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The table below is a review of notable updates that occurred in September 2025 for investigational products in development (not an inclusive list). Click on the status to view our full coverage.
| Drug | Pharmacologic Class | Proposed Indication | Status |
| Allergic Disorders | |||
| REGN1908-1909 (Regeneron) | FelD1-blocking antibody | To reduce allergic symptoms in adults with cat or birch pollen allergy. | Phase 3 results |
| Cardiovascular Disease | |||
| Enlicitide decanoate (Merck) | Proprotein convertase subtilisin/kexin type 9 | Treatment of adults with hypercholesterolemia. | Phase 3 results |
| Dermatological Disorders | |||
| Amlitelimab (Sanofi) | Anti-OX40 ligand monoclonal antibody | Treatment of moderate to severe atopic dermatitis. | Phase 3 results |
| Hematological Disorders | |||
| Mitapivat (Agios Pharmaceuticals) | Pyruvate kinase activator | Treatment of adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. | sNDA review extended |
| Infectious Diseases | |||
| Ensitrelvir (Shionogi) | SARS-CoV-2 main protease inhibitor | For the prevention of COVID-19 following exposure to an infected individual. | NDA accepted for review |
| Metabolic Disorders | |||
| SAR446268 (Sanofi) | Adeno-associated viral gene therapy designed to silence DMPK expression | Treatment of non-congenital (juvenile and adult onset) myotonic dystrophy type 1. | Fast Track designation |
| SL1009 (sodium dichloroacetate; Saol Therapeutics) | Pyruvate dehydrogenase kinase inhibitor | Treatment of pyruvate dehydrogenase complex deficiency. | Complete Response Letter issued |
| Zilganersen (Ionis) | Antisense oligonucleotide | Treatment of Alexander disease. | Phase 3 results |
| Musculoskeletal Disorders | |||
| Nanoecapsulated sirolimus plus pegadricase (Sobi) | mTOR inhibitor plus pegylated uricase | Treatment of uncontrolled gout. | BLA accepted for review |
| Neurologic Disorders | |||
| AMT-130 (uniQure) | Gene therapy that inhibits the production of mutant huntingtin protein | Treatment of Huntington disease. | Phase 1/2 results |
| Emrusolmin (Teva Pharmaceuticals)T | Small molecule that targets the alpha synuclein protein | Treatment of multiple system atrophy. | Fast Track designation |
| Oncology | |||
| Iberdomide (Bristol Myers Squibb) | Cereblon E3 ligase modulator | In combination with standard therapies to treat patients with relapsed or refractory multiple myeloma. | Phase 3 results |
| Pirtobrutinib (Eli Lilly) | Bruton tyrosine kinase inhibitor | To treat treatment-naïve patients with chronic lymphocytic leukemia or small lymphocytic lymphoma without 17p deletions. | Phase 3 results |
| Relacorilant (Corcept Therapeutics) | Glucocorticoid receptor antagonist | Treatment of platinum-resistant ovarian cancer. | NDA accepted for review |
| Trastuzumab deruxtecan (AstraZeneca) | HER2-directed antibody + topoisomerase inhibitor conjugate | In combination with ado-trastuzumab emtansine to treat patients with human epidermal growth factor receptor-positive early breast cancer. | Phase 3 results |
| Ophthalmic Disorders | |||
| Idebenone (Chiesi Global) | Synthetic analog of coenzyme Q10 | Treatment of Leber hereditary optic neuropathy. | NDA accepted for review |
| Poisoning & Drug Dependence | |||
| Cytisinicline (Achieve Life Sciences) | Plant-based alkaloid that selectively binds to nicotine acetylcholine receptors | Smoking cessation therapy for adults. | NDA accepted for review |
| Respiratory Disorders | |||
| Benralizumab (AstraZeneca) | Interleukin-5 antagonist | Treatment of chronic obstructive pulmonary disease. | Phase 3 results |
| Dexpramipexole (Areteia Therapeutics) | Small molecule that inhibits the maturation of eosinophils | Treatment of eosinophilic asthma. | Phase 3 results |
| Efzofitimod (aTyr Pharma) | tRNA synthetase derived immunomodulator | Treatment of pulmonary sarcoidosis. | Phase 3 results |
This article originally appeared on MPR
