Please login to the form below

Not currently logged in

Ionis and Roche gain PRIME status for Huntingdon’s disease candidate

More good news for Ionis after first drug approval

Ionis and Roche’s pipeline drug for Huntingdon’s disease has been granted special ‘PRIME’ status by the European Medicines Agency (EMA).

PRIME stands for ‘Priority Medicines’ and the fast-track system has been working for two years, and has similarities to the FDA’s Breakthrough Therapy designation.

The drug, known as RG6042 (formerly named IONIS-HTTRx) has shown the 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, and could be a major step forward in treating the rare, genetically-inherited progressive wasting disease.

The announcement adds to the momentum behind Ionis, which has just gained its first ever drug approval - also in Europe. On 11 July the European Union approved its first-in-class orphan drug Tegsedi (inotersen), for patients with the rare disease hereditary transthyretin amyloidosis (hATTR amyloidosis).

The company specialises in antisense drugs, which ‘silence’ RNA and target diseases much closer to their genetic origins than conventional therapies. While its close rival Alnylam is widely believed to have a superior RNA-targeting platform, Ionis is carving out a significant niche for itself.

C Frank Bennett

Ionis' C Frank Bennett

"PRIME designation for IONIS-HTTRx accelerates the review timelines and enhances interactions with the EMA, which can bring this potentially disease-modifying drug for people with Huntington's disease to regulatory approval faster. This designation will be useful as we work closely with Roche to quickly advance IONIS-HTTRx into a pivotal study," said Dr. C. Frank Bennett, senior vice president of research and franchise leader for the neurological programmes at Ionis.

He added: "This is our second antisense drug to demonstrate a strong safety profile and significant target engagement in the human central nervous system. This profile gives us further confidence in the potential of the many other drugs we have advancing in R&D for the treatment of neurological diseases."

The phase I/II study of RG6042 showed 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. 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.

The news is welcome for Roche and Ionis, particularly as the FDA hasn’t yet given the drug a Breakthrough therapy designation.  Other companies which have PRIME status include gene therapy specialists Bluebird Bio, who are expected to file their LentiGlobin therapy for beta thalassemia later this year.

Article by
Andrew McConaghie

3rd August 2018

From: Regulatory



COVID-19 Updates and Daily News

Featured jobs


Add my company
Merrill Brink International

Merrill Brink International is a leading provider of life sciences, legal, financial, manufacturing and corporate language solutions for global companies....

Latest intelligence

Diversity in clinical trials: looking back at our 2021 blogs
In this blog, we look back at the Innovative Trials' Equality & Diversity (E&D) committee blogs across 2021...
What does the future hold for Light-chain Amyloidosis?
Recent advances in the understanding and treatment are reforming pharma’s approach to the management of this rare disease. With a new standard of care rapidly developing, what does the landscape...
Securing a future for telehealth with immersive market research insights...