April 2022: Drug Pipeline Updates?

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

DrugPharmacologic ClassProposed IndicationStatusGastrohepatic DisordersDupixent (dupilumab; Regeneron Pharmaceuticals)Interleukin-4 receptor alpha antagonistTreatment of eosinphilic esophagitis in patients 12 years of age and older.sBLA accepted for Priority ReviewREC-4881 (Recursion)Non-ATP-competitive allosteric small molecule inhibitor of MEK1 and MEK2Treatment of familial adenomatous polyposis in patients who have previously undergone a colectomy or protocolectomy.Fast Track designationHematological DisordersDaprodustatHypoxia-inducible factor prolyl hypdroxylase inhibitorTreatment of anemia due to chronic kidney disease.NDA accepted for reviewImmune DisordersEfzofitimod (ATYR1923; aTyr Pharma)Selective modulator of neuropilin-2Treatment of systemic sclerosisOrphan Drug designationImmunizationV116 (Merck)21-valent pneumococcal conjugate vaccinePrevention of invasive pneumococal disease and pneumococcal pneumonia caused by S. pneumoniae.Breakthrough Therapy designationInfectious DiseaseActemera (tocilizumab; Genentech)Interleukin-6 receptor antagonistTreatment 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 ReviewEflapegrastim (Spectrum Pharmaceuticals)Long-acting granulocyte colony-stimulating factorTo 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 reviewREGEN-COV® (casirivimab and imdevimab; Regeneron Pharmaceuticals) Monoclonal antibodiesTreatment of COVID-19 in nonhospitalized patients and as prophylaxis in certain individuals.PDUFA target action date has been extendedMetabolic DisordersEdsivo (celiprolol; Acer Therapeuatics)?1-adrenoceptor antagonist with partial ?2 agonist activityTreatment of vascular Ehlers-Danlos syndrome in patients with a confirmed type III collagen mutation.Breakthrough Therapy designationMusculoskeletal DisordersPentosan polysulfate sodium (Zilosul; Paradigm Biopharmaceuticals)Semi-synthetically produced heparin-like macromolecular carbohydrate derivativeTreatment of osteoarthritis.Fast Track designationNeurological DisordersPRX012 (Prothena Corporation)Anti-amyloid beta antibodyTreatment of Alzheimer disease.Fast Track designationPXL770 (Poxel SA)Direct adenosine monophosphate-activated protein kinase activatorTreatment of adrenomyeloneuropathy.Fast Track designationOncologyADI-001 (Adicet Bio)Anti-CD20 chimeric antigen receptor (CAR) T cell therapyTreatment of relapsed or refractory B-cell Non-Hodgkin lymphoma.Fast Track designationEnhertu (Fam-trastuzumab deruxtecan-nxki; Daiichi Sankyo)HER2-directed antibody + topoisomerase inhibitor conjugateTreatment 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 designationEnhertu (Fam-trastuzumab deruxtecan-nxki; Daiichi Sankyo)HER2-directed antibody + topoisomerase inhibitor conjugateTreatment 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 grantedTremelimumab (AstraZeneca) + Imfinzi (durvalumab; AstraZeneca)CTLA-4 inhibitor + PD-L1 blocking antibodyA single priming dose of tremelimumab added to durvalumab to treat patients with unresectable hepatocellular carcinoma.Priority Review grantedTTI-101 (Tvardi Therapeutics)STAT3 inhibitorTreatment of hepatocellular carcinoma.Orphan Drug designationPsychiatric DisordersTV-46000/mdc-IRM (risperidone extended-release injectable suspension; Teva and MedinCell)Atypical antipsychoticTreatment of schizophrenia.Complete Response Letter issuedRespiratory DisordersColistimethate sodium powder for nebulization solution (CMS I–neb®; Zambon S.p.A)Polymyxin antibioticTo reduce the incidence of pulmonary exacerbations in adults with non-cystic fibrosis bronchiectasis colonized with P. aeruginosa.Complete Response Letter issued