RSV vaccinations, an Alzheimer’s medication, and the first CRISPR gene-editing therapy are examples of novel therapies.
In 2023, weight-loss medications took centre stage, but these medicinal advancements addressing other ailments are also important.
Approved for CRISPR genome editing
The world’s first CRISPR/Cas9 gene-editing medicine was approved by the US Food and Drug Administration on December 8.The Casgevy medication helps patients manufacture healthy haemoglobin, which is how it addresses sickle cell disease. The condition causes aberrant haemoglobin, which makes red blood cells rigid and crescent-shaped and can obstruct blood flow in those who have it. The FDA will make a decision by March 2024 about the use of the same medicine for the treatment of beta-thalassemia, a condition that lowers haemoglobin production.
Alzheimer’s disease slowing down
In July, the FDA fully approved lecanemab, also known as Leqembi, a medication for Alzheimer’s. Lecanemab eradicates the amyloid plaques that accumulate in the brains of Alzheimer’s patients, just like the medication aducanumab, which was approved in 2021. Lecanemab doesn’t reverse the course of the disease, but over the course of an 18-month clinical trial, it did decrease cognitive deterioration by roughly 30% when compared to a placebo.
A treatment for muscular dystrophy using genes
The first gene treatment for children with Duchenne muscular dystrophy was authorised by the FDA in June. This muscle-wasting illness is caused by a defective gene that prevents the production of the protein dystrophin, which maintains muscle cell integrity. The treatment aids in the body’s production of the protein that is lacking.
Preventing RSV
This year brought with it several methods for preventing the respiratory syncytial virus. The FDA authorised Arexvy, the first RSV vaccination for adults 60 years of age and above, in May. Abrysvo, a vaccine intended for pregnant women, was approved in August. In July, a monoclonal antibody—a synthetic antibody that resembles immune system proteins—was approved to guard against the virus that brings up to 80,000 young children to American hospitals annually and protects youngsters under the age of two. However, the U.S. Centres for Disease Control and Prevention advised in October that the medication be reserved for infants who are most at risk of developing problems from RSV due to the therapy’s limited supply.
A postpartum depression medication
Up until August, the only postpartum depression medicine available in the US required a hospital stay for a 60-hour intravenous infusion. Postpartum depression sufferers can now take an oral medicine at home called zuranolone (brand name Zurzuvae), which has FDA approval and can show relief in as little as three days.
Birth control without a prescription
The FDA decided in July that the 1973-approved oral contraceptive norgestrel will be freely accessible without a prescription. This is the country’s first over-the-counter (OTC) daily birth control pill. Reducing barriers to contraception, according to some public health professionals, is particularly crucial for reproductive autonomy now that governmental prohibitions have restricted access to abortion.
An attempt to combat chikungunya
Newborns can die from the chikungunya virus, which can also cause fever and excruciating joint pain. The first vaccination against the virus—which is spread by mosquitoes—was approved by the FDA in November. Although the virus is most common in tropical areas, the FDA alerts us about its spread to other parts of the world.
Narcan over-the-counter
Naloxone, also known as Narcan, is a nasal spray that can reverse the effects of an opioid overdose in a matter of minutes. The FDA decided in March that this life-saving medication can be supplied without a prescription. Authorities believe that making Narcan more widely available will aid in the fight against the opioid epidemic, which resulted in the overdose deaths of almost 645,000 individuals between 1999 and 2021.