A New Era in Medicine: FDA Approves Casgevy, Vertex's CRISPR-Based Therapy for Sickle Cell Disease
Food and Drug Administration(FDA) has granted approval for a powerful treatment aimed at addressing sickle cell disease. This severe condition affects over 100,000 Americans, predominantly impacting the Black population. Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, the therapy, named Casgevy, achieves a historic milestone as the first medication approved in the United States that utilizes the Nobel Prize-winning gene-editing tool, CRISPR, acknowledged in the field of chemistry in 2020. CRISPR is one the three acknowledged methods of gene editing. The other two are TALE nucleases and zinc-finger nucleases.
Casgevy serves as a treatment for sickle cell disease and beta thalassemia, both blood disorders. CRISPR Therapeutics and Vertex Pharmaceuticals jointly developed this treatment, gaining approval not only from the FDA but also from UK medical authorities. Priced at $2.2 million( about R36,788,950.00) per person, the treatment is complex, requiring patients to spend weeks or even months in the hospital both before and after the therapy is administered. Casgevy is crafted from a patient's stem cells, collected and transported to a manufacturing facility where they undergo editing using CRISPR. A precise DNA snip enables these cells, upon reinfusion, to produce a fetal form of the oxygen-carrying protein hemoglobin, crucially damaged in individuals with sickle cell.
This month has proven significant for biopharma, with the FDA clearing another genetic medicine for sickle cell, named Lyfgenia and developed by Bluebird bio. Targeting individuals aged 12 and older with a history of pain crises from the disease. Lyfgenia comes with a wholesale acquisition cost of $3.1 million(R54,951,351.00).
Despite these advancements, both treatments pose challenges. Patients undergoing either therapy must endure a preparatory chemotherapy regimen, potentially causing painful mouth sores, low blood cell counts, and organ damage. The side effects may be severe, potentially limiting treatment tolerance for older individuals or those with extensively damaged organs due to sickle cell. The chemotherapy, known as busulfan, can also induce infertility, complicating treatment choices for those planning to have children. Vertex has established a program offering fertility support to commercially insured patients, but federal restrictions prevent similar assistance for those on Medicaid or Medicare. The estimated patient pool for these treatments is around 25,000 people in both the US and the UK. Notably, some companies, including Bristol Myers Squibb with their CAR-T treatment Abecma, have encountered obstacles in the development of their treatments.
Vertex Pharmaceuticals Vertex Pharmaceuticals is Boston based biopharma company that was one of the first biotech firms to use an explicit strategy of rational drug design rather than combinatorial chemistry. The company was founded in 1989.
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