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History Is Made: FDA Approves Spark’s Vision-Restoring Gene Therapy

Spark LogoSpark Therapeutics’ vision-restoring RPE65 gene therapy has received marketing approval from the U.S. Food and Drug Administration, becoming the first gene therapy to gain regulatory approval in the U.S. for the eye or any inherited condition.

Known as LUXTURNA™ (voretigene neparvovec), the gene therapy restored vision in a clinical trial for people between the ages of 4 and 44 with Leber congenital amaurosis (LCA) caused by mutations in the gene RPE65. Study participants with severe vision loss reported putting away their navigational canes, seeing stars, being able to read, and recognizing faces of loved ones. Vision restoration has persisted for at least three years. The treatment is also designed to work for people with retinitis pigmentosa (RP) caused by RPE65 mutations.

On October 12, 2017, an FDA Advisory Committee voted 16 to 0 in recommending marketing approval for LUXTURNA.

The Foundation Fighting Blindness invested about $10 million in more than a decade of lab research that made possible the RPE65 gene therapy clinical trial at the Children’s Hospital of Philadelphia (CHOP). FFB-funded research included: studies to understand the role of RPE65 in vison and retinal disease, development of animal models with RPE65 mutations, RPE65 gene therapy testing and development, and support for early clinical research at CHOP.

“This is truly a historical moment in the fight against blindness. We are delighted by the FDA’s approval of LUXTURNA for people with RPE65 mutations,” says Stephen Rose, PhD, chief research officer at FFB. “LUXTURNA’s approval also provides affirmation that gene therapies can be a safe, effective, and commercially viable approach to treating many forms of blindness, providing a big boost to an already burgeoning field. The approval is great news for a broad spectrum of people with inherited retinal diseases.”

Retinal gene therapy clinical trials are underway for people with a wide range of retinal diseases including: X-linked RP, choroideremia, Usher syndrome type 1B, Stargardt disease, X-linked retinoschisis, and achromatopsia.

More than 10 million people in the U.S., and millions more around the world, have retinal degenerative diseases.

“We are thrilled for the patients whose lives will change because of this treatment,” says David Brint, Foundation Fighting Blindness chairman. “And we are also pleased to have this concrete example of the strength of the Foundation’s strategy of investing early in promising treatments in order to attract later industry investment that can usher the treatments through clinical trials and FDA approval.”

“LUXTURNA will be life changing for people with RP and LCA caused by RPE65 mutations. For them, the treatment may well mean the difference between relying on assistive technologies or other people and living a life of independence. Also important is the momentum this approval provides to other gene-based therapies — for the eye and other diseases — now in the pipeline,” says Benjamin Yerxa, PhD, Foundation chief executive officer.

The LUXTURNA gene therapy involves injection of healthy copies of RPE65 underneath the retina. The RPE65 copies are contained in a human-engineered virus — known as an adeno-associated virus or AAV — which is designed to readily penetrate retinal cells to deliver the therapeutic genetic cargo. A single treatment is expected to last several years.

Spark Therapeutics, which holds the biologics license for LUXTURNA and conducted the clinical trials that showed its safety and efficacy, will also manage the treatment rollout. Spark has announced that in order to ensure the treatment is safely administered, it will only be available through a small number of centers of clinical excellence around the country. Spark has also expressed its commitment to educate third-party payers about the value of LUXTURNA and to work to help ensure treatment access to all eligible patients.

Anyone in need of more information about LUXTURNA should contact Spark Therapeutics at 1-833-SPARK-PS (833-772-7577). Another resource for information is www.luxturna.com.


100 Responses to 'History Is Made: FDA Approves Spark’s Vision-Restoring Gene Therapy'

  1. SS says:

    My husband has RP. Is this current gene therapy (Luxterna) helpful for him at this time?

  2. Davida Luehrs says:

    What wonderful and exciting news. Best Christmas gift ever.

  3. Jennifer Foster says:

    Fantastic news!!! Christmas miracle 🙂

  4. Skylar Hoage says:

    How do my dad and I get this done to help our RP

  5. Carri says:

    This is amazing!! Is this only for rpe65 Gene?

  6. God bless Sparks and the Foundation Fighting Blindness and all those dedicated people who worked on the research for and development of Luxterna. My wife has lost all her vision due to RP. This brings us hope.

  7. John Chaves says:

    With two brothers with RP it is a hopeful sign their vision will be returned!

  8. Johnny Ray Gibbs says:

    congratulations!! I am possible to interest participation., but cost really is ouch!!! I am recipient of government check , I am deaf and with retina pigmontoea. I want to be free as normal people what I was used in my youth. I am current 55 years old.

  9. g b heath says:

    1. What is the cost?
    2. What age groups are targeted?
    3. At what stage of RP would treatment be considered?
    4. If already on organ transplant antirejection meds, would treat be advisable?

  10. doris molter says:

    what wonderful news.I hope this works for macular degeneration some day soon.

  11. Kandy McDonald says:

    Any plans for gene therapy for those with LHON? My husband connected Lebers at the age of 62 and is desperate for help. His mutation is 11772, that most common mutation.

  12. Priscilla underwood says:

    It’s wonderful.maybe my son will see again!thank you for all your work

  13. Nitin Dhakate says:

    Wonderful and best mankind on the earth.

  14. francisco caicedo says:

    Proud and how amaising are the científics ti find the ways to resolve a problem.
    Very thanks for your dedication

  15. Lynne Thorstenson says:

    OMG!!!!! My mom had RP and so do I!!!! I’m sooooooooooooo happy! Thank you researchers!! Your amazing, thank you for your hard work!!! Where can I sign up to have it done???? 2018 hopefully looking better. So thankful!

  16. Elizabeth Ross says:

    Will this treatment be helpful for people with dry macular degeneration?

    • EyeOnTheCure says:

      Hi Elizabeth,

      No – it isn’t for dry AMD, but this will help open the door for development of additional vision treatments for other diseases. Visit http://www.clinicaltrials.gov and search on age-related macular degeneration to learn about clinical trials for emerging dry AMD therapies.

  17. Claude David Doucet says:

    I am excited about the identity of RPE65 and the Luxturna treatment. I am 66 yrs. ld and have RP autosomal dominant. This will help me and my family members who inherited this disease.

  18. P.c.mangal says:

    Wonderful, hope this treatment will be available in more hospitals soon.

  19. Robert Smith says:

    My wife and her sister both suffer with R.P. and desperatly want treatment

  20. Kamal kharbanda says:

    Very thrilled to hear about this miracle cure. Thanks to the foundation who has been working on this research for decades . Many people will be blesse . Great Christmas gift. God bless you all.
    Like to know more about it.

  21. April D says:

    I have lost all of my sight to R P, hope your hard work will some day restore my sight, God bless each of you.

  22. Kimberly Walls says:

    My son was diagnosed with X – linked retinoschisis a few years ago. Such wonderful news about this new trial LUXTURNA! Prayers that it will help many people!

  23. With committed Researchers and Organizations we have been given hope that maybe someday our vision loss will be restored. To know that our children who may have inherited this eye disease will be able to live as it is meant to be without an eye disease holding them from having a full and purposeful life is a BLESSINGS to dos many! Thank you!! Whether I’m put on a waiting list it will be worth the wait!!
    BLESSINGS TO ALL!
    MERRY CHRISTMAS AND HAPPY BEW YEAR TO ALL!!!

  24. Gary Olcott Jr says:

    This could change mind and my entire families life. I hope this becomes a reality.

  25. Raghavendra says:

    Great news indeed. Is this therapy applicable for ABCA4 gene as well? Please let me know. Both my daughters have stargardts.

  26. Ginny Schott says:

    The best Christmas gift ever.

  27. Anita Mata says:

    The Best Christmas Present my son could have ever received!! A million thank you’s!!!

  28. Jenny ravi says:

    This is amazing news,
    I have two children with stargazers both now in their early 20s and both have suffered with day to day struggles for many of those years.

    I can now give them hope !!

    From a mother who has been strong and encouraging every day to not give up and be the best they can be considering their vision impairment

  29. john reid says:

    wonderful – i hope this will help many: will this genetic treatment help stroke related retinitis – my right eye went blind from cva some years ago, but has come back to 40% over the years. ???

    • EyeOnTheCure says:

      Hi John,

      LUXTURNA is for people with retinal disease caused by mutations in the gene RPE65, not for your retinal condition. We suggest contacting your retinal specialist to learn more about potential therapies for your condition.

  30. Narendra Patnaik says:

    Dear Sir,
    Emmencely thankful to one and all responsible for this breakthru scientific service to humanity since centuries.
    Congratulations and A MERRY CHRIST MASS to you all. Now millions of eyes can see Jesus in front of them.

  31. Judi McCoy says:

    God is so good! His guidance has led science to make this fantastic discovery. My 79 year old husband has RP….he had to retire at 47 years old. Him being able to see again would be wonderful, but if not, this should help millions of people.

  32. Athena Lewis says:

    I pray this gets to many retinal doctor’s across the U.S.A., as it is costly for some of us to travel. I also pray Medicaid, and Medicare will pay for treatment. I have RP, and only see out of one eye now. Please help, and God bless all involved.

  33. Mary Weiner says:

    Both of my sons have RP. They are now 32 and 35. What would the next step be to help them?

  34. Julie says:

    This is amazing news to everyone affected by RP. I hope this treatment will be available worldwide ASAP.

  35. Joan M. Vrettos says:

    I have retinitis Pigmentosa and have dealt with it most of my adult life. My vision is not totally gone but I can feel it everyday diminishing. To have a medical advancement like this is amazing and I would love to be a part of it. I had a brother and older sister with the same disease. My sister had to rely on a seeing eye dog and it was really sad to watch her. It would be a dream to be able to have medical help and not go totally blind.

  36. Robert Deis says:

    Spark contacted us to schedule a test to determine whether we can be helped by this treatment. I assume that they found us through the FFB patient registry. If you haven’t enrolled in the database yet, I suggest you go here: https://www.myretinatracker.org/ and register.

  37. Linda Wiliams says:

    When and where is this available. I gave 2 son’s with RP.

    • EyeOnTheCure says:

      Hi Linda,

      LUXTURNA is for people with mutations in the gene RPE65. Your sons need to be genetically tested to determine which gene is causing their RP. If you (they each) register at http://www.myretinatracker.org, you can receive notifications of FFB’s genetic testing program.

  38. Lise Nadon says:

    My son , 26 years old, has a RP, gêne USH2A. Is it possible to
    Know if this treatment will be for him or anything is coming soon.
    Best gift for ever…

  39. Gay Bowers says:

    I have RP. How does one become a participant in this?

  40. John Barrie says:

    i have 2 sons who have RP. How do we get treatment?

  41. Moji.E says:

    Hello Mr Ben Shaberman
    I have lost all of my sight to RP, hope your hard work will some day restore my sight, God bless each of you Mr Ben Shaberman and Foundation Fighting Blindness .
    This could change mind and my entire families life. I hope this becomes a reality.
    My husband, would low vision be interested in this treatment since he (and one Brother and 2 daughter) has Retinitis Pigmentosa. Please keep us informed as to when this treatment would be available. We are in the iran-tehran area.
    Please help,
    Mr.Moji.E

  42. Antoinette Tleige says:

    I have two sons in there thritys with Retinitis pegmentosa usher syndrome,it would be a miracle if they can get there sight back.i pray that will happen soon.

  43. Daniel C. Weber, OD says:

    I just read in the Colorado Springs Gazette that Spark will be charging $425,000 per injection for a total of $850,000 for bilateral injections. These costs are exhorbitant and will not be covered by insurance. And what happened to the $10 million that FFB contributed to start the effort and my donations over the years to contribute to FFB and allow Spark to gouge the public?

    • EyeOnTheCure says:

      Hi Daniel,

      Spark is working with insurance companies to gain coverage for the treatment. Spark is doing all it can to provide access to the treatment for those who need it. Visit https://luxturna.com/take-the-first-step to learn more about gaining access to LUXTURNA. FFB’s $10 million investment went to early research that made the clinical trials for LUXTURNA possible.

  44. Lenny macek says:

    Will this save the eyesight ..of people w/cone rod dystrophy? My daughter was diagnosed / this when she was 10 yrs old..

    • EyeOnTheCure says:

      Hi Lenny,

      LUXTURNA is not for people with cone-rod dystrophy. She needs genetic testing to determine what mutated gene is causing her vision loss. That will help her determine which emerging therapies may be relevant. We suggest she register at http://www.myretinatracker.org to qualify for free genetic testing (coming soon) and learn about clinical trials for which she may be eligible.

  45. F. Davis says:

    Lost the vision of my left due to a bacterial infection. I also have glaucoma in both eyes. Some vision in left eye but not much no side vision. Would a treatment like this help me?

  46. Susan Mills says:

    Would love to know if I could be a candidate. This is an answered prayer for so many.

    • EyeOnTheCure says:

      Hi Susan, LUXTURNA is specifically for people who have mutations in the gene RPE65. You need to be screened with a genetic test to determine which gene is associated with your vision loss. Sign up at http://www.MyRetinaTracker.org to make yourself eligible for free genetic testing.

  47. Linda Mancusi says:

    Wonderful news! I have RP-ushers. But I don’t know what type. I’ve been looking to see king for genetic testing but not having any luck. Thank you for your good work. Hoe can we get more infor I want my sight back

  48. Gary Willis says:

    Where do I get this treatment. I have last stages of Stargardt disease, my center vision is 20/400 in both eyes. Can anyone point me in the right direction as where to begin? Please let me know if you do.

  49. Nidhi Mishra says:

    I am suffering from RP and want to get this treatment done for my self. what is the procedure. also since I am from india, is the treatment can be performed here? do let me know, so that I can make the arrangement to come over USA and get the treatment.
    Thanks

    • EyeOnTheCure says:

      First, you should understand that this therapy is only for people with mutations in the gene RPE65. There are many genes that can cause RP – you need to have a genetic test to determine which mutated gene is causing your disease.

      We are aware of the following clinics in India, which may be helpful:
      Shankara Netralaya, Chennai
      L. V. Prasad Eye Institute, Hyderabad – They have branches in different states
      Aravind Eye Hospital, Madhurai
      All India Institute of Medical Sciences- New Delhi

      If you are interested in a clinic in a different location, contact one of these clinics and ask them for a suggested clinic in your region.

  50. David says:

    Steps in the right direction! Hats off to all in America for establishing FFB and all the fantastic people raising funds for research. I live in the U.K. and have an inherited eye disease as have many other people in my family, mother, sister, aunts, cousins etc and like many people with RDD it has taken years to get a diagnosis, from when I was a teenager in the 1980 s I was told I have RP then to be told I have AMD and recently I have been informed that I have a rod/cone like dystrophy. I have also been gene tested and the result is that I have a mutation in the GUCY2D gene. If any of your researchers are looking in this area and need a case study or anything that may help I would be more than happy to pass my medical history/records over for perusal. Thanks to all involved.

  51. Wayne says:

    OMG!! My daughter was diagnosed with Stargardt disease last Spring. I have been following the research and blogs since then. Now there is real, tangible hope. I pray we can provide this for her. Thank God Almighty…

  52. Diane says:

    I was diagnosed with RP 9 years ago. It seems that my vision is worsening at a quicker pace these last several months. Hopefully this is the answer to our prayers.

  53. ram says:

    what a great news genetic engineering will be developed

  54. This is great news but I am worried about the cost! I am a mother with Stargardt’s Disease and Congenital Stationary Night Blindness. My life would be so different. I be able to support my two kids much better. I work full-time and have a child who is Down Syndrome. I am thankful for love ones, but I am very independent. Crossing streets with a little one would be less stressful. Please tell me this would be affordable…

    • EyeOnTheCure says:

      Hi Tonnya, Spark Therapeutics has worked very closely with payers to allow commercially insured patients who seek treatment within network to be treated at no or near no patient out-of-pocket expense. If you have any further questions, please feel free to visit their Patient Support Services website (www.mysparkgeneration.com) or call their patient inquiry number at 1 833 SPARK PS (833 772 7577). Hope this helps!

  55. Dipo Salau says:

    I have two sons with RP due to usher syndrome. How can one of them become a participant in the clinical trials?
    Thank you.
    Dipo

    • EyeOnTheCure says:

      Hi Dipo, LUXTURNA restores vision for those with mutations in the gene RPE65. The first step is being genetically tested to see if your sons have the mutation that can be treated by this therapy. You can contact Spark Therapeutics on their website http://www.SparkTX.com, or by calling their patient inquiry number at 1 833 SPARK PS (833 772 7577). Their team can help answer further questions on whether someone is eligible for this treatment.

  56. Jessila Macwes says:

    Hi
    Im a private nurse of a 90 y/o man, he was diagnosed of having diabetic retinopathy. His right eye can’t see anymore and his doctor said that also the left eye is deteriorating.
    Could be this luxturna will help?

    • EyeOnTheCure says:

      Hi Jessila, LUXTURNA restores vision for those with mutations in the gene RPE65. But there are many ongoing clinical trials for diabetic retinopathy that you can find by visiting http://www.clinicaltrials.gov. The site has a search function to find trials relevant to you.

  57. Puneet says:

    Will this also help in Stargardt disease?

    • EyeOnTheCure says:

      Hi Puneet, no, LUXTURNA only treats those with the RPE65 genetic mutation. But this gene therapy approval is a historic event and will help open the door for development of additional vision treatments for other diseases, including Stargardt disease.

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