Företagen Biogen och Ionis Pharmaceuticals har nyligen publicerat en uppdatering gällande de kliniska prövningar som är aktuella för SMA. NSMA väljer att publicera detta då det ger en bra sammanfattning om några viktiga kliniska studier som pågår i världen.

Dear members of the SMA community,

In response to further requests for information, we want to provide an update on our investigational program to you and the many families around the world who are touched by SMA.  Since our last community post, we have made important progress in moving the compound development program forward.

Our regular meetings with regulators around the world have been productive and collaborative as we work to define the most expeditious path toward regulatory review and potential approval.  It is through this partnership with regulatory agencies that we continue to explore all accelerated pathways to hopefully achieve our ultimate goal of bringing a potential therapy to people with SMA in the shortest period of time.

Over the last few months, we have achieved multiple milestones that move us closer to this goal:

  • Enrollment in our clinical studies continues, which means we are making significant progress.
  • The SHINE study has started, which is the open-label extension for eligible patients who toward gathering the necessary data required for review and approval.
  • Our pivotal, Phase 3 studies, are still on track to complete in 2017.  Of importance:
    • Our childhood Phase 3 study, CHERISH, has completed its target enrollment.
    • Our infant Phase 3 study, ENDEAR, is on course with its target enrollment plan.
  • Our Phase 2 studies are also on track:
    • The EMBRACE study has almost fully enrolled.
    • The NURTURE study, which is enrolling pre-symptomatic newborns that have a genetic diagnosis of SMA, continues to progress with its enrolments.
  • The SHINE study has started, which is the open-label extension for eligible patients who have completed ENDEAR and CHERISH, ensuring all children who have completed one of these studies have the opportunity to receive active therapy.

For more information on the clinical trial studies please see https://clinicaltrials.gov/

We continue to be optimistic about the data from our open-label studies. However, it is important to remember that our well-controlled Phase 3 pivotal studies were designed to definitively prove the safety and efficacy of the investigational compound and these will be required for the approval process.

At every point along the development path, we have asked ourselves and regulators what possibilities might be available to shorten this timeline, and we continue to explore all regulatory options. The consideration of appropriate timing for early access for patients in need is deeply important to us. Our goal is to make the investigational compound available as appropriate to patients once several criteria have been met. These include confidence that doing so will not delay the clinical trials or compromise the data necessary for regulators to make a determination about the safety and efficacy of the investigational compound and after we are able to end the sham-controlled study arm.

Throughout 2016, we will continue to work tirelessly to enroll and conduct our Phase 3 studies in the most expeditious way possible and are committed to updating the SMA community when we can.  Most importantly, we remain steadfast to our goal to bring an approved therapy to the SMA community.


Biogen & Ionis Pharmaceuticals