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FDA Fast-Tracks Heart Failure Gene Therapy

Heart check

(Gerd Altmann, Pixabay)

14 December 2017. A biotechnology company developing transfers of healthy genes to treat chronic diseases is receiving an accelerated review of its heart failure therapy from the U.S. Food and Drug Administration. Renova Therapeutics in San Diego says its lead product, code-named RT-100, will receive fast-track status at FDA for treating heart failure.

Heart failure is a condition where the heart cannot pump enough blood to meet the body’s needs, a condition affecting some 5.7 million people in the U.S., according to Centers for Disease Control and Prevention. Most cases of heart failure affect both the left and right sides of the heart, although in some cases only one side is affected. The leading causes of heart failure are conditions that weaken or damage the heart, including coronary heart disease, high blood pressure, and diabetes.

Renova says RT-100 is being developed as a therapy for heart failure with reduced ejection fraction, a condition where the heart’s left ventricle, one of its main pumping chambers, cannot fill or eject blood. The treatments transfer a healthy gene, in this case a gene encoding instructions for a protein called adenylyl cyclase type 6, or AC6, that regulates heart muscle functioning, which in many heart failure patients is limited or restricted. The one-time AC6 gene transfer is made into heart muscles with an inactivated benign virus during cardiac catheterization, a commonly performed diagnostic technique.

The company tested RT-100 in an intermediate-stage trial in patients with heart failure. The results, reported in March 2016, show the treatments enable patients receiving the healthy gene to increase the capacity and ejection of their left ventricles. In addition, the gene transfer recipients saw their symptoms of heart failure decrease after 12 weeks, compared to placebo recipients. Rates of adverse effects were similar between the treatment and placebo participants.

A late-stage clinical trial, expected to begin in February 2018, will test RT-100, also against a placebo, among 536 individuals with a history of heart failure, and left-ventricle ejection fractions between 10 and 35 percent. This study will look primarily for the rate of hospitalizations for heart failure, but also scores on standard heart function scales, occurrence of any heart failure events, and deaths from cardiovascular disease or other causes in the 12 months following treatment.

Fast-track status from FDA qualifies Renova for more frequent meetings and written communications with the agency as RT-100 is reviewed, as well as rolling reviews that allow the company to submit its new drug or biologics application in sections rather than all at once. FDA designates drugs for fast-track reviews if they show superior effectiveness against serious disorders, or address emerging or anticipated public health needs.

Renova is also developing therapies with genes that encode instructions for peptides, short protein-like chains, that improve the uptake of glucose in patients with type 2 diabetes. As reported by Science & Enterprise in February 2017, a technology received a U.S. patent, which Renova licensed for producing peptides from genes that treat type 2 diabetes and heart failure.

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