February 2023 Recap: Drug Pipeline Updates

The table below is a review of notable updates that occurred in February 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
Onpattro (patisiran; Alnylam) Transthyretin-directed small interfering RNA Treatment of the cardiomyopathy of transthyretin-mediated amyloidosis. sNDA accepted for review
Gastrohepatic Disorders
Bylvay (odevixibat; Albireo Pharma) Ileal bile acid transporter inhibitor Treatment of Alagille syndrome. sNDA accepted for Priority Review
Linzess (linaclotide; Ironwood Pharmaceuticals) Guanylate cyclase-C agonist Treatment of pediatric patients 6 to 17 years of age with functional constipation. sNDA accepted for Priority Review
Immune Disorders
Benlysta (belimumab; GSK) B-lymphocyte stimulator-specific inhibitor Treatment of systemic sclerosis. Orphan Drug designation
Pozelimab (Regeneron Pharmaceuticals) Complement factor 5 inhibitor Treatment of patients 1 year of age and older with CHAPLE disease. BLA accepted for Priority Review
RSVpreF (PF-06928316; Pfizer) Bivalent respiratory syncytial virus vaccine For administration to pregnant women to help protect against the complications of RSV disease in infants from birth through 6 months. BLA accepted for Priority Review
Infectious Diseases
Prevymis (letermovir; Merck) CMV DNA terminase complex inhibitor Prophylaxis of cytomegalovirus disease in adult kidney transplant recipients at high risk (D+/R-). Also, extend use of letermovir from 100 to 200 days in adults receiving an allogeneic HSCT who are at risk for late CMV infection and disease. sBLAs accepted for review
Musculoskeletal Disorders
Taldefgrobep alfa (Biohaven) Fully human anti-myostatin recombinant protein. Treatment of spinal muscular atrophy. Fast Track designation
Neurologic Disorders
Ketarx (racemic ketamine; PharmaTher Holdings) NMDA receptor antagonist Treatment of Rett syndrome. Orphan Drug designation
Avasopasem manganese (GC4419; Galera Therapeutics) Selective dismutase mimetic To reduce the incidence of radiotherapy-induced severe oral mucositis in patients with head and neck cancer undergoing standard of care treatment. NDA accepted for Priority Review
Elranatamab (Pfizer) B-cell maturation antigen CD3-targeted bispecific antibody Treatment of patients with relapsed or refractory multiple myeloma. BLA accepted for Priority Review
mRNA-4157/V940 (Moderna and Merck) Personalized mRNA cancer vaccine In combination with Keytruda (pembrolizumab) for the adjuvant treatment of patients with high-risk melanoma following complete resection. Breakthrough Therapy designation
Nirogacestat (SpringWorks Therapeutics) Gamma secretase inhibitor Treatment of adults with desmoid tumors. NDA accepted for Priority Review
Opdivo (nivolumab; Bristol Myers Squibb) Programmed death receptor-1 blocking antibody As monotherapy in the adjuvant setting for the treatment of patients with completely resected stage IIB or IIC melanoma. sBLA accepted for review
TTX-MC138 (TransCode Therapeutics) MicroRNA-10b inhibitor Treatment of pancreatic cancer. Orphan Drug designation
Ophthalmic Disorders
Aflibercept 8mg (Regeneron Pharmaceuticals) Vascular endothelial growth factor inhibitor Treatment of patients with wet age-related macular degeneration, diabetic macular edema, and diabetic retinopathy. BLA accepted for Priority Review
Avacincaptad pegol (Iveric Bio) Complement C5 protein inhibitor Treatment of geographic atrophy secondary to AMD. NDA accepted for Priority Review
CSF-1 (pilocarpine hydrochloride; Orasis Pharmaceuticals) Muscarinic cholinergic agonist Treatment of presbyopia. NDA accepted for review
EA-2353 (Endogena Therapeutics) Selectively activates endogenous retinal stem and progenitor cells Treatment of retinitis pigmentosa. Fast Track designation
Nyxol (phentolamine ophthalmic solution 0.75%; Ocuphire Pharma) Nonselective alpha-1 and alpha-2 adrenergic antagonist Reversal of pharmacologically-induced mydriasis. NDA accepted for review
Reproxalap (Aldeyra Therapeutics) Covalently binds to free aldehydes and diminishes excessive RASP levels Treatment of the signs and symptoms of dry eye disease. NDA accepted for review
Poisoning and Drug Dependence
Narcan nasal spray (naloxone HCl; Emergent BioSolutions) Opioid antagonist For over-the-counter use in the emergency treatment of known or suspected opioid overdose. FDA Committees vote
Psychiatric Disorders
Zuranolone (Sage Therapeutics and Biogen) Oral neuroactive steroid GABA-A receptor positive allosteric modulator Treatment of major depressive disorder and postpartum depression. NDA accepted for Priority Review