The table below is a review of notable updates that occurred in October 2022 for investigational products in development (not an inclusive list). Click on the status link to view our full coverage.

Drug Pharmacologic Class Proposed Indication Status
Allergic Disorders
Neffy (epinephrine nasal spray; Silverback Therapeutics) Sympathomimetic For the emergency treatment of allergic reactions (type 1), including anaphylaxis in adults and children weighing at least 30kg. NDA accepted for review
Cardiovascular Disorders
Camzyos (mavacamten; Bristol Myers Squibb) Selective allosteric inhibitor of cardiac myosin To reduce the need for septal reduction therapy in patients with obstructive hypertrophic cardiomyopathy. sNDA accepted for review
Hematological Disorders
Takhzyro (lanadelumab-flyo; Takeda) Plasma kallikrein inhibitor For prophylaxis to prevent attacks of hereditary angioedema in pediatric patients 2 to less than 12 years of age. sBLA accepted for Priority Review
Valoctocogene roxaparvovec (BioMarin Pharmaceutical) Adeno-associated virus (AAV) gene therapy Treatment of hemophilia A in adults. Resubmitted BLA accepted for review
Infectious Diseases
SER-109 (Seres Therapeutics) Oral microbiome therapeutic containing purified bacterial spores of Firmicute species obtained from the stool of healthy human donors Treatment of recurrent Clostridioides difficile infection. BLA accepted for Priority Review
Kidney Disease
Daprodustat (GlaxoSmithKline) Hypoxia-inducible factor prolyl hydroxylase inhibitor Treatment of patients with anemia of chronic kidney disease. FDA panel voted
Sparsentan (Travere Therapeutics) Dual endothelin angiotensin receptor antagonist Treatment of immunoglobulin A nephropathy. NDA review period extended
Metabolic Disorders
Alvelestat (MPH-966; Mereo BioPharma) Neutrophil elastase inhibitor Treatment of patients with alpha-1 antitrypsin deficiency-associated lung disease. Fast Track designation
Neurologic Disorders
SPN-830 (apomorphine infusion device; Supernus Pharmaceuticals) Non-ergoline dopamine agonist Continuous treatment of motor fluctuations (off episodes) in adults with Parkinson disease. Complete Response Letter issued
Telitacicept (RemeGen Co., Ltd) Recombinant TACI-Fc (transmembrane activator and calcium modulator and cyclophilin ligand interactor) fusion protein Treatment of myasthenia gravis. Orphan Drug designation
Tofersen (Biogen) Antisense medicine to reduce the production of SOD1 protein Treatment of superoxide dismutase 1 amyotrophic lateral sclerosis. NDA review period extended
Oncology
Sapanisertib (Calithera Biosciences) Dual mTORC 1/2 inhibitor Treatment of adults with unresectable or metastatic squamous non-small cell lung cancer (sqNSCLC) whose tumors have a mutation in nuclear factor erythroid 2-related factor (NRF2) and who have received prior platinum-based chemotherapy and immune checkpoint inhibitor therapy. Fast Track designation
Trodelvy (sacituzumab govitecan-hziy; Gilead Sciences) Trop-2-directed antibody and topoisomerase inhibitor conjugate Treatment of adults with unresectable locally advanced or metastatic hormone receptor (HR) positive, human epidermal growth factor receptor 2 (HER2) negative (IHC 0, IHC 1+ or IHC 2+/ISH-) breast cancer who have received endocrine-based therapy and at least 2 additional systemic therapies in the metastatic setting. sBLA accepted for Priority Review
Ophthalmic Disorders
Eylea (aflibercept; Regeneron Pharmaceuticals) Vascular endothelial growth factor inhibitor Treatment of retinopathy of prematurity in preterm infants. sBLA accepted for Priority Review
CyclASol (cyclosporine ophthalmic solution; Novaliq) Anti-inflammatory and selective immunomodulatory drug Treatment of the signs and symptoms of dry eye disease. NDA accepted for review
Pain Management
Basimglurant (NOE-101; Noema Pharma) Metabotropic glutamate receptor 5 inhibitor Management of pain associated with trigeminal neuralgia. Fast Track designation