Huntington's "disease modification therapy"! Roche antisense RNA therapy rg6042 (ionis-httrx) was awarded orphan drug qualification in Japan!

February 19, 2020 / BIOON / -- Chugai, a Japanese pharmaceutical company controlled by Roche, a Swiss pharmaceutical giant, recently announced that MHLW has awarded rg6042 (formerly known as Ionis httrx) as a research drug to the company as an orphan eligible for treatment of Huntington's disease (hd) Rg6042 is an antisense RNA drug, which has been proved to reduce the production of mutant Huntington's protein (mhtt), the fundamental cause of HD At present, rg6042 phase III global clinical research (generation HD1) is in progress Dr Yasushi ITO, executive vice president of Sino foreign pharmaceutical and co director of project and life cycle management department, said: "Huntington's disease is a genetic disease, which is recognized as a refractory disease in Japan Currently, only symptomatic drugs are available, and there are high unmet medical needs for potential new therapies We will continue to work with Roche to advance clinical research and bring rg6042 as the first disease modification therapy to patients with Huntington's disease " Huntington's disease (hd), also known as grand chorea or Huntington's chorea, is an autosomal dominant neurodegenerative disease In addition to mental symptoms and cognitive changes, it is accompanied by involuntary movements, mainly dance movements The disease was discovered in 1872 by George Huntington, an American medical scientist, and hence the name The main cause of the disease is the mutation of huntingtin gene on chromosome 4, which produces the mutant protein mhtt Mhtt gradually gathers together in the cell, forming a large molecular mass, accumulating in the brain and damaging the function of nerve cells In general, patients develop middle-aged disease, which is characterized by dance like movements As the disease progresses, they gradually lose the ability of speaking, acting, thinking and swallowing The disease will continue to develop for about 10 to 20 years, and eventually lead to death Because there is no clear treatment for the disease, patients usually receive symptomatic treatment for involuntary movement and mental symptoms Rg6042 (formerly Ionis httrx) is an antisense RNA drug designed to reduce the production of all types of Huntington protein (HTT), including mutant (mhtt), which is the root cause of HD Rg6042 provides a unique approach to treat all HD patients, regardless of their respective htt mutations A previously published phase I / II clinical study showed that rg6042 significantly reduced mhtt levels The results showed that specific htt levels in cerebrospinal fluid (CSF) of HD patients decreased by an average of 40% (up to 60%) three months after treatment with two highest doses of rg6042 In addition, in the most recent measurement, mhtt levels continued to decline in CSF in most patients (about 70%) In this study, rg6042 was well tolerated In the European Union and the United States, rg6042 was granted orphan drug qualification (odd) to treat Huntington's disease in May and December 2015, respectively In addition, in August 2018, the EU granted rg6042 the priority drug qualification (prime) for the treatment of Huntington's disease Chugai receives orphan drug design for rg6042 in Huntington's disease from the MHLW