Janet Woodcock, M.D.
- FDA Director, Center for Drug Evaluation & Research
Janet Woodcock, M.D.
- FDA Director, Center for Drug Evaluation & Research
FDA Accelerated Approval of Genervon's GM604 for Use In ALS
My name is Nick and I am 54-years old. In October 2011, I was diagnosed with ALS. Today, I am in a wheelchair and cannot walk or stand. My arms grow weaker almost every day and my breathing is starting to be affected. I used to be very active but now I can only leave my home when I have someone with me who's strong enough to lift me into my wheelchair once I'm done scooting down to the bottom of the stairs on my butt. It can feel like I'm a 100-year old man who can't do things for himself anymore. At 54, I should still be a vibrant part of my community, but this disease is kicking me where it hurts. Through the enthusiasm generated by the Ice Bucket Challenge, many people now know that ALS is a degenerative neurological disease that currently affects over 30,000 Americans, and as it stands the disease is always fatal. Most people with ALS are given only 2-5 years to live. When I was diagnosed, I was devastated - it took me months to accept it. Although I am now resolved, I have not lost hope. Currently, there is only one drug available to treat ALS (Riluzole) and it is only able to extend life expectancy by a couple months. That's why the recent news about a small company named Genervon Biopharmaceuticals and their trial drug "GM604" has been so exciting for the Global ALS Community. While most trials have a hard time even finding a positive trend, the GM604 trial data supports the view that this drug could very well be is a game changer in the battle against ALS. There are multiple, statistically significant data. Not only in clinical data and results alone, but also correlated biomarkers data and results as well. (Please see "Files" section on the campaign website: https://sites.google.com/site/aap4gm6/) Genervon met with the FDA in February 2015 and made a potentially life-changing request on behalf of the ALS community; they asked the FDA to promote GM604 to the Accelerated Approval Program with Post-Marketing Phase 4 Requirements, so all ALS patients can have legal access to GM604 now. Under the FDA's Accelerated Approval Program, the treatments would be covered by health insurance. Although Genervon knows that this request might complicate their relationship with the FDA, they were willing to take a stand and do everything they can to help the ALS community because it is the right thing to do. If the FDA does not grant Accelerated Approval, it will likely be 3 more years before patients are able to access this drug -- meaning that most people currently living with ALS will not live to see it reach market. While acknowledging the paramount importance of public safety in the FDA’s drug approval process, I believe that ALS presents a compelling case for an exception to the usual process. I am asking you to help me appeal to the FDA through their sense of compassion for those of us with this dreadful illness. People with ALS don't have time to wait for another clinical trial to be completed. By signing this petition, you'll be helping patients like me request that the FDA expidite the way potentially life-saving treatments are made accessible to people with ALS -- starting right now with GM604. Doing so could very likely mean a change in the course of ALS progression not only for myself, but hundreds of thousands of other patients worldwide. After you sign, please follow this link to tweet and/or email the FDA: https://www.change.org/p/lisa-murkowski-fda-accelerated-approval-of-genervon-s-gm604-for-use-in-als/u/9475826 Thank you, Nick Like us on Facebook: https://www.facebook.com/gm604forals Please go to the Genervon website for a true understanding of the facts and the science behind GM604: http://www.genervon.com/genervon/about_pressreleases.php
Fight the FDA-Keep the Owlet Smart Sock on the market. Use your voice!
The Owlet Smart Sock is a monitoring device for babies so that parents can track their heart rate and oxygen saturation while they sleep. Millions of babies die suddenly due to illness and disease, while all of those deaths cannot be prevented, this device is a way to keep an eye on your babies vitals during those early newborn days when they’re so fragile. Keeping this product on the market would ensure peace of mind for millions of parents and quite literally save lives by alerting parents to an issue before it’s too late. The FDA has come after the Owlet company and forced them to stop sales of the Smart Sock in the United States. I am one mom who’s life has been greatly impacted by this product. When my one year old was a newborn I fell asleep on my side nursing her one night as countless other mothers have. Thankfully she was wearing her Smart Sock. It went off alerting me to a problem and when I woke up she was pressed up against my side and under my breast. I can’t say for sure what would have happened had the monitor not gone off but I’m thankful it did. This is a product that has given peace of mind to millions of parents. There are other products and substances on the market the FDA could be targeting rather than a baby monitor that’s sole job is to provide peace of mind and help us keep an eye on our little ones. There are also other products on the market with these same capabilities. Please use you voice to help the FDA hear from the parents they’re actually affecting.
#CutFDARedTape: Abolish Cruel, Wasteful FDA Dog Testing Requirements!
Outdated U.S. Food and Drug Administration (FDA) regulations are slowing the search for a cure to COVID-19 and other illnesses by forcing drug makers to conduct unscientific and extremely cruel drug safety tests on puppies.As a pathologist with expertise diagnosing human disease, I can say with certainty that dosing puppies with drugs designed for humans is not just inhumane, but wasteful and unnecessary. It also will not make people safer—in fact, the FDA's dog testing mandate harms human health and wastes precious resources.I hope you will join me and White Coat Waste Project to help #CutFDARedTape, and abolish the FDA's wasteful and cruel dog testing mandate!The government’s own data shows that over 90 percent of drugs that are effective in animal testing, fail in humans—because they are ineffective, or because they show dangerous side effects not predicted by the animal trials.Yet, the FDA continues to require that companies waste millions of dollars to force beagle puppies to swallow or breathe doses of experimental drugs daily for up to a year, after which they’re killed. At least 20,000 dogs per year are experimented on and killed to comply with U.S. government regulations like these, and that number may be even higher.Even when companies want to use superior alternatives to dog testing, which FDA rules allow, the agency often refuses to permit it and punishes them for challenging the requirement.Please sign my petition and join me and White Coat Waste Project in urging the FDA to spare dogs, stop waste, and save lives by cutting FDA red tape!Sincerely,Dr. Tiffani Milless
Acceso temprano a tratamientos para la ELA (Early access to treatments for ALS)
En abril de 2018 diagnosticaron a mi marido de ELA, Esclerosis Lateral Amiotrófica, una enfermedad agonizante y mortal que a día de hoy no tiene cura ni tratamiento efectivo y que igual que él sufren 500.000 personas en el mundo. La esperanza de vida es de 2 a 5 años, pero muchos pacientes fallecen a los 6 meses después del diagnóstico. En este tiempo la persona va quedando inmovilizada, encerrada en su propio cuerpo. Hoy mi marido ya no puede hablar, no puede tragar, no puede casi andar, es alimentado a través de una sonda gástrica y vive con ventilación asistida las 24 horas del día. Pero él es sólo es uno más... Esta terrible enfermedad puede tocarle a cualquiera, en cualquier momento y a cualquier edad: desconocer su origen y sus causas hace que no sea una enfermedad que podamos prevenir. Por eso creemos que es vital que todos los enfermos puedan tener acceso temprano a tratamientos, pues suponen una oportunidad de intentarlo, de ganar tiempo, de poder vivir. Llevamos casi 30 años sin que aprueben tratamientos para esta enfeemedad y es hora de cambiar esto, ya que la ELA no da tregua y el tiempo juega en nuestra contra. Para ello pedimos a la FDA, la EMA, la MHRA, Health Canadá, y hacemos esta petición extensible a todas las agencias reguladoras de los 5 continentes que trabajen con la misma flexibilidad que han demostrado tener frente al Covid19. Firma esta petición para que agilicen los trámites para la aprobación de los ensayos clínicos para la ELA, y que se otorgue la Autorización de Comercialización Condicionada a todos aquellos ensayos clínicos que desde la fase II demuestren resultados mejores de lo que tenemos ahora, NADA. También necesitamos que tengan en cuenta la heterogeneidad de esta enfermedad, la importancia de los subgrupos y las carencias, tácitamente reconocidas por los expertos, de la escala ALSFRS-R. Sabemos lo difícil que será conseguir un tratamiento perfecto, pero si hay tratamientos que funcionan sólo en un subgrupo, necesitamos su aprobación. Hay más de 500.000 pacientes en todo el mundo, por lo que si hay una mínima esperanza para alguno de esos subgrupos, ese avance podría beneficiar a miles de personas. A la industria farmacéutica les suplicamos que presenten sus resultados positivos de manera urgente y desde la fase II a las Agencias reguladoras del medicamento para así poder obtener la Autorización de Comercialización Condicionada lo antes posible y hacer llegar los tratamientos rápidamente a los pacientes. Igualmente pedimos que tengan en cuenta la heterogeneidad, los subgrupos y las carencias de la ALSFRS-R para diseñar sus ensayos y conseguir mejores resultados. Por último, queremos pedir a la Comisión Europea más inversión: la ELA es la tercera enfermedad neurodegenerativa más diagnosticada del mundo tras el alzheimer y el parkinson. Desterremos que la ELA es una enfermedad incurable: es una enfermedad sin inversión. Firma ahora esta petición para conseguir una mayor flexibilidad, agilidad e inversión en los tratamientos para la ELA. ¡¡Ayúdanos en esta lucha por la vida!!
Urge FDA to approve new treatment for Carcinoid Tumors
Hi. My name is Kerry and I’m a zebra. Now, before you think I'm crazy, I hope you'll please let me have a minute of your time to explain. In medical school, doctors-to-be are often taught the adage “If you hear hoof beats, think horse”. In other words, the most obvious answer is usually the right answer. But sometimes the hoof beats come from something rarer: a zebra perhaps. For nearly 5 years, doctors heard hoof beats as they looked at scans of my liver and saw some small “things” taking up residence. And, for nearly 5 years, they thought horse. They did blood tests, colonoscopies, upper endoscopies; all of the tests you would do looking for the “normal” cancers such as colon cancer, but the tests were negative. So they concluded that my liver had some benign growths on it. Nothing to worry about. In January of 2016 a biopsy of my liver resolved the mystery. I was diagnosed with a rare form of cancer called Carcinoid or Neuroendocrine Tumor (NET). The disease is so rare it is referred to as the zebra of cancers and represents only about 2% of cancers treated each year. In honor of its rarity, the zebra has been adopted as the official mascot of those who suffer from it and we, the patients, are called zebras. In my desperate quest for solutions to my problem, I read about a treatment called Peptide Receptor Radionuclide Therapy, or PRRT for short. It has been available in Europe for more than a decade and clinical trials here in the United States have recently concluded. The results have been very promising. According to one of the doctors who participated in the phase 3 clinical trial, “The findings were, in my opinion, extraordinarily impressive, the median progression-free survival improved by nearly 80%, which is fairly unprecedented in oncologic studies. The finding is important because limited therapeutic options exist for such patients, who comprise 20% to 45% of neuroendocrine tumor cases.” (1) As you can imagine, I have been following these trials with great interest as I am their target patient: my tumors are inoperable and are known to respond to the drug being used in the treatment. Earlier this year, there was hope the treatment would be approved by mid-year 2016. Then it was sometime in the fourth quarter of 2016. The latest timeframe for approval is now early 2017. I understand and appreciate the fact that the FDA has to perform their due diligence to insure that new drugs are safe and effective. None of us want unsafe drugs to be hurried to market. In the case of PRRT, it appears to have proven its worth, both in US clinical trials as well as in more than 10 years of use in Europe. I don’t profess to know or understand what the holdup is in obtaining approval. I’m certainly not a doctor. What I do know is that every day that passes without this treatment being approved thousands of us zebras get sicker and closer to the point where treatments won’t matter any longer. Will you please join me in urging the FDA to keep the approval of PRRT at the top of their priority list? I’m only 55 years old and I’m not ready to leave this world yet. I and my fellow zebras thank you. Kerry (1) Full article discussing PRRT can be found online at http://www.carcinoid.org/2016/05/03/one-step-closer-us-peptide-receptor-radionuclide-therapy-prrt-neuroendocrine-cancers/
Approve AstraZeneca NOW
The Federal Government cannot sit idly by while a safe and effective vaccine is denied to the American people based on nothing more than bureaucratic line-toeing. The UK approved the AstraZeneca vaccine on December 30 and millions of people have now received the vaccine that has been shown to prevent COVID-19 hospitalizations with near-100% efficacy and to be 70% effective at preventing the main variant of the disease entirely. The EU-equivalent of the FDA has now approved the vaccine in Europe and The World Health Organization has recommended it for use in all countries. there remain no serious concerns about the vaccine’s safety or efficacy after extensive trials and now public use. The U.S. helped fund the development of the AstraZeneca vaccine and secured 300 million doses of the vaccine months ago. The vaccine is relatively easy to ship and store, and very inexpensive to produce. The only barrier to starting distribution is regulatory approval — something the FDA “projects” it will do in April. It is unclear what the FDA expects (or indeed doesn’t expect) to find that so many other countries missed. The cost is the 2,000+ Americans who will die each and every day we wait. Many object that AstraZeneca has not applied for Emergency Use Authorization, but that itself is likely a product of a political decision not to rely on foreign trial data. No matter the reason, an application wouldn't change the overwhelming evidence from this vaccine's actual use around the world. It is safe. It is effective. The virus is deadly. There is no legal obstacle to immediate approval that isn’t worth hurdling. Congress can override the FDA with a two-sentence bill. The President can attempt to do so by executive order. The FDA can amend or violate its own processes. Whatever legal consequences come pale in comparison to the staggering death toll America faces. Someone needs the courage to act. The signatories of this petition urge them to do so. Now.
Tell FDA NO! You are risking the lives of those with food allergies, EoE and more!
TELL FDA NO - WE AREN’T GOING TO RISK MORE LIVES! If you or someone in your life suffers from a life threatening food allergy, or from EoE (eosinophilic esophagitis) - tell FDA to reverse their recent (Friday night of Memorial Day announcement) COVID labeling changes. These changes could kill adults and children who battle these severe conditions every day, and it is already harder during this global pandemic to secure safe food. NOW FDA WANTS TO RISK LIVES OF MORE! SAY NO! OUR COMMUNITY DEMANDS YOU REVERSE THIS DECISION NOW! Many will suffer from these so called “small changes”...many are allergic or cause disease exacerbation by ingredients not recognized in the U.S. by the top 8 food allergens. 32 million Americans, that includes millions of children, suffer from food allergies! That doesn’t include those suffering already from EoE and an unknown ingredient can cause their esophagus to close! That doesn’t include those with other autoimmune conditions (UC, Crohn’s) that can be severely impacted by eating ingredients that are not handled by the digestive/gastrointestinal system. Your voice is needed to protect those that during an uncertain and challenging time for our country and world, we don’t need to risk the health of more - especially our children! Please sign and be a voice! Thank you! EoE and allergy mom - taking one moment at a time
FDA Guidance for ALS
The US Food and Drug Administration (FDA) is responsible for developing guidance for amyotrophic lateral sclerosis (ALS). The guidance provides a roadmap to those in the ALS industry to navigate through the development of treatments. To develop new treatments for ALS, the guidance would incentivize industry sponsors by directing trials to be more efficient, predictable, and effective at assessing drug safety and efficacy outcomes. The FDA has failed to deliver the guidance in a timely fashion and is now culpable to the inhumane treatment of ALS patients. ALS is a progressive terminal neurological disease that can strike anyone at any time. The disease currently has no meaningful treatment. Patients are often told to get their affairs in order, and that they have 2-5 years left to live. You have a 1:400 chance of developing ALS in your lifetime and only 10% of patients live longer than 5 years. There is no typical progression of ALS; however, it eventually paralyzes every aspect of a patient’s ability to move. Eventually, patients are no longer able to walk, talk, swallow, and breathe on their own. A federal law in 2012 allowed for patient-focused drug development (PFDD). After extreme pressure in May of 2015, ALS patients finally met with the FDA to discuss the drafting of a guidance document. Due to the lack of urgency from the FDA, the ALS Association (ALSA) took ownership of creation to expedite the development of the guidance. In August of 2017, the ALS community submitted the results to the FDA. In February 2018, the FDA published the Draft Guidance that completely ignored the work of the ALS community. The open comment section closed in May of 2018 after receiving hundreds of comments related to the published inhumane boilerplate Draft Guidance. In July, ALSA convened with the FDA and advocates to discuss the guidance. Due to the lack of results, ALS patients and advocates met with the FDA in February 2019. At that time Dr. Janet Woodcock and Dr. Billy Dunn stated that the ALS Guidance would be published within 2 months. After this deadline passed without publishing the ALS Guidance many patients and advocates reached out to the FDA. The lack of acknowledgement by the FDA for missing their deadline truly demonstrated that the ALS community is not a priority. In June of 2019, another ALS advocacy group, No More Excuses met with some of the same people at the FDA. ALS patients and advocates are doing everything possible. However, the FDA ALS Guidance remains extremely elusive. The FDA must be held accountable and publish the ALS Guidance. We need you to sign our petition to hold the FDA accountable of their negligence to the ALS community. Below are immediate requirements of the ALS Guidance along with an example from just one current clinical trial (NurOwn): Using historical controls for placebo groups – FDA inhumanely required 100 terminal patients to get a placebo (fake medicine) injected into their spinal cord (3x) and receive bone marrow aspiration. There are clinical trials for cancer and HIV/AIDS that eliminate the requirement of placebo due to the inhumane nature of testing fake medicine on a terminal patient. Historical controls allow the use of more actual placebo data than the use of placebo groups themselves. It also prevents the deterioration that ALS patients are forced to undergo as they receive a placebo. Placebo trials in ALS are akin to forcing people to die "in the name of science". Wider onset criteria – Trials usually have inclusion criteria that are set less than 18 months from first symptom. It typically takes 12-18 months to be diagnosed with ALS, patients often have symptoms longer than that. This excludes many from clinical trials. This is inhumane and forces trials to take longer to enroll participants. Requirement of crossover or open-label – Patients have no access once they have completed the trial. Patients on placebo also have no access to the treatment after completion. There are patients who show improvement or slowing of ALS in trials that are forced to go home and continue deteriorating after the trial. Remove penalties for unmasking data to apply for Accelerated Approval – Due to the additional 100 patients and strict onset criteria, BrainStorm is still trying to complete the NurOwn trial as ALS patients die. If BrainStorm decides to unmask the data early to apply for Accelerated Approval they are at risk of jeopardizing the trial. Incentivize Agencies to provide Right to Try and Expanded Access to patients that don’t qualify for trials – Outside of one patient, BrainStorm has chosen not to allow Right to Try or Expanded Access. There are no incentives to allow terminal patients that don’t qualify for the trial access to this treatment. Innovative Guidance – The FDA routinely calls for innovative trials but requires ALS to have archaic designs. The ALS Guidance needs to be a responsive document that is quickly amendable as innovation evolves. A living breathing document will expedite updates and remove the slow federal bureaucracy that is occurring now. We ask for Executive Order or Congressional action to order the FDA to expeditiously publish humane and ethical Guidance for ALS and to hold those responsible for these failures accountable. Since 2012, roughly 50,000 US ALS patients have died waiting for humane ALS Guidance from the FDA. Please sign this petition to show your support. Authors Twitter: Corey Polen , End ALS Facebook: #StandForCorey , End ALS
Petition for FDA to grant clinical trials of gene therapy for cln2 patients.
In expectations of preparing this petition. I would like to express our concerns and be the voice of our children and all CLN2 parents. The FDA has denied access to clinical trials for gene therapy for our CLN2 children. Gene therapy has helped other rare disease patients. Here is A brief explanation of CLN2 Battens Disease: CLN2 disease (Neuronal Ceroid Lipofuscinosis Type 2) is an ultra-rare, neurodegenerative lysosomal storage disease, caused by an enzyme deficiency of tripeptidyl peptidase 1 (TPP1). CLN2 disease affects motor skills, such as sitting and walking, and speech development. This condition also causes the loss of previously acquired skills (developmental regression), intellectual disability that gradually gets worse, and behavioral problems. Individuals with this condition often require the use of a wheelchair by late childhood and typically do not survive past their teens. This is not the life that our children deserve to live. There’s better treatment for our children, and that’s gene therapy. We have rights as parents and our voices need to be heard. Our children receive enzyme replacement therapy. This produces our children with synthetic enzymes. This is a bi-weekly treatment, some kids have been receiving this treatment for over five years. To be able to have gene therapy offered to our children, would allow many children to live a normal life. If the FDA can please take some sympathy to listen to the concerned voices of all CLN2 parents, we can make a difference. Life has better options, and gene therapy is the number one step towards it for our children. This is not how any of us predicted for our life to be, and science has proven that it can make miraculous changes. Allowing clinical trials to take place, would be the most ethically responsible and humane thing to do. Science studies has also shown allowing CLN2 patients to produce natural enzymes, will improve all motor skills and mobility and speech. It is ethically right to allow scientist, to move forward with the clinical trials. It is also ethically right to want to help others, and not frown upon wanting to provide a treatment that can be extremely beneficial for our children. Scientist have already begun to work on the treatment, and it would be highly appreciated if clinical trial for gene therapy for CLN2 would be provided to our children. My question in theory is how come the FDA is giving us CLN2 parents a hard time to grant clinical trials for gene therapy? This is life threatening to our children! Time is of the essence, and with such a harmful rare genetic disease, every moment counts. Thank you for taking the time to review this petition.
Please help parents of children with corn allergies. Add corn to food allergen labeling.
I’m a parent and one of my children was recently diagnosed with a corn allergy. The first bit of advice our doctor gave us was -- avoid foods with corn or corn derivative. The task seemed simple enough -- until we learned that corn was lurking, unlabeled, in nearly every processed food product in the grocery store. Baking powder, semolina, vanilla extract, citric acid -- did you know each of these ingredients contains a corn derivative? Neither did we! And now we are tasked with memorizing a laundry list of ingredients you’d never associate with corn in order to keep our kid safe. All because manufacturers aren’t required to notify consumers of the presence of corn or corn derivatives in their food or product. Bottom line: unlabeled corn allergens in food (and medicines!) threaten the health and safety of children and adults across the country. By excluding corn from the Food Allergen Labeling act, our government is putting its citizens at risk. Keeping a child safe from food allergens shouldn’t be a daily game of chance. Parents shouldn’t have to gamble with their child’s health and well-being to keep them fed and nourished. If you agree with me, please sign this petition. It’s time for food products containing corn and corn-derivatives to be CLEARLY LABELED -- like any other common food allergen. Tell the FDA to add CORN to the Food Allergen Labeling and Consumer Protection Act today.