FDA Fast Track Designation Granted to Laszarin™
26th May 2005
NZ’s First FDA Fast Track Designation Granted to Protemix’s Laszarin™ for Heart Failure in Diabetes Patients
In what is believed to be a first for a New Zealand company, the US Food and Drug Administration (FDA) has granted fast track status for Protemix Corporation’s lead compound, Laszarin™ (trientine) for treatment of heart failure in people with diabetes. Studies by Protemix have implicated undesired copper levels as an important factor in the progression of diabetic complications, and the company has demonstrated that its Laszarin™ compound can be used to remove surplus copper.
“Fast Track” is a formal mechanism that facilitates FDA-company interactions concerning clinical testing of a proposed therapeutic. By law, Fast Track status is reserved to facilitate the development and expedite review of a drug only “if it is intended for the treatment of a serious or life-threatening condition and it demonstrates the potential to address unmet medical needs for such a condition.”
Benefits of Fast Track status include scheduled meetings to seek FDA input into development plans, the option of submitting a New Drug Application in sections rather than all components simultaneously, and the option of requesting evaluation of studies using surrogate endpoints.
Protemix co-founder Professor Garth Cooper explained: `This is an important first for New Zealand science and means that market approval for Laszarin™ in the designated indication could be hastened. The FDA has granted this important designation because Laszarin™ is being developed for the treatment of heart failure in diabetic patients and there is an unmet need for these patients. We intend to study the benefits of Laszarin™ in respect of clinically important endpoints including mortality and hospitalistion.’
Protemix CEO, Larry Ellingson commented: `This news from the FDA confirms that there is an important unmet clinical need for patients with diabetes suffering from heart failure and that Laszarin™ has a potentially important role to play. We are negotiating with several clinical research organisations to expand our clinical trials, after further discussion with the FDA.’
Diabetes is often accompanied by heart enlargement, heart dysfunction and coronary heart disease and these are major causes of death in people with type 2 diabetes.1 Previous work by Protemix researchers2 showed that six month’s treatment with trientine resulted in a significant decrease in heart size in people with diabetes that had enlarged hearts.
The FDA decision also follows the publication last month of Protemix research showing, among other things, a positive effect of Laszarin on the levels of oxidised copper in the body. The company believes that this may be as important as lowering blood glucose levels in preventing cardiovascular complications in diabetes mellitus.
If successful in remaining Phase II and III trials, Laszarin™ has a potential worldwide market of more than three million people with diabetic heart failure.
1 G.J.S. Cooper, Y.-K. Chan, A.M. Dissanayake, F.E. Leahy, G.F. Keogh, C.M. Frampton, G.D. Gamble, D.H. Brunton, J.R. Baker, and S.D. Poppitt: Demonstration of a Hyperglycemia-Driven Pathogenic Abnormality of Copper Homeostasis in Diabetes and Its Reversibility by Selective Chelation: Quantitative Comparisons Between the Biology of Copper and Eight Other Nutritionally Essential Elements in Normal and Diabetic Individuals. Diabetes 54: (5) 2005
2 G.J.S. Cooper and associates: Regeneration of the Heart in Diabetes by Selective Copper Chelation. Diabetes 53:2501-2508, 2004
Notes: Trientine (Laszarin™) is an orally-active small molecule that selectively chelates the oxidised form of copper, which has been used in the treatment of the rare genetic disorder Wilson’s disease for over 20 years.
Protemix is a biopharmaceutical company established over ten years ago, with its operations directed from offices in Auckland and San Diego and research facilities located within The University of Auckland. It discovers and develops novel therapies for cardiovascular disease, diabetes mellitus and other metabolic disorders.