FDA批准Velphoro治疗慢性肾脏病透析患者高磷血症

Vifor制药11月29日宣布，磷酸盐结合剂Velphoro（sucroferric oxyhydroxide）已获FDA批准，用于治疗慢性肾脏病（CKD）透析患者的高磷血症（hyperphosphatemia），以控制其血清磷水平。

Velphoro（以前称为PA21）是一种铁基（iron-based）、无钙、可咀嚼的磷酸盐结合剂，该药的获批，为接受透析治疗的慢性肾脏病（CKD）患者控制其血清磷水平提供了一种新的有效的治疗选择，该药将于2014年在美国上市。

Velphoro的获批，是基于一项关键性III期临床试验的结果。该项研究在正接受透析治疗的慢性肾脏病（CKD）患者中开展，研究结果表明，与标准的护理药物碳酸司维拉姆（sevelamer carbonate）相比，Velphoro以较少的药丸数量，成功地控制了患者的高磷血症，52周后，用于控制高磷血症的平均每天剂量仅为3.3粒/天，达到了研究的主要和次要终点。

高磷血症（hyperphosphatemia）是指血液中磷水平的异常升高，是正在接受透析治疗的慢性肾脏病（CKD）患者中一种常见且严重的疾病。大多数透析患者需接受磷酸盐结合剂治疗。

然而，尽管目前已有多种不同的磷酸盐结合剂可用，但高达50%的患者仍无法达到并维持其目标血清磷水平。在一些患者中，因高的服药负担导致的非依从性（non-compliance）和较差的耐受性，似乎是导致血清磷水平控制不足的关键因素。平均而言，透析患者每天需服约19粒药物，而磷酸盐结合剂占到了其中的约50%。而Velphoro的推荐起始剂量仅为每日3片（每餐1片）。

目前，Velphoro在欧盟、瑞士、新加坡的监管审查正在进行中，预计将于2014年上半年得出审查决定。进一步的监管申请正在准备中。

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