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Tofersen

New Drug Approvals

3] Tofersen is an antisense oligonucleotide that targets the production of superoxide dismutase 1 , an enzyme whose mutant form is commonly associated with amyotrophic lateral sclerosis. 3] Tofersen was approved for medical use in the United States in April 2023, [3] [6] and in the European Union in May 2024. [4] 3 November 2006.

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Gepirone

New Drug Approvals

4] It was approved for the treatment of major depressive disorder in the United States in September 2023. [4] 4] This came after the drug had been rejected by the Food and Drug Administration (FDA) three times over two decades due to insufficient evidence of effectiveness. [5] The FDA rejected approval for gepirone in 2002 and 2004. [5]

FDA
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Vamorolone

New Drug Approvals

Finally, the acetate group is cleaved under basic conditions to give crude Vamorolone, which was recrystallized from iPrOH to obtain the pure product. The product was filtered off, washed with H 2 O (3 x 0.5 The product was filtered off, washed with H 2 O/MeCN 4:1 (2 x 0.5 and DMAP (16.1 g, 0.132 mmol, 0.10 HCI (950 mL, 0.95

FDA
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Biogen Launches Phase 3 Trial of Felzartamab for Primary Membranous Nephropathy

The Pharma Data

Despite its impact, there are currently no FDA-approved therapies for PMN , and treatment options are limited to non-specific and often toxic agents such as chemotherapy or general immunosuppressants. The rights to felzartamab were licensed to Human Immunology Biosciences (HI-Bio) , which Biogen acquired in July 2024.

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2025 Merkin Prize in Biomedical Technology awarded to pioneers of CAR T-cell therapy

Broad Institute

The next step was to adapt cell culture techniques to enable the production of effective CAR T cells for infusion to patients. After their clinical results, their technology was licensed to Juno Therapeutics (later acquired by Bristol Myers Squibb.) The scientists also needed to prove that the cell therapy could work inside patients.

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Inavolisib

New Drug Approvals

3] Inavolisib was approved for medical use in the United States in October 2024. [3] The residue was purified via flash chromatography on silica gel (eluted: 15% ethyl acetate in petroleum ether) to give crude product which was washed with 2.4 g (90%) of the crude title product as a colorless oil. Hz, 1H), 3.71-3.57

FDA
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Fitusiran

New Drug Approvals

2] Fitusiran was approved for medical use in the United States in March 2025. [2] 2] The fixed dose of fitusiran is not approved because it led to excessive clotting in some participants. [2] 2] The US Food and Drug Administration (FDA) granted the application for fitusiran orphan drug and fast track designations. 26 March 2025.