Miplyffa

Application withdrawnHuman

Orphan medicine

Details

EMA number	EMEA/H/C/005203
ATC code	N07
Authorisation	-
Revision	-

EMA page

Therapeutic indication

Treatment of Niemann-Pick disease type C (NPC).

MeSH: Niemann-Pick Disease, Type C

Group: Other nervous system drugs

Active substances

arimoclomol citrate

Marketing Authorisation Holder

Orphazyme A/S

MAH

🇺🇸 Also approved in the US (FDA)

MIPLYFFA — ZEVRA DENMARK, approved 2024-09-20 (NDA214927).

Most reported adverse events (US / FAERS)

38 reports across the top reactions. These are spontaneously reported events from the FDA Adverse Event Reporting System — they reflect reporting volume, not incidence or frequency, and a report does not mean the drug caused the event.

diarrhoea

5

product size issue

4

vomiting

4

insomnia

3

death

🇺🇸 Also approved in the US (FDA)

MIPLYFFA — ZEVRA DENMARK, approved 2024-09-20 (NDA214927).

Most reported adverse events (US / FAERS)

38 reports across the top reactions. These are spontaneously reported events from the FDA Adverse Event Reporting System — they reflect reporting volume, not incidence or frequency, and a report does not mean the drug caused the event.

diarrhoea

5

product size issue

4

vomiting

4

insomnia

3

death

2

fall

2

fatigue

2

gait disturbance

2

pneumonia

2

pneumonia aspiration

2

product administration interrupted

2

product distribution issue

2

product use complaint

2

seizure

2

sepsis

2