dm+d

Unassigned

New Medicines

RayaldeeSecondary hyperparathyroidism in chronic kidney disease

Information

Rayaldee
New molecular entity
Vifor Fresenius Medical Care Renal Pharma Ltd (VFMCRP)
OPKO Health

Development and Regulatory status

Licensed but not launched
Approved (Licensed)
Launched
Jan 21Vifor Pharma UK currently has no plans to market Rayaldee in the UK [15].
Jul 20Calcifediol 30 microgram prolonged-release capsules were approved by the MHRA for the treatment of secondary hyperparathyroidism (SHPT) in adults with chronic kidney disease (CKD) Stage 3 or 4 and vitamin D insufficiency or deficiency [14].

Apr 19: Marketing Authorisation Application (MAA) filed through the EU Decentralised Procedure, seeking MA in "selected" [company description] European countries with approvals expected in 2020 [13]. In the decentralised procedure, the application is submitted to licensing agencies in both a Reference Member State, which prepares an assessment report (normally within 210 days), and also to the other countries in which the MA is hoped for (Concerned Member States, CMS); the assessment report is considered by the CMS and if they agree with it will issue a MA within 30 days. Until the MA is published, the agencies do not disclose which countries are involved.


Nov 16: launched US [11]; 2018 price $900 - 1000 for 30 capsules [12].


Jun 16: FDA approves Rayaldee to treat secondary hyperparathyroidism (SHPT) in chronic kidney disease patients. The company plans to launch the drug in the U.S. during H2 2016. In May, Opko partnered with Vifor Fresenius to develop and commercialise Rayaldee to treat SHPT in Europe, Canada, Mexico, Australia, South Korea and certain other international markets [10].


Jul 15: The FDA has accepted for review the New Drug Application for Rayaldee for the prevention and treatment of secondary hyperparathyroidism (SHPT) in patients with stage 3 or 4 chronic kidney disease (CKD) and vitamin D insufficiency. [8]


Sep 14: A New Drug Application submission in the US is planned for the end of 2014 [6].

Category

Vitamin D prohormone
In secondary hyperparathyroidism is usually seen in renal disease and seen in almost all patients with dialysis-dependent chronic renal failure. Most patients with CKD stage 5 develop secondary hyperparathyroidism (patient.co.uk)
Secondary hyperparathyroidism in chronic kidney disease
Oral

Trial or other data

Apr 16: The FDA has accepted resubmission of the NDA for Rayaldee for the treatment of secondary hyperparathyroidism in patients with stage 3 or 4 CKD and vitamin D insufficiency[9].


May 15: OPKO submitted a New Drug Application (NDA) for oral Rayaldee to the US FDA Food requesting marketing approval for Rayaldee for prevention and treatment of secondary hyperparathyroidism (SHPT) in pts with stage 3 or 4 CKD and vitamin D insufficiency. The application is supported by positive data from three randomised, double-blind, placebo-controlled studies and one open-label extension study [7].


Aug 14: Topline results from the first pivotal PIII trial of Rayaldee announced. The primary efficacy endpoint was a responder analysis in which “responder” was defined as any treated subject who demonstrated an average 30% decrease in plasma parathyroid hormone (PTH) from pre-treatment baseline during the last six weeks of the treatment period. A significantly higher response rate (p<0.001) was observed with Rayaldee which steadily increased with treatment duration. The response rate with Rayaldee was similar in CKD stages 3 and 4. Safety and tolerability data were comparable in both treatment groups. [5]


May 2014: Third phase 3 trial of Rayaldee initiated, designed to evaluate long-term safety and efficacy in treating secondary hyperparathyroidism (SHPT) in patients with stage 3 or 4 chronic kidney disease (CKD) and vitamin D insufficiency. This phase 3 trial is a 6-month open-label extension of two ongoing RCTs (n=approximately 430). Both ongoing trials will end in July 2014 and top-line data are expected during Q3 2014. In the open-label extension study, patients either continue RAYALDEE treatment or switch to RAYALDEE from placebo treatment. Additional patients will be allowed to enroll in the open-label extension study after leaving the blinded pivotal trials. The endpoints of all three phase 3 studies include vitamin D status and changes in plasma intact parathyroid hormone (PTH), serum calcium and serum phosphorus. [4]


Nov 13: First PIII study (NCT01651000) is fully enrolled. Second PIII study (NCT01704079) is not yet recruiting [3].


May 13: Over 50% enrollment in the first phase 3 trial of CTAP 101 to treat patients with secondary hyperparathyroidism (SHPT), stage 3 or 4 chronic kidney disease (CKD) and vitamin D insufficiency. This trial is the first of two identical randomised, double-blind, placebo controlled, multi-site studies intended to establish the safety and efficacy of CTAP 101 as a new treatment for SHPT in the targeted population. The endpoints of both studies, which will be conducted in parallel, include vitamin D status and changes in serum calcium, serum phosphorus and plasma intact parathyroid hormone (PTH). [2]


A first-in-class oral formulation of vitamin D prohormone, for the treatment of secondary hyperthyroidism associated with vitamin D insufficiency in patients with chronic kidney disease (CKD) stages 3 or 4. [1]