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 |
