2023 was a productive year for innovative drugs targeting aging, rare diseases, and age-related health conditions like Alzheimer’s Disease (AD). Eli Lilly, with its “weight-loss wonder drug” Mounjario (tirzepatide), became the first pharmaceutical company in history to exceed a market cap of $400 billion. Seragon’s RESTORIN, the most advanced oral-form aging intervention to date, was finally launched. Additionally, the first CRISPR/Cas9 gene editing drug, Casgevy (Vertex Pharmaceuticals), was launched globally, offering new hope to patients with rare genetic disorders. The introduction of these and other blockbuster drugs may signal a revolution in the fields of aging, obesity, AD, cancer, and immune health.
1. Leqembi: The First FDA-approved AD Drug in 20 Years
In July 2023, the FDA approved Legembi (lecanemab), a drug jointly launched by Eisai and Biogen for the treatment of Alzheimer’s Disease (AD). This marked the first FDA approval for an AD progression treatment in 20 years. Leqembi, an antibody, selectively binds to and neutralizes a toxic protein called β-amyloid (Aβ), which is a key pathogenic factor in AD, associated with contributing to the neurodegenerative process. Pivotal Phase III clinical trials have demonstrated that Leqembi not only reduces Aβ levels but also significantly improves dementia symptoms in AD patients.
2. Vowst: The World’s First Oral Fecal Microbiota Drug
In April 2023, Seres Therapeutics launched Vowst (formerly known as SER-109), the only FDA-approved oral fecal microbiota drug for preventing recurrent C. difficile infection. C. difficile infection is one of the most common healthcare-associated infections in the United States, causing between 15,000 and 30,000 deaths annually. As the first oral fecal microbiota drug, Vowst is made from the feces of healthy individuals and contains spores from a beneficial bacterial phylum known as Firmicutes. These Firmicutes may treat C. difficile infection by reducing the presence of C. difficile in the gut.
3. Tirzpatide: The Most Potent Weight-Loss Drug
In November 2023, Eli Lilly launched Mounjaro, a novel weight-loss drug also used for treating type 2 diabetes. Compared to Novo Nordisk’s semaglutide, another anti-diabetes drug, Mounjaro offers several advantages, including more significant weight loss effects and a more attractive price. The monthly cost of Mounjaro treatment is approximately $974, whereas semaglutide treatment costs about $1349 per month.
4. Casgevy: The World’s First CRISPR Gene Editing Drug
In December 2023, Casgevy, the world’s first CRISPR/Cas9 gene editing drug, was launched, jointly developed by Vertex Pharmaceuticals and CRISPR Therapeutics. Casgevy is used for patients aged 12 and older with transfusion-dependent beta-thalassemia or sickle cell disease (SCD) experiencing recurrent vaso-occlusive crises (VOC). SCD, the most common hereditary hemoglobin disease globally, sees an annual incidence of up to 300,000 new cases.
5. Arexvy: The World’s First Respiratory Syncytial Virus (RSV) Vaccine
In May 2023, GlaxoSmithKline (GSK) launched Arexvy, the world’s first vaccine for RSV (respiratory syncytial virus). RSV is a common respiratory virus that mainly causes mild, cold-like symptoms. It’s particularly serious in infants and older adults, potentially leading to bronchiolitis and pneumonia in young children. Arexvy is specifically designed for the prevention of lower respiratory tract disease caused by RSV infection in people over 60 years old.
6. Roctavian: The First Gene Therapy Drug for Hemophilia A
In June 2023, BioMarin launched Roctavian, the first gene therapy drug for treating severe hemophilia A in patients without AAV5 antibodies (FVIII <1 IU/dL). Roctavian utilizes the AAV5 virus vector to facilitate the transgenic expression of coagulation factor VIII (FVIII). Its primary advantage is the potential requirement of only a single treatment to achieve FVIII gene expression, potentially eliminating the need for long-term prophylactic coagulation factor injections in patients.
7. Fabhalta: The First Oral Monotherapy for PNH Treatment
In December 2023, Novartis launched Fabhalta (iptacopan) for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder characterized by the destruction of red blood cells. Fabhalta is the first oral monotherapy, meaning it is used alone without the need for combination with other drugs, for adult PNH. Developed by Novartis, iptacopan is an oral inhibitor that targets factor B in the complement alternative pathway. The drug acts at the upstream end of the C5 terminal pathway, thereby controlling both intravascular and extravascular hemolysis. It supplements the deficiency of anti-C5 antibodies, offering patients a convenient oral monotherapy option.
8. SRN-901N RESTORIN: The Most Potent Anti-Aging Inhibitor
In early 2023, Seragon, a pioneering biotechnology company in aging intervention and anti-cancer drugs, launched SRN-901N (RESTORIN). Seragon’s SRN-901N, combines core technologies to address the aging process at the cellular level and promote healthy aging. This includes compounds that target enhance DNA damage repair, CD38 inhibition, sirtuin activation, and mitochondrial metabolism. Developed with support from leading research institutions like Harvard University and Mayo Clinic, preclinical trials have demonstrated a significant extension in the natural lifespan of experimental animals and a notable inhibition of physical and cognitive decline in elderly animals.
9. Talvey: The World’s First GPRC5D/CD3 Bispecific Antibody Drug
In August 2023, Johnson & Johnson launched Talvey (talquetamab), a GPRC5D/CD3 bispecific antibody, for the treatment of relapsed or refractory multiple myeloma (MM) in adult patients. MM is a type of cancer that forms in plasma cells leading to bone pain, anemia, and kidney problems. MM is currently the third most common kind of blood cancer, with an estimated 35,000 cases diagnosed and over 12,000 deaths from the disease in the United States in 2023.
10. Elevidys: The world’s first gene therapy drug for Duchenne muscular dystrophy (DMD)
In June 2023, Sarepta Therapeutics launched Elevidys, the world’s first gene therapy drug for Duchenne muscular dystrophy (DMD), aimed at treating pediatric patients aged 4 to 5 with confirmed DMD gene mutations. DMD is a genetic disorder characterized by progressive muscle degeneration and weakness. It is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. Starting in early childhood, it primarily affects boys, leading to decreased mobility and respiratory difficulties. As an AAV gene therapy, Elevidys utilizes the MHCK7 promoter in combination with the AAVrh74 vector to deliver the gene encoding the microdystrophin protein specifically to muscle tissue. This process facilitates the production of microdystrophin protein, which is crucial in the treatment of DMD.
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