The year 2023 witnessed significant breakthroughs in medical science, extending beyond the realm of weight-loss drugs.
The year 2023 witnessed significant breakthroughs in medical science, extending beyond the realm of weight-loss drugs. While these treatments garnered attention, several other medical advancements have made a remarkable impact on diverse health conditions. From groundbreaking gene-editing therapies to innovations in Alzheimer’s treatment and the development of vaccines against viruses, these milestones are shaping the future of healthcare. Let’s delve into some of the notable medical advances of 2023.
CRISPR Gene Editing Receives FDA Approval:
On December 8, 2023, the U.S. Food and Drug Administration (FDA) approved Casgevy as the world’s first CRISPR/Cas9 gene-editing therapy to treat sickle cell disease. This groundbreaking one-time treatment alters a patient’s blood cell DNA, providing a lifelong remedy for the debilitating symptoms of sickle cell disease.
Specifically targeting the genetic mutation responsible for the condition, Casgevy enables the production of healthy hemoglobin. The FDA is expected to make a decision by March 2024 on whether the therapy can also be used for treating beta-thalassemia, a disorder affecting hemoglobin production.
Lecanemab Slows Down Alzheimer’s:
In July 2023, the U.S. Food and Drug Administration (FDA) granted full approval to Lecanemab (brand name Leqembi) for treating Alzheimer’s disease. Initially approved under the Accelerated Approval pathway in January 2023, Lecanemab, an amyloid beta-directed antibody, reduces amyloid plaques in the brain—a key Alzheimer’s feature.
The FDA’s decision followed confirmation from the Study 301 (CLARITY AD), showing a significant reduction in cognitive decline over 18 months compared to a placebo. Although Lecanemab doesn’t halt Alzheimer’s progression, the approval underscores its efficacy in slowing cognitive decline.
Gene Therapy for Duchenne Muscular Dystrophy:
In June 2023, the FDA granted approval for the first gene therapy designed to treat Duchenne muscular dystrophy. Developed by Sarepta and named delandistrogene moxeparvovec, or Elevidys, the therapy utilizes an adeno-associated virus (AAV) vector to deliver a microdystrophin construct encoding a 138-kDa protein that mimics some functions of the full-length dystrophin.
The FDA provided accelerated approval for Elevidys, a designation aimed at offering earlier access to therapies that demonstrate an impact on surrogate endpoints predicting potential clinical benefit. This groundbreaking therapy is specifically approved for pediatric patients aged 4 through 5 years who are diagnosed with Duchenne muscular dystrophy.
The FDA’s approval of Elevidys represents a significant milestone in the treatment of Duchenne muscular dystrophy, instilling hope for patients and their families.
RSV Protection Advances:
In medical advances of 2023, new measures to guard against respiratory syncytial virus (RSV) were introduced. The FDA gave its approval for the first RSV vaccine, named Arexvy, in May 2023, targeting adults aged 60 and above. This vaccine aims to reduce the risk of developing lower respiratory tract disease (LRTD).
Another vaccine, Abrysvo, a protein subunit vaccine devoid of live virus, received approval in August 2023 for pregnant individuals. Abrysvo is designed to safeguard infants from LRTD from birth up to 6 months, marking it as the first FDA-approved RSV vaccine for this purpose. Furthermore, a monoclonal antibody, sanctioned in July 2023, provides protection for children aged 2 and under against RSV.
Zuranolone for Postpartum Depression:
In August 2023, the FDA granted approval for zuranolone, one of medical advances of 2023, marketed as Zurzuvae, an oral medication designed for the treatment of postpartum depression. Developed through a collaboration between Sage Therapeutics and Biogen, zuranolone stands out as a neuroactive steroid affecting both synaptic and extrasynaptic GABA A receptors, distinguishing it from similar agents.
The drug is believed to exert its effects by modulating the neurotransmitter GABA, crucial in mood regulation. Clinical trials have demonstrated zuranolone’s potential as a rapid-acting remedy for postpartum depression, manifesting improvements within as little as three days.
The recommended dosage is 50 mg taken once daily in the evening for a duration of 14 days. Zuranolone marks a groundbreaking development as the first oral medication specifically indicated for postpartum depression in adults, offering an alternative to the previous intravenous infusion requiring hospitalization.
Over-the-Counter Birth Control:
In July 2023, the U.S. Food and Drug Administration (FDA) granted approval for Opill (norgestrel) tablets to be available without a prescription for the prevention of pregnancy.
This milestone of medical advances makes Opill the first daily oral contraceptive in the United States that can be obtained without a prescription. Consumers can now purchase this progestin-only oral contraceptive at various retail outlets, including drug stores, convenience stores, grocery stores, and online platforms.
The FDA’s decision is aimed at removing barriers to contraception, particularly in the context of state-imposed bans that have restricted access to abortion. The contraceptive efficacy of norgestrel was initially established when it received approval for prescription use in 1973. While Opill is now available over the counter, other formulations and dosages of oral contraceptives will remain accessible only through prescription.
Chikungunya Vaccine Approval:
In November 2023, the U.S. Food and Drug Administration (FDA) granted approval for the first vaccine targeting the chikungunya virus. This vaccine, named Ixchiq, is administered as a single-dose injection and contains a live, weakened form of the virus.
It is approved for individuals aged 18 years and older who face an elevated risk of exposure to the chikungunya virus. The virus is primarily transmitted through mosquito bites and poses a growing global health threat, with over 5 million reported cases in the past 15 years.
Common symptoms of chikungunya include fever and joint pain, and some individuals may suffer from persistent joint pain lasting for months or even years.
It is important to note that the vaccine may induce symptoms similar to those experienced by individuals with chikungunya disease. Reported side effects among vaccine recipients include headache, fatigue, muscle pain, joint pain, fever, nausea, and tenderness at the injection site.
Narcan Available Over the Counter:
In March 2023, the U.S. Food and Drug Administration (FDA) granted approval for the first over-the-counter naloxone nasal spray, known as Narcan. This nasal spray contains 4 milligrams (mg) of naloxone hydrochloride and is the initial naloxone product authorized for non-prescription use. It is designed to swiftly reverse the effects of opioid overdose within minutes.
The product can be directly sold to consumers in various retail locations such as drug stores, convenience stores, grocery stores, gas stations, and online. The FDA has encouraged the manufacturer to prioritize making the product accessible as soon as possible and at an affordable price.
Conclusion:
The medical advances of 2023 signify significant progress across various healthcare domains. Notable achievements include the approval of the world’s first CRISPR/Cas9 gene therapy for sickle cell disease, lecanemab for Alzheimer’s, zuranolone for postpartum depression, and multiple interventions against respiratory syncytial virus (RSV).
In contraception, the FDA’s decision to allow over-the-counter access to norgestrel simplifies birth control. Additionally, the approval of the first chikungunya virus vaccine (Ixchiq) and the over-the-counter naloxone nasal spray (Narcan) highlights proactive measures against infectious diseases and the opioid epidemic, respectively.
These milestones collectively contribute to a more resilient healthcare future, emphasizing innovation, accessibility, and improved outcomes.