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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 |
