Rare diseases and orphan drugs are at the forefront of novel development and groundbreaking research. Almost half of all novel medications approved by the U.S. Food and Drug Administration (FDA) are orphan drugs. Below is the list of important regulatory dates for all orphan drugs for 2022.  

Prescription Drug User Fee Act (PDUFA) dates refer to deadlines for the FDA to review new drugs. The PDUFA date is 10 months after the drug application has been accepted by the FDA or 6 months, if the drug is given a priority review designation.  As each PDFUA date approaches, CheckRare will be covering the FDA meetings and outcomes.

 

PDUFA Date Orphan Drug Indication Company Status
1.29.2022 Zonisamide oral suspension Epilepsy Eton Pharmaceuticals / Azurity Pharmaceuticals NDA accepted
1.31.2022 Somatrogon (hGH-CTP) Children – growth hormone deficiency Opko Health / Pfizer BLA accepted
2.17.2022 Mitapivat (AG-348) Pyruvate kinase deficiency Agios NDA accepted
2.23.2022 Tebentafusp Uveal melanoma Immunocore Holdings BLA accepted
2.25.2022 Bardoxolone methyl Alport Syndrome Reata Pharmaceuticals NDA accepted
2.28.2022 Filsuvez (Oleogel-S10) Epidermolysis Bullosa Amryt Pharma NDA accepted
2.28.2022 Ciltacabtagene autoleucel (cilta-cel) Multiple myeloma Legend Biotech BLA accepted
2.28.2021 Pactitinib Myelofibrosis CTI Biopharm NDA accepted
3.16.2022 Imcivree (setmelanotide) Alstrom Syndrome and Bardet-Biedl Syndrome Rhythm Pharmaceuticals NDA accepted
3.20.2022 Ganaxolone CDKL5 Deficiency Disorder (CDD) Marinus Pharmaceuticals NDA accepted
3.25.2022 FINTEPLA (fenfluramine) Lennox-Gastaut syndrome Zogenix Pharmaceuticals NDA accepted
3.25.2022 Ukoniq (umbralisib) and Ublituximab Chronic Lymphocytic Leukemia (CLL) and small lymphocytic lymphoma (SLL) TG Therapeutics BLA accepted
3.27.2022 Reblozyl (Luspatercept) Non-transfusion-dependent beta-thalassemia Acceleron Pharma BLA accepted
3.29.2022 Vadadustat Anemia due to chronic kidney disease (CKD) in patients on dialysis and not on dialysis. Akebia Therapeutics NDA accepted
4.14.2022 Vutrisiran ATTR amyloidosis Alnylam NDA accepted
4.28.2022 Mavacamten (MYK-461) Hypertrophic cardiomyopathy (HCM) BMS / LianBio NDA accepted
4.30.2022 Parsaclisib Marginal Zone Lymphoma

Follicular lymphoma

Incyte NDA accepted
4.30.2022 Surufatinib Neuroendocrine Tumors Hutchmed NDA accepted
5.20.2022 Betibeglogene autotemcel (beti-cel) (LentiGlobin) β-Thalassemia Bluebird bio BLA accepted
5.29.2022 AT-GAA – Propel Pompe Disease Amicus NDA accepted
6.5.2022 ACER-101 Urea cycle disorder (UCD) Acer Therapeutics / Relief Therapeutics NDA accepted
6.17.2022 Lenti-D (Elivaldogene Autotemcel / Eli-Cel) Cerebral Adrenoleukodystrophy (CALD) Bluebird bio BLA accepted

 

2021 Orphan Drugs: PDUFA Dates and FDA Approvals