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EMA grants 'PRIME' designation to Roche's investigational Huntington's treatment

By Josh White

Date: Friday 03 Aug 2018

EMA grants 'PRIME' designation to Roche's investigational Huntington's treatment

(Sharecast News) - Roche announced on Friday that the European Medicines Agency has granted 'Priority Medicines' (PRIME) designation for its investigational medicine RG6042 - formerly known as IONIS-HTTRx - for the treatment of people with Huntington's disease.
The Swiss drugmaker said RG6042 had demonstrated its ability to reduce the toxic mutant huntingtin protein - mHTT - which was believed to be the underlying cause of Huntington's disease (HD), in a Phase I/IIa study.

It described PRIME as 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.

"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 Roche's chief medical officer Sandra Horning.

"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 was said to have been primarily based on the data from an exploratory Phase I/IIa trial of RG6042, which demonstrated a "significant reduction" in mHTT, which breaks down the nerve cells in the brain.

The study apparently demonstrated a mean 40%, and up to 60%, reduction of the specific HD protein in the cerebrospinal fluid of adult patients treated with RG6042 for three months at the two highest doses.

Additionally, levels of mHTT measured in the cerebrospinal fluid were still declining in the majority of treated patients - around 70% - as of the last measurement in the study.

Roche said RG6042 was "well tolerated" in the short initial study.

The data was shared at the CHDI 13th Annual HD Therapeutics Conference in March, and updated results were presented at the American Academy of Neurology annual meeting in April.

Roche added that it would 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.

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