Drug Discovery and Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Women in Pharma and Biotech
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • Resources
    • Video features
    • Podcast
    • Voices
    • Views
    • Webinars
  • Pharma 50
    • 2025 Pharma 50
    • 2024 Pharma 50
    • 2023 Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50
  • Advertise
  • SUBSCRIBE

Alexion’s Soliris Gets EC Orphan Status for New Treatment

By Drug Discovery Trends Editor | April 23, 2014

AlexionAlexion Pharmaceuticals Inc. announced that the European Commission has granted an orphan drug designation (ODD) to Soliris (eculizumab), a first-in-class terminal complement inhibitor, for the prevention of graft rejection following solid organ transplantation. Graft rejection can cause severe injury to the transplanted organ and is a significant barrier to successful transplantation.
Soliris is currently approved in the United States, European Union, Japan and other countries for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two debilitating, ultra-rare and life-threatening disorders caused by chronic uncontrolled complement activation. Alexion is investigating Soliris for the prevention of acute antibody-mediated rejection (AMR) in kidney transplant recipients, and for the prevention of delayed graft function (DGF) in patients receiving deceased donor kidney transplants. Soliris is not approved in any country to prevent or treat rejection following kidney or other solid organ transplantation.
“Rejection after transplantation is a severe and potentially devastating occurrence for patients undergoing organ transplantation due to the very real risk of losing the transplanted organ,” said Martin Mackay, executive vice president, global head of R&D at Alexion. “By specifically inhibiting the terminal complement pathway, Soliris has the potential to lower the risk of rejection, a benefit that could lead to improved clinical outcomes for these patients.”
The European Commission grants orphan medicinal product status to provide incentives to develop medicinal products to treat, prevent or diagnose diseases or conditions that affect no more than five in 10,000 persons in the EU. The orphan medicinal product status designation would provide Alexion with certain benefits and incentives, including a period of marketing exclusivity if Soliris is approved in the EU for the orphan therapeutic indication of acute antibody-mediated rejection (AMR).

Filed Under: Drug Discovery

 

Related Articles Read More >

Korean team reports all-in-one cancer nanomedicine in pre-clinical studies
Nektar’s Phase 2b atopic dermatitis win triggers 1,746% analyst target surge, but legal tussle with ex-partner Lilly could complicate path forward
Dupixent approved to treat bullous pemphigoid
EVEREST lead investigator on why Dupixent sets a new bar for treating coexisting CRSwNP and asthma
“ddd
EXPAND YOUR KNOWLEDGE AND STAY CONNECTED
Get the latest news and trends happening now in the drug discovery and development industry.

MEDTECH 100 INDEX

Medtech 100 logo
Market Summary > Current Price
The MedTech 100 is a financial index calculated using the BIG100 companies covered in Medical Design and Outsourcing.
Drug Discovery and Development
  • MassDevice
  • DeviceTalks
  • Medtech100 Index
  • Medical Design Sourcing
  • Medical Design & Outsourcing
  • Medical Tubing + Extrusion
  • Subscribe to our E-Newsletter
  • Contact Us
  • About Us
  • R&D World
  • Drug Delivery Business News
  • Pharmaceutical Processing World

Copyright © 2025 WTWH Media LLC. All Rights Reserved. The material on this site may not be reproduced, distributed, transmitted, cached or otherwise used, except with the prior written permission of WTWH Media
Privacy Policy | Advertising | About Us

Search Drug Discovery & Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Women in Pharma and Biotech
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • Resources
    • Video features
    • Podcast
    • Voices
    • Views
    • Webinars
  • Pharma 50
    • 2025 Pharma 50
    • 2024 Pharma 50
    • 2023 Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50
  • Advertise
  • SUBSCRIBE