Alexion Pharmaceuticals’ ravulizumab has expanded the range of indications for treating a rare complement-mediated disease.
Rare/Orphan Diseases
Posted inAutoimmune Diseases, New Drugs, Rare/Orphan Diseases, Regulatory
Empaveli: Powerful New Drug for Paroxysmal Nocturnal Hemoglobinuria
Pegcetacoplan is more effective in treating paroxysmal nocturnal hemoglobinuria compared to Soliris.
Posted inAutoimmune Diseases, Mergers & Acquisitions, Rare/Orphan Diseases
AstraZeneca + Alexion = Rescue of Both Companies
Alexion Pharmaceuticals, which owns Soliris, one of the most expensive drugs on Earth, has agreed to sell AstraZeneca for $39 billion.
Posted inNew Drugs, Oncology, Rare/Orphan Diseases, Regulatory
Danyelza: New Drug for Neuroblastoma That Can Cure It
Naxitamab by Y-mAbs Therapeutics offers gorgeous treatment efficacy with unsafe adverse events.
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory
Koselugo: First Drug to Treat Neurofibromatosis
Selumetinib by AstraZeneca powerfully suppresses tumor growth in neurofibromatosis type 1.
Posted inNeuroscience, New Drugs, Rare/Orphan Diseases, Regulatory
Zolgensma: Gene Therapy That Cure Spinal Muscular Atrophy. The Most Comprehensive Review
Novartis revealed the most expensive drug in the world. And it is amazing.
Posted inNew Drugs, Rare/Orphan Diseases, Regulatory
Arcalyst: World’s First Drug to Treat Recurrent Pericarditis
Rilonacept by Kiniksa Pharmaceuticals will reduce recurrent pericarditis to almost nothing.