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FDA Committee Unanimously Recommends Approval for Spark’s RPE65 Gene Therapy – Final Decision Due in January 2018

Ashley and Cole Carper traveled from Little Rock, AR, to tell their family’s story at the FDA hearing.

Ashley and Cole Carper traveled from Little Rock, AR, to tell their family’s story at the FDA hearing.

Spark Therapeutics has taken a major step closer to gaining marketing approval for its vision-restoring gene therapy for people with RPE65 mutations causing Leber congenital amaurosis (LCA) and retinitis pigmentosa. At the conclusion of a public hearing on October 12, 2017, an advisory committee comprised of FDA-selected experts voted unanimously – 16 to 0 – to recommend approval. The FDA is due to make a final decision on marketing approval for the treatment, known as voretigene neparvovec, by January 12, 2018.

The event held at FDA headquarters included the presentation of trial results from Spark representatives, as well as compelling testimony from patients, family members, and industry stakeholders.

Twenty-four-year-old Katelyn Corey told hearing attendees that before receiving the treatment, her constant adaptation to dwindling vision didn’t leave time for much else in her life. But her circumstances changed dramatically in December 2013, after she received the RPE65 gene therapy in Spark’s Phase III clinical trial.

“Within days, I could see vibrant colors. I could even see the Philadelphia City Hall clock tower at night,” she said. “Also, I can go to a restaurant and see everything by candlelight, and I can see stars in the night sky.” Katelyn recently earned a master’s degree in epidemiology and works as a research analyst for the U.S. Department of Veterans Affairs.

Eleven-year-old Cole Carper, the youngest speaker at the hearing, said he loves playing with Legos now that he has better vision thanks to the gene therapy. His 13-year-old sister, Caroline, who was also in Spark’s Phase III study, enjoys reading print books instead of Braille. She’s also preparing for a role in the play “Shrek”- something her mom, Ashley, said would have been very difficult before treatment.

Cole and his mom were headed down to the National Mall in Washington, D.C., after the meeting to take in the sites. Cole was especially looking forward to checking out the Spy Museum.

The Foundation’s own chief research officer, Dr. Stephen Rose, also gave testimony at the hearing. “The approval of this gene therapy will be life-changing for people with severe vision loss due to RPE65 mutations,” he said. “FDA approval of this groundbreaking treatment would provide strong momentum for the advancement of several other vision-saving gene therapies under development in labs and clinics around the world.”

If approved, voretigene neparvovec has the potential to be the first FDA-approved gene therapy for the eye and for any inherited disease. The investigational treatment, the result of more than two decades of research and development, delivers functional copies of the RPE65 gene directly into the retina thereby compensating for nonfunctional, mutated copies. FFB was an early financial supporter of that work, investing $10 million for RPE65 lab and clinical research.

“FFB applauds the investigative teams at the University of Pennsylvania, University of Florida, Children’s Hospital of Philadelphia, and Spark Therapeutics for bringing the therapy into and through clinical trials that have demonstrated safety and strong efficacy,” added Dr. Rose


21 Responses to 'FDA Committee Unanimously Recommends Approval for Spark’s RPE65 Gene Therapy – Final Decision Due in January 2018'

  1. Katey Bader says:

    My Son has Retintis Pigmentosa and his test came out he has gene called EYS I am wondering if you have any clinical trail for that Gene.. Thanks

  2. Jone says:

    Is there any hope for PRPH2 MUTANTS?

  3. Carolyn Isley says:

    This is absolutely amazing. This is the answer to many prayers for a cure for hereditary blindness. Four generations of my family has suffered with Retinitis Pigmentosa. I have 3 siblings that are almost completely blind. I believe this is the beginning of a cure for all hereditary blindness. God bless you!!!

  4. angela stancil says:

    Please help my nephew who is 24 yrs old. He has retinoschiosis and such a wonderful person. He seeks his own life and one of the most profoundly curious and witty person I know. The gentleman lives between his aunts house and desires to work, travel, learn everything he can. He is so deserving and has such a potential.
    His name is Daniel Doggett.

  5. Linda g. Jones says:

    This is wonderful how do I get involved???
    I am in the gene therapy program at Columbia in Manhattan but have yet gotten the ok for treatment.

  6. David Jirele says:

    Very good news. Is there more progress on stem cell treatments?

  7. Jean Fowler says:

    My husband, Bob, would definitely be interested in this treatment since he (and his mother and one daughter) has Retinitis Pigmentosa. Please keep us informed as to when this treatment would be available. We are in the Houston area.

    • EyeOnTheCure says:

      Hi Jean,

      Has anyone been genetically tested? That is an important first step toward getting into a clinical trial. You can reach out to http://www.idyourird.com and see if you qualify for their no-cost genetic testing. The above trial is only for people with mutations in RPE65.

  8. Tracey says:

    Would someone with Gyrate Atrophy benefit from Gene Therapy?

  9. Tammie Wirebaugh says:

    My father was diagnosed with retinitis pigmentosa at around the age of 18 yrs. He is now 68 yrs and would love to see his grandchildren and now great grandchildren. If you have any information on how he might be able to be in one of your trials or just more information on the gene therapy that would be very much appreciated. Thank you for your time. My fathers name is Richard Wirebaugh.

  10. m.ebrahimi says:

    Thanks for the vision therapies info

  11. Nicole Maer says:

    This is ground breaking stuff; hope to millions of people out there who have congenital eye defects, and have limited pharmacologic treatment options. It is heartwarming to read about Katelyn and Cole. Hoping to hear and experience more of these success stories, after the PDUFA date of Jan 12, 2018. Keeping fingers crossed.

  12. Jasmina says:

    Can it cure also nystagmus my little girls have both LCA and nystagmus .
    I hope they can also cure nystagmus along with the LCA . My both daughters have since birth.

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