Updates

Pfrieger’s Digest – 12/2024 Edition

IntraBio Appoints Dr. Marc C. Patterson, US Chief Medical Officer

Update on PRONTO and Phase 2 RAINBOW study data with nizubaglustat, for patients with Niemann-Pick disease type C (NPC) GM1 and GM2 gangliosidoses.

Azafaros granted important regulatory designations and clearance by European authorities for global Phase 3 studies, to be initiated in 2025

Zevra NPC Community Letter – MIPLYFFA now available (USA)

IntraBio Announces U.S. FDA Approval of AQNEURSA for the Treatment of Niemann- Pick Disease Type C

• AQNEURSA™ (ak-nur-sah) is the only FDA-approved stand-alone therapy for the treatment of Niemann-Pick disease type C (NPC) • Approval follows positive Phase III data demonstrating significant improvements in neurological symptoms and functional benefits that could be seen within...

GeMDAC Votes Favorably on Arimoclomol for NPC: Key Step Toward FDA Approval

      August 2nd, 2024 Dear NPC Community, Today, the Genetic Metabolic Diseases Advisory Committee (GeMDAC) met to review Zevra Therapeutics’ arimoclomol as an orally delivered treatment for Niemann-Pick disease type C (NPC). The committee, an independent panel...

Azafaros announces positive topline Phase 2 study data with nizubaglustat

Azafaros announces positive topline Phase 2 study data with nizubaglustat in GM2 gangliosidosis and Niemann-Pick disease type C. Study primary endpoint met, with confirmed safety profile Investigators and Patient Organisations supportive of continued development Leiden, The Netherlands, 19 June...

Azafaros: Completion of 12-week Phase 2 RAINBOW study evaluating lead asset nizubaglustat in rare disease patients

Pfrieger’s Digest – 02/2024 Edition