Setback for OPKO as FDA issues CRL on Rayaldee

The US Food and Drug Administration has issued a complete response letter (CRL) to OPKO Health’s New Drug Application (NDA) for Rayaldee (calcifediol) as a treatment for secondary hyperparathyroidism (SHPT) in patients with stage 3 or 4 chronic kidney disease (CKD) and vitamin D insufficiency.

The FDA indicated in the CRL that observations of deficiencies at OPKO’s third-party contract manufacturer were issued on March 25, as a result of an FDA field inspection initiated on March 14, 2016. The observations were not specific to Rayaldee manufacturing. 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.

Though likely to be only a temporary setback, OPKO’s shares fell 8.40% to $10.14 in pre-market trading this morning, and were down 9.9% at $9.97 at 9.30am EDT. The company’s third-party manufacturer has committed to respond promptly to the FDA’s observations to ensure early resolution.