New reimbursement deal in Germany expands access to Casgevy for SCD
Gene-editing therapy to now be available to patients at low or no cost
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Casgevy (exagamglogene autotemcel) will be reimbursed by Germany’s public health system, enabling people with severe sickle cell disease (SCD) in the country, ages 12 and older, to access the one-time gene-editing therapy at low or no cost.
The reimbursement agreement, established with Vertex Pharmaceuticals — which developed the therapy alongside CRISPR Therapeutics — covers both eligible SCD patients and eligible people with transfusion-dependent beta thalassemia (TDT), a related blood condition. This matches the approved indications of Casgevy in the European Union, where the therapy was cleared for use in 2024.
“This agreement represents significant progress for people living with these two devastating and life-shortening diseases,” Ludovic Fenaux, senior vice president at Vertex International, said in a company press release announcing the deal’s signing.
“We are pleased to collaborate across the health care system to ensure the value of Casgevy is recognized and sustainable patient access is secured,” Fenaux said.
Germany joins a growing number of countries that offer reimbursement for Casgevy, including other EU nations such as Italy, as well as England, through its publicly-funded health systems. The therapy is widely indicated for use in SCD, with approvals in the U.S., Saudi Arabia, Bahrain, and other nations.
A group of rare genetic disorders, SCD is caused by the production of faulty hemoglobin, the protein that carries oxygen in red blood cells. It leads to clumping inside red blood cells, making them assume a sickle-like shape.
Sickled cells are prone to die prematurely and to block blood vessels, leading to SCD symptoms such as anemia, marked by low levels of red blood cells, and painful episodes known as vaso-occlusive crises (VOCs).
In trials, Casgevy reduced or eliminated SCD crises
Casgevy uses CRISPR-Cas9 gene-editing technology to increase the production of fetal hemoglobin in the body. This hemoglobin type, normally produced during fetal development, is more effective at carrying oxygen than adult hemoglobin.
The treatment starts by collecting a patient’s hematopoietic stem cells, which can mature into all types of blood cells, from bone marrow, the spongy tissue in the center of bones. The individual’s cells are then genetically modified in a laboratory to activate the production of fetal hemoglobin. Afterward, the edited cells are infused back into the patient, where they are expected to generate red blood cells that produce fetal hemoglobin.
Regulatory approvals for Casgevy were mainly based on results from a Phase 2/3 clinical trial dubbed CLIMB-121 (NCT03745287). In that study, all but one of 30 treated adolescents and adults with SCD were free from severe VOCs and related hospitalizations for at least one consecutive year. Some of the patients experienced no severe VOCs for up to nearly four years after dosing.
Additional trial data showed that therapy was also associated with improvements in quality of life.
Patients who completed the trial are being followed for up to 15 years in the ongoing open-label CLIMB-131 extension study (NCT04208529), which is evaluating Casgevy’s long-term safety and effectiveness.
The therapy also shown promise in younger children with SCD. Preliminary data from the Phase 3 CLIMB-151 trial (NCT05329649) showed that all Casgevy-treated children, ages 5-11, remained free of VOCs for up to two years. Vertex is planning to file applications seeking expansion of Casgevy’s current indication — covering SCD patients ages 12 and older — to those as young as 5.
