The table below is a review of notable updates that occurred in August 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
Cardiovascular Disease
Lomecel-B (Longeveron Inc.) Allogenic, bone marrow-derived medicinal signaling cell product Treatment of hypoplastic left heart syndrome in infants. Fast Track designation
MK-2060 (Merck) Factor XI inhibitor To reduce the risk of major thrombotic cardiovascular events in patients with end-stage renal disease. Fast Track designation
PF-07265803 (Pfizer) Small molecule inhibitor of the p38a mitogen activated protein kinase pathway Treatment of patients with symptomatic dilated cardiomyopathy due to a mutation of the gene encoding the lamin A/C protein. Clinical development discontinued
Dermatological Disorders
Beremagene geperpavec (B-VEC; Krystal Biotech, Inc.) Topical gene therapy designed to deliver 2 copies of the COL7A1 gene Treatment of dystrophic epidermolysis bullosa. BLA accepted for Priority Review
NexoBrid (MediWound Ltd.) Topically administered, bromelain-based biological product containing a sterile mixture of proteolytic enzymes For eschar removal (debridement) in adults with deep partial thickness and/or full thickness thermal burns. Resubmitted BLA accepted for review
Hematological Disorders
Efanesoctocog alfa (BIVV001; Sanofi – Aventis Groupe) Recombinant factor VIII therapy Treatment of hemophilia A. BLA accepted for Priority Review
Immune Disorders
TRE-515 (Trethera Corporation) Small molecule inhibitor of deoxycytidine kinase Treatment of acute disseminated encephalomyelitis (ADEM). Treatment of demyelinating optic neuritis. Orphan Drug designation for ADEM Orphan Drug designation for optic neuritis
Immunization
Novavax COVID-19 Vaccine, Adjuvanted (Novavax) Protein-based COVID-19 vaccine As a homologous and heterologous booster to prevent COVID-19 caused by SARS-CoV-2 in adults. EUA request submitted
VAX-24 (Vaxcyte, Inc.) 24-valent pneumococcal conjugate vaccine Prevention of invasive pneumococcal disease in adults. Fast Track designation
Neurologic Disorders
Pimavanserin (Acadia Pharmaceuticals Inc.) Atypical antipsychotic Treatment of hallucinations and delusions associated with Alzheimer disease psychosis. Complete Response Letter issued
Ob/Gyn
Brexafemme (ibrexafungerp; Scynexis, Inc.) Triterpenoid antifungal Prevention of recurrent vulvovaginal candidiasis. sNDA accepted for Priority Review
Fezolinetant (Astellas Pharma Inc.) Selective neurokinin-3 receptor antagonist Treatment of moderate to severe vasomotor symptoms associated with menopause. NDA accepted
Oncology
Momelotinib (GlaxoSmithKline) Inhibitor of JAK1, JAK2, and ACVR1 signaling pathways Treatment of myelofibrosis. NDA accepted
Lynparza (olaparib; AstraZeneca) + abiraterone and prednisone or prednisolone PARP inhibitor + CYP17 inhibitor + glucocorticoid Treatment of metastatic castration-resistant prostate cancer. sNDA accepted for Priority Review
Omidubicel (Gamida Cell Ltd.) Advanced nicotinamide-enabled stem cell therapy candidate Treatment of patients with hematologic malignancies in need of an allogeneic hematopoietic stem cell transplant. BLA accepted for Priority Review
Polivy (polatuzumab vedotin-piiq; Genentech) plus rituximab, cyclophosphamide, doxorubicin, and prednisone CD79b-directed antibody-drug conjugate + CD20-directed cytolytic monoclonal antibody, alkylating agent, anthracycline, glucocorticoid Treatment of adults with previously untreated diffuse large B-cell lymphoma. sNDA accepted for review
Trodelvy (sacituzumab govitecan-hziy; Gilead Sciences, Inc.) Trop-2-directed antibody and topoisomerase inhibitor conjugate Treatment of patients with HR+/HER2- metastatic breast cancer who received prior endocrine therapy, CDK4/6 inhibitors and 2 to 4 lines of chemotherapy. sBLA submitted
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
Respiratory Disorders
Efzofitimod (ATYR1923; aTyr Pharma, Inc.) Selective modulator of neuropilin-2 Treatment of pulmonary sarcoidosis. Fast Track designation
Urological Disorders
OCE-205 (Ocelot Bio, Inc.) Mixed agonist-antagonist peptide therapeutic targeting the vasopressin 1a receptor Treatment of hepatorenal syndrome. Orphan Drug designation