Key FDA Decisions Made in 2024

By Benedette Cuffari, M.Sc.Reviewed by Lily Ramsey, LLM

Major Drug Approvals
Conclusions 
References

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

Image Credit: Panchenko Vladimir/Shutterstock.com

Major Drug Approvals

1. Frostbite

In February 2024, the U.S. FDA approved Aurlumyn (iloprost), an injectable vasodilator treatment that is the first ever to be approved for the treatment of severe frostbite. This approval was granted after a randomized controlled trial (RCT) demonstrated that patients who were intravenously administered iloprost for six hours every day for up to eight days prevented patients from requiring amputation of affected fingers and/or toes.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 have a life-threatening food allergy. Since there is no cure for food allergies, most affected individuals must strictly avoid these food products and, in the event they are exposed, immediately receive epinephrine 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 patients to use following accidental exposure to foods. Omalizumab is a monoclonal antibody that prevents immunoglobulin E (IgE) from binding to its receptor, thereby preventing an allergic reaction from occurring.³

3. Gene therapy

Since 2017, the FDA has approved 19 gene therapies, many of which are used to treat rare diseases. For example, Duchenne muscular dystrophy (DMD) is a rare genetic disorder that impacts the ability to produce dystrophin, a protein that protects muscle fibers from enzymatic activity. As a result, DMD patients often experience muscle weakness that can lead to trouble walking and running, frequent falls, and fatigue, as well as reduced respiratory capacity due to weakened lung function.⁴

Originally approved in June 2023 under the FDA Accelerated Approval pathway, Elevidys is a recombinant gene therapy that utilizes 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 the approval of Elvidys also to treat non-ambulatory patients four years of age and older.⁶

Lenmeldy, which is the first gene therapy to be approved for children with metachromatic leukodystrophy, is developed from the patient’s own hematopoietic stem cells (HSCs) that have been genetically modified to include functional copies of the arylsulfatase A (ARSA) gene, which is deficient in patients with this genetic disease. Following a single dose of Lenmeldy, modified stem cells engraft within the patient’s bone marrow to ultimately provide the body with the machinery needed to produce the ARSA enzyme.⁵

Synovial sarcoma is a rare cancer that affects soft tissues throughout the body, particularly the extremities. In the United States, about 1,000 people each year will be diagnosed with synovial sarcoma, most of whom are males who are in their 30s or younger.

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

4. Medical device approvals and innovations

The U.S. FDA has authorized a total of 950 Artificial Intelligence and Machine Learning (AI/ML)-Enabled Medical Devices for public use. Each of these devices has met premarket requirements by the FDA, which involves a comprehensive evaluation of the safety and effectiveness of the devices based on their intended application.⁸

A total of 21 devices received approval from the FDA in 2024, the most common of which included lab tests and stents. Within cardiology, several devices have also received approval, including a novel heart valve, vascular closure device, heart clip implant, tricuspid valve replacement system, and defibrillator.¹⁰

Medical Device Categories Explained

5. Vaccine developments

The severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), the virus responsible for the coronavirus disease 2019 (COVID-19), continues to mutate, which has led to the emergence of numerous variants capable of evading immunity induced by early COVID-19 vaccines and immunity from infections with older SARS-CoV-2 strains.

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

The FDA has 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

Conclusions

In addition to repurposing several existing drugs for novel treatment indications, the FDA has also approved an increasing number of gene therapies, with more approvals expected over the next several years.

With over 1,000 cell and gene therapies in clinical development in the U.S. alone, as well as over 3,000 in pre-clinical stages, gene therapy will inevitably transform how diseases are treated in the future.

References

1. “What does FDA regulate?” [Online]. Available from: https://www.fda.gov/about-fda/what-we-do/what-does-fda-regulate.
2. “FDA Approves First Medication 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 Medication 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 Patients with Duchenne Muscular Dystrophy” [Online]. Available from: https://www.fda.gov/news-events/press-announcements/fda-expands-approval-gene-therapy-patients-duchenne-muscular-dystrophy.
7. “FDA Approves and Authorizes Updated mRNA COVID-19 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 Curently 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 Approvals” [Online]. Available from: https://www.fda.gov/medical-devices/software-medical-device-samd/artificial-intelligence-and-machine-learning-aiml-enabled-medical-devices.

Further Reading

• All Drug Discovery Content
• Hit to Lead (H2L) Process in Drug Discovery
• Understanding Lead Optimization
• How is AI Transforming Drug Discovery?
• Hot Melt Extrusion in the Pharmaceutical and Food Industries