Neuren and the Orphan Drug Status: An Overview

by Dr. Steve Kaminsky, Chief Science Officer,


This week’s news that Neuren Pharmaceuticals has been granted Orphan Drug Status by the FDA for NNZ-2566, now known as Trofinetide, is good news for families who live with Rett syndrome. It is good news because it means that developing an effective treatment for Rett syndrome has - for the first time - been made a priority by the manufacturer and the regulator.

Read Neuren’s press release here.

So what does an “Orphan Drug Status” designation actually mean?

The US federal government recognized the lack of incentives for pharmaceutical companies to develop cures and treatments for rare diseases, so they enacted the Orphan Drug Act in January 1983.  This law established the “orphan” designation for conditions with fewer than 200,000 patients in the U.S. In addition, it allowed the FDA to incentivize drug companies to develop drugs for orphan-designated disorders.

What are these incentives?

  1. Seven years of exclusivity for the particular indication named in the orphan designation
  2. Tax credits for qualified clinical testing.
  3. Pricing incentives that the drug is not subject to a prescription drug user fee.

What does the “Orphan Drug Status” being granted mean for the patient and in particular, Rett syndrome?

Orphan drug status does not mean that the drug is approved for use. It means that the drug company qualifies for certain benefits (described above) and needs to continue through the FDA process. Effectively, this means that the regulators and the manufacturers have made this drug a priority for Rett syndrome. More importantly:

  1. The data has been reviewed by the FDA in regards to the claims of the drug and the FDA approves what Neuren has done. *This is important for Rett syndrome because it means the FDA has looked at the data on how NNZ-2566 [Trofinetide] works and they like what they see.
  2. Neuren is committed to developing treatments for this orphan disease - they are in it with us for the long-haul. 

How can I explain the benefits of this breakthrough to friends and family? 

If you’re trying to explain why the news about Neuren being granted Orphan Drug Status for NNZ-2566 [trofinetide] is important to your friends, family and the circle of people have supported or invested in our research fundraising efforts, here are some things you can focus on:

  1. The FDA has looked at the results of the trial and what trofinetide does and they like what they see, this is good news for totally obvious reasons.
  2. Neuren is in for the long haul. They are committed to the research, the drug and Rett syndrome.
  3. There are very valid and extremely important reasons why the information on specifically what the drug does are not released to the general public. Part of the regulatory process for clinical trials involves keeping the specifics of the data unpublished to maintain maximum integrity for the rest of the process.

Along with yesterday’s announcement, Neuren created an informative video explaining the science behind Trofinetide for Rett syndrome and other neurological disorders.  Larry Glass, CSO of Neuren, narrates the video and makes the science very easy to understand.

Special Thanks

Many thanks go to all those at Neuren and the clinical trial teams at the Baylor College of Medicine, University of Alabama Birmingham and the Gillette Children's Specialty Healthcare Center for accelerating clinical research and to our donors and families, as well as Cure Rett for their continued partnership in helping to make this possible!

Next Steps? 

Neuren is working with the FDA to review all the data collected on the Phase II clinical trial in women with Rett syndrome and will discuss the design of the next clinical trial.  Most likely the next trial will be in a younger population and if found to be effective in this trial they will then work with the FDA on a Phase III clinical trial. 

We look forward to sharing further developments as they occur!