The FDA has accepted the resubmission of a new drug application (NDA) for Rayaldee (extended-release calcifediol, Opko Health) for the treatment of secondary hyperparathyroidism (SHPT) in patients with stage-3 or -4 chronic kidney disease (CKD) and vitamin D insufficiency.
Opko resubmitted the NDA after receiving a complete response letter (CRL) from the FDA in March, in which the FDA indicated that the NDA could not be approved because of deficiencies observed during a facility inspection of Opko’s third-party manufacturer. The observations were not specific to Rayaldee manufacturing, and the CRL did not cite any safety, efficacy, or labeling issues with regard to Rayaldee, nor did it request any additional studies to be conducted prior to FDA approval.
A six-month review period has been assigned for the resubmitted NDA, with a Prescription Drug User Fee Act (PDUFA) date of October 22, 2016.
Rayaldee (calcifediol) extended-release capsules have a proprietary formulation designed to increase serum total 25-hydroxyvitamin D (prohormone) concentrations to targeted levels (at least 30 ng/mL) and to reduce elevated intact parathyroid hormone (iPTH).
SHPT is a condition commonly associated with CKD in which the parathyroid glands secrete excessive amounts of parathyroid hormone (PTH). SHPT occurs as a result of vitamin D insufficiency or impaired kidney function that prevents sufficient production of vitamin D hormone to properly regulate calcium and phosphorus metabolism, and PTH secretion. Prolonged elevation of blood PTH causes excessive calcium and phosphorus to be released from bone, leading to elevated serum calcium and phosphorus, osteomalacia, and calcification of vascular and renal tissues. SHPT affects 40% to 60% of patients with moderate CKD and approximately 90% of patients with severe CKD.
Source: Opko Health; April 28, 2016.