The headquarters of the US biopharmaceutical company Vertex Pharmaceuticals in Boston, Massachusetts, on November 4, 2023.
Joseph Prezioso | Afp | Getty Images
The U.S. Food and Drug Administration has approved gene therapy from Vertex Pharmaceuticals and CRISPR Therapeutics to treat a rare blood disorder that requires regular blood transfusions in patients ages 12 and older, Vertex said Tuesday.
The decision gives the therapy, branded Casgevy, its second U.S. approval after it was approved in December for the treatment of sickle cell anemia, another inherited blood disorder.
The Food and Drug Administration's approval comes more than two months ahead of the expected March 30 effective date.
Casgevy, which requires administration by authorized treatment centers with expertise in stem cell transplantation, will be available in the U.S. early this year at a list price of $2.2 million for both approved indications, Vertex said in an email response.
Oppenheimer analyst Hartaj Singh said he expected a “slow and steady rollout” of the therapy and estimated combined peak sales at about $400 million.
“We believe Casgevy’s profile is well-suited to treatment-naïve patients,” Singh said.
Casgevy is the first treatment based on the Nobel Prize-winning gene editing technology CRISPR to receive approval for transfusion-dependent beta thalassemia (TDT) in the United States.
CRISPR, discovered by Jennifer Doudna and CRISPR Therapeutics co-founder Emmanuelle Charpentier, uses molecular “scissors” to trim faulty parts of genes, which can then be disabled or replaced with new strands of normal DNA.
Rival bluebird bio's Zynteglo became the first cell-based gene therapy to secure FDA approval in 2022 to treat adult and pediatric patients with TDT, commanding a record $2.8 million.
TDT, or Cooley anemia, the more severe form of the disease, causes children to develop life-threatening anemia that requires blood transfusions every two to five weeks.
According to Boston Children's Hospital, it is estimated that more than 100,000 people worldwide suffer from transfusion-dependent thalassemia, including at least 1,200 in the United States.