The headquarters of US biopharmaceutical company Vertex Pharmaceuticals in Boston, Massachusetts, on November 4, 2023.
Joseph Prezioso | AFP | Getty Images
The U.S. health regulator has approved Vertex Pharmaceuticals and CRISPR Therapeutics’ gene therapy to treat a rare blood disorder that requires regular blood transfusions in patients ages 12 and older, Vertex said on Tuesday.
This decision marks the second American approval for the treatment, called “Casgevi,” after it received the green light in December to treat sickle cell anemia, another genetic blood disorder.
The FDA approval comes more than two months before the expected March 30 action date.
Casgevy, which requires administration through approved treatment centers with expertise in stem cell transplantation, will be made available early this year at a list price of $2.2 million in the U.S. for both approved indications, Vertex said in an email response.
Hartaj Singh, an analyst at Oppenheimer, said he expects a “slow and steady rollout” for the treatment and estimates peak combined sales at about $400 million.
“We think the Kasjivi profile will be good for treatment-naïve patients,” Singh said.
Casgevy has become the first treatment based on the Nobel Prize-winning CRISPR gene-editing technology to gain approval for transfusion-dependent beta thalassemia, or TDT, in the United States.
CRISPR, discovered by Jennifer Doudna and CRISPR Therapeutics co-founder Emmanuel Charpentier, uses molecular “scissors” to snip out defective parts of genes that can then be inactivated or replaced with new strands of normal DNA.
Bluebird Bios competitor Zynteglo, in 2022, became the first cell-based gene therapy to gain FDA approval to treat adult and pediatric patients with TDT, reaching a record price of $2.8 million.
TDT, or Cooley’s anemia, the most serious form of the disorder, causes children to develop life-threatening anemia that requires blood transfusions every two to five weeks.
It is estimated that more than 100,000 people have transfusion-dependent thalassemia globally, including at least 1,200 people with the disorder in the United States, according to data from Boston Children’s Hospital.