Green Light for CRISPR Gene Editing Technology
On December 8, the U.S. Food and Drug Administration approved the world’s first gene editing treatment using CRISPR/Cas9 technology. The treatment, called Casgevy, targets sickle cell anemia by helping patients produce healthy hemoglobin. In people with the disease, hemoglobin is abnormal, causing red blood cells to stiffen and take on a crescent shape, which can hinder blood flow. By March 2024, the FDA will decide whether the same treatment can be used for beta-thalassemia.
Slowing Alzheimer’s Disease
The Alzheimer’s drug lecanemab (brand name Leqembi) received full FDA approval in July. Like the previously approved drug aducanumab in 2021, lecanemab removes amyloid plaques that accumulate in the brains of people with Alzheimer’s disease. The drug does not stop the disease, but in the clinical trial, lecanemab slowed cognitive decline by about 30 percent over 18 months compared to a placebo group.
Gene Therapy for Muscular Dystrophy
In June, the FDA approved the first gene therapy for children with Duchenne muscular dystrophy. Because of a defect in the gene, people with this disease do not produce the dystrophin protein that helps maintain the integrity of muscle cells. The treatment helps the body produce a copy of the missing protein.
Prevention of RSV Virus
Several methods for the prevention of respiratory syncytial virus emerged this year. In May, the FDA approved the first RSV vaccine called Arexvy in the United States for adults over the age of 60. Then in August, a vaccine for pregnant women called Abrysvo was approved. A monoclonal antibody – a lab-made antibody that mimics immune proteins – was approved in July to protect children aged 2 and under from the virus, which sends up to 80,000 toddlers to U.S. hospitals every year. However, in October, a shortage of supplies of this treatment led the Centers for Disease Control and Prevention in the U.S. to recommend reserving it for infants at the highest risk of RSV complications.
Pills for Postpartum Depression
Until August, the only targeted medication for postpartum depression in the U.S. required a 60-hour intravenous infusion in a hospital. With the FDA’s approval of zuranolone (brand name Zurzuvae), those suffering from postpartum depression can take the medication orally at home and improve their condition in just three days.
Over-the-Counter Birth Control
In July, the FDA decided that over-the-counter access to the oral contraceptive norgestrel, which was first approved in 1973, would be possible. It is the first daily birth control pill available over the counter in the U.S. Some public health experts believe that reducing barriers to contraception is particularly important for gaining reproductive autonomy now that state bans have limited access to abortion.
Vaccine Against Chikungunya Virus
The chikungunya virus can cause severe fever and joint pain, and it can be fatal for infants. In November, the FDA approved the first vaccine against the mosquito-borne virus. The virus is primarily spread in tropical areas, but the FDA warns that it is spreading to new areas around the world.
Over-the-Counter Narcan
Narcan nasal spray, also known as naloxone, can reverse the effects of an opioid overdose within minutes. In March, the FDA decided that this lifesaving drug could be sold over the counter. Officials hope that easy access to Narcan will help combat the opioid epidemic, which has claimed nearly 645,000 lives from 1999 to 2021 due to overdoses.
Source:
https://www.sciencenews.org/article/medical-advances-vaccines-drugs-2023
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