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The table below is a review of notable updates that occurred in March 2023 for investigational products in development (not an inclusive list). Click on the status to view our full coverage.
| Drug | Pharmacologic Class | Proposed Indication | Status |
| Cardiovascular Disease | |||
| Omecamtiv mecarbil (Cytokinetics) | Selective small molecule cardiac myosin activator | Treatment of heart failure with reduced ejection failure. | Complete Response Letter issued |
| Dermatological Disorders | |||
| Berdazimer gel (SB206; Novan) | Nitric oxide-releasing topical antiviral gel | Treatment of molluscum contagiosum. | NDA accepted for filing |
| Dupilumab (Dupixent; Regeneron Pharmaceuticals, Inc) | Interleukin-4 receptor alpha antagonist | Treatment of patients 12 years of age and older with chronic spontaneous urticaria that is inadequately controlled with standard-of-care H1 antihistamines. | sBLA accepted for review |
| Endocrine Disorders | |||
| Empagliflozin (Jardiance; Eli Lilly) | SGLT2 inhibitor | As an adjunct to diet and exercise to improve glycemic control in patients 10 years of age and older with type 2 diabetes. | sNDA accepted for review |
| Hematological Disorders | |||
| Valoctocogene roxaparvovec (BioMarin) | Adeno-associated virus gene therapy | Treatment of hemophilia A in adults. | FDA review period extended for BLA |
| Immunization | |||
| RSVPreF3 (GlaxoSmithKline) | RSV vaccine candidate | For active immunization to prevent RSV-associated lower respiratory tract disease in adults 60 years of age and older. | FDA panel voted |
| Metabolic Disorders | |||
| Eplontersen (Ionis Pharmaceutics) | Ligand-conjugated antisense medicine | Treatment of hereditary transthyretin-mediated amyloid polyneuropathy. | NDA accepted for review |
| Zagociguat (Cyclerion Therapeutics) | Small molecule activator of soluble guanylate cyclase | Treatment of mitochondrial disease. | Orphan Drug designation |
| Musculoskeletal Disorders | |||
| Voxzogo (vosoritide; BioMarin) | Analogue of C-type natriuretic peptide | Treatment of children less than 5 years of age with achondroplasia. | sNDA accepted for review |
| Neurologic Disorders | |||
| ABBV-951 (foscarbidopa/ foslevodopa; AbbVie) | Dopa-decarboxylase inhibitor + dopamine precursor | Treatment of motor fluctuations in adults with advanced Parkinson disease. | Complete Response Letter issued |
| Lecanemab-irmb (Leqembi; Eisai) | Humanized immunoglobulin gamma 1 monoclonal antibody | Conversion of accelerated approval status to a traditional approval for the treatment of Alzheimer disease. | sBLA accepted for Priority Review |
| Solanezumab (Eli Lilly) | Humanized monoclonal antibody | Treatment of patients with Alzheimer disease. | Clinical development discontinued |
| Tofersen (Biogen) | Antisense medicine to reduce synthesis of SOD1 protein production | Treatment of patients with superoxide dismutase 1 amyotrophic lateral sclerosis. | FDA panel voted |
| Ganaxolone (Ztalmy; Marinus Pharmaceuticals) | Neuroactive steroid GABA A receptor positive modulator | Treatment of Lennox-Gastaut syndrome. | Orphan Drug designation |
| Ob/Gyn | |||
| Opill (norgestrel; Perrigo Company plc) | Progestin-only daily birth control pill | For the prevention of pregnancy. | FDA panel to review the application for Rx to OTC switch |
| Oncology | |||
| ADX-2191 (methotrexate injection; Aldeyra Therapeutics, Inc.) | Intravitreal formulation of methotrexate | Treatment of primary vitreoretinal lymphoma. | NDA accepted for Priority Review |
| FORE8394 (FORE Biotherapeutics) | Small molecule, next-generation, orally available selective inhibitor of mutated BRAF | Treatment of primary brain and CNS malignancies. | Orphan Drug designation |
| Osemitamab (TST001; Transcenta Holding Limited) | High affinity, humanized ADCC-enhanced anti-Claudin18.2 monoclonal antibody | Treatment of patients with pancreatic cancer. | Orphan Drug designation |
| PBP1510 (ulenistamab; Prestige Biopharma) | Targets pancreatic adenocarcinoma upregulated factor | Treatment of unresectable or metastatic pancreatic adenocarcinoma that has relapsed following and/or is refractory to at least 1 line of prior therapy. | Fast Track designation |
| RRx-001 (EpicentRx) | Small molecule inflammasome NLRP3 inhibitor and Nrf2 upregulator | For the prevention/attenuation of severe oral mucositis in chemotherapy and radiation-treated head and neck cancer patients. | Fast Track designation |
| Ruxolitinib extended-release tablets (Incyte) | Janus kinase (JAK)1/2 inhibitor | For once-daily use in the treatment of certain types of myelofibrosis, polycythemia vera, and graft-vs-host disease. | Complete Response Letter issued |
| Niraparib (Zejula; GlaxoSmithKline) plus abiraterone acetate | Poly (ADP-ribose) polymerase inhibitor + CYP17 inhibitor | Treatment of patients with BRCA-positive metastatic castration-resistant prostate cancer. | NDA submitted |
| Pain Management | |||
| Bupivacaine liposome injectable suspension (Exparel; Pacira BioSciences, Inc.) | Local anesthetic (amide-type) | As a single-dose sciatic nerve block in the popliteal fossa and as a single-dose femoral nerve block in the adductor canal. | sNDA accepted for review |
| Respiratory Disorders | |||
| ARINA-1 (ascorbic acid and glutathione; Renovion, Inc.) | At-home nebulized therapy | For the prevention of bronchiolitis obliterans syndrome progression in adults with a bilateral lung transplant. | Fast Track designation |
