Orphan Diseases Companies

Latest Orphan Diseases Companies Updates:

Amgen acquires Horizon Therapeutics This $28 billion deal in September 2023 further consolidates Amgen's position in the rare disease market, adding Horizon's portfolio of innovative treatments for rare metabolic and inflammatory diseases.

Pfizer partners with MyoKardia for ATTR amyloidosis This November 2023 collaboration expands Pfizer's reach in rare cardiovascular diseases, combining their expertise with MyoKardia's promising pipeline of gene therapy solutions for ATTR amyloidosis.

Roche receives FDA approval for Evrysdi (risdiplam) for SMA types 1, 2, and 3: This oral medication offers a convenient and potentially transformative treatment option for patients with spinal muscular atrophy, a rare neuromuscular disease.

BioMarin Pharmaceuticals launches Vimizim (elapramin hydrochloride) for Niemann-Pick disease type C This drug received accelerated approval from the FDA, providing the first approved treatment for this previously untreatable neurodegenerative disease.

List of Orphan Diseases Key companies in the market:

• Bristol-Myers Squibb Company (US), 


• CELGENE CORPORATION (US), 


• Novartis AG (Switzerland), 


• Pfizer Inc. (US), 


• 
  AbbVie Inc. (US),


• F. Hoffmann-La Roche Ltd (Switzerland),


• Alexion (US), 


• Vertex Pharmaceuticals Incorporated (US), 


• Merck & Co., Inc (US),


• Sanofi (French),