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 APPROVED
1.31.2022 Somatrogon (hGH-CTP) Children – growth hormone deficiency Opko Health / Pfizer
2.17.2022 Mitapivat (AG-348) Pyruvate kinase deficiency Immunocore APPROVED
2.25.2022 Bardoxolone methyl Alport Syndrome Reata Pharmaceuticals Received CRL
2.28.2022 Filsuvez (Oleogel-S10) Epidermolysis Bullosa Amryt Pharma Received CRL
2.28.2022 Ciltacabtagene autoleucel (cilta-cel) Multiple myeloma Legend Biotech APPROVED
2.28.2021 Pactitinib Myelofibrosis CTI Biopharm APPROVED
3.16.2022 Imcivree (setmelanotide) Alstrom Syndrome and Bardet-Biedl Syndrome Rhythm Pharmaceuticals PDUFA moved to June 16
3.20.2022 Ganaxolone CDKL5 Deficiency Disorder (CDD) Marinus Pharmaceuticals APPROVED
3.25.2022 FINTEPLA (fenfluramine) Lennox-Gastaut syndrome Zogenix Pharmaceuticals APPROVED
3.29.2022 Vadadustat Anemia due to chronic kidney disease (CKD) in patients on dialysis and not on dialysis. Akebia Therapeutics Received CRL
4.2.2022 Yescarta (axicabtagene ciloleucel)

Initial Treatment of Relapsed or Refractory Large B-cell Lymphoma (LBCL)

Kite (a Gilead Company) APPROVED
4.14.2022 Vutrisiran ATTR amyloidosis Alnylam Pharmaceuticals PDUFA moved to July 14
4.28.2022 Mavacamten (MYK-461) Hypertrophic cardiomyopathy (HCM) BMS / LianBio APPROVED
4.30.2022 Parsaclisib Marginal Zone Lymphoma

Follicular lymphoma

Incyte NDA withdrawn
4.30.2022 Surufatinib Neuroendocrine Tumors Hutchmed Received CRL
5.20.2022 Dupilumab (Dupixent) Eosinophilic Esophagitis (EoE) Regeneron / Sanofi APPROVED
6.5.2022 ACER-101 Urea cycle disorder (UCD) Acer Therapeutics / Relief Therapeutics WAITING
6.14.2022 Vutrisiran (Amvuttra) Polyneuropathy observed in patients with hereditary ATTR (hATTR) amyloidosis Alnylam APPROVED

Extended to 9.16.2022

Lenti-D (Elivaldogene Autotemcel / Eli-Cel) Cerebral Adrenoleukodystrophy (CALD) Bluebird bio BLA accepted
6.24.2022 Breyanzi (lisocabtagene maraleucel) Relapsed or Refractory Large B-cell Lymphoma Bristol Myers Squibb BLA accepted
6.25.2022 Ukoniq (umbralisib) and Ublituximab Chronic Lymphocytic Leukemia (CLL) and small lymphocytic lymphoma (SLL) TG Therapeutics BLA withdrawn
6.27.2022 Reblozyl (Luspatercept) Non-transfusion-dependent beta-thalassemia Acceleron Pharma BLA accepted
6.29.2022 AMX0035 Amyotrophic Lateral Sclerosis (ALS) Amylyx Pharmaceuticals NDA accepted
8.19.2022 Betibeglogene autotemcel (beti-cel) (LentiGlobin) β-Thalassemia Bluebird bio BLA accepted
10.26.2022 Lumasiran Primary Hyperoxaluria Type 1 Alnylam Pharmaceuticals NDA accepted
10.29.2022 AT-GAA – Propel Pompe disease Amicus NDA accepted


2021 Orphan Drugs: PDUFA Dates and FDA Approvals