CBER 2025 Orphan Approvals (new BLAs)
This list reflects information regarding the applications as of the approval date. It is not updated with regard to applicant or application status changes. The applications are listed by date of approval in reverse chronological order.
| Trade Name/Proper Name | Indication | Manufacturer/License Number | Approval Date |
|---|---|---|---|
| WASKYRA | for treatment of pediatric patients aged 6 months and older and adults with Wiskott-Aldrich Syndrome (WAS) who have a mutation in the WAS gene for whom hematopoietic stem cell transplantation (HSCT) is appropriate and no suitable HLA-matched related stem cell donor is available | Fondazione Telethon ETS | 12/9/2025 |
| ITVISMA | For the treatment of spinal muscular atrophy (SMA) in adult and pediatric patients 2 years of age and older with confirmed mutation in survival motor neuron 1 (SMN1) gene | Novartis, Inc | 11/24/2025 |
| PAPZIMEOS | For the treatment of adults with recurrent respiratory papillomatosis | Precigen, Inc | 8/14/2025 |
| ZEVASKYN | For the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB) | Abeona Therapeutics Inc | 4/28/2025 |
| ENCELTO | For the treatment of adults with idiopathic macular telangiectasia type 2 (MacTel) | Neurotech Pharmaceuticals | 3/5/2025 |