TARSA Therapeutics, Inc. today announced that its New Drug Application (NDA) for TBRIATM (calcitonin-salmon [rDNA origin] delayed release tablets) submitted to the U.S. Food and Drug Administration (FDA) in July 2015, has been accepted for review, with a PDUFA date of May 30, 2016.
Calcitonin has been available as an osteoporosis treatment for more than 35 years, but only in formulations allowing for injectable or intranasal administration. TBRIA would be the first FDA approved oral calcitonin, offering a convenient route of administration and potentially providing enhanced patient acceptance and compliance.
TBRIA is a once-daily oral recombinant salmon calcitonin tablet for the treatment of postmenopausal osteoporosis in women greater than 5 years postmenopause when alternative treatments are not suitable (e.g., patients for whom other therapies are contraindicated or for patients who are intolerant or unwilling to use other therapies).
TBRIA demonstrated positive safety and efficacy as a treatment for postmenopausal osteoporosis in the global, randomized, double-blind ORACAL trial. This study, which was the subject of a Special Protocol Assessment with the FDA, is the pivotal Phase III trial that forms the basis for TARSA's NDA. In the trial, TBRIA met its primary endpoint and demonstrated statistical superiority to both placebo and nasal calcitonin spray in increasing bone mineral density at the lumbar spine after 48 weeks of use. The safety profile of TBRIA did not substantially differ from nasal calcitonin or placebo. Study results were published in the Journal of Bone and Mineral Research.
"We are pleased that the FDA has accepted our NDA for review, offering the possibility that TBRIA could be cleared for marketing in 2016," said David Brand, President and CEO of TARSA. "Osteoporosis is a common condition and a leading cause of disability in a growing older population. However, concerns about adverse events with some current therapies have resulted in millions of osteoporosis patients dropping or never initiating treatment."
An estimated 10 million people in the U.S. have osteoporosis. Perceived safety issues with several classes of once-widely used osteoporosis drugs, including bisphosphonates and hormone replacement therapies, have led to a sharp decline in patients receiving treatment. According to a study published in 2013, prescriptions for oral bisphosphonates fell by 53% between 2008 and 2012.
Mr. Brand continued, "In clinical trials TBRIA has been shown to have a positive effect on bone mineral density at the lumbar spine, and our primary market research among physicians suggests that once-daily, oral TBRIA is seen as an attractive therapeutic option for many patients with osteoporosis who cannot or will not take other therapies."
Neil Binkley, MD, who is a Professor of Geriatrics and Co-Director, Osteoporosis Clinical Center & Research Program at the University of Wisconsin School of Medicine and Public Health-Madison, noted, "The oral calcitonin NDA submission is positive news. Many women with osteoporosis are untreated, in part due to perceived safety issues with existing medications. As such, there is a clear need for additional treatment options."
TARSA's formulation of TBRIA uses proprietary, patented oral delivery technology licensed from Enteris Biopharma. TARSA has exclusive development and worldwide commercialization rights to the oral calcitonin product developed with Enteris' technology, with the current exception of China.