FDA Panel Votes Unanimously to Approve New Gene Therapy

October 13th, 2017 by Dr. Mary Sunderland

History was made on World Sight Day, October 12, 2017, when an FDA advisory panel voted unanimously to approve a new gene therapy, Luxturna, designed to treat inherited blinding eye diseases caused by mutations in the RPE65 gene.

We have been following this story closely over the past few years and celebrating every milestone along the way. Yesterday’s milestone was a true breakthrough, as it was the FIRST gene therapy to reach this regulatory stage!

As many of our long-time supporters know, gene therapies have been under development since the 1990s. This is when the work on Luxturna began, led by Dr. Jean Bennett, a physician and scientist, now at the University of Pennsylvania. The therapy’s pioneering path from Dr. Bennett’s laboratory in the 1990s to the FDA review panel yesterday is paving the way for a new era of gene therapies.

Although yesterday’s vote is not the final stage, these recommendations will play a critical role when the FDA considers the Biologics License Application for Luxturna. This application is the next regulatory step, which is being fast-tracked because Luxturna is considered a breakthrough therapy that stands to benefit children living with rare diseases. If all goes well, Luxturna will be available as a treatment in the US in early 2018.

In support of yesterday’s vote, FFB Canada submitted a letter highlighting the global importance of this new therapy, emphasizing that many Canadians are living with inherited blinding eye diseases and no treatment options.

Thank you to all of the FFB supporters who helped to make this milestone possible! I would like to give an extra-special thank you to Jack McCormick, who lives with a blinding eye disease, Lebers congenital amaurosis (LCA), that is caused by RPE65 mutations. Jack played a critically important role educating the Spark Therapeutics team about living with LCA. We are proud that Jack’s story and leadership are shaping this new era of innovative therapeutics. Moreover, we are so thankful that Jack shared his story at the FFB’s Comic Vision fundraising events this year and is also co-chairing the FFB’s 2017 Young Leaders Summit. Jack is committed to creating a future where all people have equal opportunities – his work is making a difference!

The success of Spark Therapeutic’s Luxturna has helped to revive interest and investment in the future of gene therapies, which is wonderful because there are more than 225 genetic mutations that are known to cause blindness. Currently, there are many ongoing gene therapy trials for blinding eye diseases. Notably for our FFB Canada community, the gene therapy company AGTC is currently enrolling patients living with x-linked retinoschisis from Canada and the US into ongoing gene therapy trials. You can learn more by listening to our Facebook Live session about gene therapy or attending an upcoming Vision Quest event – we will be in Calgary on October 21st!

Being the first certainly has its challenges, which is why it is important to celebrate and recognize these regulatory milestones just as we celebrate scientific discoveries. The regulatory work that Spark is doing will certainly help to expedite the regulatory process in Canada. At the FFB, we are actively working to ensure that Canadians will have access to these innovative and truly life-changing treatments.

Every day, we are getting closer to making new treatments a reality – sometimes these are just baby steps forward, but yesterday was a giant leap ahead!