The table below is a review of notable updates that occurred in September 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 |
| Dermatological Disorders | |||
| Ritlecitinib (Pfizer) | Oral covalent kinase inhibitor with high selectivity for Janus kinase 3 (JAK3) | Treatment of alopecia areata. | NDA accepted for filing |
| Hematological Disorders | |||
| NTLA-2002 (Intellia Therapeutics, Inc.) | CRISPR/Cas9 therapy to inactivate the kallikrein B1 (KLKB1) gene | Treatment of hereditary angioedema. | Orphan Drug designation |
| Immune Disorders | |||
| Leniolisib (Pharming Group N.V.) | Oral, selective phosphoinositide 3-kinase delta inhibitor | Treatment of activated phosphoinositide 3-kinase delta syndrome. | NDA accepted for Priority Review |
| Immunization | |||
| GBS6 or PF-06760805 (hexavalent anti capsular polysaccharide/cross reactive material 197 glycoconjugate; Pfizer) | Group B Streptococcus (GBS) maternal vaccine candidate | Prevention of invasive GBS disease due to the vaccine serotypes in newborns and young infants by active immunization of their mothers during pregnancy. | Breakthrough Therapy designation |
| Pfizer-BioNTech Omicron BA.4/BA.5-adapted bivalent COVID-19 vaccine | COVID-19 vaccine | Booster dose for children 5 to 11 years of age. | Emergency Use Authorization (EUA) requested |
| Infectious Diseases | |||
| Rezafungin (Cidara Therapeutics, Inc.) | Once-weekly echinocandin antifungal | Treatment of candidemia and invasive candidiasis. | NDA accepted for Priority Review |
| Metabolic Disorders | |||
| MZE001 (Maze Therapeutics, Inc.) | Oral, glycogen synthase (GYS1) inhibitor | Treatment of Pompe disease. | Orphan Drug designation |
| Velmanase alfa (Chiesi Global Rare Diseases) | Recombinant form of human alpha-mannosidosis | Treatment of alpha-mannosidosis. | BLA accepted for Priority Review |
| Neurologic Disorders | |||
| NMD670 (NMD Pharma) | Small molecule inhibitor of the skeletal muscle specific chloride ion channel | Treatment of myasthenia gravis. | Orphan Drug designation |
| Trofinetide (Acadia Pharmaceuticals Inc.) | Synthetic analogue of the amino-terminal tripeptide of IGF-1 | Treatment of Rett syndrome. | NDA accepted for Priority Review |
| Oncology | |||
| SY-5609 (Syros Pharmaceuticals, Inc.) | Cyclin-dependent kinase 7 inhibitor | Treatment of pancreatic cancer. | Orphan Drug designation |
| Tukysa (tucatinib; Seagen) in combination with trastuzumab | Tyrosine kinase inhibitor + human epidermal growth factor receptor inhibitor | Treatment of adults with HER2-positive colorectal cancer who have received at least 1 prior treatment regimen for unresectable or metastatic disease. | sNDA accepted for Priority Review |
| Ophthalmic Disorders | |||
| NOV03 (perfluorohexyloctane; Bausch + Lomb Corporation and Novaliq GmbH) | Water- and preservative- free nonsteroidal eye drop | Treatment of the signs and symptoms of dry eye disease associated with Meibomian gland dysfunction. | NDA accepted |
| Pain Management | |||
| SP-103 (lidocaine topical system 5.4%; Scilex Holding Company) | Topical anesthetic | Treatment of acute low back pain. | Fast Track designation |
| Psychiatric Disorders | |||
| REL-1017 (esmethadone; Relmada Therapeutics, Inc.) | N-methyl-D-aspartate receptor channel blocker | As monotherapy for the treatment of major depressive disorder. | Fast Track designation |
