Peter Marks, the director of the Center for Biologics Evaluation and Research within the Food and Drug Administration (FDA), unilaterally approved Sarepta Therapeutics' gene therapy.
I'd be interested to know Peter Marks career history, and a detailed financial disclosure (that we'll never get) would also go a long way towards knowing his motivations to overrule his teams decisions and unilaterally grant approval for this product.
After completing his training, Marks worked at the Brigham and Women's Hospital as a clinician-scientist, and later served as Clinical Director of Hematology. He then worked in the pharmaceutical industry, where he worked on the development of hematology and oncology products. He later managed the Adult Leukemia Service at Yale University and served as the Chief Clinical Officer of the Yale New Haven Hospital Cancer Center. Marks joined the Center for Biologics Evaluation and Research as deputy director in 2012, and was promoted to director in 2016.[3][4]
In May 2020, he was selected to serve as a member of the White House Coronavirus Task Force,[5] although he left a few days later over concerns that his participation would represent a conflict with his position at FDA.[6] Marks also played a role in establishing Operation Warp Speed, a partnership between the federal government and various private companies to develop a COVID-19 vaccine, but left the project in May 2020 shortly after it was launched because Marks believed he would be more useful in his role as chief regulator of vaccines as the Director of FDA's Center for Biologics Evaluation and Research.[7][8][9] In 2021, Marks served as a plenary speaker at the State of the Science Research Summit.[10] In 2024, Marks overruled FDA staff to approve gene therapy Elevidys to treat Duchenne muscular dystrophy (DMD) despite it failing Phase III clinical trial.[11]
What I never understand is that ambulation as an outcome is the lesser of a DMD patients worries…what about the effect of dystrophin in cardiomyocytes…the heart failure is what is most concerning for DMD. For a gene therapy to be effective you have to fix all skeletal muscle (perhaps except extraocular muscle) and the heart. How is this feasible in postmitotic tissue.
My grandson has DMD, so I have a vested interest. I had a very special opportunity to meet Dr. Marks and spend 10 minutes with him, one-on-one, after the abbreviated approval, but before the final approval. A more humble public servant you will not find. And no, he did not make his career in government. The balance that only the head of the division can take is that since these (mostly) boys HAVE NO ALTERNATIVE, the known outcome without intervention is a slow and inhumane death. Also, via clinical trials, we have learned how to administer this therapy safely. So, with safety in hand (the risks are characterized), if families wish to have this potentially life-saving therapy administered, thanks to Dr. Marks, they may do so. The upside is potentially huge for the families while the downside is manageable. I applaud and thank Dr. Marks for his leadership and courage, while appreciating the good folks who work for him who did their jobs professionally.
I appreciate your situation, and that you have acknowledged your bias/vested interest. I truly hope this gives your grandson an effective treatment option for DMD which I know is an awful diagnosis with a tragic outcome. I have worked in the clinical research industry for the past 20 years, which I admit is my entire working life. Over those 20 years I have gone from being an outspoken & strong advocate for the FDA, to just a quiet participant in the drug approval process. Especially in the past 3 years since COVID, I have witnessed the erosion of trust in the agency and in public health as an institution - for very good reason. The infiltration of the FDA by corporate stooges and the revolving door between FDA, Pharma and Lobbyist firms has tainted what was one of the few government institutions that wasn't (entirely) corrupted by greed. We were once the golden standard in drug & device approvals here in the USA, and now I'm sad to say I do not think we still are - and when I see headlines like this it deepens my cynicism. Especially considering the price tag. My best to you and your grandson, Frank! Hoping this brings him a long and happy life.
Nick, (that's my grandson's name) I 100% agree with your sentiments. I too was in the industry on the business side of things (20 yrs Ciba/Novartis, then biotech). FDA was the envy of the world, now I'm not so sure. Don't know if COVID changed things or revealed truth, but I'm brokenhearted too.
Marks is obviously corrupt which was very clear during COVID. However I personally have no issue approving it unless it's clearly harmful, so long as no tax payer pays for it
I'd be interested to know Peter Marks career history, and a detailed financial disclosure (that we'll never get) would also go a long way towards knowing his motivations to overrule his teams decisions and unilaterally grant approval for this product.
From his Wiki page:
After completing his training, Marks worked at the Brigham and Women's Hospital as a clinician-scientist, and later served as Clinical Director of Hematology. He then worked in the pharmaceutical industry, where he worked on the development of hematology and oncology products. He later managed the Adult Leukemia Service at Yale University and served as the Chief Clinical Officer of the Yale New Haven Hospital Cancer Center. Marks joined the Center for Biologics Evaluation and Research as deputy director in 2012, and was promoted to director in 2016.[3][4]
In May 2020, he was selected to serve as a member of the White House Coronavirus Task Force,[5] although he left a few days later over concerns that his participation would represent a conflict with his position at FDA.[6] Marks also played a role in establishing Operation Warp Speed, a partnership between the federal government and various private companies to develop a COVID-19 vaccine, but left the project in May 2020 shortly after it was launched because Marks believed he would be more useful in his role as chief regulator of vaccines as the Director of FDA's Center for Biologics Evaluation and Research.[7][8][9] In 2021, Marks served as a plenary speaker at the State of the Science Research Summit.[10] In 2024, Marks overruled FDA staff to approve gene therapy Elevidys to treat Duchenne muscular dystrophy (DMD) despite it failing Phase III clinical trial.[11]
What I never understand is that ambulation as an outcome is the lesser of a DMD patients worries…what about the effect of dystrophin in cardiomyocytes…the heart failure is what is most concerning for DMD. For a gene therapy to be effective you have to fix all skeletal muscle (perhaps except extraocular muscle) and the heart. How is this feasible in postmitotic tissue.
My grandson has DMD, so I have a vested interest. I had a very special opportunity to meet Dr. Marks and spend 10 minutes with him, one-on-one, after the abbreviated approval, but before the final approval. A more humble public servant you will not find. And no, he did not make his career in government. The balance that only the head of the division can take is that since these (mostly) boys HAVE NO ALTERNATIVE, the known outcome without intervention is a slow and inhumane death. Also, via clinical trials, we have learned how to administer this therapy safely. So, with safety in hand (the risks are characterized), if families wish to have this potentially life-saving therapy administered, thanks to Dr. Marks, they may do so. The upside is potentially huge for the families while the downside is manageable. I applaud and thank Dr. Marks for his leadership and courage, while appreciating the good folks who work for him who did their jobs professionally.
I appreciate your situation, and that you have acknowledged your bias/vested interest. I truly hope this gives your grandson an effective treatment option for DMD which I know is an awful diagnosis with a tragic outcome. I have worked in the clinical research industry for the past 20 years, which I admit is my entire working life. Over those 20 years I have gone from being an outspoken & strong advocate for the FDA, to just a quiet participant in the drug approval process. Especially in the past 3 years since COVID, I have witnessed the erosion of trust in the agency and in public health as an institution - for very good reason. The infiltration of the FDA by corporate stooges and the revolving door between FDA, Pharma and Lobbyist firms has tainted what was one of the few government institutions that wasn't (entirely) corrupted by greed. We were once the golden standard in drug & device approvals here in the USA, and now I'm sad to say I do not think we still are - and when I see headlines like this it deepens my cynicism. Especially considering the price tag. My best to you and your grandson, Frank! Hoping this brings him a long and happy life.
Nick, (that's my grandson's name) I 100% agree with your sentiments. I too was in the industry on the business side of things (20 yrs Ciba/Novartis, then biotech). FDA was the envy of the world, now I'm not so sure. Don't know if COVID changed things or revealed truth, but I'm brokenhearted too.
Marks is obviously corrupt which was very clear during COVID. However I personally have no issue approving it unless it's clearly harmful, so long as no tax payer pays for it