2013年11月30日訊 /生物谷BIOON/ --Vifor製藥11月29日宣布，磷酸鹽結合劑Velphoro（sucroferric oxyhydroxide）已獲FDA批准，用於治療慢性腎臟病（CKD）透析患者的高磷血症（hyperphosphatemia），以控制其血清磷水平。
Velphoro（以前稱為PA21）是一種鐵基（iron-based）、無鈣、可咀嚼的磷酸鹽結合劑，該藥的獲批，為接受透析治療的慢性腎臟病（CKD）患者控制其血清磷水平提供了一種新的有效的治療選擇，該藥將於2014年在美國上市。
Velphoro的獲批，是基於一項關鍵性III期臨床試驗的結果。該項研究在正接受透析治療的慢性腎臟病（CKD）患者中開展，研究結果表明，與標準的護理藥物碳酸司維拉姆（sevelamer carbonate）相比，Velphoro以較少的藥丸數量，成功地控制了患者的高磷血症，52周後，用於控制高磷血症的平均每天劑量僅為3.3粒/天，達到了研究的主要和次要終點。
高磷血症（hyperphosphatemia）是指血液中磷水平的異常升高，是正在接受透析治療的慢性腎臟病（CKD）患者中一種常見且嚴重的疾病。大多數透析患者需接受磷酸鹽結合劑治療。
然而，儘管目前已有多種不同的磷酸鹽結合劑可用，但高達50%的患者仍無法達到並維持其目標血清磷水平。在一些患者中，因高的服藥負擔導致的非依從性（non-compliance）和較差的耐受性，似乎是導致血清磷水平控制不足的關鍵因素。平均而言，透析患者每天需服約19粒藥物，而磷酸鹽結合劑佔到了其中的約50%。而Velphoro的推薦起始劑量僅為每日3片（每餐1片）。
目前，Velphoro在歐盟、瑞士、新加坡的監管審查正在進行中，預計將於2014年上半年得出審查決定。進一步的監管申請正在準備中。（生物谷Bioon.com）
英文原文：Velphoro® (PA21) receives US FDA approval for the treatment of hyperphosphatemia in Chronic Kidney Disease Patients on dialysis
Thursday, 28 November 2013, ↓ directly to download
_Velphoro® (sucroferric oxyhydroxide) has received US Food and Drug Administration (FDA) approval for the control of serum phosphorus levels in patients with Chronic Kidney Disease (CKD) on dialysis. Velphoro® will be launched in the US by Fresenius Medical Care North America in 2014.
_Velphoro® (previously known as PA21) is an iron-based, calcium-free, chewable phosphate binder. US approval was based on a pivotal Phase III study, which met its primary and secondary endpoints. The study demonstrated that Velphoro® successfully controls hyperphosphatemia with fewer pills than sevelamer carbonate, the current standard of care in patients with CKD on dialysis1. The average daily dose to control hyperphosphatemia was 3.3 pills per day after 52 weeks.
_Velphoro® was developed by Vifor Pharma. In 2011, all rights were transferred to Vifor Fresenius Medical Care Renal Pharma, a common company of Galenica and Fresenius Medical Care. In the US, Velphoro® will be marketed by Fresenius Medical Care North America, a company with a strong marketing and sales organization, and expertise in dialysis care. The active ingredient of Velphoro® is produced by Vifor Pharma in Switzerland.
_Hyperphosphatemia, an abnormal elevation of phosphorus levels in the blood, is a common and serious condition in CKD patients on dialysis. Most dialysis patients are treated with phosphate binders. However, despite the availability of a number of different phosphate binders, up to 50% of patients – depending on the region – are still unable to achieve and maintain their target serum phosphorus levels2. In some patients, non-compliance due to the high pill burden and poor tolerability appear to be key factors in the lack of control of serum phosphorus levels3,4. On average, dialysis patients take approximately 19 pills per day with phosphate binders5 comprising approximately 50% of the total daily pill burden. The recommended starting dose of Velphoro® is 3 tablets per day (1 tablet per meal).
_Full results from the pivotal Phase III study involving more than 1,000 patients were presented at both the 50th ERA-EDTA (European Renal Association - European Dialysis and Transplant Association) Congress in Istanbul, Turkey, in May 2013, and the American Society of Nephrology (ASN) Kidney Week in Atlanta, Georgia, in November 2013. Velphoro® was shown to be a potent phosphate binder, with lower pill burden and a good safety profile.
_Based on these data, Vifor Fresenius Medical Care Renal Pharma believes that Velphoro® offers a new and effective therapeutic option for the control of serum phosphorus levels in patients with chronic kidney disease on dialysis.
_The regulatory processes in Europe, Switzerland and Singapore are ongoing and decisions are expected in the first half 2014. Further submissions for approval are being prepared.
_Vifor Pharma, the pharma business sector of the Galenica Group, is one of the world’s leaders in the discovery, development, manufacturing and marketing of pharmaceutical products for the treatment of iron deficiency. The company also offers a diversified portfolio of prescription medicines as well as over-the-counter (OTC) products. Vifor Pharma, headquartered in Zurich, Switzerland, has an increasingly global presence and a broad network of affiliates and partners around the world.
_Vifor Fresenius Medical Care Renal Pharma Ltd., a common company of Galenica and Fresenius Medical Care develops and commercialises innovative and high quality therapies to improve the life of patients suffering from Chronic Kidney Disease (CKD) worldwide. The company was founded at the end of 2010 and is owned 55% by Galenica and 45% by Fresenius Medical Care.
_Velphoro® / PA21 is a chewable, iron-based phosphate binder containing a mixture of polynuclear iron(III)-oxyhydroxide, sucrose and starches. Each tablet of Velphoro® contains the equivalent of 500mg of iron. When taken with meals, Velphoro® adsorbs the dietary phosphate in the gastrointestinal tract and prevents its uptake into the blood. The adsorbed phosphate is subsequently eliminated through the faeces.
_The Velphoro® Phase II clinical trial met both its primary and secondary endpoints. The serum phosphorus lowering efficacy of the two lowest active doses was numerically comparable to 4.8g/day sevelamer hydrochloride. Velphoro® was also well tolerated, with a comparable overall safety and tolerability profile.
_The Phase III trial was an open-label, randomised, active controlled, parallel group study to investigate the safety and efficacy of Velphoro® compared to sevelamer carbonate, followed by a randomized comparison of Velphoro® maintenance dose versus Velphoro® inactive low-dose in dialysis patients with hyperphosphatemia.
_During the Phase III study no clinically meaningful changes for vitamins (A, D, E and K) were observed on Velphoro®. A sub-analysis is being conducted on patients who received oral vitamin D analogues in the study, in an effort to modify the package insert statement that 「Velphoro® should not be prescribed with oral vitamin D analogues」 which was based on in-vitro data. Of the dialysis patients in the US receiving vitamin D for the management of bone and mineral disease, 82% are treated with intravenous formulation6, which is approved for use with Velphoro®.
_Velphoro® is developed in collaboration with Fresenius Medical Care It is also currently undergoing Phase III clinical development in Japan by Kissei Pharmaceuticals Co., Ltd.