Results of Pegcetacoplan (APL-2) trial

A recently published study from Leeds, England, investigates the efficacy of the new drug pegcetacoplan for Paroxysmal Nocturnal Haemoglobinuria (PNH). Pegcetacoplan is not yet approved for PNH therapy in Europe, but approval is currently being considered by the European Medicines Agency (EMA). For this reason, we would like to briefly introduce the drug and the study to you and first take a brief look at the disorder of blood formation underlying PNH in order to understand how pegcetacoplan works:

Normally, certain proteins are found on the surface of the red blood cells (erythrocytes) that transport oxygen through our bodies. Among other things, these regulate the immune system and prevent the erythrocytes from being broken down prematurely as they circulate through the body. In PNH,READ MORE

Recommendations of the Ulm University Hospital on COVID-19 vaccination in AA/PNH

A few weeks ago, vaccinations against the SARS-CoV-2 virus began in Germany. As announced in our February virtual group meeting, the Ulm University Hospital, as one of the most important AA/PNH centres in Germany, has now published a recommendation letter (German) on this topic. If you need assistance with details contained in the recommendation letter please email us at info(at) or give us a call: +49 30 549094080.

We briefly summarise the most important contents for you below:

The Ulm University Hospital's vaccination advice letter for AA/PNH patients is based on the current recommendations of the Ständige Impfkommission (STIKO) of the Robert Koch Institute, previous studies on the approved vaccines, and the Ulm AA/PNH Centre's experience with COVID-19 vaccinations.


Ravulizumab available in more concentrated formulation

In Germany, Ravulizumab is now available in a more concentrated formulation (100 mg/ml compared to 10 mg/ml previously). The new formulation significantly shortens the infusion time while maintaining safety and efficacy. Previously, the infusion took at least two hours, but with the new formulation it will be about 60% faster on average, depending on the dose and weight of the patient. It became available as an infusion in Germany on 5 January 2021.