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FDA Approves J&J’s Darzalex Faspro for Rare Light Chain Amyloidosis

The Pharma Data

Food and Drug Administration (FDA) approved Janssen Pharmaceuticals ’ (a Johnson and Johnson company) Darzalex Faspro for adults with newly diagnosed light chain amyloidosis. It was approved in combination with bortezomib, cyclophosphamide and dexamethasone (D-VCd). Michael Vi/Shutterstock. It was developed with Genmab.

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The Long Road to End Tuberculosis

Codon

Still, while these developments provide cause for hope, each drug or vaccine faces a variety of challenges, ranging from financial incentives for clinical trials to the difficulty of discovering new antimicrobials that pass safety and efficacy tests. A phase 3 clinical trial for M72/AS01E, funded by the Gates Foundation, began this year.

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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.

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Topical Gene Therapy FDA-Approved for Severe Skin Disease, Dystrophic Epidermolysis Bullosa

PLOS: DNA Science

The newest FDA-approved gene therapy treats the severe, skin-peeling condition dystrophic epidermolysis bullosa (DEB). The gene treatment has been a long time coming, but it differs from the handful of other approved gene therapies: it isn’t a one-and-done. Earlier results were published in Nature Medicine.

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FDA Approves Roche’s ENSPRYNG for Neuromyelitis Optica Spectrum Disorder (NMOSD)

The Pharma Data

First and only FDA-approved subcutaneous treatment option for anti-aquaporin-4 antibody positive NMOSD that can be self-administered by a person with NMOSD or a caregiver every four weeks. First and only approved therapy for NMOSD designed to target and inhibit interleukin-6 receptor activity, using novel recycling antibody technology.

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FDA Approves Verquvo (vericiguat) for Heart Failure with Reduced Ejection Fraction

The Pharma Data

FDA Approves Verquvo (vericiguat) for Heart Failure with Reduced Ejection Fraction. Armstrong, cardiologist and Distinguished University Professor of Medicine at the Canadian VIGOUR Centre, University of Alberta, and study chair of the VICTORIA trial. KENILWORTH, N.J.–(BUSINESS Verquvo (vericiguat) 2.5

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Darzalex Faspro (daratumumab and hyaluronidase-fihj) Becomes the First FDA-Approved Treatment for Patients with Newly Diagnosed Light Chain (AL) Amyloidosis

The Pharma Data

Food and Drug Administration (FDA) approval of Darzalex Faspro ® (daratumumab and hyaluronidase-fihj), a subcutaneous formulation of daratumumab, in combination with bortezomib, cyclophosphamide and dexamethasone (D-VCd) for the treatment of adult patients with newly diagnosed light chain (AL) amyloidosis.[1]