Antitrypsin Deficiency
Apr 01, 2025
Sanofi’s Qfitlia Becomes First FDA-Approved Therapy for Hemophilia A or B; FDA Approves AstraZeneca’s IMFINZI as First Perioperative Immunotherapy for Bladder Cancer; Beam Therapeutics’ BEAM-302 for AATD Receives FDA Clearance; AstraZeneca’s CALQUENCE Combo Greenlit in EU for Mantle Cell Lymphoma; Ionis’ WAINZUA Wins EU Approval for Hereditary Transthyretin Amyloidosis
Sanofi’s Qfitlia Becomes First US-Approved Therapy for Hemophilia A or B The FDA has approved Qfitlia (fitusiran), the first antithrombin-lowering therapy for routine prophylaxis to prevent or reduce bleeding episodes in hemophilia A or hemophilia B patients (ages 12+) with or without inhibitors. Approval is bas...
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May 15, 2019
Antitrypsin Deficiency Pipeline
Many Pharmaceuticals companies are working across the globe towards the development of new treatment therapies for Alpha-1 Anti-trypsin Deficiency to fulfil the unmet needs of Anti-trypsin Deficiency Therapeutic market as discussed in the previous article. The Alpha-1 Anti-trypsin Deficiency market is expected to c...
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May 13, 2019
Unmet Needs in the treatment of Antitrypsin deficiency
Antitrypsin deficiency, also called AAT deficiency (Alpha-1 Antitrypsin deficiency) is an inherited disease characterized by severe problems that make difficult to breathe. Along with lung disease, it can also cause serious issues in the liver too. Mutations in the SERPINA1 gene, whose primary function is to transc...
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