The table below is a review of notable pipeline updates that occurred in March 2022 for investigational products in development (not an inclusive list). Click on the article title to view our full coverage.

Drug Pharmacologic Class Proposed Indication Status
Dermatological Disorders
Ruxolitinib Cream (Incyte Corporation) JAK inhibitor Treatment of vitiligo. FDA extends PDUFA target date
Endocrine Disorders
Teplizumab (Provention Bio) Anti-CD3 monoclonal antibody For the delay of clinical type 1 diabetes in at-risk individuals. Resubmitted BLA accepted for review
Gastrohepatic Disorders
Vonoprazan (Phathom Pharmaceuticals)
Small molecule potassium-competitive acid blocker Treatment of erosive esophagitis. NDA submitted
Hematological Disorders
ASC618 (ASC Therapeutics) AAV gene therapy Treatment of hemophilia A. Fast Track designation
Vadadustat (Akebia Therapeutics) Hypoxia-inducible factor prolyl hydroxylase inhibitor Treatment of anemia due to chronic kidney disease in adults. Complete Response Letter issued
Immunization
RSVpreE (PF-06482077; Pfizer Inc.) Respiratory syncytial virus (RSV) vaccine candidate Prevention of RSV-associated lower respiratory tract illness in infants from birth up to 6 months of age by active immunization of pregnant women. Breakthrough Therapy designation
RSVpreE (PF-06482077; Pfizer Inc.) RSV vaccine candidate Prevention of lower respiratory tract disease caused by RSV in adults 60 years of age and older. Breakthrough Therapy designation
Infectious Diseases
EDP-235 (Enanta Pharmaceuticals) Coronavirus 3CL protease inhibitor Treatment and prevention of COVID-19. Fast Track designation
Metabolic Disorders
Elamipretide (Stealth BioTherapeutics Inc.) Peptide compound Treatment of Friedreich ataxia. Orphan Drug designation
rAAV-Olig001-ASPA
(Myrtelle Inc.)
AAV gene therapy Treatment of Canavan disease. Fast Track, Rare Pediatric Disease, Orphan Drug designations
SBT101
(SwanBio Therapeutics)
AAV gene therapy Treatment of adrenomyeloneuropathy. Orphan Drug designations
Musculoskeletal Disorders
Pegloticase + Methotrexate (Horizon Therapeutics plc) PEGylated uric acid specific enzyme + DMARD (folic acid antagonist) Treatment of uncontrolled gout. Priority Review granted
Oncology
ALLO-316 (Allogene Therapeutics) Allogeneic chimeric antigen receptor (CAR) T solid tumor candidate targeting CD70 Treatment of advanced or metastatic clear cell renal cell carcinoma. Fast Track designation
Futibatinib (Taiho Oncology, Inc.) Selective, irreversible small molecular inhibitor of FGFR 1, 2, 3, and 4 Treatment of patients with previously treated locally advanced or metastatic cholangiocarcinoma harboring FGFR2 gene rearrangements, including gene fusions. NDA accepted for Priority Review
Respiratory Disorders
Benralizumab (AstraZeneca) Interleukin-5 receptor alpha-directed cytolytic monoclonal antibody Treatment of chronic rhinosinusitis with nasal polyps. Complete Response Letter issued