PDUFA Date | Orphan Drug | Indication | Company | Status |
First Quarter of 2024 | Brukinsa (zanubrutinib) | Relapsed or refractory follicular lymphoma | BeiGene | APPROVED |
1.12.2024 | Olbetuximab | Gastroesophageal junction (GEJ) adenocarcinoma | Astellas | Complete response letter |
1.16.2024 | Hyqvia [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase] | Chronic inflammatory demyelinating polyneuropathy (CIDP) | Takeda | APPROVED |
1.31.2024 | Dupixent (dupilumab) | Eosinophilic esophagitis (EoE) | Sanofi | APPROVED |
2.13.2024 | Onivyde (irinotecan liposome injection) | Metastatic pancreatic ductal adenocarcinoma | Ipsen | APPROVED |
3.13.2024 | Livmarli(maralixibat) | Cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) | Mirum Pharmaceuticals | APPROVED |
3.13.2024 | Tevimbra (tislelizumab-jsgr) | Unresectable or metastatic esophageal squamous cell carcinoma | BeiGene | APPROVED |
3.14.2024 | Resmetirom | Nonalcoholic steatohepatitis (NASH) | Madrigal Pharmaceuticals | APPROVED |
3.14.2024 | Breyanzi (lisocabtagene maraleucel) | Relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) | Bristol Myers Squibb | APPROVED |
3.15.2024 | Abecma | Triple-class exposed relapsed or refractory multiple myeloma | Bristol Myers Squibb and and 2seventy bio | Awaiting new action date |
3.18.2024 | Lenmeldy (atidarsagene autotemcel) | Metachromatic leukodystrophy (MLD) | Orchard Therapeutics | APPROVED |
3.21.2024 | Givinostat | duch*enne muscular dystrophy (DMD) | ITF Therapeutics | APPROVED |
3.22.2024 | OPSYNVI (macitentan and tadalafil) | Pulmonary Arterial Hypertension (PAH) | Johnson & Johnson | APPROVED |
3.25.2024 | Ultomiris (ravulizumab-cwvz) | Neuromyelitis optica spectrum disorder (NMOSD) | AstraZeneca | APPROVED |
3.26.2024 | Sotatercept | Pulmonary arterial hypertension (PAH) | Merck | APPROVED |
3.27.2024 | Vadadustat | Anemia caused by chronic kidney disease | Akebia Therapeutics | APPROVED |
3.30.2024 | Exagamglogene autotemcel (exa-cel) | Transfusion-dependent beta thalassemia | Vertex Pharmaceuticals and CRISPR Therapeutics | APPROVED |
3.31.2024 | Odronextamab (REGN1979) | Relapsed/refractory follicular lymphoma or relapsed/refractory diffuse large B-cell lymphoma (DLBCL) | Regeneron Pharmaceuticals | Complete response letter |
4.1.2024 | Voydeya (danicopan) | Extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria (PNH) | AstraZeneca | APPROVED |
4.5.2024 | Carvykti(ciltacabtagene autoleucel; cilta-cel) | Relapsed or refractory multiple myeloma | Janssen | APPROVED |
4.5.2024 | Opdivo (nivolumab) | Unresectable or metastatic urothelial carcinoma | Bristol Myers Squibb | APPROVED |
4.5.2024 | Abecma (idecabtagene vicleucel; ide-cel) | Triple-Class Exposed Relapsed or Refractory Multiple Myeloma After Two Prior Lines of Therapy | Bristol Myers Squibb & 2seventy Bio | APPROVED |
4.18.2024 | Alecensa (alectinib) | Anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC) | Genentech, Inc. | APPROVED |
4.26.2024 | Beqvez (Fidanacogene elaparvovec) | Hemophilia B | Pfizer | APPROVED |
4.30.2024 | Tovorafenib | Relapsed or progressive pediatric low-grade glioma (pLGG) | Day One Biopharmaceuticals | APPROVED |
4.30.2024 | Mavorixafor | WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome | X4 Pharmaceuticals | APPROVED |
5.23.2024 | Breyanzi (lisocabtagene maraleucel) | Relapsed or refractory follicular lymphoma (FL) | Bristol Myers Squibb | APPROVED |
5.25.2024 | Pz-cel | Recessive dystrophic epidermolysis bullosa | Abeona Therapeutics | Complete Response Letter |
5.28.2024 | Bkemv (eculizumab-aeeb) | Paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis and atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy | Amgen | APPROVED |
5.31.2024 | Breyanzi (lisocabtagene maraleucel) | Relapsed or refractory mantle cell lymphoma (MCL) | Bristol Myers Squibb | APPROVED |
6.10.2024 | Elafibranor | Primary biliary cholangitis (PBC) | Ipsen and GENFIT | APPROVED |
6.12.2024 | Tarlatamab | Advanced small cell lung cancer | Amgen | APPROVED |
6.14.2024 | Blinatumomab | CD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia | Amgen | APPROVED |
6.16.2024 | Imetelstat | Lower risk myelodysplastic syndromes (MDS) | Geron Corporation | APPROVED |
6.20.2024 | Piasky (crovalimab-akkz) | Paroxysmal nocturnal hemoglobinuria | Genentech | APPROVED |
6.21.2024 | ELEVIDYS (delandistrogene moxeparvovec-rokl) | duch*enne muscular dystrophy (DMD) | Sarepta Therapeutics | APPROVED |
6.21.2024 | VYVGART Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) | Chronic inflammatory demyelinating polyneuropathy (CIDP) | Argenx | APPROVED |
6.26.2024 | Epkinly (epcoritamab-bysp) | Relapsed or refractory follicular lymphoma | Genmab US | APPROVED |
6.30.2024 | RP-L201 (marnetegragene autotemcel) | Leukocyte adhesion deficiency-I (LAD-I) | Rocket Pharmaceuticals | Complete response letter |
7.24.2024 | Brineura (cerliponase alfa) | Neuronal ceroid lipofuscinosis type 2 (CLN2 disease) | BioMarin Pharmaceutical | APPROVED |
7.31.2024 | Dasynoc (dasatinib amorphous) | Chronic myeloid leukemia (CML) | Xspray Pharma | Complete Response Letter |
8.1.2024 | Tecelra (afamitresgene autoleucel) | Unresectable or metastatic synovial sarcoma | Adaptimmune Therapeutics | APPROVED |
8.4.2024 | Afami-cel | Advanced synovial sarcoma | Adaptimmune Therapeutics | APPROVED |
8.6.2024 | Vorasidenib | Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation | Servier Pharmaceuticals | APPROVED |
8.7.2024 | Fabhalta (iptacopan) | IgA nephropathy (IgAN) | Novartis | APPROVED |
8.13.2024 | LYMPHIR (denileukin diftitox) | Relapsed or refractory cutaneous T-cell lymphoma (CTCL) | Citius Pharmaceuticals | APPROVED |
8.14.2024 | Seladelpar | Primary biliary cholangitis (PBC) | CymaBay Therapeutics | APPROVED |
8.14.2024 | Palopegteriparatide | Hypoparathyroidism | Ascendis Pharma | APPROVED |
8.14.2024 | Niktimvo (axatilimab) | Chronic graft versus host disease | Incyte and Syndax Pharmaceuticals | APPROVED |
8.20.2024 | Lazertinib in combination with Rybrevant (amivantamab-vmjw) | EGFR+ advanced or metastatic non–small cell lung cancer | Janssen Biotech | APPROVED |
8.20.2024 | Equecabtagene autoleucel | Lupus Nephritis and Non-Renal Systemic Lupus Erythematosus | IASO Bio | APPROVED |
8.22.2024 | Linvoseltamab | Relapsed/refractory multiple myeloma | Regeneron Pharmaceuticals | Complete Response Letter |
9.5.24 | Filspari (sparsentan) | IgA Nephropathy | Travere Therapeutics | APPROVED |
9.21.2024 | Arimoclomol | Niemann-Pick disease type C | Zevra Therapeutics | NDA accepted |
9.24.2024 | IB1001 (N-acetyl-L-leucine) | Niemann-Pick disease type C | IntraBio | NDA accepted |
9.25.2024 | Keytruda (pembrolizumab) | Unresectable advanced or metastatic malignant pleural mesothelioma | Merck | sBLA accepted |
9.27.2024 | Sarclisa (isatuximab-irfc) in combination with bortezomib, lenalidomide, and dexamethasone | Transplant-ineligible multiple myeloma | Sanofi | sBLA accepted |
10.15.2024 | Ocaliva (obeticholic acid) | Primary biliary cholangitis (PBC) | Intercept Pharmaceuticals | sNDA accepted |
10.21.2024 | Octreotide (CAM2029) | Acromegaly | Camurus | NDA accepted |
11.13.2024 | Upstaza (eladocagene exuparvovec) | AADC deficiency | PTC Therapeutics | BLA accepted |
11.16.2024 | Obecabtagene autoleucel (obe-cel) | Relapsed/refractory adult B-cell acute lymphoblastic leukemia (ALL) | Autolus Therapeutics | BLA accepted |
11.28.2024 | Govorestat | Galactosemia type 1 | Applied Therapeutics | NDA accepted |
11.29.2024 | Acoramidis | Transthyretin amyloid cardiomyopathy (ATTR-CM) | BridgeBio Pharma | NDA accepted |
11.30.2024 | SH-201 | Certain forms of leukemia and other cancers | Shorla Oncology | NDA accepted |
12.19.2024 | Olezarsen | Familial chylomicronemia syndrome (FCS) | Ionis Pharmaceuticals | NDA accepted |
12.26.2024 | Revumenib | Relapsed or refractory (R/R) KMT2A-rearranged (KMT2Ar) acute leukemia | Syndax Pharmaceuticals | NDA accepted |
12.26.2024 | Imcivree (setmelanotide) | Bardet-Biedl syndrome or POMC/LEPR deficiency | Rhythm Pharmaceuticals | sNDA accepted |
12.27.2024 | DCCR (Diazoxide Choline) | Prader-Willi Syndrome | Soleno Therapeutics | NDA accepted |
12.28.2024 | Ensartinib | Metastatic ALK-positive non-small cell lung cancer (NSCLC) | Xcovery Holdings | NDA accepted |
12.28.2024 | Cosibelimab | Advanced cutaneous squamous cell carcinoma | Checkpoint Therapeutics | BLA accepted |
12.30.2024 | Crinecerfont | Congenital adrenal hyperplasia (CAH) | Neurocrine Biosciences | NDA accepted |
1.15.2025 | Tabelecleucel (Tab-cel) | Epstein-Barr Virus Positive Post-Transplant Lymphoproliferative Disease | Atara Biotherapeutics | BLA accepted |
2.17.2025 | Vimseltinib | Tenosynovial giant cell tumor (TGCT) | Deciphera Pharmaceuticals | NDA accepted |
6.17.2025 | Sebetralstat | Hereditary Angioedema | KalVista Pharmaceuticals | NDA accepted |