Radicava Approved By FDA!
May is ALS awareness month and we are pleased to announce genuine progress in modernizing the clinical trial process and in fighting this disease. On May 1, 2017 Hope NOW for ALS facilitated a meeting with key officials at the FDA and was joined by the ALS Association. Devising smarter clinical trial designs (“21st Century Clinical Trials”) will speed up the pace of discovery and lead to faster approval of viable treatments, while also funneling resources to the most promising therapeutics. The involvement of the ALS patient community is largely responsible for this progress which represents a seismic shift in trials for ALS. We will continue to collaborate with others in the ALS community to accelerate the process of finding additional treatments and a cure.
On May 5, 2017 the FDA approved edaravone (brand name Radicava) for the treatment of ALS. Developed by MT Pharma America, this drug was approved in Japan in 2001, originally to protect against the effects following ischemic stroke, and approved there for ALS in 2015. The FDA actively engaged with the sponsor and approved the treatment without requiring additional trials to be conducted in the US. We believe the FDA acted with the flexibility granted to them by Congress by accepting data from trials conducted outside the U.S. without not sacrificing the statistical rigor needed to show efficacy of the treatment. While Radicava is not a cure, it has been shown to significantly slow progression in some patients (up to 33% slower progression in PALS <2 years post-diagnosis). We believe it represents an important new treatment option for ALS patients and demonstrates to other medical sponsors and investors that effective treatments can be developed - and expediently approved - to fight ALS.
Hope NOW for ALS is committed to active engagement with the FDA, the ALS Association, and other ALS organizations to promote clinical trial designs tailored to the nature of this disease. The past few decades have demonstrated that standard trial designs have been ineffective in providing clear answers on the effectiveness of potential treatments for ALS. Inefficient trial designs make it hard to identify effective treatments and can lead to wasted investments in treatments that do not work. The FDA acknowledges the difficulties in designing trials utilizing homogeneous treatment and placebo arms for a highly variable disease like ALS, and has indicated receptivity to newer, customized trial designs. ALS has significant variations and lacks a predictable course. Some of the ideas now circulating regarding ALS-specific trial designs include incorporating historical controls, predictive algorithms, and leveraging individuals existing progression data. In addition, more information can be gleaned from trial data by controlling for severe adverse events (SAEs) unrelated to the treatment. In instances where researchers feel that a placebo arm is necessary, crossover designs can be utilized to increase patient access to the treatment, and enhance the power of the data.
We have good reason to believe that significant progress is, and will, continue to occur. Together, with the sustained involvement of the patient community, we will accelerate the pace of finding a cure.