Receive updates in your inbox.
A branch of the European Medicines Agency (EMA) has established that the experimental treatment of prilenia, pridopidine, will be designated as an orphan drug to treat other people with amyotrophic lateral sclerosis (ELS).
Medicines that have the prospect of becoming effective remedies for rare, life-threatening or debilitating chronic diseases affecting up to one user in 2000 are eligible for orphan drug designation in Europe.
The statute gives the developer of a drug several incentives designed to accelerate the commercialization of the therapy. These come with testing protocols and a 10-year era of exclusivity in the market after approval.
“This positive EMA review validates the possibility that pridopidine has an effect on the devastating evolution observed in ALS patients,” Michael Hayden, PhD, CEO and co-founder of Prilenia, said in a press release.
The designation will be given within 30 days of the positive opinion, which was issued through the EMA’s Committee for Orphan Medicinal Products (COMP).
Pridopidine is an oral treatment designed to protect nerves from damage by binding and activating the sigma-1 receptor in nerve and glial cells (a type of nerve cell). This receptor regulates biological mechanisms, such as the elimination of poisonous proteins, energy production and the relief of inflammation and cellular tension, which are mandatory for the survival and functioning of nerve cells.
According to Prilenia, mutations that absolutely cause the activity of this receptor to cause a serious juvenile bureaucracy of THEA.
Administered as a small capsule twice a day, pridopidine was found to be and well tolerated in more than 1300 participants examined, causing similar-looking effects to those noted with placebo, the company says on its website.
The remedy also demonstrated neuroprotective benefits in several preclinical models of ELA, which led to preserving the integrity of neuromuscular junctions, connections between nerve and muscle cells, and relieving muscle atrophy (shrinkage).
Comp based its resolution on those preclinical knowledge that appeared as prospects for the preservation of motor function, which the Committee considers an unmet medical necessity. COMP also considered this prospective effect as a merit over the latest treatments received.
Pridopidine is one of the 4 remedies recently included in the HEALEY ALS platform trial (NCT04297683), which studies several prospective remedies for diseases to boost the progression of the most promising ones, while reducing examination costs.
The pridopidine test group (NCT04615923), which recruited its first player in January, will come with 160 adults with sporadic or familial ALS who will get placebo or forty-five mg of the experimental remedy twice daily for 24 weeks.
Recruitment for ridopydine and other screening arms is underway at 54 sites in the United States; Here you have more data, the effects are expected in the third quarter of 2022.
“We are encouraged by the various effects of pridopidine which has a favorable effect on neurodegeneration in ALS models and look forward to reading the trial later next year,” Hayden said.
Pridopidine is already an orphan drug for Huntington’s in the United States and Europe and is being studied in a phase 3 trial of Huntington’s.
Receive updates in your inbox.
This site is strictly a site of news and data about the disease. It does not provide medical recommendations, diagnoses, or treatments. This content is not intended to update professional medical recommendations, diagnoses or treatments. Always seek the recommendation of your doctor or other qualified person. fitness care pro for any questions you may have about a fitness problem. It’s never the recommendation of a fitnesscare pro and don’t wait to look it up for anything you’ve read on this website.