In 2024, key advancements in medical research, drug development, and therapeutic interventions were achieved. Here are 11 significant medical breakthroughs from the year:
11. FDA Approval of Zepbound for Sleep Apnea
Zepbound gained FDA approval as the first drug to directly treat obstructive sleep apnea in adults with obesity, potentially impacting prevalent insurance coverage barriers.
10. Promising Parkinson’s Disease Therapy
Tavapadon demonstrated significant motor function improvements in Phase 3 trials for Parkinson’s disease, and a potential FDA submission is expected soon.
9. Ulcerative Colitis Treatment Gained Approval
In June, risankizumab-rzaa (Skyrizi) was approved for treating moderate to severe ulcerative colitis. The approval expanded treatment options beyond existing indications for psoriasis and Crohn’s disease.
8. Alzheimer’s Treatment Approved
The FDA approved donanemab (Kisunla) for early symptomatic Alzheimer’s disease in July. The drug, delivered via monthly IV infusion, aimed to slow disease progression.
7. PARP Inhibitor Extended Survival in Breast Cancer
Data from the OlympiA trial showed that olaparib improved survival in BRCA-mutated, HER2-negative early breast cancer patients. These findings positioned olaparib as a significant option in early breast cancer treatment.
6. Triple Combination Therapy Approved for Cystic Fibrosis
The FDA approved Alyftrek, a next-generation cystic fibrosis treatment this month. The therapy targets specific genetic mutations and was approved for patients aged 6 years and older.
5. Over-the-Counter COVID-19/Flu Test Released
The FDA authorized the Healgen Rapid Check COVID-19/Flu A&B Antigen Test in October. This was the first combination test for these viruses available over the counter without emergency-use authorization.
4. New Schizophrenia Drug Approved
In September, the FDA approved Cobenfy, a new drug class combining xanomeline and trospium chloride for schizophrenia. Clinical trials demonstrated efficacy in reducing symptoms like hallucinations and disorganized thinking.
3. Cause of Lupus Identified
In July, a study in Nature identified imbalances in T-cell responses among lupus patients. Brigham & Women’s Hospital and Northwestern Medicine researchers highlighted specific mediators that could potentially reverse these imbalances.
2. Groundbreaking Pig Kidney Transplant Performed
Surgeons at Massachusetts General Hospital performed the world’s first genetically edited pig kidney transplant into a living human. The procedure, conducted in March, involved connecting the pig kidney’s blood vessels and ureter to a 62-year-old patient with end-stage kidney disease.
1. Gene Therapy Restores Hearing in Children
In January, a study in The Lancet reported that gene therapy restored hearing in children with DFNB9, a hereditary deafness caused by a gene mutation. Researchers at Mass Eye and Ear delivered a functioning gene via an inactive virus into the inner ears of six children, five of whom regained hearing sufficient for normal conversations.
