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Tofersen

New Drug Approvals

Jump up to: a b c d e f g h i j k l “FDA approves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene” (Press release). Food and Drug Administration (FDA). “Biogen’s ALS Drug Gets Partial Backing From FDA Panel” Bloomberg News. January 2022).

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Inavolisib

New Drug Approvals

3] Inavolisib was approved for medical use in the United States in October 2024. [3] 3] Inavolisib was approved for medical use in the United States in October 2024. [3] 2022 Dec 22;65(24):16589-16621. Epub 2022 Dec 1. Food and Drug Administration (FDA). December 2022). J Med Chem. doi: 10.1021/acs.jmedchem.2c01422.

FDA
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Sunvozertinib

New Drug Approvals

2] [4] Sunvozertinib was approved for medical use in the United States in July 2025. [1] 5] In China, it was conditionally approved in 2023 for the treatment of NSCLC and full approval is contingent on results of phase 3 clinical trials. [6] Sunvozertinib CAS 2370013-12-8 DZD9008, 584.1 Retrieved 7 July 2025. 12 (7): 1676–1689.

FDA
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Q&A: One scientist’s bold vision to make on-demand treatments routine for life-threatening rare genetic diseases

Broad Institute

s specific mutation, creating a mouse model of the disease, determining the optimal base editor, performing extensive safety analyses, working with Danaher to manufacture the therapeutic, conducting toxicity studies, and securing FDA approval for the trial. This unprecedented feat required diagnosing K.J.’s

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Vamorolone

New Drug Approvals

Syn EuropeanJournalofMedicinalChemistry265(2024)116124 Vamorolone (Agamree) On October 26, 2023, Vamorolone, developed jointly by Santhera Pharmaceuticals and ReveraGen BioPharma, has received FDA approval to treat DMD in patients aged 2 years and older [1]. 79 (2022) 1005–1014. [71] 79 (2022)1005–1014 Bioorg. Shale, U.J.

FDA
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Pirtobrutinib

New Drug Approvals

Food and Drug Administration (FDA). FDA Approves Jaypirca (pirtobrutinib), the First and Only Non-Covalent (Reversible) BTK Inhibitor, for Adult Patients with Relapsed or Refractory Mantle Cell Lymphoma After at Least Two Lines of Systemic Therapy, Including a BTK Inhibitor” (Press release). eCollection 2022.

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Automated red blood cell exchange: bridging treatment gaps in sickle cell disease care

Drug Target Review

Currently, three FDA-approved disease-modifying drug therapies are available: hydroxyurea, crizanlizumab and L-glutamine, though each has limitations that affect patient compliance. 2022 Oct 7;71(9):1-18. The use of FDA-approved medications for preventing vaso-occlusive events in sickle cell disease. 2023.44546.