower limit of the normal range, while the conventional treatment group increased significantly. At baseline, the average serum phosphorus level and average renal phosphate re-absorption levels of patients in the Crysvita treatment group and the conventional treatment group were below the lower limit of the normal value. In the Crysvita group, serum phosphorus and renal phosphate re-absorption levels were within the normal range from baseline examination to week 64. In contrast, in the conventional treatment group, the average level of serum phosphorus and renal phosphate re-absorption remained below the lower limit of normal values by week 64. Crysvita has significant differences from conventional treatment (p<0.0001).
patients in the Crysvita treatment group and the conventional treatment group showed an increase in serum 1,25-hydroxyvita vitamin D and remained within the normal range for 64 weeks. (Bio Valley)