Glossary - Key Terms in Iron Chelation

Glossary of clinical, pharmacological, regulatory and commercial terms used across the DEFRATAJ site.


Reference glossary of clinical, pharmacological, regulatory and commercial terms used across the DEFRATAJ website.

A - B #

ACTD
ASEAN Common Technical Dossier - harmonised registration format for ASEAN.
ALT / AST
Liver-function enzymes monitored during chelation therapy.
ANVISA
Brazilian Health Regulatory Agency.
APQR
Annual Product Quality Review - GMP-required annual batch review.
BE
Bioequivalence - comparative PK study vs reference product.

C #

CAPA
Corrective and Preventive Action.
Cardiac T2*
MRI measure of myocardial iron; < 20 ms suggests cardiac iron risk.
CoA
Certificate of Analysis - batch-specific QC results.
COFEPRIS
Mexican regulatory authority.
CoPP
Certificate of Pharmaceutical Product - WHO-format for export registration.
CQA
Critical Quality Attribute.
CPP
Critical Process Parameter.
CTD
Common Technical Document - Module 1-5 dossier format.

D - E #

DDP
Delivered Duty Paid - Incoterm.
DRESS
Drug Reaction with Eosinophilia and Systemic Symptoms.
EAEU
Eurasian Economic Union harmonised registration.
eCTD
Electronic CTD format.

F - L #

FOB
Free On Board - Incoterm.
Ferritin
Serum iron-storage protein; primary iron-overload biomarker.
HDPE
High-Density Polyethylene - bottle material.
ICH
International Council for Harmonisation.
ICSR
Individual Case Safety Report.
IPSS / IPSS-R
MDS risk stratification.
LIC
Liver Iron Concentration - MRI R2/T2* gold standard.

M - P #

MAH
Marketing Authorization Holder.
MDS
Myelodysplastic Syndromes.
NTDT
Non-Transfusion-Dependent Thalassemia.
OOS
Out of Specification.
PIC/S
Pharmaceutical Inspection Co-operation Scheme.
PPQ
Process Performance Qualification.
PSUR
Periodic Safety Update Report.

Q - Z #

QA / QC
Quality Assurance / Quality Control.
QP
Qualified Person - batch release authority.
SCAR
Severe Cutaneous Adverse Reactions - SJS, TEN, DRESS.
SCD
Sickle Cell Disease.
SJS
Stevens-Johnson Syndrome - detachment < 10% BSA.
SmPC
Summary of Product Characteristics.
TEN
Toxic Epidermal Necrolysis - detachment > 30% BSA.
UGT1A1
Hepatic enzyme; deferasirox glucuronidation.
WHO-GMP
WHO Good Manufacturing Practices.
WHO-PQ
WHO Prequalification programme.