The table provided is a review of notable updates that occurred in April 2022 for investigational products in development.

Drug Pharmacologic Class Proposed Indication Status
Gastrohepatic Disorders
Dupixent (dupilumab; Regeneron Pharmaceuticals) Interleukin-4 receptor alpha antagonist Treatment of eosinphilic esophagitis in patients 12 years of age and older. sBLA accepted for Priority Review
REC-4881 (Recursion) Non-ATP-competitive allosteric small molecule inhibitor of MEK1 and MEK2 Treatment of familial adenomatous polyposis in patients who have previously undergone a colectomy or proctocolectomy. Fast Track designation
Hematological Disorders
Daprodustat (GlaxoSmithKline) Hypoxia-inducible factor prolyl hypdroxylase inhibitor Treatment of anemia due to chronic kidney disease. NDA accepted for review
Vadadustat (Akebia Therapeutics) Hypoxia-inducible factor prolyl hypdroxylase inhibitor Treatment of anemia due to chronic kidney disease. Complete Response Letter issued
Immune Disorders
Efzofitimod (ATYR1923; aTyr Pharma) Selective modulator of neuropilin-2 Treatment of systemic sclerosis Orphan Drug designation
Immunization
V116 (Merck) 21-valent pneumococcal conjugate vaccine Prevention of invasive pneumococal disease and pneumococcal pneumonia caused by S. pneumoniae. Breakthrough Therapy designation
Infectious Diseases
Actemra (tocilizumab; Genentech) Interleukin-6 receptor antagonist Treatment of COVID-19 in hospitalized adults who are receiving systemic corticosteroids and require supplemental oxygen, noninvasive mechanical ventilation, or extracorporeal membrane oxygenation. sBLA accepted for Priority Review
Eflapegrastim (Spectrum Pharmaceuticals) Long-acting granulocyte colony-stimulating factor To decrease the incidence of infection, as manifested by febrile neutropenia, in patients with nonmyeloid malignancies receiving myelosuppressive anticancer drugs associated with clinically significant incidence of febrile neutropenia. Resubmitted BLA accepted for review
REGEN-COV (casirivimab and imdevimab; Regeneron Pharmaceuticals) Monoclonal antibodies Treatment of COVID-19 in nonhospitalized patients and as prophylaxis in certain individuals. PDUFA target action date has been extended
Metabolic Disorders
Edsivo (celiprolol; Acer Therapeutics) β1-adrenoceptor antagonist with partial β2 agonist activity Treatment of vascular Ehlers-Danlos syndrome in patients with a confirmed type III collagen mutation. Breakthrough Therapy designation
Musculoskeletal Disorders
Pentosan polysulfate sodium (Zilosul; Paradigm Biopharmaceuticals) Semi-synthetically produced heparin-like macromolecular carbohydrate derivative Treatment of osteoarthritis. Fast Track designation
Neurological Disorders
PRX012 (Prothena Corporation) Anti-amyloid beta antibody Treatment of Alzheimer disease. Fast Track designation
PXL770 (Poxel SA) Direct adenosine monophosphate-activated protein kinase activator Treatment of adrenomyeloneuropathy. Fast Track designation
Oncology
ADI-001 (Adicet Bio) Anti-CD20 chimeric antigen receptor (CAR) T cell therapy Treatment of relapsed or refractory B-cell Non-Hodgkin lymphoma. Fast Track designation
Enhertu (fam-trastuzumab deruxtecan-nxki; Daiichi Sankyo) HER2-directed antibody + topoisomerase inhibitor conjugate Treatment of unresectable or metastatic HER2-low breast cancer in patients who have received a prior systemic therapy in the metastatic setting, or developed recurrence during or within 6 months of completing adjuvant chemotherapy. Breakthrough Therapy designation
Enhertu (fam-trastuzumab deruxtecan-nxki; Daiichi Sankyo) HER2-directed antibody + topoisomerase inhibitor conjugate Treatment of adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have a HER2 (ERBB2) mutation and who have received a prior systemic therapy. Priority Review granted
Tremelimumab (AstraZeneca) + Imfinzi (durvalumab; AstraZeneca) CTLA-4 inhibitor + PD-L1 blocking antibody A single priming dose of tremelimumab added to durvalumab to treat patients with unresectable hepatocellular carcinoma. Priority Review granted
TTI-101 (Tvardi Therapeutics) STAT3 inhibitor Treatment of hepatocellular carcinoma. Orphan Drug designation
Psychiatric Disorders
TV-46000/mdc-IRM (risperidone extended-release injectable suspension; Teva and MedinCell) Atypical antipsychotic Treatment of schizophrenia. Complete Response Letter issued
Respiratory Disorders
Colistimethate sodium powder for nebulization solution (CMS I–neb; Zambon S.p.A) Polymyxin antibiotic To reduce the incidence of pulmonary exacerbations in adults with non-cystic fibrosis bronchiectasis colonized with P. aeruginosa. Breakthrough Therapy designation