March 2023 Recap: Drug Pipeline Updates

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
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
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
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