Hereditary angioedema (HAE) is a rare genetic disorder characterized by recurrent episodes of severe swelling (edema), typically in the limbs, face, gastrointestinal tract, and airway. The condition is caused by a deficiency or dysfunction of C1 esterase inhibitor (C1-INH), a protein involved in regulating inflammation and the immune response. The swelling episodes can be spontaneous or triggered by stress, trauma, surgery, or infection.
BioCryst Pharmaceuticals Received FDA approval for Orladeyo™ (berotralstat) for prophylaxis of HAE attacks, offering the first and only once-daily oral medication for this indication.Announced positive late-stage clinical trial results for beronalstat in pediatric HAE patients, potentially expanding its market reach.
Takeda Pharmaceutical Company Launched Takhzyro™ (lanadelumab) in the US, providing a long-acting subcutaneous injection for HAE prophylaxis.Initiated Phase 3 clinical trials for its novel C5a receptor inhibitor TAK-994 for HAE prevention, expanding its treatment pipeline.
Shire Received FDA approval for Cinryze™ (C1 esterase inhibitor) for the treatment of acute HAE attacks, offering a rapid-acting intravenous option.Collaborated with patient advocacy groups to launch educational initiatives and improve access to HAE care.
Alexion Pharmaceuticals Continued to be a leader in the HAE market with its established medications Soliris™ (eculizumab) and Ultomiris™ (ravulizumab), offering long-term prevention and treatment options. Focused on expanding access to its therapies through patient assistance programs and advocacy partnerships.
Hereditary Angioedema Therapeutics Market Highlights:
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