The EMA has granted PRIME designation to Roche's RG6042 for treatment of Huntington’s disease.

PRIME (PRIority MEdicines) status is granted to medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options, providing a pathway for accelerated evaluation by the agency, and thus potentially enabling them to reach patients earlier.

RG6042 has the potential to be the first therapy targeting the underlying cause of Huntington’s disease, a fatal neurodegenerative rare disease.

The designation 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 (~70%) as of the last measurement in the study.3 RG6042 was well tolerated in this short initial study.

Roche will initiate a pivotal phase III study to evaluate RG6042 in a larger patient population to further characterise the safety profile and determine if it can slow the progression of HD in adults.