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FDA CBER lists six orphan-designated biologics newly licensed in 2026, from Kresladi to Voceryq

🇺🇸 FDA CBER · Tue 10 Nov 2026

The agency's 2026 orphan approvals list for new BLAs covers six products, with approval dates from 26 March to 30 October 2026.

The US FDA's Center for Biologics Evaluation and Research (CBER) has published its list of orphan-designated products that received new Biologics License Applications (BLAs) in 2026. The page was current as of 10 November 2026 and names six products, ordered by approval date from newest to oldest.

The most recent is Voceryq, from Inovio Pharmaceuticals, approved on 30 October 2026 to reduce the frequency of surgical papilloma excisions in adults with recurrent respiratory papillomatosis. Ultragenyx has two entries. Fayuvi, approved on 17 September, is for neurologic manifestations of mucopolysaccharidosis type IIIA (Sanfilippo syndrome type A) in paediatric patients with preserved neurodevelopmental function. Genglycos, approved on 19 August, is for adults and children aged 8 and over with glycogen storage disease type Ia. It is indicated to reduce daily cornstarch intake as an adjunct to nutritional management.

Orca Biosystems' Tregzi was approved on 30 June for use in matched donor haematopoietic stem cell transplantation with a myeloablative preparative regimen. It is indicated in adults with haematological malignancies, for haematopoietic and immunologic reconstitution and to improve chronic graft-versus-host disease-free survival. Regeneron's Otarmeni followed on 23 April for severe-to-profound and profound sensorineural hearing loss associated with molecularly confirmed biallelic OTOF variants. It is limited to patients with preserved outer hair cell function and no prior cochlear implant in the same ear, and it covers both paediatric and adult patients.

The earliest entry is Rocket Pharmaceuticals' Kresladi, approved on 26 March for paediatric patients with severe leukocyte adhesion deficiency-I due to biallelic ITGB2 variants. It is for those without an available HLA-matched sibling donor for allogeneic stem cell transplant. FDA notes that the list reflects each application as of its approval date and is not updated for later changes in applicant or application status.

What it means for your team

  • Pharmacovigilance teams can use the list as a checklist of newly licensed rare-disease biologics that now carry post-marketing safety reporting obligations.
  • Regulatory and quality teams should check the indication wording closely. Several are narrowly defined, for example by genetic variant, age group, donor availability or prior cochlear implant status.
  • Because the list is not updated after approval, any later changes in applicant or application status must be checked against FDA's current licence records.

Official source: FDA CBER ↗

Article written by VigiEye from the official source (FDA CBER). AI-assisted; check the source before acting.

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