Opko up on first FDA approval in chronic kidney disease associated indication

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The FDA has approved Rayaldee from Opko Health ($OPK) to treat secondary hyperparathyroidism (SHPT) in some chronic kidney disease patients. This is the first time the agency has approved a drug in this indication--which is common in chronic kidney disease patients with excessive parathyroid hormone.

The company plans to launch the drug in the U.S. during the second half of this year. In May, Opko partnered with Vifor Fresenius to develop and commercialize Rayaldee to treat SHPT in Europe, Canada, Mexico, Australia, South Korea and certain other international markets.

“Rayaldee fills a large void in the current treatment options for SHPT in pre-dialysis patients,” said Opko Renal Division CEO Dr. Charles Bishop in a statement. “The current standard of care is high dose vitamin D supplementation, an approach for treating SHPT that is neither FDA approved nor demonstrated to be safe and effective in this population. SHPT is a progressive disease that becomes increasingly debilitating and difficult to treat, necessitating timely and effective treatment.”

The drug is specifically indicated to treat SHPT in adult patients with stage 3 or 4 CKD and serum total 25-hydroxyvitamin D levels less than 30 ng/mL. It’s intended to raise serum total 25-hydroxyvitamin D (prohormone) concentrations to targeted levels (at least 30 ng/mL) and to reduce elevated plasma intact parathyroid hormone (iPTH).

In a pair of 26-week clinical trials, Rayaldee achieved more than 30% reductions in iPTH than placebo in this population. It also corrected Vitamin D insufficiency by more than 80%, versus 7% for placebo subjects.

Under the Vifor Fresenius deal, the partners are also developing a different dosage of Rayaldee that’s specifically intended to treat secondary hyperparathyroidism in dialysis patients from end stage or stage 5 renal disease--for whom Rayaldee is not indicated.

Vifor Fresenius is a joint venture between Vifor Pharma parent Galencia Group and dialysis provider Fresenius Medical Care that’s owned 55% and 45%, respectively. It has an option under the Opko deal to market the dialysis-oriented candidate in the U.S. market. To do so, it would pay up to an additional $555 million in commercial milestones, as well as double-digit royalties on net sales.

That’s a hefty bump-up on the EU and ex-U.S. deal for the newly approved Rayaldee, for which Vifor Fresenius paid $50 million up front plus an additional $232 million in regulatory and sales-based milestones--as well as tiered double-digit royalties on net sales.

Opko had received a complete response letter for Rayaldee in March. The agency had observed deficiencies with Opko’s third-party contract manufacturer. Opko resubmitted in April and had a new PDUFA date for Rayaldee of October 22.

“Rayaldee is an important new option for treating SHPT in patients with stage 3 or 4 CKD and vitamin D insufficiency,” said Kevin Martin, Director of Research, Division of Nephrology at Saint Louis University School of Medicine. “The great majority of SHPT cases in this patient population are associated with vitamin D insufficiency, a problem that Rayaldee can correct.”

Opko was up 8% in early trading on the approval news to a market cap of more than $5 billion.

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