In a groundbreaking development, the U.S. Food and Drug Administration (FDA) has granted approval to Vertex Pharmaceuticals (VRTX.O) and CRISPR Therapeutics’ (CRSP.BN) gene therapy, Casgevy, designed to treat a rare blood disorder requiring regular blood transfusions in patients aged 12 and older. The approval, announced by Vertex on Tuesday, comes as the second U.S. nod for Casgevy, following its December approval for sickle cell disease, another inherited blood disorder.
The FDA’s early decision, more than two months ahead of the expected action date of March 30, marks a significant stride in advancing innovative treatments for genetic disorders. Casgevy, a therapy that requires administration through authorized treatment centers with expertise in stem cell transplantation, is poised to be available early this year at a list price of $2.2 million in the United States for both approved indications.
Oppenheimer analyst Hartaj Singh, offering insights into the market dynamics, anticipates a “slow and steady launch” for Casgevy, projecting combined peak sales of approximately $400 million. Singh’s optimism stems from the belief that Casgevy’s profile will resonate well with patients new to gene therapy.
Casgevy’s approval is a watershed moment as it becomes the first treatment based on the Nobel Prize-winning CRISPR gene editing technology to secure approval for transfusion-dependent beta thalassemia (TDT) in the United States. The CRISPR technology, pioneered by Jennifer Doudna and CRISPR Therapeutics co-founder Emmanuelle Charpentier, employs molecular “scissors” to trim faulty parts of genes. This groundbreaking approach allows for the disabling or replacement of these segments with new strands of normal DNA, offering a novel and precise therapeutic avenue.
In the competitive landscape of gene therapy, Casgevy faces a notable rival in bluebird bio’s (BLUE.O) Zynteglo, which received FDA approval in 2022 as the first cell-based gene therapy for treating adult and pediatric patients with TDT. Zynteglo made headlines not only for its pioneering treatment approach but also for its record pricing at $2.8 million.
TDT, also known as Cooley’s anemia in its more severe form, imposes a heavy burden on affected children, leading to life-threatening anemia that necessitates blood transfusions every two to five weeks. More than 100,000 people globally are estimated to have transfusion-dependent thalassemia, with at least 1,200 individuals grappling with the disorder in the United States, according to data from Boston Children’s Hospital.
The approval of Casgevy opens up new possibilities for patients grappling with the challenges of TDT, offering a ray of hope in the form of a cutting-edge therapeutic intervention. While the list price of $2.2 million may raise eyebrows, it reflects the immense investment, research, and development efforts that have gone into bringing this revolutionary therapy to fruition.
As Casgevy prepares for its market debut, stakeholders, including healthcare providers, payers, and patients, will closely monitor its rollout. The gene therapy landscape is evolving rapidly, with each approval paving the way for future innovations. Casgevy’s success may not only redefine treatment paradigms for rare blood disorders but also serve as a testament to the transformative potential of gene editing technologies in the realm of modern medicine.