Chugai Receives Orphan Drug Designation in Japan for RG6042, a Disease-Modifying Therapy for Huntington’s Disease

February 19, 2020 / Bioon -- Chugai Pharmaceutical, a Japanese pharmaceutical company controlled by the Swiss pharmaceutical giant Roche, recently announced that the Japanese Ministry of Health, Labour and Welfare (MHLW) has granted orphan drug designation (ODD) to its investigational drug RG6042 (formerly known as IONIS-HTTRx) for the treatment of Huntington's disease (HD). RG6042 is an antisense RNA drug that has been proven to reduce the production of mutant huntingtin protein (mHTT), the underlying cause of HD. mHTT is a protein produced by the gene associated with Huntington's disease. Currently, the global Phase III clinical study of RG6042 (GENERATION HD1) is ongoing.

Dr. Yasushi Ito, Executive Vice President and Co-Head of the Project and Lifecycle Management Department at Chugai Pharmaceutical, stated: “Huntington’s disease is aHeredity“Huntington’s disease is designated as an intractable disease in Japan. Currently, only symptomatic treatments are available, and there is a high unmet medical need for potential new therapies. We will continue to collaborate with Roche to advance clinical studies, aiming to bring RG6042, the first disease-modifying therapy, to patients with Huntington’s disease.”

Huntington’s disease (HD), also known as Huntington’s chorea, is an autosomal dominantHeredityHuntington’s disease, a neurodegenerative disorder, is characterized not only by psychiatric symptoms and cognitive changes but also by involuntary movements, primarily chorea. The disease was first described by the American physician George Huntington in 1872, after whom it is named. The primary cause is a mutation in the huntingtin gene on chromosome 4, which leads to the production of mutant huntingtin protein (mHTT). mHTT gradually aggregates within cells, forming large molecular complexes that accumulate in the brain and impair neuronal function. Onset typically occurs in middle age, with initial manifestations of choreiform movements. As the disease progresses, patients gradually lose the ability to speak, move, think, and swallow. The condition typically advances over a course of 10 to 20 years, ultimately leading to death. As there are currently no disease-modifying treatments available, management primarily focuses on symptomatic relief for involuntary movements and psychiatric symptoms.

RG6042 (formerly known as IONIS-HTTRx) is an antisense RNA therapeutic designed to reduce the production of all forms of huntingtin protein (HTT), including the mutant variant (mHTT), which is the underlying cause of Huntington’s disease (HD). RG6042 offers a unique approach to treating all HD patients, regardless of their specific HTT mutation. A previously reported Phase I/II clinical study demonstrated that RG6042 significantly reduced mHTT levels. Results showed that after three months of treatment with the two highest doses of RG6042, the mean level of specific HTT in the cerebrospinal fluid (CSF) of HD patients decreased by 40% (with reductions of up to 60% in some cases). Furthermore, at the most recent assessment, mHTT levels in the CSF continued to decline in the majority of patients (approximately 70%). RG6042 was well tolerated in this study.

In the European Union and the United States, RG6042 was granted Orphan Drug Designation (ODD) for the treatment of Huntington’s disease in May and December 2015, respectively. Additionally, in August 2018, the European Union granted RG6042 Priority Medicines (PRIME) status for the treatment of Huntington’s disease. (Bioon.com)

Original Source: Chugai Receives Orphan Drug Designation for RG6042 in Huntington's Disease from the MHLW