Signals
9Osteogenesis imperfecta therapy reshapes around anti-sclerostin programs
Setrusumab pivotal data, bisphosphonate maturity, and emerging gene therapy programs are reshaping osteogenesis imperfecta management.
Achondroplasia therapy options widen past vosoritide
Vosoritide commercial maturity and follow-on CNP analogue and FGFR3-targeted programs are restructuring achondroplasia management.
Congenital adrenal hyperplasia therapy reshapes around CRF1 receptor antagonism
Crinecerfont approval, follow-on CRF1 receptor antagonist programs, and structured care infrastructure are reshaping classic congenital adrenal hyperplasia management.
Phenylketonuria therapy options mature past low-phenylalanine diet
Pegvaliase commercial maturity, sapropterin use, and emerging gene therapy programs are restructuring phenylketonuria management.
Neuromyelitis optica spectrum disorder therapy class competition matures
Eculizumab, ravulizumab, satralizumab, inebilizumab, and emerging mechanism programs define a competitive NMOSD prescribing landscape.
Hereditary angioedema oral options reach maturity
Oral plasma kallikrein inhibitors and emerging factor XIIa inhibitor programs are restructuring HAE prophylaxis and on-demand therapy.
Cell therapy moves into non-malignant rare disease
Cell-therapy approaches are beginning to land in non-malignant rare-disease indications including severe lupus, scleroderma, and inherited metabolic conditions.
FDA approves tividenofusp alfa for neurologic Hunter syndrome
The FDA approved Avlayah (tividenofusp alfa-eknm) to treat the neurologic manifestations of MPS II (Hunter syndrome), marking the first therapy specifically indicated for the CNS dimension of this rare lysosomal storage disorder.
ASO platform diversification beyond DMD is reshaping rare-disease commercial planning
The antisense oligonucleotide (ASO) platform has moved beyond the DMD foundation into multiple rare-disease indications including SMA, AdLD, Stargardt disease and adjacent conditions. The platform-level commercial logic is reshaping rare-disease portfolio strategy at multiple sponsors.
Snapshots
3Antisense oligonucleotide platform landscape (2026 reference)
Reference snapshot of approved ASO therapies, late-stage programs, and the delivery and chemistry platforms behind them.
Rare disease enzyme replacement therapy landscape, 2026 mid-year reference
Reference layout of the enzyme replacement therapy class in rare disease as of mid-2026: approved assets across lysosomal storage disease, the next-generation engineered-enzyme programs, the gene therapy alternatives, and the live commercial questions including chronic-infusion burden and CNS access.
Approved gene therapies by indication, 2026 mid-year reference
Reference layout of approved in vivo and ex vivo gene therapies as of mid-2026: indications, mechanism, delivery vector or platform, regulatory pathway, and the live commercial questions for each.