1. US Orphan Drugs Market Overview

  1.1 FDA Designation Criteria and Market Exclusivity

  1.2 Current Market Scenario

  1.3 Future Market Trends and Opportunity Assessment

2. Partnerships and Collaborations Concerning Orphan Drugs

3. US Orphan Drugs Market Trends By Indication

  3.1 Cancer

  3.2 Neurological Disorders

  3.3 Cardiovascular Disorders

  3.4 Ophthalmic Diseases

  3.5 Rare Genetic Disorders and Metabolic Disorders

  3.6 Autoimmune and Inflammatory Diseases

4. US Orphan Drugs Reimbursement Scenario

  4.1 Medicare

  4.2 Medicaid

  4.3 Private Insurers

  4.4 Pharmaceutical Companies

5. US Orphan Designated Drugs Dosage, Price and Treatment Cost Insight

This Chapter Gives Comprehensive Insights On 375 Orphan Drugs. For Representation Purpose We Have Mentioned Few Drugs Name In Table of Contents. Detailed List Is Provided In Report Sample.

  5.1 Lynparza

  5.2 Nplate

  5.3 Tafinlar

  5.4 Halaven

  5.5 Jadenu

  5.6 Xpovio

  5.7 Gavreto

  5.8 Ocaliva

  5.9 INOmax

  5.10 Truseltiq

  5.11 Lumakras

  5.12 Monjuvi

  5.13 Onpattro

  5.14 Opdivo

  5.15 Opdualag

.

.

.

.

  5.370 Colazal (Balsalazide Disodium)

  5.371 Anascorp (Centruroides (Scorpion) Immune F(ab’)2 (Equine) Injection)

  5.372 Nithiodote (sodium nitrite and sodium thiosulfate)

  5.373 BabyBIG (Botulism Immune Globulin Intravenous [Human])

  5.374 NexoBrid (anacaulase-bcdb)

  5.375 Sarclisa (Isatuximab)

6. US Orphan Designated Drugs Sales Insight (2019 – Q1’2023)

This Chapter Gives Comprehensive Insights On 106 Orphan Drugs. For Representation Purpose We Have Mentioned Few Drugs Name In Table of Contents. Detailed List Is Provided In Report Sample.

 6.1 Tafinlar

  6.2 Jadenu

  6.3 Zolgensma

  6.4 Lynparza

  6.5 Nplate

  6.6 Lumakras

  6.7 Repatha

  6.8 Otezla

  6.9 Kyprolis

  6.10 Opdivo

  6.11 Opdualag

  6.12 Empliciti

  6.13 Braftovi

  6.14 Mektovi

  6.15 Lorbrena

  .

  .

  .

  .

  6.100 Praluent

  6.101 Mozobil

  6.102 Yescarta

  6.103 Lunsumio

  6.104 Keytruda

  6.105 Pemazyre

  6.106 Camzyos

7. US Orphan Designated Drugs Clinical Trials Insight

  7.1 By Company

  7.2 By Indication

  7.3 By Patient Segment

  7.4 By Phase

  7.5 By Priority Status

8. US Orphan Drugs Clinical Trials By Company, Indication and Phase

  8.1 Research

  8.2 Preclinical

  8.3 Phase-I

  8.4 Phase-I/II

  8.5 Phase-II

  8.6 Phase-II/III

  8.7 Phase-III

  8.8 Preregistration

  8.9 Registered

9. Marketed Orphan Drugs Clinical Insight By Company and Indication

10. Competitive Landscape

  10.1 AbbVie

  10.2 Alexion AstraZeneca Rare Disease

  10.3 Alnylam Pharmaceuticals

  10.4 Amgen

  10.5 Amicus Therapeutics

  10.6 Array BioPharma

  10.7 Astellas Pharma

  10.8 AstraZeneca

  10.9 Bayer HealthCare

  10.10 Beacon Therapeutics

  10.11 BioMarin Pharmaceutical

  10.12 Bioverativ

  10.13 Boehringer Ingelheim

  10.14 Bristol-Myers Squibb

  10.15 Celgene Corporation

  10.16 Chiesi

  10.17 Chugai Pharmaceutical

  10.18 Collaborations Pharmaceuticals

  10.19 CSL

  10.20 Daiichi Sankyo Company

  10.21 Eisai Co Ltd

  10.22 Eli Lilly and Company

  10.23 Emergent BioSolutions

  10.24 Ferring Pharmaceuticals

  10.25 Fred Hutchinson Cancer Research Center

  10.26 Flavocure Biotech

  10.27 Genentech

  10.28 Genmab

  10.29 Gilead Sciences

  10.30 GlaxoSmithKline

  10.31 GSK

  10.32 Hanmi Pharmaceutical

  10.33 Horizon Therapeutics plc

  10.34 Incyte Corporation

  10.35 Ionis Pharmaceuticals

  10.36 Ipsen

  10.37 Janssen Biotech

  10.38 Jazz Pharmaceuticals plc

  10.39 Johnson and Johnson

  10.40 Kamada

  10.41 Krystal Biotech

  10.42 Kyowa Kirin

  10.43 Leadiant Biosciences

  10.44 Ligand Pharmaceuticals

  10.45 Lundbeck A/S

  10.46 MeiraGTx

  10.47 Memorial Sloan-Kettering Cancer Center

  10.48 Merck

  10.49 Moderna Therapeutics

  10.50 Nationwide Children's Hospital

  10.51 Nonindustrial Source

  10.52 Novartis

  10.53 Novo Nordisk

  10.54 Omeros Corporation

  10.55 Onyx Pharmaceuticals (Amgen)

  10.56 Otsuka Pharmaceutical

  10.57 Pfizer

  10.58 PTC Therapeutics

  10.59 Recordati

  10.60 Regeneron Pharmaceuticals

  10.61 Roche

  10.62 Sanofi

  10.63 Servier

  10.64 Shionogi

  10.65 Spark Therapeutics

  10.66 Sumitomo Pharma

  10.67 Sutro Biopharma

  10.68 Swedish Orphan Biovitrum

  10.69 Takeda

  10.70 Teva Pharmaceutical Industries

  10.71 UCB

  10.72 UniQure

  10.73 Vertex Pharmaceuticals

  10.74 XOMA

  10.75 ZymoGenetics (BMS)

Figure 1-1: US – Orphan Drugs Market Size (US$ Billion), 2022 - 2028

 Figure 3-1: TP-1287 – Study Initiation and Completion Year

 Figure 3-2: ATG-022 – Study Initiation and Completion Year

 Figure 3-3: ATG-101: Study Initiation and Completion Year

 Figure 3-4: Lacutamab – Study Initiation and Completion Year

 Figure 3-5: CART-ddBCMA – Study Initiation and Completion Year

 Figure 3-6: FORE8394 – Study Initiation and Completion Year

 Figure 3-7: ERAS-801 – Study Initiation and Completion Year

 Figure 3-8: Tirabrutinib – Study Initiation and Completion Year

 Figure 3-9: Temferon - Study Initiation and Completion Year

 Figure 3-10: RP-A601 - Study Initiation and Completion Year

 Figure 3-11: RP-A501 – Study Initiation and Completion Year

 Figure 3-12: HG004 – Study Initiation and Completion Year

 Figure 3-13: EA-2353 – Study Initiation and Completion Year

 Figure 3-14: ARD-101 – Study Initiation and Completion Year

 Figure 3-15: SYNB1934 – Study Initiation and Completion Year

 Figure 3-16: ST-920 – Study Initiation and Completion Year

 Figure 5-1: US – Lynparza Approvals by Indication

 Figure 5-2: US – Lynparza Orphan Drug Designation Years by Indication

 Figure 5-3: US – Cost of 60 and 120 Tablets Supply of 100 mg and 150 mg Lynparza

 (US$), July’2023

 Figure 5-4: US – Monthly Cost of Treatment with 100 mg and 150 mg Lynparza Tablets

 (US$), July’2023

 Figure 5-5: US – Cost of 125 mcg, 250 mcg and 500 mcg Supply Packs of Nplate (US$),

 July’2023

 Figure 5-6: US – Tafinlar Approvals by Indication

 Figure 5-7: US – Tafinlar Orphan Drug Designation Years

 Figure 5-8: US – Cost of Per Unit and Supply of 120 Capsules of 50mg and 75mg Tafinlar

 (US$), July’2023

 Figure 5-9: US – Monthly Cost of Tafinlar Treatment by Weight (US$), July’2023

 Figure 5-10: US – Halaven Approvals by Indication

 Figure 5-11: US – Monthly Cost of Treatment with Havalen Injection (US$),

 July’2023

 Figure 5-12: US – Jadenu Orphan Drug Designation Years by Indication

 Figure 5-13: US – Cost of Per Unit and Supply of 30 Tablets of 90mg, 180mg and 360mg

 Jadenu (US$), July’2023

 Figure 5-14: US – Monthly Cost of Treatment with Jadenu (US$), July’2023

 Figure 5-15: US – Xpovio Orphan Drug Designation and Approval Years by Indication

 Figure 5-16: US – Cost of Per Unit and Supply of 20mg Xpovio Tablets (US$), July’2023

 Figure 5-17: US – Cost of Per Unit and Supply of 40mg Xpovio Tablets (US$), July’2023

 Figure 5-18: US – Cost of Per Unit and Supply of 50mg and 60mg Xpovio Tablets (US$),

 July’2023

 Figure 5-19: US – Monthly Cost of Treatment with Xpovio (US$), July’2023

 Figure 5-20: US – Gavreto Orphan Drug Designation and Approval Years by Indication

 Figure 5-21: US – Cost of Per Unit and Supply of 100mg Gavreto Tablets (US$),

 July’2023

 Figure 5-22: US – Monthly Cost of Treatment with Gavreto Capsule (US$), July’2023

 Figure 5-23: US – Cost of Per Unit and Supply of 5mg and 10mg Ocaliva Tablets (US$),

 July’2023

 Figure 5-24: US – INOmax Orphan Drug Designation and Approval Years

 Figure 5-25: US – Truseltiq Orphan Drug Designation and Approval Years

 Figure 5-26: US – Cost of Per Unit and Supply of Different Truseltiq Doses (US$),

 July’2023

 Figure 5-27: US – Monthly and Yearly Cost of Treatment with Truseltiq (US$),

 July’2023

 Figure 5-28: US – Lumakras Orphan Drug Designation and Approval Years

 Figure 5-29: US – Cost of Per Unit and Supply of 120mg and 320mg Lumakras Doses

 (US$), July’2023

 Figure 5-30: US – Monjuvi Orphan Drug Designation and Approval Years by Indication

 Figure 5-31: US – Cost of Truseltiq Treatment Cycles US$), July’2023

 Figure 5-32: US – Onpattro Orphan Drug Designation and Approval Years

 Figure 5-33: US – Cost of Per Unit and Supply of 5mL Vial of Onpattro (US$), July’2023

 Figure 5-34: US – Cost of Onpattro Treatment by Weight US$), July’2023

 Figure 5-35: US – Cost of Per Unit and Supply of 10 mg/mL Opdivo (US$), July’2023

 Figure 5-36: US – Opdualag Orphan Drug Designation and Approval Years

 Figure 5-37: US – Cost of Per Unit and Supply of Opdualag Solution (US$), July’2023

 Figure 5-38: US – Unit, Monthly and Yearly Cost of Opdualag Treatment US$),

 July’2023

 Figure 5-39: US – Orladeyo Orphan Drug Designation and Approval Years

 Figure 5-40: US – Cost of Per Unit and Supply of Orladeyo Capsule (US$), July’2023

 Figure 5-41: US – Orladeyo Orphan Drug Designation and Approval Years

 Figure 5-42: US – Cost of Per Unit and Supply of Rubraca Tablet (US$), July’2023

 Figure 5-43: US – Livmarli Orphan Drug Designation and Approval Years

 Figure 5-44: US – Cost of Per Unit and Supply of Livmarli Solution (US$), July’2023

 Figure 5-45: US – Unit, Monthly and Yearly Cost of Livmarli Treatment US$), July’2023

 Figure 5-46: US – Bendeka Orphan Drug Designation and Approval Years

 Figure 5-47: US – Cost of Per Unit and Supply of Bendeka Solution (US$), July’2023

 Figure 5-48: US – Unit Cost and Cost of 1 and 6 Cycles of Bendeka Treatment for CLL

 US$), July’2023

 Figure 5-49: US – Unit Cost and Cost of 1 and 6 Cycles of Bendeka Treatment for CLL

 US$), July’2023

 Figure 5-50: US – Ryanodex Orphan Drug Designation and Approval Years

 Figure 5-51: US – Monthly Cost of Treatment with Fyarro Injection (US$), July’2023

 Figure 5-52: US – Firdapse Orphan Drug Designation and Approval Years

 Figure 5-53: US – Cost of Per Unit and Supply of 10mg Firdapse Tablet (US$),

 July’2023

 Figure 5-54: US – Braftovi Orphan Drug Designation and Approval Years

 Figure 5-55: US – Cost of Per Unit and Supply of 75mg Braftovi Capsule (US$),

 July’2023

 Figure 5-56: US – Monthly Cost of Melanoma Treatment with Braftovi Capsule (US$),

 July’2023

 Figure 5-57: US – Monthly Cost of Colorectal Cancer Treatment with Braftovi

 Capsule (US$), July’2023

 Figure 5-58: US – Cost of Per Unit and Supply of 15mg Mektovi Tablet (US$), July’2023

 Figure 5-59: US – Monthly Cost of Melanoma Treatment with Mektovi Tablet (US$),

 July’2023

 Figure 5-60: US – Evrysdi Orphan Drug Designation and Approval Years

 Figure 5-61: US – Cost of Per Unit and Supply of 0.75mg/mL Evrysdi Solution (US$),

 July’2023

 Figure 5-62: US – Hetlioz Orphan Drug Designation and Approval Years by Indication

 Figure 5-63: US – Cost of Per Unit and Supply of 20mg Hetlioz Capsule (US$),

 July’2023

 Figure 5-64: US – Cost of Per Unit and Supply of 20mg Hetlioz LQ Oral Suspension

 (US$), July’2023

 Figure 5-65: US – Monthly Cost of Melanoma Treatment with Hetlioz Capsule (US$),

 July’2023

 Figure 5-66: Yervoy – First Approval by Indication

 Figure 5-67: US – Cost of Per Unit and Supply of Yervoy Solution (US$), July’2023

 Figure 5-68: US – Lorbrena Orphan Drug Designation and Approval Years

 Figure 5-69: US – Cost of Per Unit and Supply of Lorbrena Tablet (US$), July’2023

 Figure 5-70: US – Monthly and Yearly Cost of Treatment with Lorbrena Tablet (US$),

 July’2023

 Figure 5-71: US – Terlivaz Orphan Drug Designation and Approval Years

 Figure 5-72: US – Brukinsa Orphan Drug Designation and Approval Years by Indication

 Figure 5-73: US – Cost of Per Unit and Supply of Brukinsa Capsule (US$), July’2023

 Figure 5-74: US – Monthly and Yearly Cost of Treatment with Brukinsa (US$),

 July’2023

 Figure 5-75: US – Viltepso Orphan Drug Designation and Approval Years

 Figure 5-76: US – Monthly and Yearly Cost of Treatment with Viltepso (US$), July’2023

 Figure 5-77: US – Eylea Orphan Drug Designation and Approval Years

 Figure 5-78: US – Cost of Per Unit and Supply of Eylea Solution (US$), July’2023

 Figure 5-79: US – Monthly and Yearly Cost of Treatment with Viltepso (US$), July’2023

 Figure 5-80: US – Evkeeza Orphan Drug Designation and Approval Years

 Figure 5-81: US – Cost of Per Unit and Supply of Evkeeza Solution (US$), July’2023

 Figure 5-82: US – Sogroya Orphan Drug Designation and Approval Years

 Figure 5-83: US – Cost of Per Unit and Supply of Sogroya Solution (US$), July’2023

 Figure 5-84: US – Skytrofa Orphan Drug Designation and Approval Years

 Figure 5-85: US – Cost of Per Unit and Supply of Skytrofa Injection (US$), July’2023

 Figure 5-86: US – Cost of Per Unit and Supply of Avastin Solution (US$), July’2023

 Figure 5-87: US – Vyvgart Orphan Drug Designation and Approval Years

 Figure 5-88: US – Cost of Per Unit and Supply of Vyvgart Solution (US$), July’2023

 Figure 5-89: US – Monthly Cost of Treatment with Vyvgart (US$), July’2023

 Figure 5-90: US – Tpoxx Orphan Drug Designation and Approval Years

 Figure 5-91: US – Koselugo Orphan Drug Designation and Approval Years

 Figure 5-92: US – Cost of Per Unit and Supply of 10mg Capsules of Koselugo (US$),

 July’2023

 Figure 5-93: US – Cost of Per Unit and Supply of 25mg Capsules of Koselugo (US$),

 July’2023

 Figure 5-94: US – Monthly Cost of Treatment with Koselugo by BSA (US$), July’2023

 Figure 5-95: US – Actemra Orphan Drug Designation and Approval Years

 Figure 5-96: US – Cost of Per Unit and Supply of 20 mg/mL Vials of Actemra IV

 Solution (US$), July’2023

 Figure 5-97: US – Cost of Per Unit and Supply of 162 mg/0.9 mL Prefilled Syringe of

 Actemra Subcutaneous Solution (ACTpen) (US$), July’2023

 Figure 5-98: US – Tecentriq Orphan Drug Designation and Approval Years

 Figure 5-99: US – Cost of Per Unit and Supply of Tecentriq Intravenous Solution (US$),

 July’2023

 Figure 5-100: US – Qinlock Orphan Drug Designation and Approval Years

 Figure 5-101: US – Cost of Per Unit and Supply of Qinlock Tablets (US$), July’2023

 Figure 5-102: US – Unit, Monthly and Annual Cost of Treatment with Qinlock (US$),

 July’2023

 Figure 5-103: US – Wakix Orphan Drug Designation and Approval Years

 Figure 5-104: US – Cost of Per Unit and Supply of Wakix Tablets (US$), July’2023

 Figure 5-105: US – Kimmtrak Orphan Drug Designation and Approval Years

 Figure 5-106: US – Cost of Per Unit and Supply of Kimmtrak (US$), July’2023

 Figure 5-107: US – Elahere Orphan Drug Designation and Approval Years

 Figure 5-108: US – Cost of Per Unit and Supply of 100 mg/20 mL Elahere Solution

 (US$), July’2023

 Figure 5-109: US - Price for Single Unit of Polivy 30 mg and 140 mg Intravenous

 Powder for Injection (US$), July’2023

 Figure 5-110: US – Cost of Single Dose, Single Treatment Cycle and Full Treatment of

 Hairy Cell Leukemia with 30 mg Polivy Powder (US$), July’2023

 Figure 5-111: US – Cost of Single Dose, Single Treatment Cycle and Full Treatment of

 Hairy Cell Leukemia with 140 mg Polivy Powder (US$), May’2023

 Figure 5-112: US – Enhertu Orphan Drug Designation and Approval Years

 Figure 5-113: Enhertu – Cost of Single Treatment Cycle and Annual Treatment (US$),

 July’2023

 Figure 5-114: US – Jakafi Orphan Drug Designation and Approval Years

 Figure 5-115: US – Cost of Per Unit and Supply of Different Jakafi Doses (US$),

 July’2023

 Figure 5-116: Jakafi – Cost of Monthly and Annual Treatment (US$), July’2023

 Figure 5-117: US – Kalydeco Orphan Drug Designation and Approval Years

 Figure 5-118: US – Cost of Per Unit and Supply of Kalydeco Oral Granule and Tablet

 (US$), July’2023

 Figure 5-119: Kalydeco – Cost of Monthly and Annual Treatment (US$), July’2023

 Figure 5-120: US – Cost of Per Unit and Supply of Symdeko Oral Tablet (US$),

 July’2023

 Figure 5-121: US – Trikafta Orphan Drug Designation and Approval Years

 Figure 5-122: US – Cost of Per Unit and Supply of Trikafta Oral Granule and Tablet

 (US$), July’2023

 Figure 5-123: US – Orkambi Orphan Drug Designation and Approval Years

 Figure 5-124: US – Cost of Per Unit and Supply of Orkambi Oral Granule and Tablet

 (US$), July’2023

 Figure 5-125: US – Hemgenix Orphan Drug Designation and Approval Years

 Figure 5-126: US – Voxzogo Orphan Drug Designation and Approval Years

 Figure 5-127: US – Cost of Per Unit and Supply of Voxzogo Powder (US$), July’2023

 Figure 5-128: US – Actimmune Orphan Drug Designation and Approval Years

 Figure 5-129: US – Cost of Per Unit and Supply of Actimmune Solution (US$),

 July’2023

 Figure 5-130: US – Actimmune Orphan Drug Designation and Approval Years

 Figure 5-131: US – Cost of per Unit of Reblozyl powder (US$), July’2023

 Figure 5-132: US – Monthly and Annual Cost of Treatment with Reblozyl (US$),

 July’2023

 Figure 5-133: US – Repatha Orphan Drug Designation and Approval Years

 Figure 5-134: US – Cost of Per Unit and Supply of Repatha (US$), July’2023

 Figure 5-135: US – Monthly and Annual Cost of Repatha Treatment in Adults with

 Established Cardiovascular Disease or Primary Hyperlipidemia (US$), July’2023

 Figure 5-136: US – Monthly and Annual Cost of Repatha Treatment for HoFH (US$),

 July’2023

 Figure 5-137: US – Rebyota Orphan Drug Designation and Approval Years

 Figure 5-138: US – Cost of Per Unit and Supply of Rebyota (US$), July’2023

 Figure 5-139: US – Cayston Orphan Drug Designation and Approval Years

 Figure 5-140: US – Cost of Per Unit and Supply of Cayston Powder (US$), July’2023

 Figure 5-141: US – Sylvant Orphan Drug Designation and Approval Years

 Figure 5-142: US – Cost of Per Unit and Supply of Sylvant Powder (US$), July’2023

 Figure 5-143: US – Cost of Monthly and Yearly Treatment with Sylvant US$), July’2023

 Figure 5-144: US – Mylotarg Orphan Drug Designation and Approval Years

 Figure 5-145: US – Cost of Per Unit and Supply of Mylotarg Powder (US$), July’2023

 Figure 5-146: US – Cost of Induction Cycle, Consolidation Cycles and Full Treatment

 Cost of Newly Diagnosed De Novo CD33-positive AML with Mylotarg (US$), July’2023

 Figure 5-147: US – Cost of Induction Phase and Continuation Phase and Full Treatment

 Cost of CD-33 positive AML with Mylotarg (US$), July’2023

 Figure 5-148: US – Cost of Single Dose and Full Treatment Cost of Relapsed and

 Refractory CD33 Positive AML with Mylotarg (US$), July’2023

 Figure 5-149: US – Besremi Orphan Drug Designation and Approval Years

 Figure 5-150: US – Cost of Single Dose, Monthly and Yearly Treatment with Besremi

 (US$), July’2023

 Figure 5-151: US – Ravicti Orphan Drug Designation and Approval Years

 Figure 5-152: US – Cost of Per Unit and Supply of Ravicti (US$), July’2023

 Figure 5-153: US – Cost of Per Unit and Supply of Tazverik Tablet (US$), July’2023

 Figure 5-154: US – Luxturna Orphan Drug Designation and Approval Years

 Figure 5-155: US – Zynteglo Orphan Drug Designation and Approval Years

 Figure 5-156: US – Empaveli Orphan Drug Designation and Approval Years

 Figure 5-157: US – Cost of Per Unit and Supply of Empaveli Solution (US$), July’2023

 Figure 5-158: US – Cost of Single Dose, Monthly and Yearly Treatment with Empaveli

 (US$), July’2023

 Figure 5-159: US – Ninlaro Orphan Drug Designation and Approval Years

 Figure 5-160: US – Cost of Per Unit and Supply of Ninlaro Capsule (US$), July’2023

 Figure 5-161: US – Cost of Single Dose, Monthly and Yearly Treatment with Empaveli

 (US$), July’2023

 Figure 5-162: US – Otezla Orphan Drug Designation and Approval Years

 Figure 5-163: US – Cost of Per Unit and Supply of Otezla tablet (US$), July’2023

 Figure 5-164: US – Arzerra Orphan Drug Designation and Approval Years

 Figure 5-165: US – Cost of Per Unit and Supply of Arzerra Solution (US$), July’2023

 Figure 5-166: US – Macrilen Orphan Drug Designation and Approval Years

 Figure 5-167: US – Cost of Per Unit and Supply of Macrilen Granules (US$), July’2023

 Figure 5-168: US – Tagrisso Orphan Drug Designation and Approval Years

 Figure 5-169: US – Cost of Per Unit and Supply of Tagrisso Tablets (US$), July’2023

 Figure 5-170: US – Tagrisso Monthly, Yearly and Full Course Treatment Cost (US$),

 July’2023

 Figure 5-171: US – Gammagard Liquid Orphan Drug Designation and Approval Years

 Figure 5-172: US – Cost of Per Unit and Supply of Different Gammagard Liquid Vials

 (US$), July’2023

 Figure 5-173: US – Fintepla Orphan Drug Designation and Approval Years

 Figure 5-174: US – Cost of Per Unit and Supply of Fintepla Solution (US$), July’2023

 Figure 5-175: US – Daybue Orphan Drug Designation and Approval Years

 Figure 5-176: US – Takhzyro Orphan Drug Designation and Approval Years

 Figure 5-177: US – Cost of Per Unit and Supply of Takhzyro Solution (US$), July’2023

 Figure 5-178: US – Cyramza Orphan Drug Designation and Approval Years

 Figure 5-179: US – Cost of Per Unit and Supply of Cyramza Solution (US$), July’2023

 Figure 5-180: US – Hyftor Orphan Drug Designation and Approval Years

 Figure 5-181: US – Cost of Per Unit and Supply of Hyftor Gel (US$), July’2023

 Figure 5-182: US – Prevymis Orphan Drug Designation and Approval Years

 Figure 5-183: US – Cost of Per Unit and Supply of Prevymis Solution (US$), July’2023

 Figure 5-184: US – Cost of Per Unit and Supply of Prevymis Tablets (US$), July’2023

 Figure 5-185: US – Prevymis Orphan Drug Designation and Approval Years

 Figure 5-186: US – Cost of Initial, Subsequent and Annual Treatment with Zynlonta

 (US$), July’2023

 Figure 5-187: US – Copiktra Orphan Drug Designation and Approval Years

 Figure 5-188: US – Cost of Per Unit and Supply of Copiktra Capsules (US$), July’2023

 Figure 5-189: US – Cost of Monthly and Annual Treatment with Copiktra (US$),

 July’2023

 Figure 5-190: US – Tepezza Orphan Drug Designation and Approval Years

 Figure 5-191: US – Cost of Single, Initial and Subsequent Doses of Copiktra (US$),

 July’2023

 Figure 5-192: US – Mycapssa Orphan Drug Designation and Approval Years

 Figure 5-193: US – Cost of Per Unit and Supply of Mycapssa Capsules (US$), July’2023

 Figure 5-194: US – Juxtapid Orphan Drug Designation and Approval Years

 Figure 5-195: US – Cost of Per Unit and Supply of Juxtapid Capsules (US$), July’2023

 Figure 5-196: US – Myalept Orphan Drug Designation and Approval Years

 Figure 5-197: US – Jelmyto Orphan Drug Designation and Approval Years

 Figure 5-198: US – Mitosol Orphan Drug Designation and Approval Years

 Figure 5-199: US – Cost of Per Unit and Supply of Mitosol Kit (US$), July’2023

 Figure 5-200: US – Empliciti Orphan Drug Designation and Approval Years

 Figure 5-201: US – Cost of Per Unit and Supply of Empliciti Powders (US$), July’2023

 Figure 5-202: Empliciti – Cost of Treatment (US$), July’2023

 Figure 5-203: US – Kcentra Orphan Drug Designation and Approval Years

 Figure 5-204: US – Kyprolis Orphan Drug Designation and Approval Years by Indications

 Figure 5-205: US – Cost Per Supply of 10mg, 30mg and 60mg Kyprolis (US$), July’2023

 Figure 5-206: US – Enspryng Orphan Drug Designation and Approval Years by

 Indications

 Figure 5-207: US – Cost of Treatment with Enspryng (US$), July’2023

 Figure 5-208: US – Sirturo Orphan Drug Designation and Approval Years by Indications

 Figure 5-209: US – Cost of Per Unit and Supply of 20mg and 100mg Sirturo Tablets (US$),

 July’2023

 Figure 5-210: US – Tavalisse Orphan Drug Designation and Approval Years by

 Indications

 Figure 5-211: US – Cost of Per Unit and Supply of Tavalisse Tablets (US$), July’2023

 Figure 5-212: US – Gleolan Orphan Drug Designation and Approval Years by Indications

 Figure 5-213: US – Keveyis Orphan Drug Designation and Approval Years

 Figure 5-214: US – Cost of Per Unit and Supply of Keveyis Tablets (US$), July’2023

 Figure 5-215: US – Zolgensma Orphan Drug Designation and Approval Years

 Figure 5-216: US – Darzalex Orphan Drug Designation and Approval Years

 Figure 5-217: US – Cost of Per Unit and Supply of Darzalex Solution (US$), July’2023

 Figure 5-218: US – Darzalex Orphan Drug Designation and Approval Years

 Figure 5-219: US – Doptelet Orphan Drug Designation and Approval Years

 Figure 5-220: US – Cost of Per Unit and Supply of Doptelet Tablet (US$), July’2023

 Figure 5-221: US – Endari Orphan Drug Designation and Approval Years

 Figure 5-222: US – Cost of Per Unit and Supply of Endari Powder (US$), July’2023

 Figure 5-223: US – NutreStore Orphan Drug Designation and Approval Years

 Figure 5-224: US – Cost of Per Unit and Supply of NutreStore (US$), July’2023

 Figure 5-225: US – Lymphoseek Orphan Drug Designation and Approval Years

 Figure 5-226: US – Krystexxa Orphan Drug Designation and Approval Years

 Figure 5-227: US – Cost of Treatment with Krystexxa (US$), July’2023

 Figure 5-228: US – Krystexxa Orphan Drug Designation and Approval Years

 Figure 5-229: US – Cost of Per Unit and Supply of Korlym (US$), July’2023

 Figure 5-230: US – Monthly and Yearly Korlym Treatment Cost (US$), July’2023

 Figure 5-231: US – Kalbitor Orphan Drug Designation and Approval Years

 Figure 5-232: US – Livtencity Orphan Drug Designation and Approval Years

 Figure 5-233: US – Cost of Per Unit and Supply of Livtencity (US$), July’2023

 Figure 5-234: US – Monthly and Yearly Korlym Treatment Cost (US$), July’2023

 Figure 5-235: US – Enjaymo Orphan Drug Designation and Approval Years

 Figure 5-236: US – Cost of Per Unit and Supply of Enjaymo (US$), July’2023

 Figure 5-237: US – Rethymic Orphan Drug Designation and Approval Years

 Figure 5-238: US – Coartem Orphan Drug Designation and Approval Years

 Figure 5-239: US – Cost of Per Unit and Supply of Coartem (US$), July’2023

 Figure 5-240: US – Nulibry Orphan Drug Designation and Approval Years

 Figure 5-241: US – Inmazeb Orphan Drug Designation and Approval Years

 Figure 5-242: US – Valtoco Orphan Drug Designation and Approval Years

 Figure 5-243: US – Cost of Per Unit and Supply of Valtoco (US$), July’2023

 Figure 5-244: US – Detectnet Orphan Drug Designation and Approval Years

 Figure 5-245: US – Folotyn Orphan Drug Designation and Approval Years

 Figure 5-246: US – Cost of Per Unit and Supply of Folotyn (US$), July’2023

 Figure 5-247: US – Cost of Single Infusion and 6-Cycle Treatment with Folotyn US$),

 July’2023

 Figure 5-248: US – Cystadane Orphan Drug Designation and Approval Years

 Figure 5-249: US – Cost of Per Unit and Supply of Cystadane (US$), July’2023

 Figure 5-250: US – Cystaran Orphan Drug Designation and Approval Years

 Figure 5-251: US – Cost of Per Unit and Supply of Cystaran (US$), July’2023

 Figure 5-252: US – Voraxaze Orphan Drug Designation and Approval Years

 Figure 5-253: US – Cost of Treatment with Voraxaze (US$), July’2023

 Figure 5-254: US – Mepron Orphan Drug Designation and Approval Years

 Figure 5-255: US – Cost of Per Unit and Supply of Mepron (US$), July’2023

 Figure 5-256: US – Vistogard Orphan Drug Designation and Approval Years

 Figure 5-257: US – Cost of Per Unit and Supply of Mepron (US$), July’2023

 Figure 5-258: US – Xuriden Orphan Drug Designation and Approval Years

 Figure 5-259: US – Cost of Per Unit and Supply of Mepron (US$), July’2023

 Figure 5-260: US – Xatmep Orphan Drug Designation and Approval Years

 Figure 5-261: US – Cost of Per Unit and Supply of Xatmep (US$), July’2023

 Figure 5-262: US – Purixan Orphan Drug Designation and Approval Years

 Figure 5-263: US – Cost of Per Unit and Supply of Purixan (US$), July’2023

 Figure 5-264: US – Cost of Per Unit and Supply of Erwinaze (US$), July’2023

 Figure 5-265: US – Ascor Orphan Drug Designation and Approval Years

 Figure 5-266: US – Cost of Per Unit and Supply of Ascor (US$), July’2023

 Figure 5-267: US – Tepadina Orphan Drug Designation and Approval Years

 Figure 5-268: US – Cost of Per Supply of Tepadina (US$), July’2023

 Figure 5-269: US – BAT Orphan Drug Designation and Approval Years

 Figure 5-270: US – Busulfex Orphan Drug Designation and Approval Years

 Figure 5-271: US – Cost of Per Unit and Supply of Busulfex (US$), July’2023

 Figure 5-272: US – Lipiodol Orphan Drug Designation and Approval Years

 Figure 5-273: US – Cost of Per Unit and Supply of Lipiodol (US$), July’2023

 Figure 5-274: US – Doxil Orphan Drug Designation and Approval Years

 Figure 5-275: US – Cost of Per Unit and Supply of Doxil (US$), July’2023

 Figure 5-276: US – Nymalize Orphan Drug Designation and Approval Years

 Figure 5-277: US – Cost of Per Unit and Supply of 30 mg/5 mL Nymalize (US$),

 July’2023

 Figure 5-278: US – Cost of Per Unit and Supply of 60 mg/10 mL Nymalize (US$),

 July’2023

 Figure 5-279: US – Priftin Orphan Drug Designation and Approval Years

 Figure 5-280: US – Cost of Per Unit and Supply of Priftin (US$), July’2023

 Figure 5-281: US – Gralise Orphan Drug Designation and Approval Years

 Figure 5-282: US – Cost of Per Unit and Supply of Priftin (US$), July’2023

 Figure 5-283: US – Gralise Orphan Drug Designation and Approval Years

 Figure 5-284: US – Cost of Per Unit and Supply of Galzin (US$), July’2023

 Figure 5-285: US – NeoProfen Orphan Drug Designation and Approval Years

 Figure 5-286: US – Cost of Per Unit and Supply of NeoProfen (US$), July’2023

 Figure 5-287: US – Photofrin Orphan Drug Designation and Approval Years

 Figure 5-288: US – Retisert Orphan Drug Designation and Approval Years

 Figur