Huntington's disease in the progressive limelight

Progress so far

For many decades, there have been many challenges and a level of improvement in numerous neurodegenerative conditions. Over the past two years, uniQure’s data received a green signal for therapies shaping a new future for a subtype of amyotrophic lateral sclerosis (ALS) and Alzheimer’s. The approval was justified as it highlighted 75% of reduction in the Huntington condition. The families and patients received a certain level of relief.

Learning about the severity of the life-threatening neurodegenerative disease emerging due to the CAG consistent presence in the first exon of the Huntington gene made healthcare enthusiasts aware of the consequences and the need for treatment. For around 32 years, this gene has provoked incremental progress. Following this, Sage Therapeutics, Roche, Wave Life Sciences, and more giants were not successful in tackling this disease.

uniQure pathway

uniQure's pathway towards bringing new solutions and hope among 41,000 Americans suffering from Huntington's and above 200,000 with genetic mutation was quite impressive. The company was all set to envelope biologics license application, but the FDA has some other plans for this therapy. At one of the meeting the news was out.

The news states that the company pointed out that the ball is not in our court, as the agency feels that the data from its Phase 1/2 trials is not suitable to prove the primary evidence valid and does not contribute the most to the BLA submission. The company has a long way to go to introduce new robust data to justify its pathway.

Other giants' stumbling efforts linked to Huntington

Ionis and Roche put a pause to their Phase 3 trial for their antisense oligonucleotide (ASO) tominersen. After so many reasons, the companies had to reconsider the trial geography that would allow them to obtain actual effectiveness, efficacy and safety results without any second thought. Similarly, Wave also didn’t get success in two of its ASO therapies, but the company came back with WVE-003. This next-generation ASO may be a part of the Phase 2/3 trial this quarter.

Alongside the Mass-related leading biotech, Cambridge is also in a queue to strengthen its approval by implementing the slowing of caudate atrophy. This caudate atrophy is connected to the mitigation in mutant Huntington protein. These ups and downs in the Huntington’s pathway have kept the giants striving throughout and challenged their potential. The year is awaiting new therapies, clinical progress, advancement and improvement in Huntington's condition.