Updates

EMA accepts regulatory submission for olipudase alfa, the first potential therapy for ASMD

EMA accepts regulatory submission for olipudase alfa, the first potential therapy for ASMD Olipudase alfa has been granted PRIority MEdicines (PRIME) designation in Europe, Breakthrough Therapy designation in the United States, and SAKIGAKE designation in Japan European regulatory decision anticipated second half of 2022 DECEMBER 6, 2021...

Orphazyme provides regulatory update following Type A meeting with FDA on arimoclomol in Niemann-Pick disease type C

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...

Orphazyme provides regulatory and financial updates

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...

Cyclo Therapeutics Appoints Lise Lund Kjems, MD, PhD as Chief Medical Officer

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...

NPC Listening Session Aug 3 Report

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 – 5th Issue

“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...

Orphazyme announces publication of results from its Phase 2/3 trial of arimoclomol in Niemann-Pick disease type C in the Journal of Inherited Metabolic Disease

IntraBio Reports Statistically Significant and Clinically Meaningful Improvements in the Use of IB1001 For Treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease)

NPC Letter to FDA (13th August 2021)

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...

INPDA Spotlight on Associazione Italiana Niemann Pick Onlus

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...