Q1 2026 PDUFA activity highlights continued momentum in rare diseases and specialty indications, building on trends observed in late 2025 regulatory decisions, with a notable concentration of therapies targeting ultra-rare genetic disorders and metabolic conditions. The quarter also reflects sustained growth in immunology and oncology, alongside an increasing presence of gene therapies and precision medicines. Additionally, multiple lifecycle expansions for established assets signal ongoing efforts to broaden indications and maximize commercial value across portfolios.
| PDUFA Date | Company | Orphan Drug | Indication |
| 01/10/26 | Atara Biotherapeutics | Tabelecleucel (Tab-cel) |
Epstein-Barr Virus Positive Post-Transplant Lymphoproliferative Disease
|
| 01/13/26 | Travere Therapeutics | FILSPARI (Sparsentan) | Focal Segmental Glomerulosclerosis (FSGS) |
| 01/14/26 | Sentynl Therapeutics | CUTX-101 | Menkes disease |
| 01/28/26 | Tenpoint Therapeutics | BRIMOCHOL PF | Presbyopia |
| 01/31/26 | Pharming Group | Leniolisib |
Activated Phosphoinositide 3-kinase Delta Syndrome (APDS)
|
| 01/31/26 | Aquestive Therapeutics | Anaphylm | Severe allergic reactions |
| 01/31/26 | Aquestive Therapeutics | Anaphylm™ (dibutepinephrine) | Severe Allergic Reactions |
| 02/08/26 | REGENXBIO | RGX-121 | MPS II (Hunter syndrome) |
| 02/20/26 | Merck | Keytruda | Platinum-Resistant Recurrent Ovarian Cancer |
| 02/20/26 | Vanda Pharmaceuticals | Bysanti | Bipolar I Disorder and Schizophrenia |
| 02/23/26 | Immedica Pharma | Pegzilarginase | Arginase 1 deficiency |
| 02/24/26 | Sanofi and Regeneron | Dupixent (dupilumab) | Allergic fungal rhinosinusitis (AFRS) |
| 02/25/26 | Taiho Pharmaceutical and Taiho Oncology | Inqovi + Venclexta | Acute Myeloid Leukemia (AML) |
| 02/25/26 | Eton Pharmaceuticals | ET-600 (Desmopressin Oral Solution) |
Central diabetes insipidius, also known as arginine vasopressin deficiency (AVP-D)
|
| 02/28/26 | Ascendis Pharma | Navepegritide (TransCon CNP) | Achondroplasia |
| 02/28/26 | Chiesi | Idebenone | Leber hereditary optic neuropathy (LHON) |
| 02/28/26 | Biomarin Pharmaceutical Inc. | PALYNZIQ (pegvaliase-pqpz) | Phenylketonuria |
| 03/06/26 | Lantheus Holdings | PYLARIFY TruVu (piflufolastat F 18) |
prostate-specific membrane antigen (PSMA) imaging
|
| 03/06/26 | Bristol Myers Squibb | Sotyktu (Deucravacitinib) | Psoriatic arthritis |
| 03/06/26 | GSK | Arexvy | Lower respiratory tract disease (LRTD) |
| 03/16/26 | Aldeyra Therapeutics | Reproxalap | Dry eye disease |
| 03/20/26 | Rhythm Pharmaceuticals | IMCIVREE (setmelanotide) | Acquired Hypothalamic Obesity |
| 03/24/26 | GSK | Linerixibat |
cholestatic pruritus in patients with primary biliary cholangitis (PBC)
|
| 03/28/26 | Rocket Pharmaceuticals | Kresladi | Severe Leukocyte Adhesion Deficiency-I (LAD-I) |
| 03/29/26 | Lantheus Holdings | LNTH-2501 |
PET imaging for SSTR-positive neuroendocrine tumors
|
