International Niemann–Pick Disease Alliance
Progress made in understanding potential resolution of topics outlined in Complete Response Letter, including need for additional data to support NDA FDA recommends the company provides supplemental information and analyses, and the FDA offers to have further interactions to...
Copenhagen – October 5, 2021 – Orphazyme A/S (ORPHA.CO; ORPH), a late-stage biopharmaceutical company, today announces regulatory updates for its investigational drug candidate, arimoclomol, for which it is seeking marketing authorization in both Europe and the United States for...
Sep 27, 2021 – Dr. Kjems is a leading global physician scientist with a proven pharma track record of drug development across multiple therapeutic areas, including chronic indications, oncology and rare diseases – – Gerald F. Cox, MD, PhD will...
We are pleased to share the following Niemann-Pick Type C Community Listening Session with the U.S. Food and Drug Administration (FDA), which was originally held on August 3rd 2021. The meeting arranged and the subsequent report prepared in a collaboration between...
“Pfrieger’s Digest” is written by Frank Pfrieger of Niemann-Pick Selbsthiifegruppe (Germany) to provide an overview of the latest advances in Niemann-Pick disease based on recent scientific publications. Frank Pfrieger is group leader at the Institute of Cellular and Integrative Neurosciences in...
The INPDA is committed to improving outcomes for the global Niemann-Pick community and to ensuring equitable access to safe and effective therapies. In support of the US patient community and in recognition of the global impact of FDA decision...
Sometimes in such a big organisation as the INPDA, our various member groups can be overshadowed, but we want to show just how important each and every member group is in making the INPDA work. In the INPDA Spotlight this...