- FDA Approves Drug for Gaucher Disease – Genzyme won FDA approval for Cerdelga (eliglustat) for the long-term treatment of adult patients with Type 1 form of Gaucher disease. Gaucher disease is a rare genetic disorder that results in a person producing an insufficient amount of the glucocerebrosidase enzyme which causes fatty materials to collect in the spleen, liver and bone marrow. Cerdelga inhibits the metabolic process that produces the fatty materials. Cerdelga received orphan drug designation from FDA.
- FDA Allows Marketing of New Type 1 Diabetes Diagnostic – The agency announced this week that it would be allowing the marketing of the first zinc transporter 8 autoantibody (ZnT8Ab) test to help diagnose Type 1 diabetes, the most common form of diabetes diagnosed in children and adolescents. ZnT8Ab is produced by the immune system of many people with type 1 diabetes and the test detects the presence of the antibody in a patient’s blood. The device was reviewed by the agency through the de novo premarket review pathway which is intended for low-to-moderate-risk devices that are not substantially equivalent to an already legally marketed device.
- Plan Related to Diversity and Clinical Trial Data Released – The agency released a plan and had an accompanying blog post from Commissioner Hamburg related to diversity in clinical trials. It is called the “FDA Action Plan to Enhance the Collection and Availability of Demographic Subgroup Data“. The Action Plan follows a report issued last year on the topic and includes 27 different action items that the agency is putting on its “to-do” list that the commissioner states is designed to meet three primary goals – (1) improving quality of data collection regarding sub-groups (the term used by the agency related to diversity); (2) to identify barriers to greater participation among diverse populations; and (3) to make the data collection more transparent. The plan is to be executed by a steering committee and the blog posting states that a website has been set up to track progress, but there was not a link to the website provided.
That’s it for me this week. Enjoy the waning August days.