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    • Key FDA decisions made in 2024
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    Key FDA decisions made in 2024

    Online Niel Posted on

    Major drug approvals
    ConCluSIonS
    reference

    Within the United States Department of Health and Human Services, the Food and Drug Administration (FDA) is the federal agency that regulates therapy for both humans and animals, which includes drugs, vaccines, dietary supplements, biologics, and medical devices. FDA also works to ensure the safety of cosmetics, the nation’s food supply, tobacco products, and electronic products that emit radiation.1

    Key FDA decisions made in 2024Image credit: Panchenko Vladimir/Shutterstock.com

    Major drug approvals

    1. Frostbite

    In February 2024, the US FDA approved Aurlumyn (iloprost), an injectable vasodilator treatment that is the first to be approved for the treatment of severe frostbite. This approval was granted after a randomized controlled trial (RCT) showed that patients receiving iloprost intravenously for six hours every day for up to eight days prevented patients from requiring amputation of fingers and/or from the affected legs.2

    Importantly, ilorpost is a repurposed drug for this indication, as it has been approved for clinical use since 2004 for the treatment of pulmonary arterial hypertension.

    2. Food allergies

    Current estimates indicate that up to 6% of the American population has a life-threatening food allergy. Because there is no cure for food allergies, most affected individuals must strictly avoid these foods and, if exposed, receive epinephrine immediately to prevent an anaphylactic reaction.

    In February 2024, the FDA approved Xolar (omalizumab), a drug previously approved for the treatment of moderate to severe persistent asthma, for use in patients following accidental food exposure. Omalizumab is a monoclonal antibody that prevents immunoglobulin E (IgE) from binding to its receptor, thus preventing an allergic reaction.3

    3. Gene therapy

    As of 2017, the FDA has approved 19 genetic therapiesmany of which are used to treat rare diseases. For example, Duchenne muscular dystrophy (DMD) is a rare genetic disorder that affects the ability to produce dystrophin, a protein that protects muscle fibers from enzyme activity. As a result, DMD patients often experience muscle weakness that can lead to problems walking and running, frequent falls and fatigue, and reduced breathing capacity due to weakened lung function.4

    Initially approved in June 2023 under the FDA’s accelerated approval pathway, Elevidys is a recombinant gene therapy which uses a viral vector to deliver micro-dystrophin, a shortened version of the dystrophin gene. Following observations that Elvidys led to significant improvements in creatine kinase levels as well as physical activities used to reflect skeletal muscle function, the FDA expanded approval of Elvidys to treat non-ambulatory patients four years of age and older .6

    Lenmeldy, which is the first gene therapy approved for children with metachromatic leukodystrophy, is developed from the patient’s hematopoietic stem cells (HSCs), which have been genetically modified to include functional copies of the deficient arylsulfatase A (ARSA) gene. in patients with this genetic disease. After a single dose of Lenmeldy, the modified stem cells are grafted into the patient’s bone marrow to eventually provide the body with the machinery needed to make the ARSA enzyme.5

    Synovial sarcoma is a rare cancer that affects soft tissues throughout the body, especially the extremities. In the United States, about 1,000 people each year will be diagnosed with synovial sarcoma, most of whom are men who are over 30 years old or younger.

    In August 2024, the FDA approved Tecelra, which is a T-cell receptor (TCR) gene therapy that is developed from a patient’s own T cells, to treat adults with metastatic synovial sarcoma. These T cells are then modified to specifically target melanoma-associated antigen 4 (MAGE-A4), an antigen that is highly expressed in synovial sarcoma cells.4

    4. Approvals and innovations for medical devices

    The US FDA has cleared a total of 950 artificial intelligence and machine learning (AI/ML) enabled medical devices for public use. Each of these devices has met the FDA’s premarket requirements, which involves a comprehensive evaluation of the safety and efficacy of the devices based on their intended application.8

    A total of 21 devices received FDA approval in 2024, the most common of which include lab tests and stents. In cardiology, several devices also received approval, including a new heart valve, a vascular closure device, a cardiac clamp implant, a tricuspid valve replacement system, and a defibrillator.10

    Categories of medical devices explained

    5. Development of vaccines

    Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), the virus responsible for coronavirus disease 2019 (COVID-19), continues to mutate, leading to the emergence of numerous variants capable of evading early COVID-induced immunity . -19 vaccines and immunity against infections with older strains of SARS-CoV-2.

    As a result, the FDA has approved the use of updated COVID-19 messenger ribonucleic acid (mRNA) vaccines containing a monovalent component corresponding to the SARS-CoV-2 Omicron KP.2 subvariant.7

    The FDA also granted emergency use authorization (EUA) for an updated version of the Novavax COVID-19 vaccine, which is a protein adjuvant vaccine that now contains a monovalent component corresponding to the Omicron JN.1 subvariants.8

    10 Facts About FDA Dos and Don’ts #FDAFacts (1-10)

    ConCluSIonS

    In addition to repurposing several existing drugs for new treatment indications, the FDA has also approved a growing number of gene therapies, with more approvals expected in the next few years.

    With more than 1,000 cell and gene therapies in clinical development in the US alone, as well as more than 3,000 in preclinical stages, gene therapy will inevitably transform the way diseases are treated in the future.

    reference

    1. “What does the FDA regulate?” (Online). Available from: https://www.fda.gov/about-fda/what-we-do/what-does-fda-regulate.
    2. “FDA Approves First Drug to Treat Severe Frostbite” (online). Available from: https://www.fda.gov/news-events/press-announcements/fda-approves-first-medication-treat-severe-frostbite.
    3. “FDA Approves First Drug to Help Reduce Allergic Reactions to Multiple Foods After Accidental Exposure” (online). Available from: https://www.fda.gov/news-events/press-announcements/fda-approves-first-medication-help-reduce-allergic-reactions-multiple-foods-after-accidental.
    4. “FDA approves first gene therapy to treat adults with metastatic synovial sarcoma” (online). Available from: https://www.fda.gov/news-events/press-announcements/fda-approves-first-medication-help-reduce-allergic-reactions-multiple-foods-after-accidental.
    5. “FDA approves first gene therapy for children with metachromatic leukodystrophy” (online). Available from: https://www.fda.gov/news-events/press-announcements/fda-approves-first-medication-help-reduce-allergic-reactions-multiple-foods-after-accidental.
    6. “FDA Expands Approval of Gene Therapy for Duchenne Muscular Dystrophy Patients” (online). Available from: https://www.fda.gov/news-events/press-announcements/fda-expands-approval-gene-therapy-patients-duchenne-muscular-dystrophy.
    7. “FDA Approves and Licenses Updated COVID-19 mRNA Vaccines to Better Protect Against Currently Circulating Variants” (online). Available from: https://www.fda.gov/news-events/press-announcements/fda-approves-and-authorizes-updated-mrna-covid-19-vaccines-better-protect-against-currently.
    8. “FDA authorizes updated Novavax COVID-19 vaccine to better protect against currently circulating variants” (online). Available from: https://www.fda.gov/news-events/press-announcements/fda-approves-and-authorizes-updated-mrna-covid-19-vaccines-better-protect-against-currently.
    9. “Artificial Intelligence and Machine Learning (AI/ML) Enabled Medical Devices” (online). Available from: https://www.fda.gov/medical-devices/software-medical-device-samd/artificial-intelligence-and-machine-learning-aiml-enabled-medical-devices.
    10. “2024 Device Approval” (online). Available from: https://www.fda.gov/medical-devices/software-medical-device-samd/artificial-intelligence-and-machine-learning-aiml-enabled-medical-devices.

    Further reading

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