According to a story from GlobeNewswire, The pharmaceutical company AveXis, Inc., a company that specializes in the development of gene therapies, recently announced that its candidate AVXS-101 was awarded SAKIGAKE designation. AVXS-101 is in development for treating type 1 spinal muscular atrophy. The designation was given after preliminary data results from phase I clinical trials.
Spinal muscular atrophy (SMA) is a rare genetic disorder in which a deficiency in the expression of the SMN protein causes the death of the motor neurons, which are the nerve cells that are responsible for muscle movement. The result is if the progressive wasting and degeneration of the skeletal muscles, which can ultimately become fatal in its most severe types onces the breathing muscles become involved. Type 1, for which AVXS-101 has received SAKIGAKE designation, is the most severe type of spinal muscular atrophy. Symptoms first appear at six months old and muscle wasting progresses rapidly. Most people with this type do not survive beyond their second birthday, though in about ten percent of cases people may manage to survive into adulthood with breathing assistance. To learn more about spinal muscular atrophy, click here.
SAKIGAKE designation is the Japanese equivalent of Breakthrough Therapy designation in the U.S. or the EU’s PRIority MEdicines (PRIME) scheme. SAKIGAKE was first implemented in 2014 and is meant to help encourage development and research which will allow qualifying treatments access to an expedited period of regulatory review. In order to qualify, for example, a drug must address a severe disease and must demonstrate promising results in trials. SAKIGAKE allows for an application review period that is halved from the typical year-long period to six months.
