PDUFA DateOrphan DrugIndicationCompanyStatus 1.5.2026Cablivi (caplacizumab)Acquired thrombotic thrombocytopenic purpura (aTTP)SanofiAPPROVED 1.10.2026TabelecleucelEpstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD)Atara BiotherapeuticsComplete Response Letter 1.13.2026Cerezyme (imiglucerase)Gaucher disease type 3SanofiAPPROVED 1.14.2026Copper histidinate (CUTX-101)Menkes diseaseSentynl TherapeuticsAPPROVED 1.27.2026Darzalex Faspro with D-VRdMultiple myelomaJohnson & JohnsonAPPROVED 1.31.2026LeniolisibActivated phosphoinositide 3-kinase delta syndrome (APDS)Pharming GroupComplete Response Letter 2.8.2026Clemidsogene lanparvovec (RGX-121)Mucopolysaccharidosis II (MPS II)REGENXBIOComplete Response Letter 2.13.2026BitopertinErythropoietic protoporphyria (EPP)Disc MedicineComplete Response Letter 2.20.2026Venetoclax plus acalabrutinibChronic lymphocytic leukemiaAbbVieAPPROVED 2.23.2026PegzilarginaseArginase 1 deficiency (ARG1-D)Immedica Pharma ABAPPROVED 2.25.2026Inqovi (decitabine and cedazuridine) plus Venclexta (venetoclax)Acute myeloid leukemiaTaiho OncologysNDA accepted 2.25.2026ET-600Arginine vasopressin deficiency (AVP-D)Eton PharmaceuticalsAPPROVED 2.28.2026Palynziq (pegvaliase)Phenylketonuria (PKU)BioMarin APPROVED 2.28.2026TransCon CNP (navepegritide)AchondroplasiaAscendis PharmaAPPROVED 2.28.2026IdebenoneLeber hereditary optic neuropathy (LHON)ChiesiComplete Response Letter 3.5.2026Tec-Dara [Tecvayli(teclistamab) plus Darzalex Faspro (daratumumab and hyaluronidase)]Multiple myelomaJohnson & JohnsonAPPROVED 3.3.2026Juxtapid (lomitapide)Homozygous familial hypercholesterolemia (HoFH)ChiesiAPPROVED 3.10.2026Wellcovorin (leucovorin calcium)Cerebral folate deficiencyGSKAPPROVED 3.24.2026LinerixibatPrimary biliary cholangitis AlfasigmaAPPROVED 3.28.2026Kresladi (marnetegragene autotemcel)Leukocyte adhesion deficiency-I (LAD-I)Rocket PharmaceuticalsAPPROVED 4.2.2026Tecartus (brexucabtagene autoleucel)Mantle cell lymphomaGilead/Kite OncologyAPPROVED 4.3.2026Nusinersen high-dose regimenSpinal muscular atrophyBiogenAPPROVED 4.5.2026Tividenofusp alfaHunter syndrome (MPS II)Denali TherapeuticsAPPROVED 4.8.2026Opdivo (nivolumab)Stage III or IV classical Hodgkin Lymphoma (cHL)Bristol Myers SquibAPPROVED 4.13.2026FILSPARI (sparsentan)Focal segmental glomerulosclerosis (FSGS)Travere TherapeuticsAPPROVED 4.23.2026Otarmeni (lunsotogene parvec-cwha)OTOF-related hearing lossRegeneron PharmaceuticalsAPPROVED 5.1.2026Jakafi XR (ruxolitinib)Myelofibrosis, polycythemia vera, and graft-versus-host diseaseIncyteAPPROVED 5.10.2026Vyvgart (efgartigimod alfa-fcab)AChR-Ab seronegative generalized myasthenia gravis (gMG)ArgenxAPPROVED 5.27.2026Pivekimab sunirineBlastic plasmacytoid dendritic cell neoplasm (BPDCN)AbbVieAPPROVED 6.2.2026Cavhanza (nilotinib)Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) Cycle PharmaAPPROVED 6.10.2026Oclaiz (CAM2029)AcromegalyCamurusComplete Response Letter 6.18.2026XS003 (nilotinib)Chronic myeloid leukemia (CML)Xspray PharmaComplete Response Letter 6.29.2026LNTH-2501 (Gallium-68 edotreotide)Somatostatin receptor-positive (SSTR+) neuroendocrine tumors (NETs)Lantheus HoldingsComplete Response Letter 6.30.2026VeligrotugThyroid eye disaseViridian TherapeuticsAPPROVED Q3 2026RusfertidePolycythemia vera (PV)Takeda and Protagonist TherapeuticsNDA accepeted 7.6.2026Tregzi (Orca-T)Acute myeloid leukemia, acute lymphoblastic leukemia, and myelodysplastic syndromesOrca BioAPPROVED 7.7.2026AtaciceptIgA NephropathyVera TherapeuticsAPPROVED 7.23.2026SarclisaMultiple MyelomaSanofiAPPROVED 8.17.2026IberdomideMultiple MyelomaBristol Myers SquibNDA accepted 8.22.2026DeramiocelDuchenne muscular dystrophyCapricor TherapeuticsCRL lifted
The matrix physically blocks drug penetration, and bacteria inside biofilms alter their metabolic state in ways that make standard antibiotics far less effective
The amount of unencapsulated GHK-Cu which diffused into the solution was determined via HPLC (Agilent 1100, Agilent Technologies, Inc., Santa Clara, CA, USA) using a UV-Vis detector at a wavelength of 220 nm using a 0.2% trifluoroacetic acid solution (TFA) and the ODS C18 column (Keystone Scientific, Inc
Oxidized phospholipids are ligands for LRP6
Molecular mechanisms involved in doxorubicin-induced cardiotoxicity: a bibliometrics analysis by VOSviewer
