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| Manufacturer ▼ | Drug Name | Indication | Stage | Status | Route of Administration | Drug Class |
|---|---|---|---|---|---|---|
| Avidity Biosciences Inc. | Delpacibart braxlosiran (del-brax) - (FORTITUDE) | Facioscapulohumeral Muscular Dystrophy (FSHD) | BLA Filing | Data Released | Subcutaneous | Genetic Disorder |
| Avidity Biosciences Inc. | Delpacibart etedesiran (del-desiran) - (MARINA-OLE) | Myotonic dystrophy type 1 (DM1) | NDA Filing | Ongoing | Intravenous | Genetic Disorder |
| Avidity Biosciences Inc. | del-desiran (AOC 1001) - (HARBOR) | Myotonic dystrophy type 1 (DM1) | Phase 3 | Hold Removed | Intravenous | Genetic Disorder |
| Avidity Biosciences Inc. | del-desiran (AOC 1001) - (HARBOR) | Myotonic dystrophy type 1 (DM1) | Phase 3 | Hold Removed | Intravenous | Genetic Disorder |
| Avidity Biosciences Inc. | Delpacibart Etedesiran (del-desiran) - (HARBOR) | Myotonic dystrophy type 1 (DM1) | Phase 3 | Enrollment Conclusion | Intravenous | Genetic Disorder |
| Axcella Health Inc. | AXA1665 - (EMMPOWER) | Hepatic encephalopathy | Phase 2 | Oral | Gastroenterology | |
| Axcella Health Inc. | AXA1125 - (EMMPACT) | Non-alcoholic steatohepatitis (NASH) / non-alcoholic fatty liver disease (NAFLD) | Phase 2b | Oral | Gastroenterology | |
| Axsome Therapeutics Inc. | AXS-05 (dextromethorphan HBr and bupropion HCl) - (ACCORD-2) | Agitation in patients with Alzheimer’s disease (AD) | PDUFA priority review | Ongoing | oral | Psychiatric |
