Latest news with #Barth
Politico
2 days ago
- Business
- Politico
FDA denies ultra-rare drug but offers path to ‘yes'
Presented by With help from Chelsea Cirruzzo Driving The Day ANOTHER BLOW TO BARTH'S TREATMENT — The FDA rejected Stealth BioTherapeutics' latest bid to market its proposed treatment for an ultra-rare genetic condition, though regulators have suggested a 'path' toward accelerated approval, the company said Thursday. Stealth said the agency's complete response letter, which came four months after the FDA was initially scheduled to make a decision, didn't raise concerns with clinical safety data for the drug, known as elamipretide, to treat Barth syndrome by targeting underlying dysfunction in mitochondria, which are small energy-producing structures in cells. CEO Reenie McCarthy told Prescription Pulse that agency requests for additional data in late 2024 led the biotech to expect approval, and the biotech believes they should have gotten accelerated approval based on what the FDA conveyed in the letter. 'They have all the information they need to make that decision,' she said. HHS didn't respond to a request for comment. Background: Barth syndrome — which can cause an enlarged and weakened heart, muscle weakness, recurrent infections and delayed growth — typically affects males and shortens their life expectancy by decades. About 150 Americans have the condition. Stealth had suggested two ways the FDA could consider approving elamipretide — granting full approval based on the company's natural history study of disease progression absent medical intervention or endorsing accelerated approval based on a trial suggesting long-term improvements in muscle function. The FDA has agreed to consider muscle strength around the knees as a potential intermediate marker by which it could support accelerated approval and has asked Stealth to resubmit its application based on that endpoint, McCarthy said. External FDA advisers voted last year to recommend approval for elamipretide despite persistent questions about its effectiveness. Stealth, rare disease advocates and patients with Barth syndrome and their caregivers have advocated for years for FDA approval, highlighting the difficulties in conducting trials for potential rare disease treatments that can't be studied in large populations. Thursday's rejection seems 'inconsistent' with FDA Commissioner Marty Makary's recent comments about approving rare disease therapies based on plausible mechanisms and patient demand, McCarthy said. 'There's a bit of a disconnect' between those statements and the FDA's feedback, she said. What's next: Stealth expects to meet with the FDA next month to discuss a postmarketing study it proposed in 2022 and 'gauge how serious they are,' McCarthy said. The company laid off 30 percent of its staff to save money for a potential FDA resubmission and to maintain elamipretide access for critically ill patients under the agency's compassionate use pathway. McCarthy also raised concern about the FDA's stance that an accelerated approval would make the drug inappropriate to administer to newborns in acute cardiac distress, a population she said makes up two-thirds of Stealth's expanded access program. Half of early Barth syndrome deaths occur by age 1, per the company. IT'S FRIDAY. WELCOME BACK TO PRESCRIPTION PULSE. Do you work at HHS? We want to learn more about the decision to cancel Moderna's bird flu vaccine funding. Send your tips to David Lim (dlim@ @davidalim or davidalim.49 on Signal) and Lauren Gardner (lgardner@ @Gardner_LM or gardnerlm.01 on Signal). Eye on the FDA REPLACING COLOR ADDITIVES IN MEDS — The FDA quietly released draft recommendations for drugmakers that want to replace color additives in their approved drugs — potentially hinting at where the Trump administration will next focus HHS Secretary Robert F. Kennedy Jr.'s Make America Healthy Again energies. The agency acknowledged that changing the formulation of a drug — including its inactive ingredients — is generally considered a major change. But the agency said replacing a color additive with another already listed in the FDA's color-additive regulations in many cases 'is unlikely to have an adverse effect on the identity, strength, quality, purity, or potency of the drug product.' Replacing a color additive can 'generally be considered a moderate change' that can be requested via a 30-day supplement known as a CBE-30, according to the draft guidance published Thursday. 'If a manufacturer or an applicant intends to use a color additive that is not already listed in FDA's color additive regulations for the particular use, a petition must be sent to the Human Foods Program,' the draft guidance says. 'If FDA finds the color additive safe and suitable for such use, the additive will be listed in the color additive regulations.' What's next: The FDA is taking comments on the draft guidance for 60 days. AROUND THE AGENCIES LAYOFFS ON HOLD — CDC layoffs planned for early June are being paused in the wake of a preliminary injunction, two employees at the agency, granted anonymity for fear of retribution, confirmed to POLITICO's Sophie Gardner. CDC employees who were sent termination notices in April received an email from the agency's Office of Human Resources on Thursday, informing them that, because of a preliminary injunction, HHS 'is staying further action on any existing Reduction in Force (RIF) notices, including final separation of employees, at this time.' The laid-off employees will remain on paid administrative leave 'or in their current employment status' until further notice, the email said. An HHS spokesperson did not immediately respond to a request for comment. Background: On April 1, amid a massive restructuring of HHS, around 18 percent of the CDC's workforce received termination notices. But legal challenges have complicated the reduction in force and the HHS reorganization, meaning the vast majority of those employees are still technically employed at the CDC, with most remaining on administrative leave. Key context: On May 22, Judge Susan Illston of the federal district court in San Francisco issued a preliminary injunction that bars the administration from carrying out the reduction in force across 22 agencies — including HHS — that are defendants in the case. Federal employee unions, nonprofits and local governments are the plaintiffs. The email sent to CDC employees cites Illston's injunction as the reason for the decision. PROOFREADER WANTED — The White House blamed 'formatting issues' for errors in the citations of the recently released Make America Healthy Again report after a media report found studies in it that do not exist. 'We have complete confidence in [Secretary Robert F. Kennedy Jr.] and his team at HHS. I understand there were some formatting issues with the MAHA report,' White House press secretary Karoline Leavitt said Thursday. Her remarks came after NOTUS reported that seven of the sources cited in the report, which said children's health is in crisis and blamed chemicals, lack of exercise and ultraprocessed foods, don't exist. The report now lists five of its citations as 'corrected' or 'updated,' though that doesn't include the citations NOTUS reported as false. 'Some of the hundreds of citations in the report were formatted incorrectly or mistakenly referenced something other than what was actually intended,' a White House spokesperson told Pulse. 'That said, the content of the report is fully substantiated, and there is nothing in there that cannot be backed up; we did not conjure up any facts. Report has been corrected now.' Pharma Moves Laura Akowuah has joined Cooley as special counsel. She previously worked at the FDA for more than a decade in enforcement roles. Document Drawer The FDA approved Eton Pharmaceuticals' hydrocortisone oral solution Wednesday as a replacement therapy in patients ages 5 and older with adrenocortical insufficiency. WHAT WE'RE READING POLITICO's Erin Schumaker spoke with Dr. Wafik El-Deiry, a Brown University cancer researcher, about his campaign to lead the National Cancer Institute amid NIH funding cuts. Two pharmacy benefit managers filed lawsuits Thursday challenging a recently passed Arkansas law they say would force pharmacies across the state to close, Modern Healthcare's Hayley DeSilva writes. Texas lawmakers have sent legislation to Republican Gov. Greg Abbott's desk that would make it easier for parents to start the process of exempting their children from school vaccine mandates, The Associated Press' Jim Vertuno reports. Several House Republicans are raising concerns about broad pharmaceutical tariffs potentially being placed on the industry, POLITICO's Ari Hawkins reports.
Yahoo
3 days ago
- Business
- Yahoo
Barth Syndrome Foundation Responds to FDA's Proposed 'Path Forward' for Elamipretide
Families face more delay despite dire need, strong data and years and years of effort BOSTON, May 29, 2025--(BUSINESS WIRE)--The Barth Syndrome Foundation (BSF) today responded to new developments from the U.S. Food and Drug Administration (FDA) regarding elamipretide, a potential treatment for the ultra-rare and life-threatening condition. Despite an already lengthy review and a 10-6 favorable FDA Cardiovascular and Renal Drugs Advisory Committee vote last October, the FDA now has indicated to Stealth BioTherapeutics that the agency will not approve the current application for this drug. However, for the first time, the agency issued a written recommendation outlining a potential path to accelerated approval based on previously submitted data showing more than 45 percent improvement in muscle strength and its correlation with increased physical function. "It's unconscionable that it now will take even longer for the FDA to rule on this drug for our very small population with no other specific therapies," said Emily Milligan, executive director, Barth Syndrome Foundation. "This administration has sent strong signals it's serious about defining a process that works for rare disease families. Now is the time to deliver. We need FDA leadership to take strong, decisive action and put an end to the hemming and hawing. This literally translates into life, death and better lives for our children." Barth syndrome is an ultra-rare, life-threatening genetic disorder primarily affecting males. There are no FDA-approved treatments and no other therapies in late-stage clinical development. Clinical studies show elamipretide can improve mitochondrial function and boost muscle strength by 45 percent and heart function by 40 percent in patients with Barth syndrome. Most patients from the original trial continue to take and benefit from this therapy more than eight years after initiating treatment. Many more individuals, mostly infants, in acute cardiac distress have received elamipretide through emergency access requests approved by the FDA. Despite the extensive safety record and strong vote of the advisory committee, the FDA has not granted approval. Nearly 20 percent of U.S. patients with Barth syndrome are currently receiving elamipretide through an expanded access program. The FDA's current proposed path, unfortunately, excludes the sickest babies, who make up a majority of those currently receiving the drug through emergency access. Over half of deaths in Barth syndrome occur in the first few years of life. Yet the FDA has declined to include these patients in the potential approval path, citing a lack of feasibility for pre-approval studies. "No safety concerns have been raised, and the agency acknowledged the difficulty of conducting traditional trials in a disease this rare," Milligan said. "Still, families are left in limbo, waiting on a treatment that medical experts, patient experiences and common sense overwhelmingly support." More than 50 clinicians and nearly 20,000 people from across the globe have signed letters and petitions over the last few years urging the FDA to approve elamipretide without further delay. Their views echo the lived experience of families and patients who have endured years of uncertainty. "With no approved therapies and limited access to treatment, every day of inaction carries a devastating cost," Milligan said. "The FDA made a promise to our community—to conduct a fair, thorough and equitable review of elamipretide through the advisory committee process. That promise remains unfulfilled." Stories Behind the Science Families from across the country continue to fight for access to a treatment that could mean survival. Their stories offer a powerful reminder of what is at stake: In Mississippi, a mother describes her desperate effort to keep her son alive with no other options: "Mississippi's Heartbeat" In California, one physician's 3 a.m. call from the ICU leads to a life-saving intervention: "In the ICU at 3 a.m." In Ohio, a family spanning two generations shares what this drug means to them: "Two Generations, One Drug" In Texas, a newborn's life is saved just in time by emergency access: "A Life Saved in the Nick of Time" In Georgia, one family measures survival in milligrams and urgency: "A Life Measured in Milligrams" In New York, a 13-year fight shows what resilience in rare disease looks like: "Too Rare to Care" And in Oklahoma, one young man explains why elamipretide is the drug that gave him his life back: "Walker Burger's Fight" Each of these families speaks with one voice: the time to approve this treatment is now. About Barth Syndrome Foundation (BSF) Barth Syndrome Foundation and our international affiliates comprise the only global network of families, health care providers, and researchers solely driven by the mission to save lives through education, advances in treatments, and finding a cure for Barth syndrome. Considered a role model in rare disease advocacy, BSF has funded $7 million and catalyzed more than $41.4 million in funding from other sources to advance global scientific discoveries. BSF also provides a lifeline to families around the world through individualized support, educational conferences, a patient registry, and collaborations with expert providers to define standards of care, treatment, and rapid diagnosis. View source version on Contacts BSF Media Contact: Matt Burnsmatt@ 518-423-5907
Reuters
3 days ago
- Business
- Reuters
US FDA asks Stealth BioTherapeutics to resubmit application for rare genetic condition therapy
May 29 (Reuters) - The U.S. Food and Drug Administration has asked for a resubmission of Stealth BioTherapeutics' marketing application for its experimental therapy targeting a rare genetic condition, Barth syndrome, the company said on Thursday. The drug developer added that it has implemented a 30% workforce reduction to conserve resources for a potential resubmission. The federal health regulator's decision marks another setback in Stealth's prolonged effort to bring the therapy, elamipretide, to market after several delays. Stealth received the FDA's so-called 'complete response letter' after a 16.5-month review, during which the FDA extended its review from January to April to assess additional data. Last month, the regulator missed the extended deadline. The Needham, Massachusetts-based company is seeking approval for elamipretide, a potential treatment for Barth syndrome - a condition that typically affects boys and causes heart, muscle weakness and delays growth. Stealth had submitted data from a mid-stage trial, which showed improvement in knee muscle strength, which was correlated with improvements on the six-minute walk test. The FDA has agreed to consider knee extensor muscle strength, for which the company previously submitted data, as a goal to support accelerated approval, the company said. However, the regulator is hesitant to extend the accelerated pathway to critically ill newborns, who make up nearly two-thirds of the therapy's expanded access program participants. Barth syndrome affects fewer than 150 individuals in the United States and fewer than 300 worldwide, and currently, no approved therapies exist.
Yahoo
3 days ago
- Business
- Yahoo
Stealth BioTherapeutics Announces "Path Forward" Despite Disappointing Delay for Ultra-rare Barth Syndrome
Following five years of discussions and a positive advisory committee recommendation, FDA proposes a potential accelerated approval pathway requiring resubmission of the elamipretide NDA Barth syndrome, a progressive, lethal, pediatric cardioskeletal disease often diagnosed at birth and with no approved therapies, affects ~150 individuals in the U.S. NEEDHAM, Mass., May 29, 2025 /PRNewswire/ -- Stealth BioTherapeutics Inc. (the "Company" or "Stealth"), a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced that the U.S. Food and Drug Administration ("FDA") has identified a potential path forward for elamipretide for the treatment of Barth syndrome following years of discussions with the Agency and a 2024 Cardiovascular and Renal Drugs Advisory Committee meeting ("Ad Com") that concluded that elamipretide is effective for the treatment of Barth syndrome. The Company submitted data in its January 2024 new drug application ("NDA") showing that knee extensor muscle strength, which improved by over 45 percent in the Company's Phase 2 clinical trial, was significantly correlated with improvements on the six-minute walk test, an FDA-recognized indicator of clinical benefit. The Company proposed in its NDA and the FDA has now agreed to consider knee extensor muscle strength, for which the Company previously submitted data, as a potential intermediate clinical endpoint to support accelerated approval. "We recognize that the FDA's recommendation for an accelerated approval for elamipretide in Barth syndrome offers a path forward for this incurable pediatric disease that affects an incredibly small number of individuals worldwide," said Reenie McCarthy, CEO. "Elamipretide, which targets the cardiolipin deficit central to Barth syndrome, is the only agent in clinical development to treat this ultra-rare disease for which the FDA has acknowledged that additional pre-approval randomized controlled trials are unfeasible. We hope the FDA will also prioritize ensuring rapid access for neonates affected by the disease subject to appropriate post-marketing safety monitoring. We are experiencing a sharp increase in emergency access requests for critically ill infants from medical experts worldwide following the recent publication of several positive case study reports." Stealth received the FDA's complete response letter after a 16.5-month priority review cycle during which the FDA extended its review from January to April 2025 then missed its April 29 extended prescription drug user fee action date. The FDA also cited observations arising from a May 2025 CGMP surveillance inspection of a third-party manufacturing facility for elamipretide, although no specific deficiencies related to the elamipretide manufacturing process were identified. The FDA did not raise concerns with the clinical safety data, requesting only that a resubmission include any additional safety data collected since NDA submission. The Company will meet with the FDA next month to discuss the proposed post-marketing study, with which the Agency previously expressed alignment when originally proposed by the Company in 2022. The regulatory pathway for this development effort has been complex, entailing four different FDA review divisions since data was first presented to the FDA in 2019. The ultra-rare nature of Barth syndrome, the FDA's reservations regarding the positive data from SPIBA-001, a Phase 3 natural history control study assessing the functional endpoints utilized in the TAZPOWER open-label extension, and the FDA's prior refusal to consider an accelerated approval pathway all contributed to the challenges of the review. In its NDA submission, the Company proposed accelerated approval on the basis of a muscle strength intermediate endpoint to address the FDA's desire, echoed by several members of the Ad Com, to see additional confirmatory data. The FDA's decision to now consider this pathway offers hope for access to treatment for the U.S. Barth syndrome patient community, approximately 20% of whom already receive elamipretide under the Company's expanded access program. However, the Agency has expressed reluctance to extend the accelerated pathway to critically ill neonates, who comprise nearly two-thirds of the Company's expanded access program. Half of early deaths reported in this lethal pediatric disease occur by age one. The FDA's recommendation to resubmit on the basis of this new advice will further delay the potential approval and launch of elamipretide. The Company has implemented a 30% reduction in its personnel to conserve resources to fund a potential NDA resubmission and avoid interrupting patients' access to elamipretide through the Company's expanded access program. About Barth SyndromeBarth syndrome is an ultra-rare genetic condition characterized by cardiac abnormalities leading to exercise intolerance, muscle weakness, debilitating fatigue, heart failure, recurrent infections, and delayed growth. The disease is associated with reduced life expectancy, with 85% of early deaths occurring by age 5. Barth syndrome occurs primarily in males and is estimated to affect one in 1,000,000 males worldwide or around 150 individuals in the United States. There are currently no FDA- or EMA-approved therapies for patients with Barth syndrome. Elamipretide has Orphan Drug, Fast Track, Priority Review, and Rare Pediatric Designation from the FDA and Orphan Drug Designation from the EMA for the treatment of Barth syndrome. About Stealth BioTherapeuticsOur mission is to develop novel therapies to improve the lives of patients living with diseases of mitochondrial dysfunction. Our lead product candidate, elamipretide, is under review for Barth syndrome and in late-stage development for primary mitochondrial myopathy and dry age-related macular degeneration. We are also evaluating a topical ophthalmic formulation of our second-generation clinical-stage candidate, bevemipretide (SBT-272), for dry age-related macular degeneration, and have a deep pipeline of novel compounds under evaluation for rare neurological and cardiac disease indications. Media ContactAnna Stallmann CommunicationsAnna Stallmannanna@ Investor ContactPrecision AQAustin View original content to download multimedia: SOURCE Stealth BioTherapeutics Inc. Sign in to access your portfolio
Yahoo
3 days ago
- Business
- Yahoo
Barth Syndrome Foundation Responds to FDA's Proposed 'Path Forward' for Elamipretide
Families face more delay despite dire need, strong data and years and years of effort BOSTON, May 29, 2025--(BUSINESS WIRE)--The Barth Syndrome Foundation (BSF) today responded to new developments from the U.S. Food and Drug Administration (FDA) regarding elamipretide, a potential treatment for the ultra-rare and life-threatening condition. Despite an already lengthy review and a 10-6 favorable FDA Cardiovascular and Renal Drugs Advisory Committee vote last October, the FDA now has indicated to Stealth BioTherapeutics that the agency will not approve the current application for this drug. However, for the first time, the agency issued a written recommendation outlining a potential path to accelerated approval based on previously submitted data showing more than 45 percent improvement in muscle strength and its correlation with increased physical function. "It's unconscionable that it now will take even longer for the FDA to rule on this drug for our very small population with no other specific therapies," said Emily Milligan, executive director, Barth Syndrome Foundation. "This administration has sent strong signals it's serious about defining a process that works for rare disease families. Now is the time to deliver. We need FDA leadership to take strong, decisive action and put an end to the hemming and hawing. This literally translates into life, death and better lives for our children." Barth syndrome is an ultra-rare, life-threatening genetic disorder primarily affecting males. There are no FDA-approved treatments and no other therapies in late-stage clinical development. Clinical studies show elamipretide can improve mitochondrial function and boost muscle strength by 45 percent and heart function by 40 percent in patients with Barth syndrome. Most patients from the original trial continue to take and benefit from this therapy more than eight years after initiating treatment. Many more individuals, mostly infants, in acute cardiac distress have received elamipretide through emergency access requests approved by the FDA. Despite the extensive safety record and strong vote of the advisory committee, the FDA has not granted approval. Nearly 20 percent of U.S. patients with Barth syndrome are currently receiving elamipretide through an expanded access program. The FDA's current proposed path, unfortunately, excludes the sickest babies, who make up a majority of those currently receiving the drug through emergency access. Over half of deaths in Barth syndrome occur in the first few years of life. Yet the FDA has declined to include these patients in the potential approval path, citing a lack of feasibility for pre-approval studies. "No safety concerns have been raised, and the agency acknowledged the difficulty of conducting traditional trials in a disease this rare," Milligan said. "Still, families are left in limbo, waiting on a treatment that medical experts, patient experiences and common sense overwhelmingly support." More than 50 clinicians and nearly 20,000 people from across the globe have signed letters and petitions over the last few years urging the FDA to approve elamipretide without further delay. Their views echo the lived experience of families and patients who have endured years of uncertainty. "With no approved therapies and limited access to treatment, every day of inaction carries a devastating cost," Milligan said. "The FDA made a promise to our community—to conduct a fair, thorough and equitable review of elamipretide through the advisory committee process. That promise remains unfulfilled." Stories Behind the Science Families from across the country continue to fight for access to a treatment that could mean survival. Their stories offer a powerful reminder of what is at stake: In Mississippi, a mother describes her desperate effort to keep her son alive with no other options: "Mississippi's Heartbeat" In California, one physician's 3 a.m. call from the ICU leads to a life-saving intervention: "In the ICU at 3 a.m." In Ohio, a family spanning two generations shares what this drug means to them: "Two Generations, One Drug" In Texas, a newborn's life is saved just in time by emergency access: "A Life Saved in the Nick of Time" In Georgia, one family measures survival in milligrams and urgency: "A Life Measured in Milligrams" In New York, a 13-year fight shows what resilience in rare disease looks like: "Too Rare to Care" And in Oklahoma, one young man explains why elamipretide is the drug that gave him his life back: "Walker Burger's Fight" Each of these families speaks with one voice: the time to approve this treatment is now. About Barth Syndrome Foundation (BSF) Barth Syndrome Foundation and our international affiliates comprise the only global network of families, health care providers, and researchers solely driven by the mission to save lives through education, advances in treatments, and finding a cure for Barth syndrome. Considered a role model in rare disease advocacy, BSF has funded $7 million and catalyzed more than $41.4 million in funding from other sources to advance global scientific discoveries. BSF also provides a lifeline to families around the world through individualized support, educational conferences, a patient registry, and collaborations with expert providers to define standards of care, treatment, and rapid diagnosis. View source version on Contacts BSF Media Contact: Matt Burnsmatt@ 518-423-5907
