One-time treatment costs $2 million!: Swiss biotechnology Crispr received approval in the USA

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In a statement released Friday evening, the FDA announced the first approval of a gene therapy that could be used to treat patients with sickle cell anemia. Approval is valid for patients 12 years of age and older.

Sickle cell anemia is an inherited blood disease in which defective red blood cells carry too little oxygen and can clog the arteries. This causes tissue and organ damage that can result in death. According to the FDA, approximately 100,000 people in the United States suffer from this disease.

Vertex’s Casgevy therapy uses Crispr/Cas9 technology, which can be used to cut the genetic material (DNA) of blood-producing bone marrow cells. This eliminates faulty DNA fragments. The modified blood-producing bone marrow cells are then reintroduced into the patient. The FDA wrote that it stimulates the production of healthy red blood cells.

This stem cell transplant is expensive: A one-time treatment with Casgevy costs $2.2 million, according to the Reuters news agency.

Both companies announced that as a result of the FDA approval, the Zug-based biotech will now receive a $200 million milestone payment from Vertex. Crispr Therapeutics gets 40 percent of the profits and Vertex gets 60 percent. (SDA)

Source :Blick