U.S. Tuberous Sclerosis Drugs Market - Industry Trends and Forecast to 2028

TABLE OF CONTENT

TABLE OF CONTENTS 1 INTRODUCTION 13 1.1 OBJECTIVES OF THE STUDY 13 1.2 MARKET DEFINITION 13 1.3 OVERVIEW OF THE U.S. TUBEROUS SCLEROSIS DRUGS MARKET 13 1.4 CURRENCY AND PRICING 15 1.5 LIMITATIONS 15 1.6 MARKETS COVERED 15 2 U.S. TUBEROUS SCLEROSIS DRUGS MARKET SEGMENTATION 18 2.1 MARKETS COVERED 18 2.2 GEOGRAPHICAL SCOPE 19 2.3 YEARS CONSIDERED FOR THE STUDY 19 2.4 DBMR TRIPOD DATA VALIDATION MODEL 20 2.5 PRIMARY INTERVIEWS WITH KEY OPINION LEADERS 23 2.6 MULTIVARIATE MODELLING 24 2.7 GENE LIFELINE CURVE 24 2.8 DBMR MARKET POSITION GRID 25 2.9 MARKET END USER COVERAGE GRID 27 2.10 SECONDARY SOURCES 28 2.11 ASSUMPTIONS 28 3 EXECUTIVE SUMMARY 29 4 THE U.S. TUBEROUS SCLEROSIS DRUG MARKET: REGULATORY FRAMEWORK 33 4.1 THE TUBEROUS SCLEROSIS REGISTRY TO CREATE DISEASE AWARENESS (TOSCA) 33 4.1.1 DATA ANALYSIS 33 4.2 LEGAL AND REGULATORY LAWS GOVERNING CANNABIDIOL AND EPIDIOLEX DRUG CLASSES IN THE U.S. 34 4.3 CONTROLLED SUBSTANCES ACT OF 1970 34 4.4 FDA APPROVAL OF EVEROLIMUS FOR TUBEROUS SCLEROSIS COMPLEX-ASSOCIATED PARTIAL-ONSET SEIZURES 34 4.5 CLINICAL AND REGULATORY PROGRAM UPDATES WHICH CONSISTS OF PLANNED CLINICAL PROGRAM IN TUBEROUS SCLEROSIS COMPLEX 34 5 PIPELINE ANALYSIS 35 6 PRICING ANALYSIS 36 7 MARKET OVERVIEW 37 7.1 DRIVERS 39 7.1.1 INCREASING RESEARCH FOR TUBEROUS SCLEROSIS 39 7.1.2 ROBUST PIPELINES FOR DEVELOPMENT OF NEWER TREATMENT 39 7.1.3 PREVALENCE OF TUBEROUS SCLEROSIS IN THE U.S. 40 7.1.4 INCREASING AWARENESS ABOUT TUBEROUS SCLEROSIS 40 7.1.5 INCREASE IN INNOVATIVE TECHNIQUES 41 7.2 RESTRAINTS 41 7.2.1 HIGH COST ASSOCIATED WITH TREATMENT 41 7.2.2 DELAYED OR MISDIAGNOSIS OF DISEASE 42 7.2.3 AVAILABILITY OF SURGICAL INTERVENTION CAN LIMIT THE DRUGS USAGE 42 7.3 OPPORTUNITIES 42 7.3.1 RISING DISPOSABLE INCOME 42 7.3.2 STRATEGIC INITIATIVES BY THE MARKET PLAYERS 43 7.3.3 INCREASING USAGE OF CBD OIL FOR THE TREATMENT 43 7.3.4 GROWING HEALTHCARE EXPENDITURE IN THE U.S. 44 7.4 CHALLENGES 44 7.4.1 COMPLICATIONS ASSOCIATED WITH THE DISEASE AND TREATMENT 44 7.4.2 REIMBURSEMENT HURDLES DUE TO COMPLEX TREATMENT REGIME 44 7.4.3 PATENT CLIFF OF DRUGS 45 8 IMPACT OF COVID-19 ON THE U.S. TUBEROUS SCLEROSIS DRUGS MARKET 46 8.1 IMPACT ON PRICE 46 8.2 IMPACT ON DEMAND 46 8.3 IMPACT ON SUPPLY CHAIN 46 8.4 STRATEGIC DECISION BY MANUFACTURERS 47 8.5 KEY INITIATIVES BY MARKET PLAYERS DURING COVID-19 47 8.6 CONCLUSION 48 9 U.S. TUBEROUS SCLEROSIS DRUGS MARKET, GENE 49 9.1 OVERVIEW 50 9.2 TSC2 53 9.3 TSC1 53 10 U.S. TUBEROUS SCLEROSIS DRUGS MARKET, BY SITE 54 10.1 OVERVIEW 55 10.2 BRAIN 58 10.2.1 CORTICAL TUBERS 58 10.2.2 SUBEPENDYMAL NODULES (SEN) 58 10.2.3 SUBEPENDYMAL GIANT-CELL ASTROCYTOMAS (SEGA) 59 10.2.4 OTHERS 59 10.3 KIDNEY 59 10.3.1 CYSTS 60 10.3.2 ANGIOMYLIOPOMAS 60 10.3.3 OTHERS 60 10.4 SKIN 60 10.4.1 FACIAL ANGIOFIBROMAS 61 10.4.2 FOREHEAD PLAQUES 61 10.4.3 UNGUAL OR SUBUNGUAL FIBROMAS 61 10.4.4 SHAGREEN PATCHES 61 10.4.5 OTHERS 61 10.5 HEART 61 10.6 LUNGS 61 10.6.1 LYMPHANGIOLEIOMYOMATOSIS (LAM) 62 10.6.2 MULTINODULAR MULTIFOCAL PNEUMOCYTE HYPERPLASIA (MMPH) 62 10.7 OTHERS 62 11 U.S. TUBEROUS SCLEROSIS DRUGS MARKET, BY DRUG CLASS 64 11.1 OVERVIEW 65 11.2 MTOR INHIBITORS 68 11.2.1 VIGABATRIN 68 11.2.2 EVEROLIMUS 68 11.2.3 SIROLIMUS 68 11.2.4 OTHERS 69 11.3 ANTICONVULSANTS 69 11.3.1 LAMOTRIGINE 69 11.3.2 VALPORIC ACID 69 11.3.3 CLONAZEPAM 70 11.3.4 OXCARBAZEPINE 70 11.3.5 TOPIRAMATE 70 11.3.6 CARBAMEZEPINE 70 11.3.7 PHENYTOIN 70 11.3.8 OTHERS 70 11.4 CANNABIDIOLS/EPIDIOLEX 70 11.5 OTHERS 71 12 U.S. TUBEROUS SCLEROSIS DRUGS MARKET, BY DRUG TYPE 72 12.1 OVERVIEW 73 12.2 BRANDED 76 12.2.1 AFINITOR/VOTUBIA 76 12.2.2 EPIDIOLEX 76 12.2.3 ZORTRESS 76 12.2.4 DEPAKOTE 76 12.2.5 OTHERS 77 12.3 GENERICS 77 13 U.S. TUBEROUS SCLEROSIS DRUGS MARKET, BY POPULATION TYPE 78 13.1 OVERVIEW 79 13.2 CHILDREN 82 13.3 ADULTS 82 14 U.S. TUBEROUS SCLEROSIS DRUGS MARKET, BY ROUTE OF ADMINISTRATION 83 14.1 OVERVIEW 84 14.2 ORAL 87 14.2.1 TABLETS 87 14.2.2 PILLS 87 14.2.3 OTHERS 87 14.3 PARENTERAL 88 14.3.1 SUBCUTANEOUS 88 14.3.2 INTRAMUSCULAR 88 14.3.3 OTHERS 88 14.4 OTHERS 89 15 U.S. TUBEROUS SCLEROSIS DRUGS MARKET, BY END USER 90 15.1 OVERVIEW 91 15.2 HOSPITALS 94 15.3 SPECIALTY CLINICS 94 15.4 HOME HEALTHCARE 94 15.5 OTHERS 95 16 U.S. TUBEROUS SCLEROSIS DRUGS MARKET, BY DISTRIBUTION CHANNEL 96 16.1 OVERVIEW 97 16.2 HOSPITAL PHARMACY 100 16.3 RETAIL PHARMACY 100 16.4 ONLINE PHARMACY 100 17 U.S. TUBEROUS SCLEROSIS DRUGS MARKET: COMPANY LANDSCAPE 102 17.1 COMPANY SHARE ANALYSIS: U.S. 102 18 SWOT 103 19 COMPANY PROFILES 104 19.1 NOVARTIS AG 104 19.1.1 COMPANY SNAPSHOT 104 19.1.2 REVENUE ANALYSIS 104 19.1.3 PRODUCT PORTFOLIO 105 19.1.4 RECENT DEVELOPMENT 105 19.2 GLAXOSMITHKLINE PLC 106 19.2.1 COMPANY SNAPSHOT 106 19.2.2 REVENUE ANALYSIS 106 19.2.3 PRODUCT PORTFOLIO 107 19.2.4 RECENT DEVELOPMENT 107 19.3 ABBVIE INC. 108 19.3.1 COMPANY SNAPSHOT 108 19.3.2 REVENUE ANALYSIS 108 19.3.3 PRODUCT PORTFOLIO 109 19.3.4 RECENT DEVELOPMENTS 109 19.4 PFIZER INC. 110 19.4.1 COMPANY SNAPSHOT 110 19.4.2 REVENUE ANALYSIS 110 19.4.3 PRODUCT PORTFOLIO 111 19.4.4 RECENT DEVELOPMENTS 111 19.5 JANSSEN PHARMACEUTICALS, INC. (A SUBSIDIARY OF JOHNSON & JOHNSON SERVICES, INC.) 112 19.5.1 COMPANY SNAPSHOT 112 19.5.2 REVENUE ANALYSIS 112 19.5.3 PRODUCT PORTFOLIO 113 19.5.4 RECENT DEVELOPMENT 113 19.6 ALEMBIC PHARMACEUTICALS LIMITED 114 19.6.1 COMPANY SNAPSHOT 114 19.6.2 REVENUE ANALYSIS 115 19.6.3 PRODUCT PORTFOLIO 115 19.6.4 RECENT DEVELOPMENT 115 19.7 AMNEAL PHARMACEUTICALS LLC 116 19.7.1 COMPANY SNAPSHOT 116 19.7.2 REVENUE ANALYSIS 116 19.7.3 PRODUCT PORTFOLIO 117 19.7.4 RECENT DEVELOPMENTS 117 19.8 APOTEX INC. 118 19.8.1 COMPANY SNAPSHOT 118 19.8.2 PRODUCT PORTFOLIO 118 19.8.3 RECENT DEVELOPMENTS 118 19.9 BIOPHARMA 119 19.9.1 COMPANY SNAPSHOT 119 19.9.2 PRODUCT PORTFOLIO 119 19.9.3 RECENT DEVELOPMENT 119 19.10 DR. REDDYS LABORATORIES LTD. 120 19.10.1 COMPANY SNAPSHOT 120 19.10.2 REVENUE ANALYSIS 120 19.10.3 PRODUCT PORTFOLIO 121 19.10.4 RECENT DEVELOPMENT 121 19.11 GW PHARMACEUTICALS PLC 122 19.11.1 COMPANY SNAPSHOT 122 19.11.2 REVENUE ANALYSIS 122 19.11.3 PRODUCT PORTFOLIO 123 19.11.4 RECENT DEVELOPMENTS 123 19.12 HIKMA PHARMACEUTICALS PLC 124 19.12.1 COMPANY SNAPSHOT 124 19.12.2 REVENUE ANALYSIS 124 19.12.3 PRODUCT PORTFOLIO 125 19.12.4 RECENT DEVELOPMENTS 125 19.13 LANNETT 126 19.13.1 COMPANY SNAPSHOT 126 19.13.2 REVENUE ANALYSIS 126 19.13.3 PRODUCT PORTFOLIO 127 19.13.4 RECENT DEVELOPMENTS 127 19.14 PAR PHARMACEUTICAL (A SUBSIDIARY OF ENDO INTERNATIONAL PLC) 128 19.14.1 COMPANY SNAPSHOT 128 19.14.2 REVENUE ANALYSIS 128 19.14.3 PRODUCT PORTFOLIO 129 19.14.4 RECENT DEVELOPMENTS 129 19.15 SUN PHARMACEUTICAL INDUSTRIES LTD. 130 19.15.1 COMPANY SNAPSHOT 130 19.15.2 REVENUE ANALYSIS 130 19.15.3 PRODUCT PORTFOLIO 131 19.15.4 RECENT DEVELOPMENT 131 19.16 TEVA PHARMACEUTICALS USA, INC. (A SUBSIDIARY OF TEVA PHARMACEUTICAL INDUSTRIES LTD.) 132 19.16.1 COMPANY SNAPSHOT 132 19.16.2 REVENUE ANALYSIS 132 19.16.3 PRODUCT PORTFOLIO 133 19.16.4 RECENT DEVELOPMENTS 133 19.17 VIATRIS INC. 134 19.17.1 COMPANY SNAPSHOT 134 19.17.2 PRODUCT PORTFOLIO 134 19.17.3 RECENT DEVELOPMENTS 135 19.18 VISTAPHARM, INC. 136 19.18.1 COMPANY SNAPSHOT 136 19.18.2 PRODUCT PORTFOLIO 136 19.18.3 RECENT DEVELOPMENT 136 19.19 WOCKHARDT USA (A SUBSIDIARY OF WOCKHARDT) 137 19.19.1 COMPANY SNAPSHOT 137 19.19.2 REVENUE ANALYSIS 137 19.19.3 PRODUCT PORTFOLIO 138 19.19.4 RECENT DEVELOPMENT 138 20 QUESTIONNAIRE 139 21 RELATED REPORTS 142