Market Research Logo

Breakthrough Therapies: Market Dynamics and Investment Opportunities

Breakthrough Therapies: Market Dynamics and Investment Opportunities

The global market for breakthrough therapy designation 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.

Report Includes
An overview of the global markets for breakthrough therapies, their dynamics, and investment opportunities
Analyses of global market trends, with data from 2015, 2016, and projections of compound annual growth rates (CAGRs) through 2022
A look at the materials used in the industry ranging from inorganic clays and concrete to iron and steel, and to commodity and specialty polymers
The challenges and opportunities of developing breakthrough therapies
A compare and contrast of different fast track drug development approaches (logistics, criteria and implications) and the potential risk and rewards of gaining break through designation
Analysis of the regulatory environment
Profiles of major players in the industry


This report highlights the challenges and opportunities of developing breakthrough therapies, it compares and contrasts difference fast track drug development approaches (logistics, criteria, and implications) and the potential risk and rewards of gaining BTD. It analyzes BTD in different therapy areas (cardiovascular, central nervous systems and neurology, rare diseases, oncology and other diseases), leading companies, approved and pipeline products, pricing market access and product revenues. It reviews BTD successes and failures and discusses the different business strategies that companies have adopted in order to maximize the competitive advantage of BTD. It summarizes the current regulatory framework and the potential application of BTD in other regions of the world, and the potential to combine BTD with early access to medicine schemes to improve patient access to medicine to treat rare diseases and address areas of high unmet clinical need.

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
SUMMARY TABLE GLOBAL MARKET FOR BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY THERAPY, THROUGH 2022 ($ MILLIONS)
SUMMARY FIGURE GLOBAL MARKET FOR BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY THERAPY, 2015-2022 ($ MILLIONS)
CHAPTER 3 OVERVIEW
DEFINING BREAKTHROUGH THERAPY DESIGNATION
QUALIFYING CRITERIA FOR BREAKTHROUGH THERAPY DESIGNATION
Serious Condition
Existing (or Available) Therapies
Preliminary Clinical Evidence
May Demonstrate Substantial Improvement on Clinically Significant Endpoint(s)
THE BENEFITS OF BREAKTHROUGH THERAPY DESIGNATION
FDA'S APPROACHES TO EXPEDITED DRUG DEVELOPMENT
DRUGS GRANTED BREAKTHROUGH THERAPY DESIGNATION
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, 2014–SEPTEMBER 30, 2015
FIGURE 1 BREAKTHROUGH THERAPY REQUESTS GRANTED BY DIVISION, 2015* (%)
PRIME (PRIORITY MEDICINE) SCHEME
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
TABLE 3 DRUGS GRANTED PRIME DESIGNATIONS, MARCH 7, 2016-AUGUST 24, 2016
CHAPTER 4 COMMERCIAL APPLICATION OF BREAKTHROUGH THERAPY
DESIGNATION
INTRODUCTION
FIGURE 5 FDA APPROVALS, BREAKTHROUGH THERAPY DESIGNATION AND DESIGNATION RESCINDED, BY STATUS, 2012-2016 (NUMBER)
FIGURE 6 FDA APPROVALS, BREAKTHROUGH DESIGNATION AND DESIGNATION RESCINDED, BY THERAPY AREA, 2012-2016
(NUMBER)
CARDIOVASCULAR
FIGURE 7 CARDIOVASCULAR BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY STAGE (NUMBER)
TABLE 4 GLOBAL MARKET FOR CV BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
CV - BTD DRUGS APPROVED
TABLE 5 FDA APPROVED CV BREAKTHROUGH THERAPY DESIGNATION
DRUGS
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
TABLE 6 GLOBAL MARKET FOR PROMACTA, THROUGH 2022 ($ MILLIONS/%)
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
TABLE 7 GLOBAL MARKET FOR PRAXBIND, THROUGH 2022 ($ MILLIONS/%)
CV - BTD IN DEVELOPMENT
TABLE 8 UNDER DEVELOPMENT CV BREAKTHROUGH THERAPY
DESIGNATION DRUGS
Reasanz (Serelaxin; Novartis)
BTD: June 21, 2013
Drug Class: Human Relaxin 2 Hormone (Biologic)
Indication: Acute Heart Failure (AHF; CV)
Clinical Stage
Forecast
TABLE 9 GLOBAL MARKET FOR REASANZ, THROUGH 2022 ($ MILLIONS/%)
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
TABLE 10 GLOBAL MARKET FOR ACTEMRA/ROACTEMRA, THROUGH 2022 ($ MILLIONS/%)
CENTRAL NERVOUS SYSTEM AND NEUROLOGICAL DISORDERS
FIGURE 8 CNS BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY STAGE, 2016 (NUMBER)
TABLE 11 GLOBAL MARKET FOR CNS BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
CNS - BTD DRUGS APPROVED
TABLE 12 FDA APPROVED CNS BREAKTHROUGH THERAPY DESIGNATION
DRUGS
Nuplazid (Primavanserin: Acadia Pharmaceuticals)
BTD: February 9, 2014
Drug Class: Selective Serotonin Inverse Agonist (Small Molecule)
Indication: Major Depressive Disorder
Clinical Stage: Approved
Forecast
TABLE 13 GLOBAL MARKET FOR NUPLAZID, THROUGH 2022 ($ MILLIONS/%)
CNS - BTD IN DEVELOPMENT
TABLE 14 UNDER DEVELOPMENT CNS BREAKTHROUGH THERAPY DESIGNATION DRUGS
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
TABLE 15 GLOBAL MARKET FOR DEUTETRABENAZINE ER, THROUGH 2022 ($ MILLIONS/%)
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
Kinect 2 - Phase 2b
Kinect 3 - Phase 3
Forecast
TABLE 16 GLOBAL MARKET FOR INGREZZA, THROUGH 2022 ($ MILLIONS/%)
Arzerra (Ofatumumab; Novartis/Genmab)
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
TABLE 17 GLOBAL MARKET FOR OF OFATUMUMAB, THROUGH 2022 ($ MILLIONS/%)
Oliceridine (TRV130; Trevena)
BTD: February 22, 2016
Drug Class: M Opioid Receptor Agonist (Small Molecule)
Indication
Clinical Stage: Phase 3
Forecast
TABLE 18 GLOBAL MARKET FOR OLICERIDINE, THROUGH 2022 ($ MILLIONS/%)
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
TABLE 19 GLOBAL MARKET FOR RAPASTINEL, THROUGH 2022 ($ MILLIONS/%)
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
TABLE 20 GLOBAL MARKET FOR SEPRANOLONE, THROUGH 2022 ($ MILLIONS/%)
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
TABLE 21 GLOBAL MARKET FOR SND-13, THROUGH 2022 ($ MILLIONS/%)
AVXS-101 (AveXis)
BTD: July 20, 2016
Drug Class: Gene Therapy (Biologic)
Indication: Spinal Muscular Atrophy (SMA; Rare Disease)
Clinical Stage: Phase 1
Forecast
TABLE 22 GLOBAL MARKET FOR AVXS-101, THROUGH 2022 ($ MILLIONS/%)
Esketamine (Johnson & Johnson)
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
TABLE 23 GLOBAL MARKET FOR ESKETAMINE, THROUGH 2022 ($ MILLIONS/%)
RBP-8000 (Indivior)
BTD: November 18, 2014
Drug Class: Cocaine Esterase Rescue Blocker (Small Molecule)
Indication: Cocaine Abuse
Clinical Stage: Phase 2
Forecast
TABLE 24 GLOBAL MARKET FOR RBP-8000, THROUGH 2022 ($ MILLIONS/%)
INFECTIOUS DISEASES
FIGURE 9 INFECTIOUS DISEASE BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY STAGE, 2016 (NUMBER)
TABLE 25 GLOBAL MARKET FOR INFECTIOUS DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
INFECTIOUS DISEASES - BTD APPROVED
TABLE 26 FDA APPROVED INFECTIOUS DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, 2016
Sovaldi (Gilead Sciences)
BTD: December 6, 2013
Drug Class: Nucleotide Analog Inhibitor (Small Molecule)
Indication: Hepatitis C Virus (Infectious Diseases)
Clinical Stage: Approved
Forecast
TABLE 27 GLOBAL MARKET FOR SOLVADI, THROUGH 2022 ($ MILLIONS/%)
Epclusa (Gilead Sciences)
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
TABLE 28 GLOBAL MARKET FOR EPCLUSA, THROUGH 2022 ($ MILLIONS/%)
Technivie/Viekirax (AbbVie)
BTD: June 30, 2014
Drug Class: Fixed Dose Triple Antiviral (Small Molecule)
Indication: Hepatitis C Virus (Infectious Disease)
Clinical Stage: Phase 3
Forecast
TABLE 29 GLOBAL MARKET FOR VIEKERA/TECHNIVIE, THROUGH 2022 ($ MILLIONS/%)
Zepatier (Elbasvir/Grazoprevir; Merck)
BTD: October 1, 2013
Drug Class: NS5A Inhibitor, NS3/4A Protease Inhibitor (Small Molecule)
Indication: Hepatitis C Virus (Infectious Disease)
Clinical Stage: Approved
Forecast
TABLE 30 GLOBAL MARKET FOR ZEPATIER, THROUGH 2022 ($ MILLIONS/%)
Harvoni (Gilead Sciences)
BTD: October 10, 2014
Drug Class: NS5A Inhibitor NS5B Inhibitor (Small Molecule)
Indication: Hepatitis C Virus (Infectious Disease)
Clinical Stage: Approved
Forecast
TABLE 31 GLOBAL MARKET FOR HARVONI, THROUGH 2022 ($ MILLIONS/%)
INFECTIOUS DISEASES - BTD IN DEVELOPMENT
TABLE 32 UNDER DEVELOPMENT INFECTIOUS DISEASE BREAKTHROUGH THERAPY DESIGNATION DRUGS, 2016
Arikayce (Amikacin Sulfate: Insmed)
BTD: June 20, 2014
Drug Class: Aminoglycoside Antibiotic. (Small Molecule)
Indication: Nontuberculous Mycobacterial (NTM; Infectious Disease)
Clinical Stage: Phase 3
Forecast
TABLE 33 GLOBAL MARKET FOR ARIKAYCE, THROUGH 2022 ($ MILLIONS/%)
Fostemsavir (ViiV Healthcare)
BTD: July 22, 2015
Drug Class: Fusion Inhibitor (Small Molecule)
Indication: Human Immunodeficiency Virus (HIV)
Clinical Stage: Phase 3
Forecast
TABLE 34 GLOBAL MARKET FOR FOSTEMSAVIR, THROUGH 2022 ($ MILLIONS/%)
Glecaprevir/Pibrentasvir (ABT-493/ABT-530; AbbVie/Enanta Pharmaceuticals)
BTD: September 30, 2016
Drug Class: NS3 Protein Inhibitor (Small Molecule)
Indication: Hepatitis C Virus (HCV; Infectious Disease)
Clinical Stage: Phase 2
Forecast
TABLE 35 GLOBAL MARKET FOR GLECAPREVIR/PIBRENTASVIR, THROUGH 2022 ($ MILLIONS/%)
Ibalizumab (TaiMed Biologics/Theratechnologies)
BTD: February 2015
Drug Class: CD4 mAb (Biologic)
Indication: Human Immunodeficiency Virus (HIV)
Clinical Stage: Phase 3
Forecast
TABLE 36 GLOBAL MARKET FOR IBALIZUMAB, THROUGH 2022 ($ MILLIONS/%)
Tafenoquine (GlaxoSmithKline/MMV)
BTD: 20 December, 2013
Drug Class: 8-Aminoquinoline Derivative (Small Molecule)
Indication: Hepatitis C Virus (HCV; Infectious Disease)
Clinical Stage: Phase 2
Forecast
TABLE 37 GLOBAL MARKET FOR TAFENOQUINE, THROUGH 2022 ($ MILLIONS/%)
V920 (Merck & Co./NewLink Genetics Corporation)
BTD: 25, July 2016
Drug Class: Ebola Vaccine (Biologic)
Indication: Ebola Zane (Infectious Disease)
Clinical Stage: Phase 3
Forecast
TABLE 38 GLOBAL MARKET FOR V920, THROUGH 2022 ($ MILLIONS/%)
ONCOLOGY
FIGURE 10 CANCER DRUGS WITH BREAKTHROUGH THERAPY DESIGNATION, BY CANCER TYPE, 2016 (NUMBER OF APPROVED BTD DRUGS)
FIGURE 11 CANCER BREAKTHROUGH THERAPY DESIGNATION, BY COMPANY, 2016 (NO. OF BTD IN ONCOLOGY)
TABLE 39 GLOBAL MARKET FOR ONCOLOGY BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
ONCOLOGY - BTD DRUGS APPROVED
TABLE 40 FDA APPROVED ONCOLOGY BREAKTHROUGH THERAPY DESIGNATION DRUGS, 2013-2015
Alecensa (Alectinib: Genentech/Roche)
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
Second Line Therapy - NP28761 (Study 1)
Second Line Therapy - NP28673 (Study 2)
First Line Therapy - J-ALEX Study
Forecast
TABLE 41 GLOBAL MARKET FOR ALENCENSA, THROUGH 2022 ($ MILLIONS/%)
Arzerra (Ofatumumab; GlaxoSmithKline/Genmab)
BTD: September 13, 2013
Drug Class: mAb Target CD20 (Biologic)
Indication: CLL
Clinical Stage: Approved
Forecast
TABLE 42 GLOBAL MARKET FOR ARZERRA, THROUGH 2022 ($ MILLIONS/%)
Blincyto (Blinatumomab; Amgen)
BTD: December 3, 2014
Drug Class: CD3/CD19 BiTE Antibody (Biologic)
Indication: B-Cell ALL (Oncology)
FIGURE 12 TREATMENT ALGORITHM FOR ALL PATIENTS
Clinical Stage: Approved
Forecast
TABLE 43 GLOBAL MARKET FOR BLINCYTO, THROUGH 2022 ($ MILLIONS/%)
Darzalex (Daratumumab; Genmab/Johnson & Johnson)
BTD: May 1, 2013, May 27, 2013, and July 2016
Drug Class: mAb CD38 (Biologic)
Indication: Multiple Myeloma (Oncology)
Clinical Stage: Phase 3
CASTOR Trial-Second Line
POLLUX Trial - Second Line
Forecast
TABLE 44 GLOBAL MARKET FOR DARZALEX, THROUGH 2022 ($ MILLIONS/%)
Gazyva/Gazyvaro (Obinutuzumab; Genentech/Roche)
BTD: May 2013
Drug Class: mAb TargetingCD20 (Biologic)
Indication: CLL
Clinical Stage: Approved
Forecast
TABLE 45 GLOBAL MARKET FOR GAZYVA, THROUGH 2022 ($ MILLIONS/%)
Ibrance (Palbociclib; Pfizer)
BTD: February 3, 2015
Indication: Her2 Metastatic Breast Cancer (Oncology)
Drug Class: CDK4/6 Inhibitor (Small Molecule)
Clinical Stage: Phase 3
PALOMA-2 Trial - Combination Therapy First-Line
PALOMA-3 - Combination Therapy
Forecast
TABLE 46 GLOBAL MARKET FOR IBRANCE, THROUGH 2022 ($ MILLIONS/%)
Imbruvica (Ibrutinib; Johnson & Johnson/ Pharmacyclic)
BTD: January 2013
Drug Class: BTK Inhibitor (Small Molecule)
Indication: CLL (Oncology)
Clinical Phase: Approved
Forecast
TABLE 47 GLOBAL MARKET FOR IMBRUVICA, THROUGH 2022 ($ MILLIONS/%)
Keytruda (Pembrolizumab; Merck)
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
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
Classical Hodgkin Lymphoma
Colorectal Cancer
Forecast
TABLE 48 GLOBAL MARKET FOR KEYTRUDA, THROUGH 2022 ($ MILLIONS/%)
Lartruvo (Olaratumab; Eli Lilly)
BTD: August 6, 2015
Drug Class: Platelet Derived Growth Factor Receptor Alpha Antagonist
(Small Molecule)
Indication: Soft Tissue Sarcoma
Clinical Stage: Approved
Forecast
TABLE 49 GLOBAL MARKET FOR LARTRUVO, THROUGH 2022 ($ MILLIONS/%)
Lenvima/Kisplyx (Lenvatinib; Eisai)
BTD: August 6, 2015
Drug Class: Tyrosine Kinase Inhibitor (Small Molecule)
Indication: Thyroid Cancer (Oncology)
Clinical Stage: Phase 3
Forecast
TABLE 50 GLOBAL MARKET FOR LENVIMA/KISPLYX, THROUGH 2022 ($ MILLIONS/%)
Lynparza (Olaparib; AstraZeneca)
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
TABLE 51 GLOBAL MARKET FOR LYNPARZA, THROUGH 2022 ($ MILLIONS/%)
Midostaurin (PKC412; Novartis)
BTD: February 19, 2016
Drug Class: Oral, Multi-Targeted Kinase Inhibitor (Small Molecule)
Indication: Acute Myeloid Leukemia (AML; Oncology)
Clinical Stage: Phase 3
Forecast
TABLE 52 GLOBAL MARKET FOR MIDOSTAURIN, THROUGH 2022 ($ MILLIONS/%)
Opdivo (Nivolumab; BMS)
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
TABLE 53 GLOBAL MARKET FOR OPDIVO, THROUGH 2022 ($ MILLIONS/%)
Tagrisso (Osimertinib; AstraZeneca)
BTD Date; April 24, 2104
Drug Class: EGFR- Tyrosine Kinase Inhibitor (Small Molecule)
Indication: Non-Small-Cell Lung Cancer (Oncology)
Clinical Stage: Approved
Forecast
TABLE 54 GLOBAL MARKET FOR TAGRISSO, SALES FORECASTS, THROUGH 2022 ($ MILLIONS/%)
Tafinlar/Mekinist (Dabrafenib/Trametinib; GlaxoSmithKline)
BTD: January 2014 (Monotherapy) and July 23, 2015 (Combination
Therapy)
Drug Class: BRAF Inhibitor plus MEK Inhibitor (Biologic)
Indication: NSCLC (Oncology).
Clinical Stage: Approved
Monotherapy Trial
Combination Therapy Trial
Forecast
TABLE 55 GLOBAL MARKET FOR TAFINLAR/MEKINIST, THROUGH 2022 ($ MILLIONS/%)
Tecentriq (Atezolizumab; Genentech/Roche)
BTD: January 23, 2014
Drug Class: Programmed Cell Death–1 Ligand 1 Monoclonal
Antibody (Biologic)
Indication: Metastatic Urothelial Carcinoma (Oncology)
Clinical Stage: Approved
Forecast
TABLE 56 GLOBAL MARKET FOR TECENTRIQ, THROUGH 2022 ($ MILLIONS/%)
Venclexta (Venetoclax; AbbVie/Roche/Genentech)
BTD: April 2015 and January 2016
Drug Class: Oral B-cell Lymphoma-2 Inhibitor (Small Molecule)
Indication: CLL (Oncology)
Clinical Stage: Approved
Forecast
TABLE 57 GLOBAL MARKET FOR VENCLEXTA, THROUGH 2022 ($ MILLIONS/%)
Xalkori (Crizotinib; Pfizer)
BTD: August 26, 2011
Drug Class: Anaplastic Lymphoma Kinase (ALK) Tyrosine Kinase
Receptor Inhibitor (Small Molecule)
Indication: ALK+ NSCLC (Oncology)
Clinical Stage: Approved
First-Line Therapy - Study 1
Second-Line Therapy - Study 2
Forecast
TABLE 58 GLOBAL MARKET FOR XALKORI, THROUGH 2022 ($ MILLIONS/%)
Zydelig (Idelalisib, Gilead Sciences)
BTD: November 18, 2013
Drug Class: Oral Phosphoinositide 3-Kinase (PI3K) Delta Inhibitor
(Small Molecule)
Indication: CLL
Clinical Stage: Approved
Forecast
TABLE 59 GLOBAL MARKET FOR ZYDELIG, THROUGH 2022 ($ MILLIONS/%)
Zykadia (Ceritinib; Novartis)
BTD: March 15, 2013
Drug Class: Anaplastic Lymphoma Kinase (ALK) Tyrosine Kinase
Receptor Inhibitor (Small Molecule)
Indication: ALK+ NSCLC (Oncology)
Clinical Stage: Approved
Forecast
TABLE 60 GLOBAL MARKET FOR ZYKADIA, THROUGH 2022 ($ MILLIONS/%)
ONCOLOGY - BTD IN DEVELOPMENT
TABLE 61 UNDER DEVELOPMENT ONCOLOGY BREAKTHROUGH THERAPY
DESIGNATION DRUGS
Abemaciclib (Eli Lilly)
BTD: August 8, 2015
Indication: Her2 Metastatic Breast Cancer (Oncology)
Drug Class: CDK4-6 Inhibitor (Small Molecule)
Clinical Stage: Phase 3
Forecast
TABLE 62 GLOBAL MARKET FOR ABEMACICLIB, THROUGH 2022 ($ MILLIONS/%)
Avelumab (Merck KGaA/PFizer)
BTD: November 18, 2015
Drug Class: Fully Human Monoclonal Antibody Targeting
Programmed Death Ligand-1 (Biologic)
Indication: Metastatic Merkel Cell Carcinoma (Oncology)
Clinical Stage: Phase 3 NSCL/ Phase 2 MCC/ Phase 1 Solid Tumors
Forecast
TABLE 63 GLOBAL MARKET FOR AVELUMAB, THROUGH 2022 ($ MILLIONS/%)
Brigatinib (AP26113; ARIAD Pharmaceuticals)
BTD: October 1, 2014
Drug Class: Oral Inhibitor of Anaplastic Lymphoma Kinase (ALK)
Inhibitor (Small Molecule)
Indication: ALK+ NSCLC (Oncology)
Clinical Stage: Filed
Forecast
TABLE 64 GLOBAL MARKET FOR BRIGATINIB, THROUGH 2022 ($ MILLIONS/%)
CTL019 (Novartis)
BTD: July 2014
Drug Class: Autologous Chimeric Antigen Receptor (CAR)- T Tell
Therapy targeting CD19 (Biologic)
Indication: CLL
Clinical Stage: Phase 2
Forecast
TABLE 65 GLOBAL MARKET FOR CTL019, THROUGH 2022 ($ MILLIONS/%)
CRS-207 and GVAX (Aduro Biotech)
BTD: 17 February, 2016
Drug Class: Tumor-Associated Antigen Mesothelin (Biologic)
Indication: Metastatic Pancreatic Cancer (Oncology)
Clinical Stage: Phase 2b
Forecast
TABLE 66 GLOBAL MARKET FOR CRS-207 AND GVAX PANCREAS, THROUGH 2022 ($ MILLIONS/%)
Durvalumab (MEDI4736; AstraZeneca-MedImmune)
BTD: February 17, 2016
Drug Class: Humanized Monoclonal Antibody Targeting Programmed
Death Ligand-1 (Biologic)
Indication: Metastatic Urothelial Bladder (Oncology)
Clinical Stage: Phase 3
Forecast
TABLE 67 GLOBAL MARKET FOR DURVALUMAB, THROUGH 2022 ($ MILLIONS/%)
EBV-CTL (Atara and MSKCC)
BTD: February 3, 2015
Drug Class: Allogeneic Hematopoietic Cell Transplant (Biologic)
Indication: EBV-Associated Lymphoproliferative Disease (Oncology)
Clinical Stage: Phase 3
Forecast
TABLE 68 GLOBAL MARKET FOR EBV-CTL, THROUGH 2022 ($ MILLIONS/%)
Entinostat (Syndax Pharmaceuticals)
BTD: September 11, 2013
Drug Class: Antibody Histone Deacetylase Inhibitor (Small Molecule)
FIGURE 13 ENTINOSTAT MECHANISM OF ACTION
Indication: Advanced HER2 Breast Cancer
Clinical Stage: Phase 3
Forecast
TABLE 69 GLOBAL MARKET FOR ENTINOSTAT, THROUGH 2022 ($ MILLIONS/%)
Inotuzumab Ozogamicin (Pfizer, Celltech)
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
TABLE 70 GLOBAL MARKET FOR INOTUZUMAB OZOGAMICIN, THROUGH 2022 ($ MILLIONS/%)
JCAR015 (Juno Therapeutics)
BTD: November 24, 2014
Drug Class: Autologous Chimeric Antigen Receptor (CAR) - T Cell
Therapy Targeting CD19 (Biologic)
Indication: Relapse-Refractory B-Cell ALL
Clinical Stage: Phase 1-2
Forecast
TABLE 71 GLOBAL MARKET FOR JCAR015, THROUGH 2022 ($ MILLIONS/%)
KTE-C19 (Kite Pharma)
BTD: December 7, 2015
Drug Class: Allogenic Chimeric Antigen Receptor (CAR) - T Cell
Therapy Targeting CD19 (Biologic)
Indication: Refractory, Diffuse Large B-Bell Lymphoma (DLBCL; Oncology)
Clinical Stage: Phase 2
Forecast
TABLE 72 GLOBAL MARKET FOR KTE-C19, THROUGH 2022 ($ MILLIONS/%)
LOXO-101 (Loxo Oncology, Inc)
BTD: July 13, 2016
Drug Class: Pan Tropomyosin Receptor Kinase Inhibitor (Small Molecules)
Indication: Solid Tumors (Oncology)
Clinical Stage: Phase 2
Forecast
TABLE 73 GLOBAL MARKET FOR LOXO-101, THROUGH 2022 ($ MILLIONS/%)
Nicord (Elbit Imaging/Gamida Cell)
BTD: October 11, 2016
Drug Class: Allogeneic Hematopoietic Cell Transplant (Biologic)
Indication: haematological malignancies (Oncology)
Clinical Stage: Phases 1 and 2
Forecast
TABLE 74 GLOBAL MARKET FOR NICORD, THROUGH 2022 ($ MILLIONS/%)
Olmutinib (Boehringer Ingelheim)
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
TABLE 75 GLOBAL MARKET FOR OLMUTINIB, THROUGH 2022 ($ MILLIONS/%)
NY-ESO Peptide (Affinity Enhanced T-Cell Therapy; Adaptimmune)
BTD: February 9, 2016
Drug class: T-cell Therapy (TCR; Biologic)
Indication: Synovial Sarcoma (Oncology)
Clinical Stage: Phases 1 and 2
Forecast
TABLE 76 GLOBAL MARKET FOR NY-ESO, THROUGH 2022 ($ MILLIONS/%)
Pexidartinib (Daiichi Sankyo/Plexxikon)
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
TABLE 77 GLOBAL MARKET FOR PEXIDARTINIB, THROUGH 2022 ($ MILLIONS/%)
Pracinostat (MEI Pharma)
BTD: August 1, 2016
Drug Class: HDAC Inhibitor (Small Molecule)
Indication: AML (Oncology)
Clinical Stage: Phase 2
Forecast
TABLE 78 GLOBAL MARKET FOR PRACINOSTAT, THROUGH 2022 ($ MILLIONS/%)
PVS-RIPO (Duke Medicine)
BTD: May 16, 2016
Drug Class: Genetically Modified Poliovirus (Biologic)
Indication: Glioblastoma (Oncology)
Clinical Stage: Phase 1
Forecast
TABLE 79 GLOBAL MARKET FOR PVS-RIPO, THROUGH 2022 ($ MILLIONS/%)
Ribociclib (LEE011; Novartis)
BTD: August 3, 2016
Indication: Her2 Metastatic Breast Cancer (Oncology)
Drug Class: CDK4-6 Inhibitor (Small Molecule)
Clinical Stage: Phase 3
Forecast
TABLE 80 GLOBAL MARKET FOR RIBOCICLIB, THROUGH 2022 ($ MILLIONS/%)
Rintega (Celldex)
BTD: February 23, 2015
Indication: Glioblastoma (Oncology)
Drug class: Cancer Vaccine (Biologic)
Clinical Stage: Discontinued
Forecast
TABLE 81 GLOBAL MARKET FOR RINTEGA, THROUGH 2022 ($ MILLIONS/%)
Rociletinib (Clovis Oncology)
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
TABLE 82 GLOBAL MARKET FOR ROCILETINIB, THROUGH 2022 ($ MILLIONS/%)
Rucaparib (Clovis Pharmaceuticals)
BTD: April 2015
Drug Class: Oral PARP Inhibitor (PARP; Small Molecule)
Indication: Ovarian Cancer (Oncology)
Clinical Stage: Phase 2
Forecast
TABLE 83 GLOBAL MARKET FOR RUCAPARIB, THROUGH 2022 ($ MILLIONS/%)
Sacituzumab Govitecan (IMMU-132; Immunomedics)
BTD: February 8, 2016
Drug Class: Antibody-Drug Conjugate (ADC; Biologic)
Indication: Triple-Negative Breast Cancer (TNBC; Oncology)
Clinical Stage: Phase 2
Forecast
TABLE 84 GLOBAL MARKET FOR SACITUZUMAB GOVITECAN, THROUGH 2022 ($ Millions/%)
Volasertib (Boehringer Ingelheim)
BTD: September 17, 2013
Drug Class: Polo-Like Kinase (Plk) Inhibitor (Biologic)
Indication: Acute Myeloid Leukemia (AML)
Clinical Stage: Phase 3
Forecast
TABLE 85 GLOBAL MARKET FOR VOLASERTIB, THROUGH 2022 ($ MILLIONS/%)
Vyxeos (Cpx-351; Celator Pharmaceuticals-Jazz Pharmaceuticals)
BTD: May 19, 2016
Drug Class: Nano-Scale Liposomal Formulation of Cytarabine: Daunorubicin (Small Molecule)
Indication: AML
Clinical Stage: Phase 3
Forecast
TABLE 86 GLOBAL MARKET FOR VYXEOS, THROUGH 2022 ($ MILLIONS/%)
RARE DISEASES
FIGURE 14 RARE DISEASE BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY STAGE, 2016
RARE DISEASES - BTD APPROVED
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 ($ MILLIONS/%)
Ilaris (Canak Inumab, Novartis)
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
TABLE 89 GLOBAL MARKET FOR ILARIS, THROUGH 2022 ($ MILLIONS/%)
Kanuma (Sebelipase Alfa; Alexion Pharmaceuticals)
BTD May 20, 2013
Drug Class: Enzyme Replacement Therapy (Biologic)
Indication: Lysosomal Acid Lipase Deficiency (Rare Disease)
Clinical Stage: Approved
Forecast
TABLE 90 GLOBAL MARKET FOR KANUMA, THROUGH 2022 ($ MILLIONS/%)
Strensiq (Asfotase Alfa; Alexion Pharmaceuticals)
BTD: May 28, 2013
Drug Class: Enzyme Replacement Therapy (Biologic)
Indication: Hypophosphatasia (Rare Disease)
Clinical Stage: Approved
Forecast
TABLE 91 GLOBAL MARKET FOR STRENSIQ, THROUGH 2022 ($ MILLIONS/%)
RARE DISEASES - BTD IN DEVELOPMENT
TABLE 92 UNDER DEVELOPMENT RARE DISEASES BREAKTHROUGH
THERAPY DESIGNATION DRUGS, 2013-2016
ALXN1101 (Alexion Pharmaceuticals)
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
TABLE 93 GLOBAL MARKET FOR ALXN-1101, THROUGH 2022 ($ MILLIONS/%)
Firdapse (Amifampridine; Catalyst Pharmaceuticals)
BTD: February 22, 2016
Drug Class: Potassium channel blocker (Small Molecule)
Indication: Lambert-Eaton Myasthenic Syndrome (LEMS; Rare Disease)
Clinical Stage: Phase 3
Forecast
TABLE 94 GLOBAL MARKET FOR FIRDAPSE, THROUGH 2022 ($ MILLIONS/%)
Olipudase Alfa (Sanofi Genzyme)
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
TABLE 95 GLOBAL MARKET FOR OLIPUDASE ALFA, THROUGH 2022 ($ MILLIONS/%)
Zorblisa (SD-101; -Amicus Therapeutics /Scioderm
BTD: April 23, 2013
Drug Class: Topical Cream (Small Molecule)
Indication: Epidermolysis Bullosa (Rare Disease)
Clinical Stage: Phase 2
Forecast
TABLE 96 GLOBAL MARKET FOR ZORBLISA, THROUGH 2022 ($ MILLIONS/%)
Setmelanotide (RM-493; Rhythm Pharmaceuticals)
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
TABLE 97 GLOBAL MARKET FOR SETMELANOTIDE, THROUGH 2022 ($ MILLIONS)
VTS-270 (Vtesse Pharmaceuticals)
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
TABLE 98 GLOBAL MARKET FOR VTS-270, THROUGH 2022 ($ MILLIONS/%)
OTHER DISEASES
FIGURE 15 OTHER DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, BY STAGE, 2016 (NUMBER)
OTHER - BTD APPROVED
TABLE 99 FDA APPROVED OTHER BREAKTHROUGH THERAPY DESIGNATION DRUGS, 2015
TABLE 100 GLOBAL MARKET FOR OTHER DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
Rapamune (Sirolimus, Pfizer)
BTD Date: May 28, 2015
Drug Class: Macrolide Lactam Immunosuppressant (Small Molecule)
Indication: Lymphangioleiomyomatosis (LAM)
Clinical Stage: Approved
Forecast
TABLE 101 GLOBAL MARKET FOR RAPAMUNE BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
Lucentis (Ranibizumab Genentech-Roche)
BTD Date: December 15, 2014
Drug Class: (EGF Inhibitor (Biologic)
Indication Diabetic Retinopathy (DR)
Clinical Stage: Approved
Forecast
TABLE 102 GLOBAL MARKET FOR LUCENTIS BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
Eylea (Aflibercept; Bayer)
BTD Date: September 16, 2014
Drug Class: VEGF Fusion Protein (Biologic)
Indication Diabetic Retinopathy (DR)
Clinical Stage: Approved
Forecast
TABLE 103 GLOBAL MARKET FOR EYLEA BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
OTHER DISEASES - BTD IN DEVELOPMENT
TABLE 104 OTHER DISEASES BREAKTHROUGH THERAPY DESIGNATION DRUGS AWAITING APPROVAL, 2015-2022
Dupilumab (Sanofi-Regeneron)
BTD Date: November 20, 2014
Drug Class: IL-4 mAb (Biologic)
Indication: Severe Atopic Dermatitis
Clinical Stage: Filed
Forecast
TABLE 105 GLOBAL MARKET FOR DUPILUMAB BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
AR101 (Aimmune Therapeutics)
BTD Date: June 18, 2015
Indication: Peanut Allergen (Small Molecule)
Clinical Stage: Phase 3
Forecast
TABLE 106 GLOBAL MARKET FOR AR101 BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
Ocaliva (Obeticholic acid; Intercept Pharmaceuticals)
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
TABLE 107 GLOBAL MARKET FOR OCALIVA BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
Viaskin Peanut (DBV Technologies)
BTD: April 9, 2015
Drug Class: Peanut Allergen (Small Molecule)
Indication: Peanut Allergen (Small Molecule)
Clinical Stage: Phase 2
Forecast
TABLE 108 GLOBAL MARKET FOR VIASKIN PEANUT BREAKTHROUGH THERAPY DESIGNATION DRUGS, THROUGH 2022 ($ MILLIONS/%)
CHAPTER 5 IMPLICATIONS OF BREAKTHROUGH THERAPY DESIGNATION
ON BUSINESS STRATEGY
INTRODUCTION
FIGURE 16 COMPARISON OF APPROVAL TIMES STANDARD VERSUS BREAKTHROUGH DESIGNATION (MONTHS)
IMPLICATIONS FOR CLINICAL DEVELOPMENT
STRATEGIES FOR EXPEDITING CLINICAL DEVELOPMENT
Process and Formulation Development
Process Validation
Analytical Development
Availability of Stability Data
IMPLICATIONS FOR MANUFACTURING
Companion Diagnostics
STRATEGIES FOR EXPEDITING MANUFACTURING CAPABILITIES
MANAGING TECHNICAL RISKS VERSUS PATIENT BENEFIT
LOOKING AHEAD
CHAPTER 6 REGULATORY IMPLICATIONS AND BREAKTHROUGH
THERAPY DESIGNATION
EXPEDITED PROGRAMS
TABLE 109 AN OVERVIEW OF THE KEY FEATURES OF THE FDA'S
EXPEDITED PROGRAMS
REGULATORY PATHWAYS
TABLE 110 AN OVERVIEW OF THE FDA'S EXPEDITED DEVELOPMENT
AND REVIEW PROGRAMS
FAST TRACK DESIGNATION
Qualifying Criteria
Serious Condition
Demonstrates the Potential to Address Unmet Medical Need
Key Features of Fast Track Designation
BREAKTHROUGH THERAPY DESIGNATION
Qualifying Criteria
Key Features
Intensive Guidance on an Efficient Drug Development Program, Beginning as early as Phase 1
TABLE 111 BREAKTHROUGH THERAPIES CONSIDERATIONS: DRUG DEVELOPMENT
Organizational Commitment Involving Senior Managers
Submission of Portions of an Application (Rolling Review)
TABLE 112 BREAKTHROUGH THERAPY CONSIDERATIONS: MARKETING APPLICATION REVIEW
Other Actions to Expedite Review
When to Make a BTD Request Submission
The BTD Review Process
FIGURE 17 COMPARISON OF STANDARD AND BREAKTHROUGH THERAPY APPROVAL TIMES (MONTHS)
Rescinding BTD Status
ACCELERATED APPROVAL
Qualifying Criteria
ACCELERATED APPROVAL ENDPOINTS
Conditions of Accelerated Approval
PRIORITY REVIEW DESIGNATION
Qualifying Criteria
Key Features
DRUGS GRANTED BREAKTHROUGH THERAPY DESIGNATION
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
SIGNIFICANCE OF BTD FOR THE PHARMACEUTICAL AND BIOTECHNOLOGY
INDUSTRIES
CHAPTER 7 COMPANY PROFILES
ABBVIE INC.
ACADIA PHARMACEUTICALS INC.
ADAPTIMMUNE THERAPEUTICS PLC
ADURO BIOTECH INC.
AIMMUNE THERAPEUTICS INC.
ALEXION PHARMACEUTICALS INC.
ALLERGAN PLC
AMGEN INC.
ANATARA LIFESCIENCES LIMITED
ARIAD PHARMACEUTICALS INC.
ASTRAZENECA PLC
AVEXIS INC.
BOEHRINGER INGELHEIM GMBH
CELLDEX THERAPEUTICS INC.
CLOVIS ONCOLOGY INC.
DAIICHI SANKYO COMPANY, LIMITED
DBV TECHNOLOGIES
EISAI CO. LTD.
ELI LILLY AND COMPANY
EXELIXIS INC.
F. HOFFMANN-LA ROCHE LTD.
GILEAD SCIENCES INC.
GLAXOSMITHKLINE PLC
IMMUNOMEDICS INC.
INDIVIDOR PLC
JAZZ PHARMACEUTICALS
JOHNSON & JOHNSON
JUNO THERAPEUTICS INC.
KITE PHARMA INC.
MERCK & CO. INC.
MERCK KGAA
NOVARTIS AG
PFIZER INC.
PHARMACYCLICS INC.
PROGENICS PHARMACEUTICALS INC.
REGENERON PHARMACEUTICALS INC.
RHYTHM PHARMACEUTICALS INC.
SYNDAX PHARMACEUTICALS INC.
SYNEURX INTERNATIONAL CORPORATION
TEVA PHARMACEUTICALS INC.
TREVENA INC.
VERTEX PHARMACEUTICALS INC.
VTESSE INC.
WELLSTAT THERAPEUTICS CORPORATION
CHAPTER 8 REFERENCES
CHAPTER 3
CHAPTER 4
CHAPTER 5
CHAPTER 6
CHAPTER 9 ACRONYMS
CHAPTER 10 ENDNOTES

Download our eBook: How to Succeed Using Market Research

Learn how to effectively navigate the market research process to help guide your organization on the journey to success.

Download eBook

Share this report