The European Medicines Agency ( EMA ) has granted PRIME ( PRIority MEdicines ) designation for an investigational medicine RG6042 ( formerly known as IONIS-HTTRx ) for the treatment of people with Huntington’s disease ( HD ).
RG6042 has demonstrated its ability to reduce the toxic mutant huntingtin protein ( mHTT ), which is believed to be the underlying cause of Huntington’s disease, in a phase I/IIa study.
PRIME is a designation implemented by the EMA to support data generation and development plans for promising medicines, providing a pathway for accelerated evaluation by the Agency, and thus potentially enable them to reach patients earlier.
PRIME designation for RG6042 is primarily based on the data from an exploratory phase I/IIa trial of RG6042 that demonstrated a significant reduction in mHTT, which breaks down the nerve cells in the brain.
The study demonstrated a mean 40% ( up to 60% ) reduction of the specific HD protein in the cerebrospinal fluid ( CSF ) of adult patients treated with RG6042 for three months at the two highest doses.
Furthermore, levels of mHTT measured in the CSF were still declining in the majority of treated patients ( approximately 70% ) as of the last measurement in the study.
RG6042 was well tolerated in this short initial study.
RG6042 is a second-generation modified antisense oligonucleotide ( ASO ) designed to reduce the production and levels of mHTT protein by targeting human HTT mRNA.
RG6042 is the most advanced compound in clinical development to target toxic mutant huntingtin protein, which is believed to be the underlying cause of HD.
Treatment with RG6042 has the potential to slow or stop disease progression in all people with Huntington’s disease.
Huntington’s disease is a rare genetic, progressive condition that causes the nerve cells in the brain to break down, which severely affects a person’s everyday functions such as mobility and thinking.
It has a devastating impact on people living with the disease, and the hereditary nature of Huntington’s disease means it profoundly affects entire families.
As the disease progresses, people with Huntington’s disease may develop personality changes, difficulty walking and swallowing, as well as having a significant cognitive impact.
Survival ranges from approximately 10-20 years following motor onset of the disease.
There is no known cure for Huntington’s disease and no approved therapies that treat the underlying cause.
The estimates for the number of people affected by Huntington’s vary between geographic regions. Huntington’s disease is the most common monogenic neurological disorder in the developed world, with an estimated prevalence of approximately 3.5–7/100,000 in North America, Western Europe, and Australia. ( Xagena )
Source: Roche, 2018