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FDA Commissioner: ‘We want to focus on food'

FDA Commissioner: ‘We want to focus on food'

The Hill11-07-2025
As part of the 'Make America Healthy Again' movement, Food and Drug Administration (FDA) Commissioner Dr. Marty Makary says one of the agency's top targets is eliminating artificial food dyes by the end of 2026.
Makary, a surgical oncologist tapped by President Trump to serve as the the 27th FDA chief, joined NewsNation on Thursday to discuss his first 100 days in the post. He said the agenda to phase out synthetic food dyes from the U.S. food supply is due to a suspected link that the dyes can cause and/or exacerbate health issues and disorders like Attention-Deficit/Hyperactivity Disorder (ADHD).
'There's 1,000 chemicals in the U.S. food supply that are not in the food supply of Europe, and so we're doing an inventory of all of them, and these petroleum-based food dyes, that is artificial food dyes, have been implicated in Attention Deficit Disorder [ADD], and other problems in the health of children,' Makary said.
'So, we're saying we want to see natural ingredients,' he added. 'We want to see people eat less ultra-processed foods.'
A 2022 report from the National Institutes of Health (NIH) looked into the association of blue food coloring with ADHD. The review ultimately said the cause of the disorder was unknown, and found no direct link between it and artificial food dyes — though some findings showed exacerbated symptoms.
Makary told NewsNation that the agency is trying to authorize alternative options, like natural dyes. He says the agency expects to approve one such dye in days.
The FDA announced in April that they are working on phasing out all petroleum-based synthetic dyes in the U.S. food supply as part of the 'Make America Healthy Again' commission.
'We want to focus on food. People forget that the 'F' in FDA stands for food,' Makary said.
Makary noted that the processed foods are often targeted toward children and said the agency has focused on fighting childhood obesity and diabetes.
'The maker of Skittles announced they're going to get rid of titanium dioxide, another chemical that we'd like to see out of candy, and so we're going to keep going like this,' Makary said. 'I don't believe our childhood diabetes epidemic is a willpower problem. We've got to look at what we are doing to children.'
Testing the impact of things like artificial dyes on minors is a challenge, the FDA commissioner noted, and said his agency will rely on hearing from patients and their parents to understand more.
'We've got to listen to parents when they say, my kid had a lot of bad behavior, aggressive behavior, was diagnosed with ADHD. We cut out all the petroleum, artificial dyes from the diet, and the kid's behavior improved,' Makary said. 'That's a data point.'
He added, 'It's not a randomized control trial, but when you have enough of those, and you do have some good, robust medical studies in the literature, we can take action.'
Health and Human Services (HHS) Secretary Robert F. Kennedy Jr. and the FDA want to remove synthetic food dyes by the end of 2026. Companies like Kraft Heinz, General Mills, PepsiCo, Nestle and ConAgra said they will phase out artificial dyes.
But enforcing those bans could prove to be difficult, with at least one major brand already pushing back.
'I think you win more bees with honey than you do with fire. I think we're starting off with a very diplomatic approach, making this request on behalf of the public,' Makary said. 'Remember, this is a very popular issue among parents in the United States; over 90 percent of parents want to see these petroleum-based food dyes removed from the food supply of children.'
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FDA Approves Apellis' EMPAVELI® (pegcetacoplan) as the First C3G and Primary IC-MPGN Treatment for Patients 12 and Older
FDA Approves Apellis' EMPAVELI® (pegcetacoplan) as the First C3G and Primary IC-MPGN Treatment for Patients 12 and Older

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FDA Approves Apellis' EMPAVELI® (pegcetacoplan) as the First C3G and Primary IC-MPGN Treatment for Patients 12 and Older

Proven efficacy across all three key markers of disease—68% reduction in proteinuria, stabilization of kidney function, and substantial clearance of C3 deposits Broad label includes adults and adolescents with C3G or primary IC-MPGN, and post-transplant C3G disease recurrence Well-established safety profile, consistent across >2,200 patient years in approved indications C3G and primary IC-MPGN are rare kidney diseases with high risk of kidney failure Conference call tomorrow at 8:00 a.m. ETWALTHAM, Mass., July 28, 2025 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that the U.S. Food and Drug Administration (FDA) has approved EMPAVELI® (pegcetacoplan) as the first treatment for C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients 12 years of age and older, to reduce proteinuria. C3G and primary IC-MPGN are rare kidney diseases, affecting 5,000 people in the United States.1 'I'm excited to now have a highly effective therapy for a broad range of patients living with C3G and primary IC-MPGN,' said Carla Nester, M.D., MSA, FASN, lead principal investigator for the VALIANT study, professor of internal medicine and pediatrics and director of pediatric nephrology, University of Iowa Stead Family Children's Hospital. 'With standard of care, patients living with these rare and severe diseases frequently progress to kidney failure, necessitating lifelong dialysis and/or a kidney transplant. Given the urgent need, particularly in children, the approval of EMPAVELI marks a pivotal moment in the treatment of rare kidney diseases.' In the Phase 3 VALIANT study, EMPAVELI demonstrated an unprecedented 68% reduction in proteinuria, stabilization of kidney function, and substantial clearance of C3 deposits as measured by C3 staining, compared to placebo. The positive results were consistent across adolescent and adult patients with C3G and primary IC-MPGN, and in C3G patients with post-transplant disease recurrence. 'EMPAVELI has the potential to be truly transformational for patients with C3G and primary IC-MPGN, who until now have had very few treatment options. In the largest pivotal study of these diseases, EMPAVELI demonstrated its potential to preserve kidney function by controlling all three key markers of disease,' said Cedric Francois, M.D., Ph.D., co-founder and chief executive officer, Apellis. 'As Apellis' third approval in four years, this milestone underscores the unique ability of targeting C3 to improve patients' lives. We are deeply grateful to everyone who made this approval possible and look forward to building on this momentum as we advance pivotal studies of EMPAVELI in other rare kidney diseases.' 'The approval of EMPAVELI is a historic milestone for people living with C3G and primary IC-MPGN, many of whom are adolescents or young adults,' said Josh Tarnoff, chief executive officer, NephCure. 'We recognize Apellis' commitment to these patients and their families, and to the research and innovation that will bring this life-changing treatment into the hands of patients that need it most.' The approval of EMPAVELI is based on positive six-month results from the VALIANT study, demonstrating benefits across all three key markers of disease: Proteinuria reduction: The study met its primary endpoint, demonstrating a statistically significant 68% (p<0.0001) proteinuria reduction in EMPAVELI-treated patients compared to placebo. Stabilization of kidney function: EMPAVELI-treated patients achieved stabilization of kidney function compared to placebo (nominal p=0.03) as measured by estimated glomerular filtration rate (eGFR). Reduction of C3 staining: A majority of EMPAVELI-treated patients achieved a reduction in C3 staining intensity (nominal p<0.0001) compared to placebo. 71% of EMPAVELI-treated patients achieved zero C3 staining intensity, demonstrating complete clearance of C3 deposits. The safety profile of EMPAVELI is well-established, with >2,200 patient years across all approved indications. The most common adverse reactions in the VALIANT study (≥10%) were infusion site reactions, pyrexia, nasopharyngitis, influenza, cough, and nausea. Apellis is committed to helping patients with treatment access and support. ApellisAssist® is a program designed to help EMPAVELI patients along their treatment journey by providing a comprehensive system of support including help navigating insurance coverage, financial assistance for eligible patients, disease education, and ongoing product support. Patients and healthcare providers can call 1-888-273-5547 for more information. Conference Call and WebcastApellis will host a conference call and webcast to discuss the FDA's approval of EMPAVELI tomorrow, July 29, 2025 at 8:00 a.m. ET. To access the live call by phone, please pre-register for the call here. A live audio webcast of the event and accompanying slides may also be accessed through the 'Events and Presentations' page of the 'Investors and Media' section of the company's website. A replay of the webcast will be available for 30 days following the event. About C3 Glomerulopathy (C3G) and Primary Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN)C3G and primary IC-MPGN are rare and debilitating kidney diseases that can lead to kidney failure. Excessive C3 deposits are a key marker of disease activity, which can lead to kidney inflammation, damage, and failure. Approximately 50% of people living with C3G and primary IC-MPGN suffer from kidney failure within five to 10 years of diagnosis, requiring a burdensome kidney transplant or lifelong dialysis therapy.2-4 Additionally, approximately 90% of patients who previously received a kidney transplant will experience disease recurrence.5 The diseases are estimated to affect 5,000 people in the United States and up to 8,000 in Europe.1 About the VALIANT StudyThe VALIANT Phase 3 study (NCT05067127) was a randomized, placebo-controlled, double-blinded, multi-center study that evaluated EMPAVELI® (pegcetacoplan) efficacy and safety in 124 patients who were 12 years of age and older with C3G or primary IC-MPGN. It is the largest single trial conducted in these populations and the only study to include pediatric and adult patients, with native and post-transplant kidneys. Study participants were randomized to receive EMPAVELI or placebo twice weekly for 26 weeks. Following this 26-week randomized controlled period, patients were able to proceed to a 26-week open-label phase in which all patients received EMPAVELI. The primary endpoint of the study was the log transformed ratio of urine protein-to-creatinine ratio (UPCR) at Week 26 compared to baseline. About EMPAVELI®/Aspaveli® (pegcetacoplan)EMPAVELI®/Aspaveli® (pegcetacoplan) is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. It is approved for the treatment of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in the United States and paroxysmal nocturnal hemoglobinuria (PNH) in the United States, European Union, and other countries globally. The therapy is also under investigation for other rare diseases. U.S. Important Safety Information for EMPAVELI BOXED WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA EMPAVELI, a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria, such as , , and type B. Life-threatening and fatal infections with encapsulated bacteria have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early. Complete or update vaccination for encapsulated bacteria at least 2 weeks prior to the first dose of EMPAVELI, unless the risks of delaying therapy with EMPAVELI outweigh the risks of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against encapsulated bacteria in patients receiving a complement inhibitor. Patients receiving EMPAVELI are at increased risk for invasive disease caused by encapsulated bacteria, even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious infections and evaluate immediately if infection is suspected. Because of the risk of serious infections caused by encapsulated bacteria, EMPAVELI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the EMPAVELI REMS. CONTRAINDICATIONS Hypersensitivity to pegcetacoplan or to any of the excipients For initiation in patients with unresolved serious infection caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B WARNINGS AND PRECAUTIONS Serious Infections Caused by Encapsulated Bacteria EMPAVELI, a complement inhibitor, increases a patient's susceptibility to serious, life-threatening, or fatal infections caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis (caused by any serogroup, including non-groupable strains), and Haemophilus influenzae type B. Life-threatening and fatal infections with encapsulated bacteria have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors. The initiation of EMPAVELI treatment is contraindicated in patients with unresolved serious infection caused by encapsulated bacteria. Complete or update vaccination against encapsulated bacteria at least 2 weeks prior to administration of the first dose of EMPAVELI, according to the most current ACIP recommendations for patients receiving a complement inhibitor. Revaccinate patients in accordance with ACIP recommendations considering the duration of therapy with EMPAVELI. Note that ACIP recommends an administration schedule in patients receiving complement inhibitors that differs from the administration schedule in the vaccine prescribing information. If urgent EMPAVELI therapy is indicated in a patient who is not up to date with vaccines against encapsulated bacteria according to ACIP recommendations, provide the patient with antibacterial drug prophylaxis and administer these vaccines as soon as possible. The benefits and risks of treatment with EMPAVELI, as well as the benefits and risks of antibacterial drug prophylaxis in unvaccinated or vaccinated patients, must be considered against the known risks for serious infections caused by encapsulated bacteria. Vaccination does not eliminate the risk of serious encapsulated bacterial infections, despite development of antibodies following vaccination. Closely monitor patients for early signs and symptoms of serious infection and evaluate patients immediately if an infection is suspected. Inform patients of these signs and symptoms and instruct patients to seek immediate medical care if these signs and symptoms occur. Promptly treat known infections. Serious infection may become rapidly life-threatening or fatal if not recognized and treated early. Consider interruption of EMPAVELI in patients who are undergoing treatment for serious infections. EMPAVELI is available only through a restricted program under a REMS. EMPAVELI REMS EMPAVELI is available only through a restricted program under a REMS called EMPAVELI REMS, because of the risk of serious infections caused by encapsulated bacteria. Notable requirements of the EMPAVELI REMS include the following: Under the EMPAVELI REMS, prescribers must enroll in the program. Prescribers must counsel patients about the risks, signs, and symptoms of serious infections caused by encapsulated bacteria, provide patients with the REMS educational materials, ensure patients are vaccinated against encapsulated bacteria at least 2 weeks prior to the first dose of EMPAVELI, prescribe antibacterial drug prophylaxis if patients' vaccine status is not up to date and treatment must be started urgently, and provide instructions to always carry the Patient Safety Card both during treatment, as well as for 2 months following last dose of EMPAVELI. Pharmacies that dispense EMPAVELI must be certified in the EMPAVELI REMS and must verify prescribers are certified. Further information is available at or 1-888-343-7073. Infusion-Related Reactions Systemic hypersensitivity reactions (eg, facial swelling, rash, urticaria, pyrexia) have occurred in patients treated with EMPAVELI, which may resolve after treatment with antihistamines. Cases of anaphylaxis leading to treatment discontinuation have been reported. If a severe hypersensitivity reaction (including anaphylaxis) occurs, discontinue EMPAVELI infusion immediately, institute appropriate treatment, per standard of care, and monitor until signs and symptoms are resolved. Interference with Laboratory Tests There may be interference between silica reagents in coagulation panels and EMPAVELI that results in artificially prolonged activated partial thromboplastin time (aPTT); therefore, avoid the use of silica reagents in coagulation panels. ADVERSE REACTIONS Most common adverse reactions in adult and pediatric patients 12 years of age and older with C3G or primary IC-MPGN (incidence ≥10%) were infusion-site reactions, pyrexia, nasopharyngitis, influenza, cough, and nausea. USE IN SPECIFIC POPULATIONS Females of Reproductive Potential EMPAVELI may cause embryo-fetal harm when administered to pregnant women. Pregnancy testing is recommended for females of reproductive potential prior to treatment with EMPAVELI. Advise female patients of reproductive potential to use effective contraception during treatment with EMPAVELI and for 40 days after the last dose. Please see full , including Boxed WARNING regarding serious infections caused by encapsulated bacteria, and . About Apellis Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company leading the way in complement science to develop life-changing therapies for some of the most challenging diseases patients face. We ushered in the first new class of complement medicine in 15 years and now have two C3-targeting medicines approved to treat four serious diseases. Breakthroughs for patients include the first-ever therapy for geographic atrophy, a leading cause of blindness, and the first treatment for patients 12 and older with C3G or primary IC-MPGN, two severe, rare kidney diseases. We believe we have only begun to unlock the potential of targeting C3 across many serious diseases. For more information, please visit or follow us on LinkedIn and X. Apellis Forward-Looking Statement Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute 'forward-looking statements' within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements regarding the potential market opportunity of EMPAVELI for C3G and IC-MPGN. The words 'anticipate,' 'believe,' 'continue,' 'could,' 'estimate,' 'expect,' 'intend,' 'may,' 'plan,' 'potential,' 'predict,' 'project,' 'should,' 'target,' 'will,' 'would' and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including whether the clinical trial results of EMPAVELI for C3G and IC-MPGN indicate an effect that is greater than the actual positive effect; and any other factors discussed in the 'Risk Factors' section of Apellis' Annual Report on Form 10-K with the Securities and Exchange Commission on February 28, 2025 and the risks described in other filings that Apellis may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Apellis specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. Media:Tracy Vineismedia@ Investors: Neil Carnahan, References1. Data on file using literature consensus. 2. Smith RJH, et al. Nat Rev Nephrol. 2019;15(3):129-143.3. Servais A, et al. Kidney Int. 2012;82(4):454-464.4. Zand L, et al. J Am Soc Nephrol. 2014;25(5):1110-1117.5. Tarragón, B, et al. C3 Glomerulopathy Recurs Early after Kidney Transplantation in Serial Biopsies Performed within the First 2 Years after Transplantation. Clinical Journal of the American Society of Nephrology. August 2024; 19(8)1005-1015. doi: 10.2215/CJN.0000000000000474. A photo accompanying this announcement is available at

Sarepta will resume gene therapy shipments after FDA review of recent patient death
Sarepta will resume gene therapy shipments after FDA review of recent patient death

The Hill

timean hour ago

  • The Hill

Sarepta will resume gene therapy shipments after FDA review of recent patient death

WASHINGTON (AP) — Shares of beleaguered drugmaker Sarepta Therapeutics jumped in afterhours trading Monday after the company said it would resume shipping its gene therapy for some patients, following a brief pause requested by regulators. The Food and Drug Administration said it recommended lifting the hold for young patients with Duchenne's muscular dystrophy who are still able to walk. Regulators had requested the pause after the deaths of two older teenagers who were taking the therapy. The FDA also said in a statement it determined that a recently reported death of an 8-year-old boy was unrelated to the therapy. Company shares surged more than 16% after the announcement to $13.86 in afterhours trading. The jump is the latest in a series of drastic stock movements triggered by changing fortunes for the company's best-selling product. Elevidys is the first gene therapy approved in the U.S. for Duchenne's muscular dystrophy, the fatal muscle-wasting disease that affects boys and young men, resulting in early death. It received accelerated approval in 2023 for a narrow range of young patients and was expanded last year for use in older patients, including those who can no longer walk. The FDA decision Monday 'significantly improves Elevidys' sales outlook in the near-term,' Jefferies analyst Andrew Tsai told investors, in a note after the announcement. 'The street will feel relieved about the situation, suggesting meaningful stock upside potential.' Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver injury, a known side effect of the treatment. The FDA then requested a pause in shipments of the drug after the death of a third patient taking a different Sarepta therapy. FDA officials have suggested the company will need to provide new study data on safety to resume Elevidys' use in older patients. 'The FDA will continue to work with the sponsor regarding non-ambulatory patients, which remains subject to a voluntary hold, following two deaths,' FDA said in its statement. ___ The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Department of Science Education and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.

Sarepta will resume gene therapy shipments after FDA review of recent patient death
Sarepta will resume gene therapy shipments after FDA review of recent patient death

San Francisco Chronicle​

timean hour ago

  • San Francisco Chronicle​

Sarepta will resume gene therapy shipments after FDA review of recent patient death

WASHINGTON (AP) — Shares of beleaguered drugmaker Sarepta Therapeutics jumped in afterhours trading Monday after the company said it would resume shipping its gene therapy for some patients, following a brief pause requested by regulators. The Food and Drug Administration said it recommended lifting the hold for young patients with Duchenne's muscular dystrophy who are still able to walk. Regulators had requested the pause after the deaths of two older teenagers who were taking the therapy. The FDA also said in a statement it determined that a recently reported death of an 8-year-old boy was unrelated to the therapy. Company shares surged more than 16% after the announcement to $13.86 in afterhours trading. The jump is the latest in a series of drastic stock movements triggered by changing fortunes for the company's best-selling product. Elevidys is the first gene therapy approved in the U.S. for Duchenne's muscular dystrophy, the fatal muscle-wasting disease that affects boys and young men, resulting in early death. It received accelerated approval in 2023 for a narrow range of young patients and was expanded last year for use in older patients, including those who can no longer walk. The FDA decision Monday 'significantly improves Elevidys' sales outlook in the near-term,' Jefferies analyst Andrew Tsai told investors, in a note after the announcement. 'The street will feel relieved about the situation, suggesting meaningful stock upside potential.' Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver injury, a known side effect of the treatment. The FDA then requested a pause in shipments of the drug after the death of a third patient taking a different Sarepta therapy. FDA officials have suggested the company will need to provide new study data on safety to resume Elevidys' use in older patients. 'The FDA will continue to work with the sponsor regarding non-ambulatory patients, which remains subject to a voluntary hold, following two deaths,' FDA said in its statement. ___ The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Department of Science Education and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.

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