FDA approves Casgevy, pioneering CRISPR therapy for sickle cell disease – What Is It & Everything You Need To Know!
Casgevy: The biggest breaking news is here. A lot of people have been waiting for this announcement for a very long time and now it is finally here. FDA has now officially approved the Casgevy which is a pioneering CRISPR therapy for sickle cell disease. This utilizes gene editing to alleviate pain crises in patients 12 and older. Vertex’s $2.2M therapy provides hope for 16,000 with severe sickle cell cases. Now check out this entire article to learn everything about Casgevy.
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FDA Approved Casgevy
Finally, after a lot of waiting, the UD FDA has granted the historic approval to the first CRISPR-based gene-editing treatment for sickle cell disease, offering groundbreaking hope. This is one of the biggest breaking news of current time. This is a very happy news. Now to know what exactly “Casgevy” is. So scroll down to the next paragraph of this article.
What is Casgevy?
Recently, the United States Food and Drug Administration (FDA) has officially approved Casgevy. It is a pioneering CRISPR-based gene-editing treatment for sickle cell disease. Developed collaboratively by Vertex Pharmaceuticals and CRISPR Therapeutics, Casgevy marks a significant milestone in the field of genetic medicine, utilizing CRISPR technology, which earned its discoverers a Nobel Prize in Chemistry.
What is Sickle Cell Disease?
Sickle cell disease is an inherited blood disorder affecting approximately 100,000 Americans, resulting in misshapen red blood cells causing painful blockages in blood vessels. Casgevy employs CRISPR to edit a patient’s DNA, activating fetal hemoglobin to maintain healthy blood cell shapes. Clinical trials have shown promising results, eliminating pain crises in a majority of patients.
Approval of FDA Guidelines
There are some guidelines on which the FDA has approved the Casgevy. So it has been approved that it is going to cover people who are aged 12 or above. It is offering hope to those grappling with the rare and life-threatening blood disorder.
Doctor Statement Regarding Casgevy
Dr. Nicole Verdun, Director of the Office of Therapeutic Products at the FDA’s Center for Biologics Evaluation and Research, highlighted the potential of gene therapy while stating the unmet needs, especially for those with limited treatment options. Despite the transformative potential of Casgevy, the intricate process involves months of treatment.
Vertex Pricing Casgevy for Patient
Blood stem cells are extracted, genetically modified in Vertex’s lab, and then reintroduced to the patient after chemotherapy. Vertex, estimating around 16,000 eligible candidates with severe sickle cell cases, will lead the drug’s launch. However, concerns arise about the extensive timeline, potential infertility risks, and the substantial cost, with Vertex pricing Casgevy at $2.2 million per patient.
