Roche:EMA Grants PRIME Designation For RG6042 For Huntington’s Disease Treatment

Swiss drug major Roche (RHHBY) said that the European Medicines Agency or EMA has granted PRIME or PRIority MEdicines designation for the company’s investigational medicine RG6042 (formerly known as IONIS-HTTRx) for the treatment of people with Huntington’s disease or HD.

RG6042 has demonstrated its ability to reduce the toxic mutant huntingtin protein (mHTT), which is believed to be the underlying cause of HD, 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.

Roche noted that it 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.

by RTTNews Staff Writer

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