The new patent (US Pat. No. 8,778,373) provides OPKO with additional intellectual property protection covering controlled release administration of a vitamin D compound. OPKO is on schedule to announce top-line results from the first pivotal phase 3 trial for RAYALDEE in the third quarter of 2014 and file a New Drug Application (NDA) with the FDA in the first quarter of 2015.
RAYALDEE is a first-in-class oral vitamin D prohormone treatment being developed for SHPT in stage 3 and 4 CKD patients with vitamin D insufficiency.
It has a proprietary modified-release formulation designed to gradually and reliably raise serum total 25-hydroxyvitamin D (prohormone) concentrations to targeted levels (at least 30 ng/mL) while avoiding upregulation of CYP24, a cytochrome P-450 enzyme that reduces the PTH lowering potency of current vitamin D supplements.
Activation of calcifediol, the active ingredient in RAYALDEE, by the kidney is tightly regulated, preventing excessive elevation of serum calcium and related side effects which encumber current vitamin D hormone therapies and promote vascular and renal calcification.
Once approved, RAYALDEE is expected to address an approximate 4 million CKD stage 3 and 4 patients in the U.S. and many more, elsewhere, with SHPT and vitamin D insufficiency.