Earlier this week at the annual PPMD Connect Conference in Orlando, Akashi Therapeutics CMO Dr. Diana Escolar presented an update on the HT-100 program. “We are confident that we will have a path to allow the program to move forward safely,” she explained. The company plans to submit a data package to the FDA by the end of July and expects to receive guidance from the Agency no more than 30 days later. The plan is to resume dosing for those patients who had safe and well tolerated long-term therapy with HT-100 and to continue development of this experimental treatment with new patients.
This graph shows the muscle strength scores of boys on HT-100 (blue line) vs. an untreated control group. Boys on HT-100 got stronger!
Meanwhile, Marathon Pharma has submitted a New Drug Application for deflazacort, a steroid that appears to have better efficacy and fewer side effects than prednisone. Deflazacort is currently only available outside of the US, which means patients are forced to battle international red tape and pay out of pocket for the best standard of care. Thankfully, Marathon is taking the essential steps to bring this much-needed steroid to US families.