The table below is a review of notable updates that occurred in November 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
Endocrine Disorders
Evinacumab-dgnb (Evkeeza; Regeneron Pharmaceuticals, Inc.) Angiopoietin-like 3 inhibitor As an adjunct to other lipid-lowering therapies to treat children aged 5 to 11 years with homozygous familial hypercholesterolemia. sBLA accepted for Priority Review
TransCon PTH (palopegteriparatide; Ascendis Pharma) Once-daily long-acting prodrug of parathyroid hormone Hormone replacement therapy in adult patients with hypoparathyroidism. NDA accepted for Priority Review
Gastrohepatic Disorders
AMT-101 (Applied Molecular Transport) Novel, GI-selective, oral recombinant biologic fusion protein of human IL-10 Treatment of pouchitis that develops following ileal pouch-anal anastomosis surgery in patients with ulcerative colitis. Orphan Drug designation
LB1148 (Palisade Bio) Broad-spectrum serine protease inhibitor For the acceleration of time to return of bowel function following surgery. Fast Track designation
Musculoskeletal Disorders
SRP-9001 (delandistrogene moxeparvovec; Sarepta Therapeutics, Inc.) Investigational gene transfer therapy delivering micro-dystrophin-encoding gene Treatment of Duchenne muscular dystrophy. BLA accepted for Priority Review
Neurologic Disorders
IPX203 (carbidopa/ levodopa ER capsules; Amneal Pharmaceuticals) Combination of carbidopa, an aromatic amino acid decarboxylation inhibitor, and levodopa, an aromatic amino acid Treatment of Parkinson disease. NDA accepted
Subcutaneous formulation of Vyvgart (efgartigimod; Argenx SE) Neonatal Fc receptor blocker Treatment of generalized myasthenia gravis in adult patients. BLA accepted for Priority Review
Zilucoplan (UCB, Inc.) Macrocyclic peptide inhibitor of complement component 5 Treatment of generalized myasthenia gravis in adult patients who are acetylcholine receptor antibody positive. NDA accepted
Oncology
Epcoritamab (Genmab A/S) IgG1-bispecific antibody designed to bind to CD3 on T cells and CD20 on B cells Treatment of relapsed/refractory large B-cell lymphoma. BLA accepted for Priority Review
Omburtamab (Y-mAbs Therapeutics, Inc.) Radiolabeled monoclonal antibody to target tumor cells expressing B7-H3 Treatment of CNS/leptomeningeal metastasis from neuroblastoma. FDA panel votes; Complete Response Letter issued
Poziotinib (Spectrum Pharmaceuticals) Tyrosine kinase inhibitor Treatment of patients with previously treated locally advanced or metastatic non-small cell lung cancer with HER2 exon 20 insertion mutations. Complete Response Letter issued
Psychiatric Disorders
KP1077 (serdexmethylphenidate; KemPharm, Inc.) CNS stimulant, a prodrug of dexmethylphenidate Treatment of idiopathic hypersomnia. Orphan Drug designation
Respiratory Disorders
PT027 (albuterol/ budesonide inhaler; AstraZeneca)  Inhaled, fixed-dose combination of albuterol, a short-acting beta2-agonist, and budesonide, an inhaled corticosteroid As-needed treatment or prevention of bronchoconstriction and for the prevention of exacerbations in patients 18 years of age and older with asthma. FDA panel voted
Urological Disorders
Tenapanor (Xphozah; Ardelyx) First-in-class oral sodium/hydrogen exchanger 3 (NHE3) inhibitor As monotherapy for the control of serum phosphorus in adults with chronic kidney disease on dialysis, and as combination therapy with phosphate binders. FDA panel voted