January 2023 Recap: Drug Pipeline Updates

The table below is a review of notable updates that occurred in January 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
Olezarsen (Ionis Pharmaceuticals, Inc.) ApoC-III inhibitor Treatment of familial chylomicronemia syndrome. Fast Track designation
Tecarfarin (Cadrenal Therapeutics) Vitamin K antagonist Prevention of systemic thromboembolism of cardiac origin in patients with end-stage renal disease and atrial fibrillation. Fast Track designation
Gastrohepatic Disorders
Obeticholic acid (Intercept Pharmaceuticals) Farnesoid X receptor agonist Treatment of patients with pre-cirrhotic liver fibrosis due to nonalcoholic steatohepatitis. Resubmitted NDA accepted for review
Vonoprazan (Phathom Pharmaceuticals) Oral small molecule potassium-competitive acid blocker Treatment of erosive esophagitis. FDA’s decision delayed for NDA
Hematological Disorders
Avapritinib (Blueprint Medicines) Tyrosine kinase inhibitor Treatment of indolent systemic mastocytosis in adults. sNDA accepted for Priority Review
Prevnar 20 (Pfizer) 20-valent pneumococcal conjugate vaccine Prevention of invasive pneumococcal disease in infants and children 6 weeks through 17 years of age, and prevention of otitis media caused by 7 of the 20 serotypes. sBLA accepted for Priority Review
Musculoskeletal Disorders
AOC 1020 (Avidity Biosciences) Monoclonal antibody that binds to the transferrin receptor 1 conjugated with siRNA targeting DUX4 mRNA. Treatment of facioscapulohumeral muscular dystrophy. Fast Track designation
Vamorolone (Santhera and ReveraGen) Dissociative steroidal drug Treatment of Duchenne muscular dystrophy. NDA accepted for filing
Neurologic Disorders
Donanemab (Eli Lily and Company) Antibody therapy that targets a modified form of deposited amyloid-beta peptide called N3pG Treatment of patients with early symptomatic Alzheimer disease. Complete Response Letter issued
Rozanolixizumab (UCB) Humanized monoclonal antibody that binds with high affinity to human neonatal Fc receptor Treatment of adults with generalized myasthenia gravis who are anti-acetylcholine receptor or anti-muscle-specific tyrosine kinase antibody positive. BLA accepted for Priority Review
Poisoning and Drug Dependence
OPNT003 (nasal nalmefene; Opiant Pharmaceuticals) Opioid antagonist Treatment of opioid overdose. NDA accepted for Priority Review
Psychiatric Disorders
Gepirone HCI extended-release (Exxua; Fabre-Kramer Pharmaceuticals) Serotonin 1A receptor agonist Treatment of major depressive disorder. Resubmitted NDA accepted for review
Brexpiprazole (Otsuka and Lundbeck) Atypical antipsychotic Treatment of agitation associated with Alzheimer dementia. sNDA accepted for Priority Review
Urological Disorders
Empagliflozin (Eli Lily and Company) Sodium-glucose co-transporter 2 inhibitor To reduce the risk of kidney disease progression and cardiovascular death in adults with chronic kidney disease. sNDA accepted for review