Eculizumab Receives Orphan Drug Status

Eculizumab Receives Orphan Drug Status

EculizumabAt the end of January 2014 the powerful drug Eculizumab received orphan drug status from the FDA as a preventative treatment of delayed graft function (DGF) for renal transplant patients. Eculizumab is already approved to treat paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. Paroxysmal nocturnal hemoglobinuria or PNH is a rare form of anemia that until the approval of Eculizumab in 2007 was untouchable in terms of viable treatment options for patients with this atypical disease. Additionally, Eculizumab was approved to treat atypical hemolytic uremic syndrome, or aHUS, which is an extremely uncommon kidney disorder affecting roughly one in every million people worldwide.

Eculizumab has provided a beacon of hope for patients suffering from rare disorders, and the latest orphan drug status from the FDA demonstrates the continued benefits of this innovative drug. The National Organization for Rare Disorders supports the use of the monoclonal antibody Eculizumab and offers financial assistance for this drug for patients with financial hardship.

The latest FDA nod for Eculizumab for its use after a kidney transplant  offers patients options for safe and effectual treatment should they experience delayed graft function post- surgery. DGF is a common problem for kidney transplant patients, and it often occurs soon after surgery is complete. When DGF occurs the transplanted organ does not immediately begin functioning normally, thus leading to the unfortunate need for lifelong dialysis for the patients. However, the use of Eculizumab in kidney transplant patients, as well as other solid organ transplant patients, has been shown to inhibit the terminal complement pathway and effectively lower the risk of DGF.

The typical dosage for Eculizumab is dispensed post-transplant surgery once a week for five consecutive weeks, and then once every two weeks. The length of time a patients receives Eculizumab is determinate on their specific medical condition, physical well-being and their type of transplant surgery. As an I.V. infusion Eculizumab is dispensed in a hospital or healthcare setting. Adverse effects are not common, and typical side effects from Eculizumab include headache, nausea and diarrhea.

LGM Pharma provides Eculizumab CAS# 219685-50-4, for research and development purposes. Clients can be assured of quality API products and continuous support throughout the R&D process.

Products currently covered by valid US Patents are offered for R&D use in accordance with 35 USC 271(e) +A13(1). Any patent infringement and resulting liability is solely at buyer risk.


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LGM Pharma Acquires CDMO

On July 27, 2020, LGM Pharma announced its acquisition of the formulation development and drug product contract manufacturing business of Nexgen Pharma, Inc. As a result, you will notice our new logo and visuals throughout the website. We’re working on updates to reflect the exciting, expanded CDMO capabilities and services we now can offer you.

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