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What FDA’s Newest Gene Therapy Approval Tells Us About Durability: How Long is Long Enough?

FDA Law Blog: Drug Discovery

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

Therapies 119
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Intellia Reports Positive 3-Year Phase 1 Data for Lonvo-z in Hereditary Angioedema

The Pharma Data

Durable Benefit and Positive Clinical Outcomes “Today’s results underscore the promising potential of Intellia’s approach to genome-editing therapy — a one-time treatment that has been well tolerated and offers a highly differentiated, durable effect for patients suffering from a serious disease,” said John Leonard, M.D.,

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Radioligand therapy pipeline with in-license for compounds targeting Fibroblast Activation Protein (FAP)

The Pharma Data

Novartis Oncology continues to reimagine cancer care through development of robust radioligand therapy portfolio. We continue to invest in radioligand therapy as one of the four unique platforms of Novartis Oncology. Broad expression of FAP demonstrated in tumors or in tumor stroma across many solid tumors 1 ,2 ,3.

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Fitusiran

New Drug Approvals

1] [2] Adverse effects The US Food and Drug Administration prescription label for fitusiran contains a boxed warning for thrombotic events (blood clotting) and gallbladder disease (with some recipients requiring gallbladder removal). [2] Fitusiran 1711.0g/mol, 1] It is an antithrombin-directed small interfering ribonucleic acid. [1]

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The future of mental health treatment: Zelquistinel’s role

Drug Target Review

However, a deeper understanding of brain function particularly the role of synaptic plasticity is now opening the door to innovative therapies. Event-driven pharmacology Donello highlights the growing recognition of synaptic plasticity’s crucial role in the biology of depression.

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Gamida Cell Provides Regulatory Update on Biologics License Application for Omidubicel

The Pharma Data

Nasdaq: GMDA), an advanced cell therapy company committed to cures for blood cancers and serious hematologic diseases, today announced that the company conducted a Type B Meeting for omidubicel with the U.S. Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the U.S. About Omidubicel.

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Otsuka’s Sibeprenlimab Shows Significant Proteinuria Reduction in Phase 3 IgAN Trial

The Pharma Data

Otsuka Unveils Promising Phase 3 Results for Sibeprenlimab in IgA Nephropathy, Marking Significant Proteinuria Reduction and Advancing a Novel APRIL-Targeted Therapy Otsuka Pharmaceutical Development & Commercialization, Inc., Treatment-emergent adverse events (TEAEs) occurred in 76.3% in the placebo group.

Trials 40