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
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
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