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 |
