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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 |
| Immunization | |||
| 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 |
| Oncology | |||
| 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 |
