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NPt520-34 Was granted orphan drug status!
The Orphan Drug Act was passed in 1983 with the intention of motivate pharmaceutical companies to target drugs that would benefit people with rare diseases. When you look at the government website https://rarediseases.info.nih.gov/diseases/fda-orphan-drugs they list 12-13 kinds of ALS; however, under each there are only two drugs approved for the treatment of ALS.
- “Edaravone (Brand name: Radicava) – Manufactured by Mitsubishi Tanabe Pharma Corporation
FDA-approved indication: May 2017, edaravone (Radicava) was approved for the treatment of amyotrophic lateral sclerosis (ALS).
- Riluzole (tablet) (Brand name: Rilutek) – Manufactured by Sanofi
FDA-approved indication: December 1995, riluzole (Rilutek) was approved for the treatment of patients with amyotrophic lateral sclerosis. Riluzole extends survival and/or time to tracheostomy.”
Knowing that there are only two approved treatments for ALS, that makes research for new medications to slow the progression, or more importantly stop the onset or cure ALS even more important. Since ALS is classified as a “Rare disease” new drugs or drugs in the research phase such as NPT520-34 are, in my opinion, of great importance.
ALS News Today recently published an article on NPT520-34. This is the drug that has success crossing the blood brain barrier and hopefully will not only benefit pALS, but people who suffer from other rare disease such as Parkinson.
Are any of our members involved with the clinical trials for NPT520-34 or have you been keeping up with the research on this drug? What are your thoughts?
If you are interested in reading the article on ALS News Today the link is https://alsnewstoday.com/2019/08/20/npt520-34-granted-fda-orphan-drug-designation-als/
Also, this article was made into a flash briefing!
Let us know your thoughts and opinions.
- “Edaravone (Brand name: Radicava) – Manufactured by Mitsubishi Tanabe Pharma Corporation
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