EXECUTIVESUMMARY2.docVIP

PAGE  PAGE 63  TOC \o 1-4 EXECUTIVE SUMMARY  PAGEREF _To\h 2 I. Background  PAGEREF _To\h 2 II. Brief Description of Clinical Trials and Patient Exposure  PAGEREF _To\h 2 III. Efficacy Conclusions  PAGEREF _To\h 2 IV. Safety Conclusions  PAGEREF _To\h 3 V. Growth Hormone for Non-growth Hormone Deficient Short Stature – Further Considerations  PAGEREF _To\h 4 SUMMARY OF CLINICAL FINDINGS  PAGEREF _To\h 5 1. INTRODUCTION  PAGEREF _To\h 5 2. BRIEF OVERVIEW OF THE CLINICAL PROGRAM  PAGEREF _To\h 5 3. CLINICAL TRIAL DESCRIPTION  PAGEREF _To\h 6 3.1 Pivotal Clinical Study GDCH  PAGEREF _To\h 6 3.2 Supportive Clinical Study E001  PAGEREF _To\h 10 4. EFFICACY  PAGEREF _To\h 14 4.1 Pivotal Study GDCH  PAGEREF _To\h 14 4.2 Supportive Study E001  PAGEREF _To\h 16 4.3 Supportive Studies from Peer-reviewed Medical Literature  PAGEREF _To\h 19 5. SAFETY  PAGEREF _To\h 20 6. DOSING  PAGEREF _To\h 22 7. General Comments on NGHDSS as a New Indication  PAGEREF _To\h 23 APPENDIX 1  PAGEREF _To\h 25 A. Efficacy Analyses  PAGEREF _To\h 25 A.1 Trial GDCH  PAGEREF _To\h 25 A.2 Trial E001  PAGEREF _To\h 29 B. Safety analyses  PAGEREF _To\h 34 B1.Deaths  PAGEREF _To\h 34 B.2. Serious Adverse Events  PAGEREF _To\h 35 B.3. Patient Discontinuations Due to Adverse Events  PAGEREF _To\h 38 B.4. Treatment-Emergent Adverse Events  PAGEREF _To\h 40 B.5. Clinically Significant Treatment-Emergent Adverse Events  PAGEREF _To\h 44 B.6. Clinical Laboratory Data  PAGEREF _To\h 47 B.6.1. Carbohydrate Metabolism  PAGEREF _To\h 48 B.6. 2. Thyroid Function  PAGEREF _To\h 55 B.6.3 Insulin-Li