Amgen's First Small Molecule - Cinacalcet HCl - Receives FDA Priority Review
SAN DIEGO, Nov. 15 -- Amgen (Nasdaq: AMGN), the world's largest biotechnology company, announced results of phase 3 studies evaluating the efficacy and safety of cinacalcet HCl, a first-in-class oral calcimimetic, administered once daily for the treatment of secondary hyperparathyroidism (HPT) associated with chronic kidney disease (CKD). These results were presented at an American Society of Nephrology (ASN) sponsored symposium in San Diego.
Amgen recently received notification that the U.S. Food and Drug Administration (FDA) granted a priority review for cinacalcet HCl, a novel therapeutic that modulates the activity of the calcium-sensing receptor, the primary regulator of PTH secretion. Cinacalcet HCl is the first investigational agent to demonstrate simultaneous reductions in parathyroid hormone (PTH), calcium-phosphorus product, phosphorus and calcium levels to successfully control secondary HPT. Moreover, cinacalcet HCl may allow patients to achieve PTH and mineral targets in accordance with the new Kidney Disease Outcomes Quality Initiative (K/DOQI) Guidelines for Bone Metabolism and Disease recently published by the National Kidney Foundation.
Secondary HPT develops early during the course of CKD and continues to progress as kidney function declines and patients begin dialysis. Patients with secondary HPT can suffer from bone disease, bone pain and fractures, soft tissue calcification and vascular calcification. The current treatment for patients with secondary HPT includes phosphate binders and vitamin D sterols, which may elevate blood calcium and/or phosphorus levels. As a consequence, treatment is frequently interrupted -- resulting in inadequate control of PTH, which contributes to disease progression.
"Cinacalcet HCl is an exciting new approach to treating secondary HPT," said Dr. Geoffrey A. Block, director of clinical research, Denver Nephrologists, PC. "Achieving the new K/DOQI guidelines will be challenging with existing therapies, which often require clinicians to make trade-offs regarding therapeutic goals. With cinacalcet HCl we may now have the ability to simultaneously maintain target levels of parathyroid hormone, calcium- phosphorus product, phosphorus and calcium."
Three phase 3 double-blind, randomized, placebo-controlled studies including more than 1,100 patients on hemodialysis and peritoneal dialysis with uncontrolled secondary HPT were conducted in Australia, Canada, Europe and the U.S. Cinacalcet HCl was titrated to achieve a target PTH level, which is within the new K/DOQI guidelines. A greater proportion of patients in the cinacalcet HCl group (36 to 48 percent), compared to those receiving standard therapy (four to seven percent) achieved the primary endpoint of PTH less than or equal to 250 pg/mL. Across the phase 3 program, clinically relevant reductions in calcium-phosphorus product (13 to 17 percent) calcium (six to eight percent) and phosphorus (seven to 10 percent) occurred in patients receiving cinacalcet HCl, while they remained at baseline levels in those receiving standard therapy and placebo. In a separate study in patients with CKD not requiring dialysis, cinacalcet HCl resulted in reductions in PTH comparable to those observed in patients with end-stage renal disease receiving dialysis.
Cinacalcet HCl was highly efficacious regardless of age, gender, race or disease severity. Cinacalcet HCl was also effective in patients receiving vitamin D as well as those contraindicated to vitamin D due to hypercalcemia or hyperphophatemia. The most commonly reported side effects in the clinical trials with cinacalcet HCl were nausea and vomiting, which were generally mild to moderate in severity and brief in duration.
"We are pleased that cinacalcet HCl, the company's first small molecule, has received priority review by the FDA. This represents an important milestone for Amgen, furthering the company's commitment to the patients suffering from CKD," said Dr. Beth Seidenberg, senior vice president of development and chief medical officer at Amgen.
Amgen licensed cinacalcet HCl from NPS Pharmaceuticals Inc. and filed a new drug application (NDA) with the FDA for cinacalcet HCl. Additionally, Amgen has filed cinacalcet HCl for regulatory approval in Australia, Canada, the European Union and New Zealand.
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