PRIME designation granted by European Medicines Agency for RG6042 for treatment of Huntington’s disease
¨ European Medicines Agency PRIME (PRIority MEdicines) status is granted to medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options
¨ RG6042 has the potential to be the first therapy targeting the underlying cause of Huntington’s disease, a fatal neurodegenerative rare disease
¨ Third PRIME designation for a Roche medicine
Roche today announced that the European Medicines Agency (EMA) has granted PRIME (PRIority MEdicines) designation for the company’s 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 HD, in a Phase I/IIa study.1 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.2
“We are very pleased that the European Medicines Agency has granted PRIME designation for RG6042, as there is an urgent medical need to find treatment options for families affected by Huntington’s disease,” said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. “Preliminary data on RG6042 were the first to show that levels of toxic mutant huntingtin protein can be lowered in adults with Huntington’s disease, and we are working closely with the EMA and other health authorities to initiate a global phase III study as soon as possible.”
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.1 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.1 These data were shared at the CHDI 13th Annual HD Therapeutics Conference in March 2018,3 and updated results were presented at the American Academy of Neurology (AAN) Annual Meeting in April 2018.4
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.