The U.S. Food and Drug Administration (FDA) has made several key decisions in 2024, approving novel therapies and expanding the use of existing drugs for new indications, marking significant advancements in treating various conditions from rare diseases to common ailments.
First-Ever Treatment for Severe Frostbite
In February 2024, the FDA approved Aurlumyn (iloprost), an injectable vasodilator, as the first-ever treatment for severe frostbite. The approval was based on a randomized controlled trial demonstrating that intravenous administration of iloprost for six hours daily for up to eight days significantly reduced the need for amputation of affected fingers and toes. Iloprost, initially approved in 2004 for pulmonary arterial hypertension, has been repurposed for this critical new indication.
Mitigating Food Allergies with Omalizumab
Recognizing that up to 6% of the American population suffers from life-threatening food allergies, the FDA approved omalizumab (Xolair) in February 2024 to help reduce allergic reactions after accidental exposure to food. Omalizumab, a monoclonal antibody previously approved for moderate to severe persistent asthma, prevents immunoglobulin E (IgE) from binding to its receptor, thus preventing allergic reactions. This approval offers a new layer of protection for individuals at risk of anaphylaxis.
Advancements in Gene Therapy
The FDA has been increasingly active in approving gene therapies, with 19 approvals since 2017, many targeting rare diseases. One notable example is Duchenne muscular dystrophy (DMD), a rare genetic disorder. The FDA expanded the approval of Elevidys, a recombinant gene therapy delivering a shortened version of the dystrophin gene, to include non-ambulatory patients four years of age and older, following observations of significant improvements in creatine kinase levels and physical activities.
Lenmeldy, the first gene therapy approved for children with metachromatic leukodystrophy, utilizes the patient’s own hematopoietic stem cells (HSCs) genetically modified to include functional copies of the arylsulfatase A (ARSA) gene. A single dose of Lenmeldy allows modified stem cells to engraft within the patient’s bone marrow, providing the body with the machinery needed to produce the ARSA enzyme.
Novel Gene Therapy for Synovial Sarcoma
In August 2024, the FDA approved Tecelra, a T-cell receptor (TCR) gene therapy, for adults with metastatic synovial sarcoma, a rare cancer affecting soft tissues. Tecelra is developed based on a patient’s T-cells, modified to specifically target melanoma-associated antigen 4 (MAGE-A4), an antigen highly expressed in synovial sarcoma cells. This approval marks a significant advancement in personalized cancer treatment.
AI/ML in Medical Devices
The FDA has authorized 950 Artificial Intelligence and Machine Learning (AI/ML)-Enabled Medical Devices for public use, with 21 devices approved in 2024 alone. These devices, which have undergone comprehensive evaluation for safety and effectiveness, include lab tests, stents, and several cardiology devices such as novel heart valves, vascular closure devices, heart clip implants, tricuspid valve replacement systems, and defibrillators. This reflects the increasing integration of AI into healthcare, promising improved diagnostics and treatments.
Updated COVID-19 Vaccines
To combat the evolving landscape of SARS-CoV-2 variants, the FDA has approved updated mRNA COVID-19 vaccines containing a monovalent component corresponding to the SARS-CoV-2 Omicron KP.2 subvariant. Additionally, the FDA granted emergency use authorization (EUA) for an updated version of the Novavax COVID-19 vaccine, a protein adjuvant vaccine containing a monovalent component corresponding to the Omicron JN.1 subvariants.
These approvals and authorizations reflect the FDA's commitment to adapting to emerging health threats and ensuring access to effective vaccines.
With over 1,000 cell and gene therapies in clinical development in the U.S. and over 3,000 in pre-clinical stages, gene therapy is expected to transform how diseases are treated in the future.
