Issued to Orphazyme A/S. Application reviewed but not approved; deficiencies must be corrected.
Issued
June 17, 2021Application
NDA 505b1 • 214927
Product
MIPLYFFA
Company
Orphazyme A/S
Review center
Other
Stage
Final Decision
Assyro regulatory brief
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FDA's complete response letter contains source-text evidence related to Clinical efficacy and substantial evidence, Nonclinical evidence, Statistical analysis, Safety and adverse events. Theme counts identify where the letter discusses a topic; they do not replace review of the exact redacted letter.
Review the exact redacted FDA letter and address each stated deficiency before resubmission.
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Application reviewed but not approved; deficiencies must be corrected. Requires resubmission addressing deficiencies. FDA issued this letter to Orphazyme A/S on June 17, 2021.
NDA 214927 is the application under which FDA issued this complete response letter for MIPLYFFA to Orphazyme A/S. It was reviewed by FDA Other. The letter is dated June 17, 2021.
FDA will not approve the application in its present form. The sponsor can resubmit after addressing every deficiency listed in the letter — which starts a Class 1 (two-month) or Class 2 (six-month) resubmission review clock — withdraw the application, or request an opportunity for a hearing.