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Pharma & Medications
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Fierce Pharma
An update on the pharma industry’s reshoring effort
Fierce PharmaFDA Approves Foundayo: First New Molecular Entity Under National Priority Voucher Program
The U.S. Food and Drug Administration (FDA) has approved Foundayo (orforglipron), marking a significant milestone as the first new molecular entity (NME) approved under the National Priority Voucher (CNPV) pilot program. This approval, which occurred just 50 days after the application was filed, is the fastest for an NME since 2002. Foundayo is indicated for adults with obesity or overweight who have at least one weight-related comorbidity, and is intended to be used alongside a reduced-calorie diet and increased physical activity to aid in weight loss and maintenance. The CNPV program, launched in 2025, aims to expedite the approval process for drugs that address critical national health priorities. The FDA has issued 18 vouchers and made six decisions under this program, which emphasizes enhanced communication and rolling reviews to shorten approval times without compromising safety. The target timeline for decisions is two months, reflecting the FDA's commitment to efficiency and thoroughness in drug evaluation. Foundayo is a glucagon-like peptide-1 (GLP-1) receptor agonist available in tablet form, with a starting dosage of 0.8 mg, which can be increased based on patient response. Clinical trials demonstrated that treatment with Foundayo, in conjunction with lifestyle changes, led to significant weight loss compared to placebo. However, the drug is associated with potential side effects, including gastrointestinal issues and warnings for serious conditions such as pancreatitis and thyroid tumors. The FDA plans to hold a public meeting on June 4, 2026, to gather feedback on the CNPV program, including its eligibility criteria and review processes. This meeting aims to refine the program and ensure it continues to meet the needs of patients and healthcare providers effectively. Eli Lilly and Company received the approval for Foundayo, providing a new option for individuals struggling with obesity and related health issues.
FDA News · 2d agoEMA Proposes Virtual Control Groups to Minimize Animal Use in Drug Development
The European Medicines Agency's Committee for Human Medicinal Products (CHMP) has released a draft qualification opinion advocating for the use of virtual control groups in preclinical research. This innovative methodology aims to reduce the number of animals, specifically rats, used in dose-range finding studies by replacing traditional concurrent animal control groups with virtual counterparts. The CHMP's endorsement of this new approach signifies a pivotal step towards minimizing animal testing in the pharmaceutical industry, aligning with the 3Rs principles of replacement, reduction, and refinement. The proposed virtual control groups are established through a statistical framework that characterizes control data and identifies suitable 'virtual comparator animals' for treated subjects. This method is designed to ensure that the integrity of study outcomes remains intact while enhancing the ethical standards of drug development. The CHMP's qualification opinion is expected to pave the way for future applications of this methodology in toxicological studies, potentially leading to a significant decrease in the overall number of animals required for testing. Public consultation on the draft opinion is open until May 12, 2026, inviting feedback from the scientific community and stakeholders. This initiative not only supports the EMA's commitment to ethical research practices but also aligns with global efforts led by the International Coalition of Medicines Regulatory Authorities (ICMRA) to promote alternative testing methods. As the EMA progresses towards integrating virtual control groups, it aims to enhance the predictability and relevance of non-clinical testing, ultimately fostering innovation while ensuring human and animal safety in clinical trials.
EMA News · 3d agoEMA Recommends Imdylltra for Relapsed Extensive-Stage Small Cell Lung Cancer Treatment
The European Medicines Agency (EMA) has recommended the marketing authorization of Imdylltra (tarlatamab) for adults with extensive-stage small cell lung cancer (ES-SCLC) who have relapsed after platinum-based chemotherapy. SCLC is a rare and aggressive cancer with a poor prognosis, and treatment options post-relapse are limited. Imdylltra represents a new targeted immunotherapy that engages T-cells to attack tumor cells by binding to the DLL3 protein on cancer cells and the CD3 protein on T-cells, leading to tumor cell death. The recommendation is based on a phase 3 study involving 509 adults, which demonstrated a significant improvement in median overall survival (OS) of 13.6 months for patients treated with Imdylltra compared to 8.3 months for those receiving standard care. Additionally, the median progression-free survival (PFS) was 4.2 months for Imdylltra versus 3.2 months for standard treatments. This translates to a 40% reduction in the risk of death for patients on Imdylltra. While Imdylltra offers promising results, it is associated with serious side effects, including cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS). These conditions require early recognition and management, and patients will receive a card detailing symptoms and when to seek urgent care. Other common side effects include decreased appetite, fever, and fatigue. The EMA's positive opinion is a crucial step towards patient access, pending a decision from the European Commission on the marketing authorization. Following this, individual Member States will determine pricing and reimbursement, considering the drug's role within their healthcare systems. Imdylltra has also been designated as an orphan medicinal product, and its orphan status will be reassessed by the Committee for Orphan Medicinal Products (COMP).
EMA News · Mar 27FDA Approves Avlayah for Neurologic Symptoms of Hunter Syndrome in Pediatric Patients
The U.S. Food and Drug Administration (FDA) has granted approval for Avlayah (tividenofusp alfa-eknm) to treat neurologic manifestations of Hunter syndrome, a rare genetic disorder affecting primarily males. This milestone approval, announced on March 25, 2026, is significant for the approximately 500 individuals in the U.S. diagnosed with this condition, which leads to the accumulation of glycosaminoglycans in cells, resulting in severe physical and mental developmental issues. Avlayah is administered as a weekly intravenous infusion and is intended for use in pediatric patients weighing at least 5 kg, either presymptomatic or symptomatic, prior to advanced neurologic impairment. FDA Commissioner Dr. Marty Makary emphasized the agency's commitment to accelerating treatments for rare diseases, highlighting the importance of regulatory flexibility and substantial evidence of effectiveness. The approval was based on a surrogate endpoint, specifically the reduction of cerebrospinal fluid heparan sulfate (CSF HS), which is linked to the organ damage associated with Hunter syndrome. In a clinical trial involving 47 pediatric patients, Avlayah demonstrated a significant average reduction of 91% in CSF HS levels at 24 weeks, with 93% of patients achieving levels below the upper limit of normal. Denali Therapeutics, the drug's manufacturer, is currently conducting a randomized clinical trial to further evaluate Avlayah's clinical benefits. The approval includes a boxed warning for potential allergic reactions, necessitating that treatment begins in a healthcare setting with appropriate monitoring. Common side effects reported include upper respiratory infections, anemia, and gastrointestinal issues, with specific monitoring required for hemoglobin levels and kidney function due to associated risks. This approval marks a critical advancement in the treatment options available for children suffering from Hunter syndrome, potentially altering the disease's trajectory during a crucial developmental period.
FDA News · Mar 25FDA Announces Public Hearing for National Priority Voucher Pilot Program on June 4, 2026
FDA News · Mar 20FDA Approves Higher Dose Semaglutide for Weight Loss Under National Priority Voucher Program
FDA News · Mar 19EMA Introduces New PRIME Tools to Enhance Medicine Development in the EU
EMA News · Mar 18FDA Draft Guidance Promotes Alternatives to Animal Testing in Drug Development
On March 18, 2026, the U.S. Food and Drug Administration (FDA) released a draft guidance aimed at facilitating the validation of new approach methodologies (NAMs) that can replace traditional animal testing in drug development. This initiative is part of the FDA's broader strategy to reduce reliance on animal testing and enhance the use of human-relevant data in the drug approval process. The guidance outlines the Center for Drug Evaluation and Research's (CDER) recommendations for validating NAMs, which include innovative testing methods such as in vitro studies, organoids, and computer simulations. The FDA emphasizes that NAMs can provide more reliable predictions of human responses to drugs, addressing the limitations of animal models that often fail to replicate complex human conditions. The draft guidance establishes four core validation principles: Context of Use, Human Biological Relevance, Technical Characterization, and Fit-for-Purpose, which aim to ensure that NAMs are scientifically robust and applicable in regulatory decision-making. This shift towards NAMs is significant for drug developers, as it aligns with the recommendations from the MAHA Commission's Strategy Report, which advocates for reducing animal studies. The FDA encourages developers to engage with the appropriate review divisions when considering NAMs for specific applications. This guidance follows previous efforts to minimize animal testing, including a draft released in December 2025 regarding non-human primate testing for monoclonal antibodies. In addition to the NAMs guidance, the FDA has also announced a revision to its guidance on Pyrogen Endotoxins Testing, further clarifying its stance on the use of recombinant reagents in testing. These developments reflect the FDA's commitment to advancing drug safety and efficacy through innovative, ethical research methodologies.
FDA News · Mar 18EMA Management Board Reviews 2025 Achievements and Future Initiatives in March Meeting
During the March 2026 meeting, the European Medicines Agency (EMA) Management Board reviewed the agency's annual report for 2025, which highlighted a successful year in medicines regulation within the EU. EMA issued 104 positive recommendations for new human medicines, including 38 with new active substances, and 30 for veterinary medicines, marking a record for the second consecutive year. The report emphasizes EMA's commitment to optimizing medicine assessments, enhancing access, and addressing shortages, alongside its role in implementing the Health Technology Assessment Regulation. The Board also discussed the governance structure for the new EU pharmaceutical legislation, which is nearly finalized. This structure involves collaboration among the European Commission, EMA, and national authorities to ensure smooth implementation once the legislation is formally adopted. EMA plans to provide guidance to stakeholders to facilitate compliance with the new legal framework. Additionally, the Board noted the draft roadmap for the rollout of electronic product information (ePI) for human medicines, aimed at improving the accessibility of up-to-date information for patients and healthcare professionals. This initiative will become mandatory for newly authorized medicines once the new legislation is in effect. The impact of the ongoing conflict in the Middle East on medicine supply was also addressed, with EMA monitoring potential disruptions. While no critical shortages have been reported, companies are experiencing varying levels of supply chain interruptions. The Board concluded with updates on clinical trials and the upcoming vacancy for the Executive Director position, emphasizing EMA's ongoing efforts to enhance the clinical trial environment in the EU.
EMA News · Mar 13CVMP Meeting Highlights: New Vaccines Approved and Updates on Veterinary Medicines
The Committee for Veterinary Medicinal Products (CVMP) convened from March 10-12, 2026, and made significant advancements in veterinary medicine. Notably, the Committee granted positive opinions for the marketing authorization of AviGate S. Infantis, aimed at immunizing healthy chickens against Salmonella infantis, and VeroBlue-3, designed to immunize sheep against bluetongue virus serotype 3. These approvals are crucial for enhancing animal health and reducing disease transmission in livestock. Additionally, the CVMP recommended a one-year extension for the marketing authorization of Syvazul BTV-3, an inactivated bluetongue virus vaccine, under exceptional circumstances. The Committee also addressed the withdrawal of Intervet International B.V.'s application for Mometamax Ultra, with further details to be published in a public assessment report. In response to a request from Germany, the CVMP concluded a procedure on quarter-based selective antibiotic dry cow therapy, affirming its alignment with current scientific knowledge aimed at minimizing antibiotic use while maintaining animal health. This guidance will assist veterinarians and farmers in making informed decisions regarding antibiotic therapies. The Committee also adopted five scientific advice reports for various veterinary products and recommended updates to Librela's product information due to rare musculoskeletal disorders observed in treated dogs. These updates are part of ongoing pharmacovigilance efforts to ensure the safety and efficacy of veterinary medicines. The CVMP's commitment to revising guidelines and templates for novel therapies and quality assurance reflects its dedication to advancing veterinary medicine and safeguarding animal health.
EMA News · Mar 13FDA Unveils New Adverse Event Monitoring System to Enhance Drug and Product Safety
On March 11, 2026, the U.S. Food and Drug Administration (FDA) launched the FDA Adverse Event Monitoring System (AEMS), a unified platform designed to streamline the analysis of adverse event reports across various regulated products, including drugs, vaccines, and cosmetics. This initiative aims to modernize the FDA's approach to postmarket surveillance, addressing the inefficiencies of previous fragmented systems that processed approximately 6 million reports annually across seven databases, costing the agency around $37 million each year. The new AEMS will consolidate these reports into a single, user-friendly dashboard, allowing for real-time access to adverse event data while ensuring patient confidentiality. By the end of May 2026, the system is expected to include comprehensive adverse event reports for all FDA-regulated products, significantly improving transparency and reducing the number of Freedom of Information Act (FOIA) requests for unreleased data. FDA Commissioner Dr. Marty Makary emphasized the importance of this modernization, stating that the previous systems created blind spots in safety monitoring. The AEMS is anticipated to save the agency approximately $120 million over the next five years, enhancing the ability to identify potential safety signals and risks associated with products. The transition to AEMS will replace several legacy systems, including the FDA Adverse Event Reporting System (FAERS) and the Vaccine Adverse Event Reporting System (VAERS), which will now be integrated into this new platform. This significant technical transformation is seen as a critical step in the FDA's commitment to ensuring public health and safety.
FDA News · Mar 11FDA Approves Tec-Dara for Relapsed Multiple Myeloma Under National Priority Voucher Program
The U.S. Food and Drug Administration (FDA) has granted approval for the drug combination teclistamab and daratumumab hyaluronidase-fihj, branded as Tec-Dara, to treat adult patients with relapsed or refractory multiple myeloma who have undergone at least one prior line of therapy. This decision, made just 55 days after the application was filed, represents the third approval under the Commissioner’s National Priority Voucher (CNPV) pilot program, aimed at expediting access to innovative therapies for critical health needs. The approval follows promising results from a Phase 3 clinical trial, which demonstrated that Tec-Dara significantly improved both progression-free survival and overall survival compared to the standard of care. Specifically, the trial indicated an 83% reduction in the risk of disease progression or death, marking a significant advancement in the treatment of a notoriously difficult-to-treat cancer. FDA Commissioner Dr. Marty Makary emphasized the agency's commitment to accelerating the availability of effective treatments for patients facing serious health challenges. The CNPV program is designed to facilitate the approval of therapies that address substantial unmet medical needs, enhance domestic manufacturing, and improve affordability. Companies participating in this program benefit from expedited review processes and enhanced communication with the FDA. The approval of Tec-Dara is particularly noteworthy as it not only provides a new treatment option for multiple myeloma but also supports the existing indication for Tecvayli (teclistamab-cqyv), which will transition from accelerated to traditional approval. Despite its potential benefits, Tec-Dara comes with significant risks, including life-threatening cytokine release syndrome (CRS) and neurologic toxicity. Due to these risks, Tec-Dara is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). The FDA has also noted common side effects associated with the treatment, which include hypogammaglobulinemia, infections, and gastrointestinal issues. The approval was granted to Janssen Biotech, Inc., marking a critical step forward in the fight against multiple myeloma.
FDA News · Mar 5FDA Approves Hernexeos for Lung Cancer Treatment Under National Priority Voucher Program
The U.S. Food and Drug Administration (FDA) has granted accelerated approval for Hernexeos (zongertinib), a new lung cancer treatment, under its National Priority Voucher (CNPV) pilot program. This decision, made on February 26, 2026, follows a clinical trial where 76% of previously untreated patients experienced a significant reduction in tumor size, a notable improvement compared to the 30-45% response rate seen with existing therapies. Lung cancer remains the leading cause of cancer-related deaths in the U.S., with an estimated 226,650 new cases and 124,730 deaths reported in 2025, highlighting the urgent need for effective treatments. Hernexeos is specifically approved for adults with unresectable or metastatic non-squamous non-small cell lung cancer (NSCLC) harboring HER2 (ERBB2) tyrosine kinase domain activating mutations, as identified by an FDA-authorized test. This approval expands treatment options for patients who have not previously received systemic therapy, building on the drug's earlier accelerated approval for those who had. The CNPV pilot program aims to fast-track the approval of therapies that meet critical health priorities, addressing significant unmet medical needs and promoting domestic manufacturing. Hernexeos has received Breakthrough Therapy and Priority Review designations from the FDA, which facilitates a more efficient review process. However, healthcare professionals should be aware of serious side effects associated with the drug, including hepatotoxicity and interstitial lung disease, as well as common side effects like diarrhea and fatigue. The approval was granted to Boehringer Ingelheim Pharmaceuticals, Inc., marking a significant advancement in lung cancer treatment options.
FDA News · Feb 26FDA Unveils Guidance to Expedite Development of Individualized Therapies for Ultra-Rare Diseases
On February 23, 2026, the U.S. Food and Drug Administration (FDA) released draft guidance aimed at facilitating the approval of targeted individualized therapies for ultra-rare diseases. This initiative is particularly significant as it addresses the challenges posed by small patient populations, where traditional randomized controlled trials may not be feasible. The guidance, developed by the FDA's Center for Biologics Evaluation and Research and Center for Drug Evaluation and Research, emphasizes the importance of therapies that directly target the genetic, cellular, or molecular abnormalities causing specific diseases. Health and Human Services Secretary Robert F. Kennedy, Jr. highlighted the urgency of this initiative, stating that it aims to accelerate the delivery of cures for families, especially children suffering from ultra-rare conditions. The FDA Commissioner, Dr. Marty Makary, reinforced the agency's commitment to removing regulatory barriers and fostering scientific innovation to provide meaningful treatments for patients with rare diseases. Key criteria outlined in the guidance include the identification of disease-causing abnormalities, demonstration of the therapy's targeting capabilities, and reliance on well-characterized natural history data. The FDA also acknowledges the unique nature of genome editing technologies, which may allow for the evaluation of multiple product variations within a single trial framework. This approach is expected to inspire the industry to focus more on individualized therapies, ultimately improving safety and access to life-saving treatments. The draft guidance is now open for public comment, with the FDA encouraging stakeholders to provide feedback within 60 days of its publication in the Federal Register. This move marks a significant step in the FDA's ongoing efforts to advance safe and effective therapies for rare diseases, ensuring that no family is overlooked due to the uncommon nature of their conditions.
FDA News · Feb 23FDA Updates Labeling for Menopausal Hormone Therapy, Easing Risk Warnings
The U.S. Food and Drug Administration (FDA) has approved significant labeling changes for six menopausal hormone therapy (HRT) products, aimed at clarifying risk considerations associated with their use. The revisions involve the removal of risk statements related to cardiovascular disease, breast cancer, and probable dementia from the 'boxed warning' section, which is the FDA's most prominent safety warning. This decision, announced on February 12, 2026, reflects the agency's commitment to align drug information with current scientific evidence, as stated by Health and Human Services Secretary Robert F. Kennedy, Jr. The FDA's initiative to revise these warnings began in November 2025, following an extensive review of scientific literature. In response, 29 pharmaceutical companies submitted proposed labeling changes. The approved products encompass all four categories of HRT for menopausal women, including systemic combination therapy, systemic estrogen-alone therapy, systemic progestogen-alone therapy, and topical vaginal estrogen therapy. FDA Commissioner Marty Makary emphasized that this action aims to provide women with accurate, scientifically grounded information about the benefits of HRT, which can significantly alleviate menopausal symptoms. Menopause is a natural phase in a woman's life, but it can lead to symptoms that severely impact quality of life, such as hot flashes, night sweats, and vaginal dryness. The FDA has previously approved various hormone therapies to address these symptoms and prevent osteoporosis. Research indicates that women who start HRT within 10 years of menopause onset experience lower all-cause mortality and reduced fracture risk. Despite the potential benefits, only a small percentage of eligible women are currently utilizing these therapies; in 2020, approximately 41 million U.S. women aged 45-64 existed, yet only about 2 million women aged 46-65 received HRT prescriptions. The recent labeling changes are expected to empower women to make informed decisions regarding their treatment options for menopausal symptoms, in consultation with their healthcare providers. Women are encouraged to review the updated drug labels for comprehensive information on the benefits and risks associated with these therapies.
FDA News · Feb 12FDA Initiates Comprehensive Review of BHA, a Common Food Preservative Amid Safety Concerns
The U.S. Food and Drug Administration (FDA) has launched a thorough re-assessment of butylated hydroxyanisole (BHA), a widely used food preservative, to evaluate its safety based on the latest scientific evidence. This initiative is part of the FDA's broader strategy to review chemical additives in the food supply, which gained momentum with the establishment of a strengthened program in May 2025. BHA, which has been classified as 'reasonably anticipated to be a human carcinogen' by the National Toxicology Program, has been a subject of concern among health advocates for decades. The FDA's review will include a Request for Information regarding the use and safety of BHA, which has been a staple in food products since it was designated as Generally Recognized as Safe (GRAS) in 1958. Despite a decline in its usage in recent years, BHA remains present in various food items, including those marketed to children, raising alarms about potential health risks. Health and Human Services Secretary Robert F. Kennedy, Jr. emphasized that this reassessment signifies a shift towards a more transparent and science-based approach to food safety, stating that if BHA fails to meet current safety standards, it will be removed from the market. FDA Commissioner Marty Makary, M.D., M.P.H., reiterated the agency's commitment to ensuring that food chemicals do not pose harm to consumers. Following the BHA assessment, the FDA plans to evaluate other preservatives such as butylated hydroxytoluene (BHT) and azodicarbonamide, indicating a comprehensive strategy to enhance food safety. The FDA's proactive measures reflect a growing recognition of the need for rigorous scrutiny of food additives to protect public health, particularly for vulnerable populations such as children. As the agency continues its assessment, stakeholders are encouraged to participate in the dialogue surrounding food safety and chemical additives.
FDA News · Feb 10