Rett Syndrome Drug Receives Fast Track Status

As originally reported in Financial Buzz, Rett Syndrome has received promising news: a drug undergoing review, ANAVEX®2-73 (blarcamesine),  has received fast track designation, meaning patients may be able to receive access sooner to the promising pharmaceutical.

Rett Syndrome

Rett syndrome is a rare neurological disorder which causes extreme symptoms such as difficulty or the inability to walk, talk, or develop cognitively. The disease, mainly affecting females, starts showing symptoms once the girl has already reached milestones, who then regress, losing the abilities. The syndrome is known for its characteristic hand movements whenever awake.

New Drug

The new treatment has so far been shown to improve their measures of hand movements, the Rett Syndrome Behaviour Questionaire, and the Clinical Global Impression – Improvement. While the same biopharmaceutical company has other experimental drugs in trials, the rare disorder still has no approved treatments. This creates an urgency to meet the serious needs of the patients.

Fast Track Designation

Not just any promising drug can receive ‘fast track’ status, an authorization that speeds up approval processes for drugs with promising results while in trials that respond to urgent unmet needs. Rett syndrome is qualified to receive such a drug because of the debilitating nature of the disease and the lack of current treatment options. The ‘fast track’ designation is meant to help fill unmet needs for extreme cases. It comes with other benefits such as more frequent meetings for the FDA to review the progression, overall more communication and assistance from the FDA to promote an expedited drug process, and allowing accelerated approval or priority review of the drug at later stages.

 

The Rett syndrome community has received various advances in research in recent years that has caused excitement for the future of the disease. This may speed that up.

 


What are your thoughts on this story? Share your stories, thoughts, and hopes with the Patient Worthy community!

Share this post

Share on facebook
Share on google
Share on twitter
Share on linkedin
Share on pinterest
Share on print
Share on email
Close Menu