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Breakthrough Therapies: Market Dynamics and Investment Opportunities

Published By :

BCC Research

Published Date : Jan 2017

Category :

Pharmaceutical

No. of Pages : 250 Pages


The global market for breakthrough therapy designation (BTD) drugs should reach $99.2 billion by 2022 from $48.8 billion in 2017 at a compound annual growth rate (CAGR) of 15.2%, from 2017 to 2022.
The cancer therapy segment of the breakthrough therapy designation drugs industry is the largest market. The market is expected to grow from $19.6 billion in 2017 to $58.6 billion in 2022 at a CAGR of 24.5% for the period 2017-2022.
The CNS and neurology therapy segment of the breakthrough therapy designation drugs industry is expected to grow from $956 million in 2017 to $8.4 billion in 2022 at a CAGR of 54.3% for the period 2017-2022.

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Chapter 1 INTRODUCTION
STUDY GOALS AND OBJECTIVES
REASONS FOR DOING THE REPORT
SCOPE OF REPORT
INTENDED AUDIENCE
METHODOLOGY AND INFORMATION SOURCES
ANALYSTS' CREDENTIALS
RELATED BCC RESEARCH REPORTS
BCC RESEARCH WEBSITE
DISCLAIMER
Chapter 2 SUMMARY
Chapter 3 OVERVIEW
DEFINING BREAKTHROUGH THERAPY DESIGNATION
QUALIFYING CRITERIA FOR BREAKTHROUGH THERAPY DESIGNATION
THE BENEFITS OF BREAKTHROUGH THERAPY DESIGNATION
FDA'S APPROACHES TO EXPEDITED DRUG DEVELOPMENT
DRUGS GRANTED BREAKTHROUGH THERAPY DESIGNATION
PRIME (PRIORITY MEDICINE) SCHEME
Chapter 4 COMMERCIAL APPLICATION OF BREAKTHROUGH THERAPY DESIGNATION
INTRODUCTION
Serious Condition
Existing (or Available) Therapies
Preliminary Clinical Evidence
May Demonstrate Substantial Improvement on Clinically Significant Endpoint(s)
CARDIOVASCULAR
CENTRAL NERVOUS SYSTEM AND NEUROLOGICAL DISORDERS
INFECTIOUS DISEASES
ONCOLOGY
CV - BTD DRUGS APPROVED
Promacta/Revolade (Eltrombopag Olamine: Novartis/GlaxoSmithKline)
BTD: February 6, 2014
Drug Class: Thrombopoietin Receptor Agonist (Small Molecule)
Indication: Severe Aplastic Anemia (SAA; CV)
Clinical Stage: Approved
Forecast
Praxbind (Idarucizumab; Boehringer Ingelheim)
BTD: June 30, 2014
Drug Class: Humanized Antibody Fragment (Biologic)
Indication: Reversal of Bleeding and Clotting Associated with Pradaxa (CV)
Clinical Stage: Approved
Forecast
CV - BTD IN DEVELOPMENT
Reasanz (Serelaxin; Novartis)
BTD: June 21, 2013
Drug Class: Human Relaxin 2 Hormone (Biologic)
Indication: Acute Heart Failure (AHF; CV)
Clinical Stage
Forecast
Actemra/RoActemra (Tocilizumab; Roche)
BTD: June 10, 2015, and August 5, 2016
Drug Class: Humanized Anti Interleukin L-6 Receptor mAb (Biologic)
Indication: Systemic Sclerosis and Giant Cell Arteritis (CNS/Rare Disease)
Clinical Stage: Approved/Phase 3 BTD
SSC Trial
CGA Trials
Forecast
CNS - BTD DRUGS APPROVED
Nuplazid (Primavanserin: Acadia Pharmaceuticals)
BTD: February 9, 2014
Drug Class: Selective Serotonin Inverse Agonist (Small Molecule)
Indication: Major Depressive Disorder
Clinical Stage: Approved
Forecast
CNS - BTD IN DEVELOPMENT
Deutetrabenazine ER (Teva Pharmaceuticals)
BTD: November 9, 2015
Drug Class: Vesicular Monoamine 2 Transporter Inhibitor (Small Molecule)
Indication: Tardive Dyskinesia (CNS)
Clinical Stage: Phase 2/3
Forecast
Ingrezza (Valbenazine; Neuroscience Biosceinces)
BTD: October 30, 2014
Drug Class: Vesicular Monoamine 2 Transporter Inhibitor (Small Molecule)
Indication: Tardive Dyskinesia (CNS)
Clinical Stage: Phase 3
Forecast
Arzerra (Ofatumumab; Novartis/Genmab)
Kinect 2 - Phase 2b
Kinect 3 - Phase 3
BTD: October 11, 2013
Drug Class: Human Monoclonal Antibody Targeting CD20-Positive B Cells (Biologic)
Indication: Relapse Remitting Multiple Sclerosis (CNS/Autoimmune)
Clinical Stage: Phase 3
Forecast
Oliceridine (TRV130; Trevena)
BTD: February 22, 2016
Drug Class: M Opioid Receptor Agonist (Small Molecule)
Indication
Clinical Stage: Phase 3
Forecast
Rapastinel (GLYX-13; Allergan)
BTD: January 31, 2016
Drug Class: NMDA Partial Receptor Antagonist (Small Molecule)
Indication: Major Depressive Disorder (CNS)
Clinical Stage: Phase 2
Forecast
Sepranolone (UC1010; Asarina Pharma)
TD: February 22, 2016
Drug Class: GABA-A Modulating Steroid Antagonist (Small Molecule)
Indication: Premenstrual Dysphoric Disorder (PMDD)
Clinical Stage: Phase 2
Forecast
NaBen (SND-13; SyneuRx International Corporation)
BTD: December 10, 2014
Drug class: D-Amino Acid Oxidase (DAAO) Inhibitor (Small Molecule)
Indication: Schizophrenia (CNS)
Clinical Stage: Phase 2/3
Forecast
AVXS-101 (AveXis)
BTD: July 20, 2016
Drug Class: Gene Therapy (Biologic)
Indication: Spinal Muscular Atrophy (SMA; Rare Disease)
Clinical Stage: Phase 1
Forecast
Esketamine (Johnson & Johnson)
RBP-8000 (Indivior)
BTD: November 2013 and August 16, 2016
Drug Class: N-methyl-D-aspartate (NMDA) Receptor Antagonist (Small Molecule)
Indication: Major Depressive Disorder (MDD)
Clinical Stage: Phase 3
Forecast
BTD: November 18, 2014
Drug Class: Cocaine Esterase Rescue Blocker (Small Molecule)
Indication: Cocaine Abuse
Clinical Stage: Phase 2
Forecast
INFECTIOUS DISEASES - BTD APPROVED
INFECTIOUS DISEASES - BTD IN DEVELOPMENT
ONCOLOGY - BTD DRUGS APPROVED
ONCOLOGY - BTD IN DEVELOPMENT
Sovaldi (Gilead Sciences)
Epclusa (Gilead Sciences)
Technivie/Viekirax (AbbVie)
Zepatier (Elbasvir/Grazoprevir; Merck)
Harvoni (Gilead Sciences)
Arikayce (Amikacin Sulfate: Insmed)
Fostemsavir (ViiV Healthcare)
Glecaprevir/Pibrentasvir (ABT-493/ABT-530; AbbVie/Enanta Pharmaceuticals)
Ibalizumab (TaiMed Biologics/Theratechnologies)
Tafenoquine (GlaxoSmithKline/MMV)
V920 (Merck & Co./NewLink Genetics Corporation)
BTD: December 6, 2013
Drug Class: Nucleotide Analog Inhibitor (Small Molecule)
Indication: Hepatitis C Virus (Infectious Diseases)
Clinical Stage: Approved
Forecast
BTD: April 22, 2014
Drug Class: Nucleotide Analog Inhibitor + Pan-Genotypic NS5A Inhibitor (Small Molecule)
Indication: Hepatitis C Virus (Infectious Disease)
Clinical Stage: Approved
Forecast
BTD: June 30, 2014
Drug Class: Fixed Dose Triple Antiviral (Small Molecule)
Indication: Hepatitis C Virus (Infectious Disease)
Clinical Stage: Phase 3
Forecast
BTD: October 1, 2013
Drug Class: NS5A Inhibitor, NS3/4A Protease Inhibitor (Small Molecule)
Indication: Hepatitis C Virus (Infectious Disease)
Clinical Stage: Approved
Forecast
BTD: October 10, 2014
Drug Class: NS5A Inhibitor NS5B Inhibitor (Small Molecule)
Indication: Hepatitis C Virus (Infectious Disease)
Clinical Stage: Approved
Forecast
BTD: June 20, 2014
Drug Class: Aminoglycoside Antibiotic. (Small Molecule)
Indication: Nontuberculous Mycobacterial (NTM; Infectious Disease)
Clinical Stage: Phase 3
Forecast
BTD: July 22, 2015
Drug Class: Fusion Inhibitor (Small Molecule)
Indication: Human Immunodeficiency Virus (HIV)
Clinical Stage: Phase 3
Forecast
BTD: September 30, 2016
Drug Class: NS3 Protein Inhibitor (Small Molecule)
Indication: Hepatitis C Virus (HCV; Infectious Disease)
Clinical Stage: Phase 2
Forecast
BTD: February 2015
Drug Class: CD4 mAb (Biologic)
Indication: Human Immunodeficiency Virus (HIV)
Clinical Stage: Phase 3
Forecast
BTD: 20 December, 2013
Drug Class: 8-Aminoquinoline Derivative (Small Molecule)
Indication: Hepatitis C Virus (HCV; Infectious Disease)
Clinical Stage: Phase 2
Forecast
BTD: 25, July 2016
Drug Class: Ebola Vaccine (Biologic)
Indication: Ebola Zane (Infectious Disease)
Clinical Stage: Phase 3
Forecast
Alecensa (Alectinib: Genentech/Roche)
Arzerra (Ofatumumab; GlaxoSmithKline/Genmab)
Blincyto (Blinatumomab; Amgen)
Darzalex (Daratumumab; Genmab/Johnson & Johnson)
Gazyva/Gazyvaro (Obinutuzumab; Genentech/Roche)
Ibrance (Palbociclib; Pfizer)
Imbruvica (Ibrutinib; Johnson & Johnson/ Pharmacyclic)
Keytruda (Pembrolizumab; Merck)
Lartruvo (Olaratumab; Eli Lilly)
Lenvima/Kisplyx (Lenvatinib; Eisai)
Lynparza (Olaparib; AstraZeneca)
Midostaurin (PKC412; Novartis)
Opdivo (Nivolumab; BMS)
Tagrisso (Osimertinib; AstraZeneca)
Tafinlar/Mekinist (Dabrafenib/Trametinib; GlaxoSmithKline)
Tecentriq (Atezolizumab; Genentech/Roche)
Venclexta (Venetoclax; AbbVie/Roche/Genentech)
Xalkori (Crizotinib; Pfizer)
Zydelig (Idelalisib, Gilead Sciences)
Zykadia (Ceritinib; Novartis)
Abemaciclib (Eli Lilly)
Avelumab (Merck KGaA/PFizer)
Brigatinib (AP26113; ARIAD Pharmaceuticals)
CTL019 (Novartis)
CRS-207 and GVAX (Aduro Biotech)
Durvalumab (MEDI4736; AstraZeneca-MedImmune)
EBV-CTL (Atara and MSKCC)
Entinostat (Syndax Pharmaceuticals)
Inotuzumab Ozogamicin (Pfizer, Celltech)
JCAR015 (Juno Therapeutics)
KTE-C19 (Kite Pharma)
LOXO-101 (Loxo Oncology, Inc)
Nicord (Elbit Imaging/Gamida Cell)
Olmutinib (Boehringer Ingelheim)
NY-ESO Peptide (Affinity Enhanced T-Cell Therapy; Adaptimmune)
Pexidartinib (Daiichi Sankyo/Plexxikon)
Pracinostat (MEI Pharma)
PVS-RIPO (Duke Medicine)
Ribociclib (LEE011; Novartis)
Rintega (Celldex)
Rociletinib (Clovis Oncology)
Rucaparib (Clovis Pharmaceuticals)
Sacituzumab Govitecan (IMMU-132; Immunomedics)
Volasertib (Boehringer Ingelheim)
Vyxeos (Cpx-351; Celator Pharmaceuticals-Jazz Pharmaceuticals)
RARE DISEASES
BTD: June 2013 and October 2016
Drug Class: Oral Inhibitor of Anaplastic Lymphoma Kinase (ALK) Inhibitor (Small Molecule)
Indication: ALK+ NSCLC (Oncology)
Clinical Stage: Approved
Forecast
Second Line Therapy - NP28761 (Study 1)
Second Line Therapy - NP28673 (Study 2)
First Line Therapy - J-ALEX Study
BTD: September 13, 2013
Drug Class: mAb Target CD20 (Biologic)
Indication: CLL
Clinical Stage: Approved
Forecast
BTD: December 3, 2014
Drug Class: CD3/CD19 BiTE Antibody (Biologic)
Indication: B-Cell ALL (Oncology)
Clinical Stage: Approved
Forecast
BTD: May 1, 2013, May 27, 2013, and July 2016
Drug Class: mAb CD38 (Biologic)
Indication: Multiple Myeloma (Oncology)
Clinical Stage: Phase 3
Forecast
CASTOR Trial-Second Line
POLLUX Trial - Second Line
BTD: May 2013
Drug Class: mAb TargetingCD20 (Biologic)
Indication: CLL
Clinical Stage: Approved
Forecast
BTD: February 3, 2015
Indication: Her2 Metastatic Breast Cancer (Oncology)
Drug Class: CDK4/6 Inhibitor (Small Molecule)
Clinical Stage: Phase 3
Forecast
PALOMA-2 Trial - Combination Therapy First-Line
PALOMA-3 - Combination Therapy
BTD: January 2013
Drug Class: BTK Inhibitor (Small Molecule)
Indication: CLL (Oncology)
Clinical Phase: Approved
Forecast
BTD: October 27, 2014, November 2, 2015, April 18, 2016
Drug Class: Anti-PD-1 Monoclonal Antibody (Biologic)
Indication: Solid Tumors (Oncology)
Clinical Stage: Approved
Classical Hodgkin Lymphoma
Colorectal Cancer
Forecast
Melanoma
KEYNOTE-001: Phase 1b
KEYNOTE-002: Phase 2
KEYNOTE-028 Study: Phase 1b
KEYNOTE-006 Study: Phase 3
NSCLC - First-Line KEYNOTE-010 Phase 2/3
BTD: August 6, 2015
Drug Class: Platelet Derived Growth Factor Receptor Alpha Antagonist (Small Molecule)
Indication: Soft Tissue Sarcoma
Clinical Stage: Approved
Forecast
BTD: August 6, 2015
Drug Class: Tyrosine Kinase Inhibitor (Small Molecule)
Indication: Thyroid Cancer (Oncology)
Clinical Stage: Phase 3
Forecast
BTD: January 27, 2016
Drug Class: Poly ADP-Ribose Polymerase Inhibitor (PARP Oncology)
Indication: Metastatic Castration-Resistant Prostate Cancer (mCRPC; Oncology)
Clinical Stage: Phase 2
Forecast
BTD: February 19, 2016
Drug Class: Oral, Multi-Targeted Kinase Inhibitor (Small Molecule)
Indication: Acute Myeloid Leukemia (AML; Oncology)
Clinical Stage: Phase 3
Forecast
BTD: April 25, 2016, and June 27, 2016
Drug Class: PD-1 Inhibitor (Small Molecule)
Indication: Solid Tumors (Oncology)
Clinical Stage: Advanced Melanoma Checkmate 067: Phase 3
Checkmate 069: Phase 2
Bladder Cancer CheckMate-032:
Advanced Squamous Non-Small Cell Lung Cancer: CheckMate -017: Phase 3
CheckMate -063: Phase 2
Second-Line Treatment of Metastatic Non-Squamous NSCLC
Metastatic Renal Cell Carcinoma CheckMate 025 Trial: Phase 3
CheckMate-057: Phase 3
Forecast
BTD Date; April 24, 2104
Drug Class: EGFR- Tyrosine Kinase Inhibitor (Small Molecule)
Indication: Non-Small-Cell Lung Cancer (Oncology)
Clinical Stage: Approved
Forecast
BTD: January 2014 (Monotherapy) and July 23, 2015 (Combination Therapy)
Drug Class: BRAF Inhibitor plus MEK Inhibitor (Biologic)
Indication: NSCLC (Oncology).
Clinical Stage: Approved
Combination Therapy Trial
Forecast
Monotherapy Trial
BTD: January 23, 2014
Drug Class: Programmed Cell Death1 Ligand 1 Monoclonal Antibody (Biologic)
Indication: Metastatic Urothelial Carcinoma (Oncology)
Clinical Stage: Approved
Forecast
BTD: April 2015 and January 2016
Drug Class: Oral B-cell Lymphoma-2 Inhibitor (Small Molecule)
Indication: CLL (Oncology)
Clinical Stage: Approved
Forecast
BTD: August 26, 2011
Drug Class: Anaplastic Lymphoma Kinase (ALK) Tyrosine Kinase Receptor Inhibitor (Small Molecule)
Indication: ALK+ NSCLC (Oncology)
Clinical Stage: Approved
Forecast
First-Line Therapy - Study 1
Second-Line Therapy - Study 2
BTD: November 18, 2013
Drug Class: Oral Phosphoinositide 3-Kinase (PI3K) Delta Inhibitor (Small Molecule)
Indication: CLL
Clinical Stage: Approved
Forecast
Chapter 5 IMPLICATIONS OF BREAKTHROUGH THERAPY DESIGNATION ON BUSINESS STRATEGY
Chapter 6 REGULATORY IMPLICATIONS AND BREAKTHROUGH THERAPY DESIGNATION
Chapter 7 COMPANY PROFILES
Chapter 8 REFERENCES
BTD: March 15, 2013
Drug Class: Anaplastic Lymphoma Kinase (ALK) Tyrosine Kinase Receptor Inhibitor (Small Molecule)
Indication: ALK+ NSCLC (Oncology)
Clinical Stage: Approved
Forecast
CHAPTER 3
CHAPTER 4
BTD: August 8, 2015
Indication: Her2 Metastatic Breast Cancer (Oncology)
Drug Class: CDK4-6 Inhibitor (Small Molecule)
CHAPTER 5
Clinical Stage: Phase 3
Forecast
BTD: November 18, 2015
Drug Class: Fully Human Monoclonal Antibody Targeting Programmed Death Ligand-1 (Biologic)
CHAPTER 6
Indication: Metastatic Merkel Cell Carcinoma (Oncology)
Clinical Stage: Phase 3 NSCL/ Phase 2 MCC/ Phase 1 Solid Tumors
Forecast
BTD: October 1, 2014
Drug Class: Oral Inhibitor of Anaplastic Lymphoma Kinase (ALK) Inhibitor (Small Molecule)
Indication: ALK+ NSCLC (Oncology)
Clinical Stage: Filed
Forecast
BTD: July 2014
Drug Class: Autologous Chimeric Antigen Receptor (CAR)- T Tell Therapy targeting CD19 (Biologic)
Indication: CLL
Clinical Stage: Phase 2
ABBVIE INC.
Forecast
BTD: 17 February, 2016
Drug Class: Tumor-Associated Antigen Mesothelin (Biologic)
ACADIA PHARMACEUTICALS INC.
Indication: Metastatic Pancreatic Cancer (Oncology)
Clinical Stage: Phase 2b
Forecast
ADAPTIMMUNE THERAPEUTICS PLC
BTD: February 17, 2016
Drug Class: Humanized Monoclonal Antibody Targeting Programmed Death Ligand-1 (Biologic)
ADURO BIOTECH INC.
Indication: Metastatic Urothelial Bladder (Oncology)
Clinical Stage: Phase 3
AIMMUNE THERAPEUTICS INC.
ALEXION PHARMACEUTICALS INC.
ALLERGAN PLC
AMGEN INC.
ANATARA LIFESCIENCES LIMITED
Forecast
ARIAD PHARMACEUTICALS INC.
BTD: February 3, 2015
ASTRAZENECA PLC
AVEXIS INC.
Drug Class: Allogeneic Hematopoietic Cell Transplant (Biologic)
BOEHRINGER INGELHEIM GMBH
Indication: EBV-Associated Lymphoproliferative Disease (Oncology)
CELLDEX THERAPEUTICS INC.
CLOVIS ONCOLOGY INC.
DAIICHI SANKYO COMPANY, LIMITED
DBV TECHNOLOGIES
EISAI CO. LTD.
Clinical Stage: Phase 3
ELI LILLY AND COMPANY
Forecast
EXELIXIS INC.
F. HOFFMANN-LA ROCHE LTD.
GILEAD SCIENCES INC.
GLAXOSMITHKLINE PLC
IMMUNOMEDICS INC.
INDIVIDOR PLC
BTD: September 11, 2013
JAZZ PHARMACEUTICALS
JOHNSON & JOHNSON
Drug Class: Antibody Histone Deacetylase Inhibitor (Small Molecule)
JUNO THERAPEUTICS INC.
KITE PHARMA INC.
Indication: Advanced HER2 Breast Cancer
Clinical Stage: Phase 3
Forecast
BTD: October 19, 2015
Drug Class: Antibody Drug Conjugate (ADC) mAb Targeting CD22 plusC Agent Inotuzumab (Biologic)
Indication: B-cell ALL
Clinical Stage: Phase 3
Forecast
BTD: November 24, 2014
Drug Class: Autologous Chimeric Antigen Receptor (CAR) - T Cell Therapy Targeting CD19 (Biologic)
MERCK & CO. INC.
Indication: Relapse-Refractory B-Cell ALL
Clinical Stage: Phase 1-2
MERCK KGAA
Forecast
NOVARTIS AG
BTD: December 7, 2015
PFIZER INC.
Drug Class: Allogenic Chimeric Antigen Receptor (CAR) - T Cell Therapy Targeting CD19 (Biologic)
Indication: Refractory, Diffuse Large B-Bell Lymphoma (DLBCL; Oncology)
PHARMACYCLICS INC.
PROGENICS PHARMACEUTICALS INC.
Forecast
REGENERON PHARMACEUTICALS INC.
RHYTHM PHARMACEUTICALS INC.
SYNDAX PHARMACEUTICALS INC.
SYNEURX INTERNATIONAL CORPORATION
TEVA PHARMACEUTICALS INC.
TREVENA INC.
VERTEX PHARMACEUTICALS INC.
Clinical Stage: Phase 2
VTESSE INC.
WELLSTAT THERAPEUTICS CORPORATION
BTD: July 13, 2016
Drug Class: Pan Tropomyosin Receptor Kinase Inhibitor (Small Molecules)
Indication: Solid Tumors (Oncology)
Clinical Stage: Phase 2
Forecast
BTD: October 11, 2016
Drug Class: Allogeneic Hematopoietic Cell Transplant (Biologic)
Indication: haematological malignancies (Oncology)
Clinical Stage: Phases 1 and 2
Forecast
BTD: December 21, 2015
Drug Class: Fully Human Monoclonal Antibody Targeting Programmed Death Ligand-1 (Biologic)
Indication: T790M Mutation NSCLC (Oncology)
Clinical Stage: Phases 1 and 2 (Approved in South Korea)
Forecast
BTD: February 9, 2016
Drug class: T-cell Therapy (TCR; Biologic)
Indication: Synovial Sarcoma (Oncology)
Clinical Stage: Phases 1 and 2
Forecast
BTD: October 30, 2015
Drug Class: Multitargeted Receptor Tyrosine Kinase (RTK) Inhibitor (Small Molecule)
Indication: Tenosynovial Giant Cell Tumor (TGCT; Oncology)
Clinical Stage: Phase 1
Forecast
BTD: August 1, 2016
Drug Class: HDAC Inhibitor (Small Molecule)
Indication: AML (Oncology)
Clinical Stage: Phase 2
Forecast
BTD: August 3, 2016
Indication: Her2 Metastatic Breast Cancer (Oncology)
Drug Class: CDK4-6 Inhibitor (Small Molecule)
Clinical Stage: Phase 3
Forecast
BTD: May 16, 2016
Drug Class: Genetically Modified Poliovirus (Biologic)
Indication: Glioblastoma (Oncology)
Clinical Stage: Phase 1
Forecast
BTD: February 23, 2015
Indication: Glioblastoma (Oncology)
Drug class: Cancer Vaccine (Biologic)
Clinical Stage: Discontinued
Forecast
BTD: August 3, 2016
Indication: Her2 Metastatic Breast Cancer (Oncology)
Drug Class: Epidermal Growth Factor Receptor Inhibitor (Small Molecule)
Indication: Non-small Cell Lung Cancer (Oncology)
Clinical Stage: Discontinued
Forecast
BTD: April 2015
Drug Class: Oral PARP Inhibitor (PARP; Small Molecule)
Indication: Ovarian Cancer (Oncology)
Clinical Stage: Phase 2
Forecast
BTD: February 8, 2016
Drug Class: Antibody-Drug Conjugate (ADC; Biologic)
Indication: Triple-Negative Breast Cancer (TNBC; Oncology)
Clinical Stage: Phase 2
Forecast
BTD: September 17, 2013
Drug Class: Polo-Like Kinase (Plk) Inhibitor (Biologic)
Indication: Acute Myeloid Leukemia (AML)
Clinical Stage: Phase 3
Forecast
BTD: May 19, 2016
Drug Class: Nano-Scale Liposomal Formulation of Cytarabine: Daunorubicin (Small Molecule)
Indication: AML
Clinical Stage: Phase 3
Forecast
RARE DISEASES - BTD APPROVED
Ilaris (Canak Inumab, Novartis)
Kanuma (Sebelipase Alfa; Alexion Pharmaceuticals)
Strensiq (Asfotase Alfa; Alexion Pharmaceuticals)
RARE DISEASES - BTD IN DEVELOPMENT
ALXN1101 (Alexion Pharmaceuticals)
Firdapse (Amifampridine; Catalyst Pharmaceuticals)
Olipudase Alfa (Sanofi Genzyme)
Zorblisa (SD-101; -Amicus Therapeutics /Scioderm
Setmelanotide (RM-493; Rhythm Pharmaceuticals)
VTS-270 (Vtesse Pharmaceuticals)
OTHER DISEASES
BTD: April 27, 2016
Drug Class: Human Monoclonal Antibody Targeting Interleukin-1 Beta (Biologic)
Indication: PFS, TRAPS and FMF (Rare Disease)
Clinical Stage: Approved
Forecast
BTD May 20, 2013
Drug Class: Enzyme Replacement Therapy (Biologic)
Indication: Lysosomal Acid Lipase Deficiency (Rare Disease)
Clinical Stage: Approved
Forecast
BTD: May 28, 2013
Drug Class: Enzyme Replacement Therapy (Biologic)
Indication: Hypophosphatasia (Rare Disease)
Clinical Stage: Approved
Forecast
BTD: October 24, 2013
Drug Class: Enzyme Co-Factor Replacement Therapy (Biologic)
Indication: Molybdenum Cofactor Deficiency (MoCD; Rare Diseases)
Clinical Stage: Phase 2 and Phase 3
Forecast
BTD: February 22, 2016
Drug Class: Potassium channel blocker (Small Molecule)
Indication: Lambert-Eaton Myasthenic Syndrome (LEMS; Rare Disease)
Clinical Stage: Phase 3
Forecast
BTD: June 4, 2014
Drug Class: Enzyme Replacement Therapy (Biologic)
Indication: Acid Sphingomyelinase Deficiency-Niemann-Pick Disease Type B
Clinical Stage: Phase 2 and Phase 3
Forecast
BTD: April 23, 2013
Drug Class: Topical Cream (Small Molecule)
Indication: Epidermolysis Bullosa (Rare Disease)
Clinical Stage: Phase 2
Forecast
BTD: January 7, 2016
Drug Class: Melanocortin-4 Receptor (MC4R) Agonist (Small Molecule)
Indication: Pro-Opiomelanocortin (POMC) Deficiency Obesity (Rare Diseases)
Clinical Stage: Phase 2
Forecast
BTD: January 6, 2016
Drug Class: 2-Hydroxypropyl-B-Cyclodextrin (Small Molecule)
Indication: Niemann-Pick Type C1 (NPC) Disease (Rare Disease)
Clinical Stage: Phase 2 and Phase 3
Forecast
OTHER - BTD APPROVED
Rapamune (Sirolimus, Pfizer)
Lucentis (Ranibizumab Genentech-Roche)
Eylea (Aflibercept; Bayer)
OTHER DISEASES - BTD IN DEVELOPMENT
Dupilumab (Sanofi-Regeneron)
AR101 (Aimmune Therapeutics)
Ocaliva (Obeticholic acid; Intercept Pharmaceuticals)
Viaskin Peanut (DBV Technologies)
BTD Date: May 28, 2015
Drug Class: Macrolide Lactam Immunosuppressant (Small Molecule)
Indication: Lymphangioleiomyomatosis (LAM)
Clinical Stage: Approved
Forecast
BTD Date: December 15, 2014
Drug Class: (EGF Inhibitor (Biologic)
Indication Diabetic Retinopathy (DR)
Clinical Stage: Approved
Forecast
BTD Date: September 16, 2014
Drug Class: VEGF Fusion Protein (Biologic)
Indication Diabetic Retinopathy (DR)
Clinical Stage: Approved
Forecast
BTD Date: November 20, 2014
Drug Class: IL-4 mAb (Biologic)
Indication: Severe Atopic Dermatitis
Clinical Stage: Filed
Forecast
BTD Date: June 18, 2015
Indication: Peanut Allergen (Small Molecule)
Clinical Stage: Phase 3
Forecast
EXPEDITED PROGRAMS
BTD Date: January 1, 2015
Drug Class: Farnesoid X Receptor Agonist (Small Molecule)
Indication: Nonalcoholic Steatohepatitis (NASH) with Liver Fibrosis (Rare Disease)
Clinical Stage: Phase 3
Forecast
BTD: April 9, 2015
Drug Class: Peanut Allergen (Small Molecule)
SIGNIFICANCE OF BTD FOR THE PHARMACEUTICAL AND BIOTECHNOLOGY INDUSTRIES
Indication: Peanut Allergen (Small Molecule)
Clinical Stage: Phase 2
Forecast
INTRODUCTION
IMPLICATIONS FOR CLINICAL DEVELOPMENT
STRATEGIES FOR EXPEDITING CLINICAL DEVELOPMENT
IMPLICATIONS FOR MANUFACTURING
STRATEGIES FOR EXPEDITING MANUFACTURING CAPABILITIES
MANAGING TECHNICAL RISKS VERSUS PATIENT BENEFIT
LOOKING AHEAD
Process and Formulation Development
Process Validation
Analytical Development
Availability of Stability Data
Companion Diagnostics
Chapter 9 ACRONYMS
Chapter 10 ENDNOTES
REGULATORY PATHWAYS
FAST TRACK DESIGNATION
BREAKTHROUGH THERAPY DESIGNATION
ACCELERATED APPROVAL
ACCELERATED APPROVAL ENDPOINTS
PRIORITY REVIEW DESIGNATION
DRUGS GRANTED BREAKTHROUGH THERAPY DESIGNATION
Qualifying Criteria
Key Features of Fast Track Designation
Qualifying Criteria
Key Features
When to Make a BTD Request Submission
The BTD Review Process
Rescinding BTD Status
Qualifying Criteria
Conditions of Accelerated Approval
Qualifying Criteria
Key Features
Serious Condition
Demonstrates the Potential to Address Unmet Medical Need
Intensive Guidance on an Efficient Drug Development Program, Beginning as early as Phase 1
Organizational Commitment Involving Senior Managers
Submission of Portions of an Application (Rolling Review)
Other Actions to Expedite Review

List of Table

Summary Table : GLOBAL MARKET FOR BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY THERAPY, THROUGH 2022
Table 1 : CDER BREAKTHROUGH THERAPY DESIGNATION REQUESTS RECEIVED BY FISCAL YEAR COHORT: JULY 9, 2012*-SEPTEMBER 30, 2015
Table 2 : CBER BREAKTHROUGH THERAPY REQUESTS FOR FINAL YEAR, OCTOBER 1, 2014SEPTEMBER 30, 2015
Table 3 : DRUGS GRANTED PRIME DESIGNATIONS, MARCH 7, 2016-AUGUST 24, 2016
Table 4 : GLOBAL MARKET FOR CV BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022
Table 5 : FDA APPROVED CV BREAKTHROUGH THERAPY DESIGNATION DRUGS
Table 6 : GLOBAL MARKET FOR PROMACTA, THROUGH 2022
Table 7 : GLOBAL MARKET FOR PRAXBIND, THROUGH 2022
Table 8 : UNDER DEVELOPMENT CV BREAKTHROUGH THERAPY DESIGNATION DRUGS
Table 9 : GLOBAL MARKET FOR REASANZ, THROUGH 2022
Table 10 : GLOBAL MARKET FOR ACTEMRA/ROACTEMRA, THROUGH 2022
Table 11 : GLOBAL MARKET FOR CNS BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022
Table 12 : FDA APPROVED CNS BREAKTHROUGH THERAPY DESIGNATION DRUGS
Table 13 : GLOBAL MARKET FOR NUPLAZID, THROUGH 2022
Table 14 : UNDER DEVELOPMENT CNS BREAKTHROUGH THERAPY DESIGNATION DRUGS
Table 15 : GLOBAL MARKET FOR DEUTETRABENAZINE ER, THROUGH 2022
Table 16 : GLOBAL MARKET FOR INGREZZA, THROUGH 2022
Table 17 : GLOBAL MARKET FOR OF OFATUMUMAB, THROUGH 2022
Table 18 : GLOBAL MARKET FOR OLICERIDINE, THROUGH 2022
Table 19 : GLOBAL MARKET FOR RAPASTINEL, THROUGH 2022
Table 20 : GLOBAL MARKET FOR SEPRANOLONE, THROUGH 2022
Table 21 : GLOBAL MARKET FOR SND-13, THROUGH 2022
Table 22 : GLOBAL MARKET FOR AVXS-101, THROUGH 2022
Table 23 : GLOBAL MARKET FOR ESKETAMINE, THROUGH 2022
Table 24 : GLOBAL MARKET FOR RBP-8000, THROUGH 2022
Table 25 : GLOBAL MARKET FOR INFECTIOUS DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022
Table 26 : FDA APPROVED INFECTIOUS DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, 2016
Table 27 : GLOBAL MARKET FOR SOLVADI, THROUGH 2022
Table 28 : GLOBAL MARKET FOR EPCLUSA, THROUGH 2022
Table 29 : GLOBAL MARKET FOR VIEKERA/TECHNIVIE, THROUGH 2022
Table 30 : GLOBAL MARKET FOR ZEPATIER, THROUGH 2022
Table 31 : GLOBAL MARKET FOR HARVONI, THROUGH 2022
Table 32 : UNDER DEVELOPMENT INFECTIOUS DISEASE BREAKTHROUGH THERAPY DESIGNATION DRUGS, 2016
Table 33 : GLOBAL MARKET FOR ARIKAYCE, THROUGH 2022
Table 34 : GLOBAL MARKET FOR FOSTEMSAVIR, THROUGH 2022
Table 35 : GLOBAL MARKET FOR GLECAPREVIR/PIBRENTASVIR, THROUGH 2022
Table 36 : GLOBAL MARKET FOR IBALIZUMAB, THROUGH 2022
Table 37 : GLOBAL MARKET FOR TAFENOQUINE, THROUGH 2022
Table 38 : GLOBAL MARKET FOR V920, THROUGH 2022
Table 39 : GLOBAL MARKET FOR ONCOLOGY BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022
Table 40 : FDA APPROVED ONCOLOGY BREAKTHROUGH THERAPY DESIGNATION DRUGS, 2013-2015
Table 41 : GLOBAL MARKET FOR ALENCENSA, THROUGH 2022
Table 42 : GLOBAL MARKET FOR ARZERRA, THROUGH 2022
Table 43 : GLOBAL MARKET FOR BLINCYTO, THROUGH 2022
Table 44 : GLOBAL MARKET FOR DARZALEX, THROUGH 2022
Table 45 : GLOBAL MARKET FOR GAZYVA, THROUGH 2022
Table 46 : GLOBAL MARKET FOR IBRANCE, THROUGH 2022
Table 47 : GLOBAL MARKET FOR IMBRUVICA, THROUGH 2022
Table 48 : GLOBAL MARKET FOR KEYTRUDA, THROUGH 2022
Table 49 : GLOBAL MARKET FOR LARTRUVO, THROUGH 2022
Table 50 : GLOBAL MARKET FOR LENVIMA/KISPLYX, THROUGH 2022
Table 51 : GLOBAL MARKET FOR LYNPARZA, THROUGH 2022
Table 52 : GLOBAL MARKET FOR MIDOSTAURIN, THROUGH 2022
Table 53 : GLOBAL MARKET FOR OPDIVO, THROUGH 2022
Table 54 : GLOBAL MARKET FOR TAGRISSO, SALES FORECASTS, THROUGH 2022
Table 55 : GLOBAL MARKET FOR TAFINLAR/MEKINIST, THROUGH 2022
Table 56 : GLOBAL MARKET FOR TECENTRIQ, THROUGH 2022
Table 57 : GLOBAL MARKET FOR VENCLEXTA, THROUGH 2022
Table 58 : GLOBAL MARKET FOR XALKORI, THROUGH 2022
Table 59 : GLOBAL MARKET FOR ZYDELIG, THROUGH 2022
Table 60 : GLOBAL MARKET FOR ZYKADIA, THROUGH 2022
Table 61 : UNDER DEVELOPMENT ONCOLOGY BREAKTHROUGH THERAPY DESIGNATION DRUGS
Table 62 : GLOBAL MARKET FOR ABEMACICLIB, THROUGH 2022
Table 63 : GLOBAL MARKET FOR AVELUMAB, THROUGH 2022
Table 64 : GLOBAL MARKET FOR BRIGATINIB, THROUGH 2022
Table 65 : GLOBAL MARKET FOR CTL019, THROUGH 2022
Table 66 : GLOBAL MARKET FOR CRS-207 AND GVAX PANCREAS, THROUGH 2022
Table 67 : GLOBAL MARKET FOR DURVALUMAB, THROUGH 2022
Table 68 : GLOBAL MARKET FOR EBV-CTL, THROUGH 2022
Table 69 : GLOBAL MARKET FOR ENTINOSTAT, THROUGH 2022
Table 70 : GLOBAL MARKET FOR INOTUZUMAB OZOGAMICIN, THROUGH 2022
Table 71 : GLOBAL MARKET FOR JCAR015, THROUGH 2022
Table 72 : GLOBAL MARKET FOR KTE-C19, THROUGH 2022
Table 73 : GLOBAL MARKET FOR LOXO-101, THROUGH 2022
Table 74 : GLOBAL MARKET FOR NICORD, THROUGH 2022
Table 75 : GLOBAL MARKET FOR OLMUTINIB, THROUGH 2022
Table 76 : GLOBAL MARKET FOR NY-ESO, THROUGH 2022
Table 77 : GLOBAL MARKET FOR PEXIDARTINIB, THROUGH 2022
Table 78 : GLOBAL MARKET FOR PRACINOSTAT, THROUGH 2022
Table 79 : GLOBAL MARKET FOR PVS-RIPO, THROUGH 2022
Table 80 : GLOBAL MARKET FOR RIBOCICLIB, THROUGH 2022
Table 81 : GLOBAL MARKET FOR RINTEGA, THROUGH 2022
Table 82 : GLOBAL MARKET FOR ROCILETINIB, THROUGH 2022
Table 83 : GLOBAL MARKET FOR RUCAPARIB, THROUGH 2022
Table 84 : GLOBAL MARKET FOR SACITUZUMAB GOVITECAN, THROUGH 2022
Table 85 : GLOBAL MARKET FOR VOLASERTIB, THROUGH 2022
Table 86 : GLOBAL MARKET FOR VYXEOS, THROUGH 2022
Table 87 : FDA APPROVED RARE DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, 2015-2016
Table 88 : GLOBAL MARKET FOR RARE DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022
Table 89 : GLOBAL MARKET FOR ILARIS, THROUGH 2022
Table 90 : GLOBAL MARKET FOR KANUMA, THROUGH 2022
Table 91 : GLOBAL MARKET FOR STRENSIQ, THROUGH 2022
Table 92 : UNDER DEVELOPMENT RARE DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, 2013-2016
Table 93 : GLOBAL MARKET FOR ALXN-1101, THROUGH 2022
Table 94 : GLOBAL MARKET FOR FIRDAPSE, THROUGH 2022
Table 95 : GLOBAL MARKET FOR OLIPUDASE ALFA, THROUGH 2022
Table 96 : GLOBAL MARKET FOR ZORBLISA, THROUGH 2022
Table 97 : GLOBAL MARKET FOR SETMELANOTIDE, THROUGH 2022
Table 98 : GLOBAL MARKET FOR VTS-270, THROUGH 2022
Table 99 : FDA APPROVED OTHER BREAKTHROUGH THERAPY DESIGNATION DRUGS, 2015
Table 100 : GLOBAL MARKET FOR OTHER DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022
Table 101 : GLOBAL MARKET FOR RAPAMUNE BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022
Table 102 : GLOBAL MARKET FOR LUCENTIS BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022
Table 103 : GLOBAL MARKET FOR EYLEA BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022
Table 104 : OTHER DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS AWAITING APPROVAL, 2015-2022
Table 105 : GLOBAL MARKET FOR DUPILUMAB BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022
Table 106 : GLOBAL MARKET FOR AR101 BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022
Table 107 : GLOBAL MARKET FOR OCALIVA BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022
Table 108 : GLOBAL MARKET FOR VIASKIN PEANUT BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022
Table 109 : AN OVERVIEW OF THE KEY FEATURES OF THE FDA'S EXPEDITED PROGRAMS
Table 110 : AN OVERVIEW OF THE FDA'S EXPEDITED DEVELOPMENT AND REVIEW PROGRAMS
Table 111 : BREAKTHROUGH THERAPIES CONSIDERATIONS: DRUG DEVELOPMENT
Table 112 : BREAKTHROUGH THERAPY CONSIDERATIONS: MARKETING APPLICATION REVIEW
Table 113 : CDER BREAKTHROUGH THERAPY DESIGNATION REQUESTS RECEIVED BY FISCAL YEAR COHORT: JULY 9, 2012*-SEPTEMBER 30, 2015
Table 114 : CDER BREAKTHROUGH THERAPY DESIGNATION REQUESTS RECEIVED BY FISCAL YEAR COHORT: OCTOBER 1, 2015-SEPTEMBER 30, 2016
Table 115 : CDER BREAKTHROUGH THERAPY DESIGNATION APPROVALS DATA AS OF DECEMBER 31, 2015
Table 116 : CUMULATIVE CBER BREAKTHROUGH THERAPY DESIGNATION REQUESTS RECEIVED BY FISCAL YEAR COHORT: JULY 9, 2012*-DECEMBER 31, 2015
Table 117 : CBER BREAKTHROUGH THERAPY DESIGNATION REQUESTS RECEIVED BY FISCAL YEAR COHORT: OCTOBER 1, 2015-SEPTEMBER 30, 2016
Table 118 : CUMULATIVE CBER BREAKTHROUGH THERAPY APPROVAL DATA AS OF SEPTEMBER 30, 2016
Table 119 : MOST RECENTLY ANNOUNCED BREAKTHROUGH THERAPY DESIGNATIONS DATA AS OF FEBRUARY 29, 2016

List of Chart

Summary Figure : GLOBAL MARKET FOR BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY THERAPY, 2015-2022
Figure 1 : BREAKTHROUGH THERAPY REQUESTS GRANTED BY DIVISION, 2015*
Figure 2 : PRIME SCHEME APPLICATIONS AND ELIGIBILITY
Figure 3 : PRIME DRUG BY TYPE OF APPLICATION AND THERAPEUTIC AREA
Figure 4 : PRIME DRUG APPLICATIONS, THERAPEUTIC AREA
Figure 5 : FDA APPROVALS, BREAKTHROUGH THERAPY DESIGNATION AND DESIGNATION RESCINDED, BY STATUS, 2012-2016
Figure 6 : FDA APPROVALS, BREAKTHROUGH DESIGNATION AND DESIGNATION RESCINDED, BY THERAPY AREA, 2012-2016
Figure 7 : CARDIOVASCULAR BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY STAGE
Figure 8 : CNS BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY STAGE, 2016
Figure 9 : INFECTIOUS DISEASE BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY STAGE, 2016
Figure 10 : CANCER DRUGS WITH BREAKTHROUGH THERAPY DESIGNATION, BY CANCER TYPE, 2016
Figure 11 : CANCER BREAKTHROUGH THERAPY DESIGNATION, BY COMPANY, 2016
Figure 12 : TREATMENT ALGORITHM FOR ALL PATIENTS
Figure 13 : ENTINOSTAT MECHANISM OF ACTION
Figure 14 : RARE DISEASE BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY STAGE, 2016
Figure 15 : OTHER DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY STAGE, 2016
Figure 16 : COMPARISON OF APPROVAL TIMES STANDARD VERSUS BREAKTHROUGH DESIGNATION
Figure 17 : COMPARISON OF STANDARD AND BREAKTHROUGH THERAPY APPROVAL TIMES

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