This week, Niemann-Pick UK (NPUK) and The International Niemann-Pick Disease Alliance (INPDA) met with Zevra following the disappointing CHMP opinion on the EU marketing authorisation for arimoclomol. Zevra reaffirmed its commitment to the NPC community and its intention to request a re-examination. Further updates will be shared when available.
Zevra’s press release:
https://investors.zevra.com/news-releases/news-release-details/zevra-therapeutics-provides-update-regulatory-submission
EMA letter (attached) and the announcement:
https://www.ema.europa.eu/en/medicines/human/EPAR/meplyffa
https://www.npuk.org/wp-content/uploads/2026/07/questions-answer-refusal-marketing-authorisation-meplyffa-arimoclomol_en.pdf
If you have any questions please contact the NPUK Team by phone on 0191 415 06 93 or by email at info@npuk.org.