OPKO wins FDA approval for Rayaldee after Catalent quickly resolves plant concerns

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Miami-based OPKO said today that the FDA has approved its chronic kidney disease drug Rayaldee. The approval comes just three months after OPKO received a complete response letter (CRL), tied to concerns that the FDA had about the plant where CMO Catalent was producing the drug for the biotech.

Rayaldee becomes the first drug approved to treat secondary hyperparathyroidism in adults with stage 3 or 4 chronic kidney disease and serum total 25-hydroxyvitamin D levels less than 30 ng/mL. Rayaldee is an extended release capsule containing 30 mcg of a prohormone called calcifediol, the company said. It expects to launch the drug in the second half of the year. 

In an email, a Catlanet spokesperson said, "The company was already implementing measures to address some of the areas noted in the FDA observations prior to submitting its response, and the FDA accepted Catalent’s full response in May, indicating it would take no further action.”

PKO ($OPK) and Catalent ($CTLT) were surprised in March when approval of the drug was sidelined by the CRL. The FDA held off on approval after an inspection of Catalent’s plant in St. Petersburg, FL turned up issues. While the concerns were not directly tied to the manufacturing of Rayaldee, they were serious enough that the agency held off on approving the drug until it was satisfied with Catalent’s response.

A Form 483 released by the agency indicated the FDA was unhappy with Catalent investigations into several issues at the 453,000-square-foot softgel plant. In one, it was reported that an unidentified “foreign dark matter” was coming down from the shaft of a gel receiver. The FDA said no one tied the “dark particles” back to seal change on the machinery two days before when a piece of equipment “had leaked all of its lubricant due to a broken seal.” There also was no investigation, the FDA said, to find out if any gel batches produced when the machinery was leaking were affected by the lubricant.

Catalent responded quickly, submitting an action plan to the FDA on April 15 and OPKO resubmitted its application for approval. On April 22, just 5 weeks after OPKO got the CRL, the FDA accepted the application as complete and set an October 22, PDUFA date. Instead of taking 6 months to approve the drug, it was done in two.

- here’s the OPKO press release

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UPDATED: Catalent issues lead to CRL for OPKO