A to Z: Drugs in Highlight: C is for CERDELGA

Welcome to “A to Z: Drugs in Highlight”

This engaging series is designed to delve into the intricate and dynamic realm of pharmaceutical drugs. As the world of pharmaceuticals continues to evolve with new discoveries and emerging challenges, we aim to shed light on the diverse array of medications available today, ranging from blockbuster medications to those that may not be as familiar.

Join us on this educational journey through the alphabet of pharmaceutical drugs. Whether you’re a healthcare professional, a student, or simply someone with a curious mind, “A to Z: Drugs in Highlight” promises to be an enlightening and engaging series.

C is for CERDELGA®

CERDELGA® (discovered at University of Michigan, developed by Genzyme Corp) uses eliglustat for the treatment of type-1 Gaucher’s disease. Gaucher’s disease is a genetic disorder characterized by the accumulation of a fatty substance known as glycosylceramide (or glucocerebroside) within the body, primarily affecting the liver, spleen, and bones. The buildup of this fat can lead to a range of symptoms, including anaemia, fatigue, easy bruising, and enlargement of the spleen and liver, as well as bone pain and fractures. CERDELGA® was designated an ‘organ medicine’ on 4 December 2007.

How it works:

The pharmaceutical active ingredient in CERDELGA®, eliglustat, works by blocking the enzyme involved in the production of the glycosylceramide fat. This helps prevent the build-up of the fat and thus helps the affected organs to function better.

Marketing approval:

CERDELGA® is approved for marketing in both the United States (approved by the FDA in August 2014) and Europe (approved by the EMA in January 2015).

Patent protection:

There are a number of patent rights protecting aspects of CERDELGA® in the United States and Europe (as well as in many other countries around the world). For example, granted EP patent no. 2504332 relates a pharmaceutical composition comprising the hemitartrate salt of a compound represented by the following structural formula:

and a pharmaceutically acceptable carrier or diluent, wherein at least 70% by weight of the salt is crystalline. Notably, the grant of EP2504332 was opposed by Sandoz. The patent was finally maintained in its current amended form by the European Patent Office’s Board of Appeal in 2021.