|Rockwell Announces FDA Advisory Committee Review Of Triferic; Horzion Pharma Presents Results of ACTIMMUNE(R) Phase 2 Data|
|By William Kent|
|Thursday, 09 October 2014 19:24|
Rockwell Medical, Inc. (Nasdaq: RMTI), a fully integrated biopharmaceutical company targeting end-stage renal disease (ESRD) and chronic kidney disease (CKD) with innovative products and services for the treatment of iron replacement, secondary hyperparathyroidism and hemodialysis, announced the Oncologic Drugs Advisory Committee (ODAC) of the U.S. Food & Drug Administration (FDA) is scheduled to review the Company's New Drug Application (NDA) for Triferic on November 6, 2014 in an afternoon session.
Triferic is Rockwell's investigational drug for the treatment of iron replacement and maintenance of hemoglobin in hemodialysis patients. Triferic is under review by the Office of Hematology and Oncology Products (OHOP) Division of Hematology products. The proposed indication for Triferic is for the treatment of iron loss or iron deficiency to maintain hemoglobin in patients with hemodialysis dependent chronic kidney disease and to reduce the prescribed dose of erythropoiesis stimulating agents (ESAs) required to maintain desired hemoglobin levels.
The FDA accepted the Triferic NDA for review on May 28, 2014. The PDUFA date for the FDA to complete its review of the Triferic NDA is January 24, 2015.
"We are pleased to discuss the potential efficacy benefit and safety profile of Triferic with the Advisory Committee, and we will continue to work closely with the FDA during the review of the NDA," said Mr. Robert L. Chioini, Founder, Chairman and CEO of Rockwell. "Triferic has a novel mechanism of action and mode of administration which addresses the unmet need of maintenance iron therapy for the hemodialysis population."
Horizon Pharma plc (NASDAQ: HZNP) announced the presentation of data from a Phase 2 clinical study of ACTIMMUNE (interferon gamma-1b) treatment in children with Friedreich's ataxia (FA). An abstract of the data has been published in a supplement to the Annals of Neurology and is being presented as a poster during the 139th Annual Meeting of the American Neurological Association in Baltimore, MD on Monday, October 13.
This single center, Phase 2 open-label study of 12 children ages five to 17 years with genetically confirmed FA with treatment of up to 12 weeks, evaluated the safety, tolerability and efficacy of ACTIMMUNE. The study measured frataxin levels, a biomarker of disease and clinical measures (Friedreich's Ataxia Rating Scale [FARS] score and other neurological evaluations) as efficacy markers. Additional details on the study design may be found at www.clinicaltrials.gov.
The results, as noted in the published abstract, showed ACTIMMUNE was well tolerated with no serious adverse events and two subjects reporting severe events and subsequent dose reductions. The safety findings generally reflected the labeled safety profile for ACTIMMUNE. Changes in frataxin protein levels, the primary study endpoint, were statistically significant in red blood cells, white blood cells and platelets. The magnitude of change observed was small and varied between tissues. Mean improvement in the FARS score, a clinically validated measurement of patient performance and secondary endpoint, was statistically significant (p=0.008) and was equivalent to two years of extension of disease progression. No other changes were observed.
"The highly significant improvement in the FARS score, a key measure of improvement in disease progression, supports further study of ACTIMMUNE in Friedreich's ataxia," said Timothy P. Walbert, chairman, president and chief executive officer, Horizon Pharma plc. "We plan to work with the Collaborative Clinical Research Network in Friedreich's Ataxia (CCRN in FA) and the Friedreich's Ataxia Research Alliance (FARA) to rapidly move into a registration program for ACTIMMUNE in FA. We plan to meet with the FDA this quarter regarding the clinical development and regulatory pathway for ACTIMMUNE in FA."
The study was sponsored by FARA and conducted by David Lynch, M.D., Ph.D., professor of neurology at Children's Hospital of Philadelphia (CHOP) and principal investigator of the CCRN in FA. Investigational product was provided by Vidara Therapeutics Research Limited, now part of Horizon Pharma plc.
"The FA community has been working toward a viable treatment option and we were proud to be able to sponsor this initial pilot study. We are encouraged that ACTIMMUNE was well tolerated and there was a significant improvement observed in a clinical outcome measure," said Ronald Bartek, president, director and co-founder of FARA. "We are very pleased to partner with Horizon Pharma and look forward to working together to explore the potential for ACTIMMUNE in FA.
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