Due to an error in the study phase numbering, the study phase was amended from Phase 3 to Phase 2.

1. SPD602-301 replaces FBS0701-CTP-15 2. Updated selection of study population, study assessments 3. Updated the description of the study design to allow entry (further to meeting all qualification criteria) of subjects who were unable to transition directly into this extension study due to administrative reasons, and who had initiated treatment with a chelator other than SSP-004184 4. Replaced glucose intolerance test with fasting glucose test 5. Added pharmacokinetic parameters to the study objectives 6. Updated the description of the study period and timelines 7. Added information regarding adverse events (AEs) of particular interest including paresthesia and hypoesthesia and extended neurological examinations 8. Updated Inclusion and exclusion criteria for subjects who did not transition directly from their feeder protocol 9. Updated the description of the study period and timelines to include timelines for qualification for subjects who did not transition directly from their feeder protocol and who had initiated treatment with a chelator other than SSP-004184 10. Qualified that the measurable data demonstrating qualification into the extension study were to be taken from the end of treatment visit from the feeder protocol for those subjects who transitioned directly from their feeder protocol only 11. Updated the follow-up period from 30 days to 7 days 12. Added withdrawal criteria: Cardiac left ventricular ejection fraction (LVEF) <50% 13. Updated the management of study toxicities section 14. Inserted endocrine laboratory tests for subjects 10 years and over who enrolled in their feeder protocol <18-years old, visual acuity test and eye examination for all subjects 15. Amended the timing of AE and serious AE collection to detail that AEs were to be recorded from the time of signed informed consent until 7 days after the last dosing 16. Added data monitoring committee

1. Clarified that echocardiography was acceptable for LVEF if MRI information was not available 2. To replace extended neurological examinations with total neuropathy score nurse (TNSn) assessments at Qualification and Enrolment, Visits 4, 8, and end of treatment/early discontinuation 3. Subjects who had either completed 1 of the previous feeder studies or who discontinued (with agreement from the investigator and Shire physician) and who met qualification criteria were permitted to enrol subjects who had either completed a previous SSP-004184AQ study or prematurely discontinued 1 of the SSP-004184AQ studies (with agreement from the investigator and Shire physician) were permitted to enrol 4. Subjects who had either completed a previous SSP-004184AQ study or prematurely discontinued 1 of the SSP-004184AQ studies (with agreement from the investigator and Shire physician) were permitted to enrol 5. Excluded subjects with cardiac T2* MRI <10.0 milliseconds (from <8msec), LVEF below the locally determined normal range or LVEF <50% at baseline testing by MRI or echocardiography 6. “Chronic cholecystitis” was no longer exclusionary 7. Subjects were to be withdrawn from the study and classified as treatment failures if they continued to accumulate iron based on cardiac iron concentration determined by MRI 8. Replaced previous AE severity assessment with those reflecting National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 criteria 9. Added statement indicating that Qualification and Enrollment physical examination, TNSn and ECG are required if assessment was not completed at the last feeder study visit (as well as for subjects who did not transition directly from their feeder protocol)