Use of Identical INN "Imiglucerase" for Different Drug Products: Impact Analysis of Adverse Events in a Proprietary Global Safety Database.

Introduction: Approved in 1994 and assigned the International Nonproprietary Name (INN) imiglucerase by the World Health Organization, Cerezyme (Sanofi Genzyme) is an enzyme replacement therapy used to treat Gaucher disease in > 90 countries. At least two therapies approved outside the USA and the European Union, Abcertin and Asbroder, have adopted the identical INN imiglucerase. Both drugs were approved via regulatory pathways not aligned with World Health Organization Similar Biotherapeutic Product guidelines.

Throwing a spotlight on under-recognized manifestations of Gaucher disease: Pulmonary involvement, lymphadenopathy and Gaucheroma.

Background: Gaucher disease (GD) is a rare lysosomal storage disorder classically subdivided into type 1 (non-neuronopathic) GD, and types 2 and 3 (neuronopathic) GD. It is typically characterized by clinical manifestations including anemia, thrombocytopenia, hepatosplenomegaly, bone lesions, and (in more severe forms) neurological impairment. However, less-commonly reported and often under-recognized manifestations exist, which potentially have a significant impact on patient outcomes.

Long-term adverse event profile from four completed trials of oral eliglustat in adults with Gaucher disease type 1.

Background: Eliglustat is a first-line oral treatment for adults with Gaucher disease type 1 who have an extensive, intermediate or poor CYP2D6 metabolizer phenotype (> 90% of patients). Whereas enzyme replacement therapy for Gaucher disease has been widely used for more than two decades, eliglustat has only been in commercial use since 2014. Clinicians and patients want to better understand which adverse events are most commonly associated with eliglustat, as well as their severity, frequency, and duration.