One of the major medical breakthroughs in 2023 was the approval of the first CRISPR gene-editing therapy by the U.S. Food and Drug Administration.

 This therapy, known as Casgevy, was developed to treat sickle cell disease by helping patients produce healthy hemoglobin. Patients with sickle cell disease have abnormal hemoglobin, leading to hard and crescent-shaped red blood cells that can block blood flow. The FDA is expected to make a decision by March 2024 on whether the same therapy can be used to treat beta-thalassemia, a disorder that reduces hemoglobin production.

Alzheimer’s Progress:

 In July, the FDA granted full approval to the Alzheimer’s drug lecanemab, also known as Leqembi. Similar to the 2021-approved drug aducanumab, lecanemab targets and removes amyloid plaques that accumulate in the brains of individuals with Alzheimer’s disease. While the drug does not halt the progression of the disease, clinical trials demonstrated that lecanemab slowed cognitive decline by approximately 30% over an 18-month period compared to a placebo.

Muscular Dystrophy Gene Therapy:

In June, the FDA authorized the first gene therapy designed for children with Duchenne muscular dystrophy. This genetic disorder prevents the production of dystrophin protein due to a faulty gene, leading to muscle degeneration. The therapy aims to stimulate the body to generate a form of the missing protein.

 RSV Protection:

Throughout the year, various methods to guard against respiratory syncytial virus (RSV) emerged. In May, the FDA approved Arexvy, the initial RSV vaccine in the United States, for adults aged 60 and above. Subsequently, in August, a vaccine named Abrysvo was authorized for pregnant individuals. Additionally, a monoclonal antibody received approval in July to shield children aged 2 and under from RSV, a virus that annually causes numerous young children to be hospitalized in the U.S.

 Postpartum Depression Treatment:

 Until August, the sole medication specifically targeting postpartum depression in the U.S. necessitated a lengthy intravenous infusion in a hospital setting. With the FDA’s approval of zuranolone, branded as Zurzuvae, individuals grappling with postpartum depression can now take an oral medication at home and potentially experience improvement within three days.

Over-the-Counter Birth Control:

 In July, the FDA decided that the oral contraceptive norgestrel, initially sanctioned in 1973, should be available without a prescription. This marked the introduction of the first over-the-counter daily birth control pill in the U.S. Some public health experts argue that reducing barriers to contraception is crucial for reproductive autonomy, particularly amidst state restrictions on abortion access.

Chikungunya Vaccine:

In November, the FDA greenlit the first vaccine against chikungunya virus, which can cause fever, severe joint pain, and pose a risk to newborns. Transmitted by mosquitoes, this virus is most prevalent in tropical regions but is increasingly spreading to new areas globally according to FDA warnings.

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