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The table below is a review of notable updates that occurred in February 2024 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 | |||
| Acoramidis (BridgeBio Pharma) | Small molecule designed to stabilize tetrameric transthyretin | Treatment of patients with transthyretin amyloid cardiomyopathy. | NDA accepted |
| CardiolRx (Cardiol Therapeutics) | Cannabidiol | Treatment of recurrent pericarditis. | Orphan Drug designation |
| Dermatologic Disorders | |||
| Nemolizumab (Galderma) | Humanized monoclonal antibody that antagonizes IL-31 receptor alpha | Treatment of prurigo nodularis and moderate to severe atopic dermatitis. | BLAs accepted for review |
| Endocrine Disorders | |||
| Diamyd (Diamyd Medical) | Antigen-specific immunotherapy | To improve glycemic control in recently diagnosed stage 3 type 1 diabetes patients with the genotype HLA DR3-DQ2. | Fast Track designation |
| Gastrohepatic Disorders | |||
| Apraglutide (Ironwood Pharmaceuticals) | Long-acting synthetic glucagon-like peptide-2 analog | To reduce parenteral support dependency in patients with short bowel syndrome with intestinal failure. | Phase 3 trial results |
| Seladelpar (CymaBay Therapeutics) | Peroxisome proliferator-activated receptor delta agonist | For the management of primary biliary cholangitis including pruritus in adults without cirrhosis or with compensated cirrhosis (Child Pugh A) who are inadequate responders or intolerant to ursodeoxycholic acid. | NDA accepted |
| Hematological Disorders | |||
| DISC-0974 (Disc Medicine) | Anti-hemojuvelin monoclonal antibody | Treatment of anemia in patients with nondialysis dependent chronic kidney disease. | Fast Track designation |
| Ruxoprubart (NovelMed) | Anti-Bb humanized antibody | Treatment of paroxysmal nocturnal hemoglobinuria. | Orphan Drug designation |
| Immune Disorders | |||
| Axatilimab (Incyte) | Monoclonal antibody that targets colony stimulating factor-1 receptor | Treatment of graft-vs-host-disease after failure of at least 2 prior lines of systemic therapy. | BLA accepted for Priority Review |
| Efgartigimod alfa plus hyaluronidase-qvfc (argenx SE) | Neonatal Fc receptor blocker + endoglycosidase | Treatment of chronic inflammatory demyelinating polyneuropathy. | sBLA accepted for Priority Review |
| Immunization | |||
| Arexvy (GlaxoSmithKline) | Respiratory syncytial virus vaccine, adjuvanted | For the prevention of respiratory syncytial virus disease in adults aged 50 to 59 years who are at increased risk for RSV disease. | sBLA accepted for Priority Review |
| Infectious Diseases | |||
| Gepotidacin (GlaxoSmithKline) | Triazaacenaphthylene antibiotic | Treatment of urogenital gonorrhea. | Phase 3 trial results |
| Kidney Disease | |||
| Sibeprenlimab (Otsuka and Visterra) | Humanized IgG2 monoclonal antibody that binds to and neutralizes a proliferation-inducing ligand | Treatment of immunoglobulin A nephropathy. | Breakthrough Therapy designation |
| Metabolic Disorders | |||
| Govorestat (Applied Therapeutics) | Aldose reductase inhibitor | Treatment of sorbitol dehydrogenase deficiency. | Phase 3 trial results |
| Pitolisant (Harmony Biosciences) | Histamine-3 receptor antagonist/inverse agonist | Treatment of Prader-Willi syndrome. | Orphan Drug designation |
| Musculoskeletal Disorders | |||
| CBL-514 (Caliway Biopharmaceuticals) | Small molecule drug designed to induce adipocyte apoptosis and lipolysis | Treatment of Dercum disease. | Fast Track designation |
| PGN-EDODM1 (PepGen) | Peptide-conjugated antisense oligonucleotide | Treatment of myotonic dystrophy type 1. | Fast Track designation |
| Neurologic Disorders | |||
| Deudextromethorphan hydrobromide and quinidine sulfate (Otsuka Pharmaceutical) | Uncompetitive NMDA receptor antagonist and sigma-1 agonist plus CYP2D6 inhibitor | Treatment of agitation associated with dementia due to Alzheimer disease. | Phase 3 trial results |
| Latozinemab (Alector) | Human monoclonal antibody designed to modulate progranulin | Treatment of frontotemporal dementia due to a progranulin gene mutation. | Breakthrough Therapy designation |
| Obstetrics and Gynecology | |||
| Nipocalimab (Johnson & Johnson) | Anti-FcRn, aglycosylated IgG1 monoclonal antibody | Treatment of alloimmunized pregnant individuals at high risk for severe hemolytic disease of the fetus and newborn. | Breakthrough Therapy designation |
| Oncology | |||
| Adagrasib in combination with cetuximab (Bristol Myers Squibb) | KRASG12C inhibitor |
In combination with cetuximab, for the treatment of patients with previously treated KRASG12C-mutated locally advanced or metastatic colorectal cancer.
|
NDA accepted for Priority Review |
| Afamitresgene autoleucel (Adaptimmune Therapeutics) | Autologous T-cell therapy | Treatment of advanced synovial sarcoma. | BLA accepted for Priority Review |
| Datopotamab deruxtecan (AstraZeneca and Daiichi Sankyo) |
TROP2-directed DXd antibody drug conjugate
|
Treatment of adult patients with locally advanced or metastatic nonsquamous non-small cell lung cancer who have received prior systemic therapy.
|
BLA accepted |
| Linvoseltamab (Regeneron) | CD3-targeted bispecific antibody | Treatment of adults with relapsed/refractory multiple myeloma. | BLA accepted for Priority Review |
| Repotrectinib (Bristol Myers Squibb) |
Kinase inhibitor
|
Treatment of adult and pediatric patients 12 years of age and older with solid tumors that have a neurotrophic tyrosine receptor kinase gene fusion, and are locally advanced or metastatic or where surgical resection is likely to result in severe morbidity. | sNDA accepted for Priority Review |
| Vepdegestrant (Arvinas and Pfizer) | Proteolysis targeting chimera ER degrader | Treatment of adults with estrogen receptor positive/human growth epidermal growth factor 2 negative locally advanced or metastatic breast cancer previously treated with endocrine-based therapy. | Fast Track designation |
| Vorasidenib (Servier) | Isocitrate dehydrogenase 1 and 2 inhibitor | Treatment of isocitrate dehydrogenase-mutant diffuse glioma. | NDA accepted for Priority Review |
| Psychiatric Disorders | |||
| Midomafetamine (Alector) | Psychoactive drug | In combination with psychological intervention for the treatment of post-traumatic stress disorder. | NDA accepted for Priority Review |
| Roluperidone (Minerva Neurosciences) |
Antipsychotic
|
Treatment of negative symptoms in patients with schizophrenia. | Complete Response Letter issued |
| Respiratory Disorders | |||
| Dupilumab (Sanofi) | Interleukin-4 receptor alpha antagonist | For maintenance treatment in certain adult patients with uncontrolled chronic obstructive pulmonary disease. | sBLA accepted for Priority Review |
