Novartis Mavoglurant (AFQ056)Earlier this month Novartis released results of the Phase IIb/III studies with mavoglurant (AFQ056) in adolescents with Fragile X syndrome (FXS). The trial results from the study of adults were released late last year. Both studies did not meet the primary endpoint of showing significant improvement in abnormal behaviors compared to placebo. Earlier today the company announced that it will no longer continue development of mavoglurant in FXS, nor will it continue the long-term extension studies of mavoglurant in FXS. The company did however make clear that given the significant unmet medical need, they remain committed to continuing their search for new treatments for rare conditions.

Company representatives acknowledged that this news will be disheartening for those affected by and involved in the management of FXS and have assured us that this decision was not taken lightly. They also acknowledged the collaborative efforts of the broader Fragile X community in the studies and noted the scientific community has gained important knowledge from the AFQ clinical trial program in FXS – including learning about the science, the condition and how to conduct clinical trials of this magnitude in our patient population.

All study participants will be contacted by their individual clinic. Please remember there are many people for each site to contact and this could take some time. Many of you will read about this development here before hearing it from your clinic. We are assured that participants will be given instructions on the process of stopping AFQ056. All participants will then need to work with their own doctor to determine the next, best step in treatment. If caregivers and participants involved in the extension studies wish to seek immediate information they are instructed to contact their investigator and study site.

In the short time since learning of this we have reached out to prominent members of the research community for their initial perspective on this news. One message we’ve been asked to pass-on is that it is important for the community to understand that the fact that these particular trials did not show benefit does not address the question of whether the drug modifies FXS and does not rule out the validity of the mGluR5 theory. Other feedback we’ve received is that these studies did not address whether it is possible to modify FXS in much younger individuals (as only adolescents and adults were studied), and with regard to the older individuals studied, three months of treatment could not be reasonably expected to reverse 12-45 years of altered signaling in the brain.

All of us at the National Fragile X Foundation (NFXF) know that the news that your family member will need to soon stop taking AFQ056 is very upsetting. We have heard your descriptions of the improvements you have observed in your family members during the trial, or the extension, and we know how worried you will likely be about the change.

Over the past few months I have had the opportunity to meet with Novartis and monitor the course of these trials and all of us at the NFXF are committed to helping our community cope and make the transition to the next chapter in the quest for safe and effective treatments for FXS.

Please, stayed tuned, check back for updates at often, work closely with your doctor and remain hopeful and optimistic that new and better treatments are still forthcoming. It is only through our participation in the process that science moves forward.

Lastly, this is sure to be a hot topic at the NFXF conference in July. Novartis will be presenting their results and the top docs in-the-know will be there to share their perspective. As always, you’ll learn about what’s really happening well in advance of any official publication and get perspective from the experts themselves that you can’t get anywhere else. We hope you can join us.


Jeffrey Cohen
Interim Executive Director
National Fragile X Foundation