May 2022: Drug Pipeline Updates

The table below is a review of notable updates that occurred in May 2022 for investigational products in development (not an inclusive list).

 

DrugPharmacologic ClassProposed IndicationStatusCardiovascular DiseaseFuroscix® (furosemide; scPharmaceuticals Inc.)pH-neutral formulation of furosemide delivered via wearable, pre-programmed on-body drug delivery systemTreatment of congestion due to fluid overload in adults with NYHA Class II and Class III chronic heart failure who display reduced responsiveness to oral diuretics and who do not require hospitalization.NDA accepted for filingDermatological DisordersBimekizumab (UCB)Humanized monoclonal lgG1 antibody that inhibits IL-17A and IL-17FTreatment of adults with moderate to severe plaque psoriasis.Complete Response Letter issuedDupilumab (Regeneron Pharmaceuticals, Inc.)Interleukin-4 receptor alpha antagonistTreatment of adults with prurigo nodularis.Priority Review grantedGastrohepatic DisordersErvogastat/clesacostat (Pfizer Inc.)Diaclyglycerol O-acyltransferase 2 inhibitor and acetyl-CoA carboxylase inhibitorTreatment of nonalcoholic steatohepatitis (NASH) with liver fibrosis.Fast Track designationVonoprazan (Phathom Pharmaceuticals)Oral small molecule potassium-competitive acid blockerTreatment for the healing of all grades of erosive esophagitis and relief of heartburn, and maintenance of healing of all grades of erosive esophagitis and relief of heartburn.NDA accepted for reviewHematological DisordersEtranacogene dezaparvovec (CSL Behring)AAV gene therapyTreatment of adults with hemophilia B.Priority Review grantedGPH101 (Graphite Bio, Inc.)Gene-edited autologous hematopoietic stem cell therapy (HSCT)Treatment of sickle cell disease.Fast Track designationImmunizationBLB-201 (Blue Lake Biotechnology)Respiratory syncytial virus (RSV) vaccinePrevention of RSV associated disease in adults aged over 60 and children aged under 2 years.Fast Track designationInfectious DiseasesFluvoxamine maleate (various generic manufacturers)SSRIOutpatient treatment of COVID-19 in adults to prevent progression to severe disease and/or hospitalization.EUA request declinedKidney DiseaseSparsentan (Travere Therapeutics, Inc.)Dual endothelin angiotensin receptor antagonistTreatment of immunoglobulin A (lgA) nephropathy.Priority Review grantedNeurologic DisordersABBV-951 (foscarbidopa/foslevodopa; AbbVie)Dopa-decarboxylase inhibitor + dopamine precursorTreatment of motor fluctuations in patients with advanced Parkinson disease.NDA submittedOmaveloxolone (Reata Pharmaceuticals)Nrf2 activatorTreatment of Friedreich ataxia.Priority Review grantedUB-311 (Vaxxinity)Immunotherapeutic vaccine candidate that targets toxic forms of aggregated amyloid beta in the brainTreatment of Alzheimer disease.Fast Track designationOb/GynMyfembree (relugolix, estradiol, and norethindrone acetate; Myovant Sciences)GnRH antagonist + estrogen + progestinManagement of moderate to severe pain associated with endometriosis.PDUFA target action date has been extendedOncologyImfinzi (durvalumab; AstraZeneca)PD-L1 blocking antibodyIn combination with standard of care chemotherapy for treatment of patients with locally advanced or metastatic biliary tract cancer.Priority Review designationMirvetuximab soravtansine (ImmunoGen)Antibody-drug conjugate comprised of a FR?-binding antibody and a tubulin-targeting agent (maytansinoid payload DM4)Treatment of patients with folate receptor alpha (FR?)-high platinum-resistant ovarian cancer who have been previously treated with 1 to 3 prior systemic treatments.Priority Review grantedNubeqa (darolutamide; Bayer)Androgen receptor inhibitorIn combination with docetaxel for the treatment of patients with metastatic hormone-sensitive prostate cancer.Priority Review grantedTasquinimod (Active Biotech AB)Small molecule immune modulatorTreatment of myelofibrosis.Orphan Drug designationPain ManagementAXS-07 (meloxicam/rizatriptan; Axsome Therapeutics)NSAID (COX-2 inhibitor) + selective 5-HT1B/1D receptor agonistAcute treatment of migraine.Complete Response Letter issuedZavegepant (Biohaven)Calcitonin gene-related peptide (CGRP) receptor antagonistAcute treatment of migraine.NDA accepted for reviewRespiratory DisordersPT027 (albuterol/budesonide; Avillion LLP) Short-acting beta2-agonist + inhaled corticosteroidAs-needed treatment or prevention of bronchoconstriction and for the prevention of exacerbations in patients with asthma 4 years of age and older.NDA accepted for filingTT-101 (Tvardi Therapeutics) Small molecule STAT3 (signal transducer and activator of transcription 3) inhibitorTreatment of idiopathic pulmonary fibrosis.Orphan Drug designation