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28453911000001100

New Medicines

Velphoro Hyperphosphataemia in children aged 2 years and older with CKD stages 4-5 or with CKD on dialysis - oral powder in sachets

Information

Velphoro
New formulation
Vifor Fresenius Medical Care Renal Pharma Ltd (VFMCRP)
Vifor Pharma

Development and Regulatory status

Licensed but not launched
Licensed but not launched
Phase III Clinical Trials
Sep 21Company has no immediate plans to make this available in the UK [6].
Nov 20Approved in EU and UK [5].
Sep 20EU positive opinion for a new indication and a new pharmaceutical form and strength – powder for oral suspension 125 mg. The proposed new indication is control of serum phosphorus levels in paediatric patients 2 years of age and older with CKD stages 4-5 (defined by a glomerular filtration rate <30 mL/min/1.73 m²) or with CKD on dialysis [2].

Category

A mixture of polynuclear iron(III)-oxyhydroxide (pn-FeOOH), sucrose and starches. Phosphate binding takes place by ligand exchange between hydroxyl groups and/or water and the phosphate ions throughout the physiological pH range of the GI tract.
Hyperphosphataemia occurs because of insufficient filtering of phosphate from the blood by poorly functioning kidneys. High serum phosphate levels can directly and indirectly increase PTH secretion, leading to secondary hyperparathyroidism. Left untreated, secondary hyperparathyroidism increases morbidity and mortality and may lead to renal bone disease with bone/muscular pain, increased incidence of fracture, bone and joint abnormalities and vascular and soft tissue calcification [1].
Hyperphosphataemia in children aged 2 years and older with CKD stages 4-5 or with CKD on dialysis - oral powder in sachets
Oral

Trial or other data

Sep 20PIII trial (NCT02688764) completed in Feb 2019 [4].
Sep 20The EMA human medicines committee (CHMP) based its recommendation for Velphoro on the assessment of an open-label, randomised phase 3 clinical study, which included 85 children from 2 years of age with CKD and hyperphosphatemia. The results showed normal phosphorus range after treatment in a large proportion of patients, comparable to what was observed for adults [2].

Evidence based evaluations