Casgevy
Casgevy, Infusionsdispersion
Casgevy is a medicine with orphan drug status in Switzerland, active substance exagamglogenum autotemcelum, authorisation holder Vertex Pharmaceuticals (CH) GmbH. Swissmedic publishes 2 orphan indications for this product.
The authorisation is temporary under Art. 9a TPA and runs until 05/09/2026. It was granted with conditions because no equivalent alternative is available for a life-threatening disease.
Data
Data from the Swissmedic list of medicinal products with orphan drug status (Art. 7 TPLO), as of 2026-09-18. Swissmedic updates the list every month.
| Active substances | exagamglogenum autotemcelum |
|---|---|
| Authorisation holder | Vertex Pharmaceuticals (CH) GmbH |
| Authorisation number | 68978 |
| Authorisation status | Temporarily authorised |
| First authorised | 06/09/2024 |
| Temporary until | 05/09/2026 |
| Authorised indication | Beta Thalassämie: Casgevy wird angewendet zur Behandlung von transfusionsabhängiger Beta Thalassämie (TDT) bei Patienten ab 12 Jahren, die für eine Transplantation von hämatopoetischen Stammzellen (HSZ) geeignet sind und für die kein humaner Leukozyten-Antigen (HLA)-kompatibler, verwandter HSZ-Spender zur Verfügung steht. Sichelzellkrankheit: Casgevy wird angewendet zur Behandlung von schwerer Sichelzellkrankheit (SCD) bei Patienten ab 12 Jahren mit rezidivierenden vasookklusiven Krisen (VOC), die für eine Transplantation von hämatopoetischen Stammzellen (HSZ) geeignet sind und für die kein humaner Leukozyten-Antigen (HLA)-kompatibler, verwandter HSZ-Spender zur Verfügung steht. |
Orphan designations
Swissmedic publishes one row per medicine and orphan indication: the indication in the wording of the decision, the date the status was granted and, where the status was withdrawn, the withdrawal date. The table below reproduces those rows unchanged and links every indication to the matching rare disease in the directory.
| Orphan indication | Disease | Status granted | Status withdrawn |
|---|---|---|---|
| Behandlung der Sichelzellkrankheit | Sickle cell disease | 12/09/2022 | Status active |
| Behandlung von Beta-Thalassämie intermedia und major | Beta thalassaemia | 12/09/2022 | Status active |
Reimbursement
Whether compulsory health insurance pays for this medicine depends on the FOPH specialities list (SL). Medicines that are not listed can be reimbursed case by case under Art. 71a-71d KVV.
Frequently asked questions
Is Casgevy authorised in Switzerland?
The Swissmedic list of medicinal products with orphan drug status shows no valid authorisation for Casgevy. The status alone does not allow placing on the market: that requires an authorisation under Art. 9 ff. TPA.
What does orphan drug status mean for Casgevy?
The status opens the simplified authorisation procedure (Art. 14 para. 1 let. f TPA), priority assessment, a waiver of the flat-rate fee for the new authorisation application (Art. 9 let. a FeeO-Swissmedic) and, on request, 15 years of document protection (Art. 11b para. 4 TPA).
Is Casgevy reimbursed by Swiss health insurance?
That depends on the FOPH specialities list (SL), not on the orphan drug status. If the product is not listed, reimbursement in an individual case under Art. 71a to 71d KVV can apply. Check the current SL entry and public price in the FOPH specialities list.
Bring your orphan drug to Switzerland
You can enquire about orphan drug status, authorisation and reimbursement in Switzerland. Any specialist service and its scope are agreed separately with the external partner.
- Public Swissmedic data
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