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FDA Breakthrough Therapy Designation Chart

May 20, 2013
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Updated01/29/15 for Breakthrough Therapy #60.  02/02/15 for Breakthrough Therapies #61 & #62.   02/06/15 for Breakthrough Therapy #63 (BMS’s September 2014 BTD for Opdivo (Nivolumab) that was missing).  02/23/15 for Breakthrough Therapy #64.  03/02/15 for Breakthrough Therapy #65 & #66.  04/06/15 for Breakthrough Therapy #67.  04/21/15 for Breakthrough Therapy #68.  05/06/15 for Breakthrough Therapy #69.  05/28/15 for Breakthrough Therapy #70.  06/04/15 for Breakthrough Therapy #71.

71 FDA Breakthrough Therapy Designations

The 71 Breakthrough Therapy Designations granted for drugs that have been identified by the sponsor companies (as of 06/04/15) are as follows:

# Drug Name Sponsor

 Company

Indication
1 Daratumumab Genmab/Janssen Multiple

Myeloma

(MM)

2 Ibrutinib Pharmacyclics Relapsed Or

Refractory

Mantle

Cell

Lymphoma (MCL)

3 Ibrutinib Pharmacyclics Waldenstroms

Macroglobulinemia

4 Ibrutinib Pharmacyclics Chronic or Small

Lymphocytic

Leukemia

(CLL/SLL)

5 Kalydeco Vertex Pharmaceuticals Cystic Fibrosis
6 Kalydeco + Lumacaftor (VX-809) Vertex Pharmaceuticals Cystic Fibrosis
7 Lambrolizumab Merck Melanoma
8 LDK378 Novartis ALK+

Non-Small

Cell    Lung

Cancer (NSCLC)

9 Palbociclib Pfizer Breast Cancer
10 SD-101 Scioderm Epidermolysis

Bullosa

11 3-drug regimen Bristol-Myers Squibb Hepatitis C
12 3-drug combination AbbVie Hepatitis C
13 Obinutuzumab Genentech Chronic

Lymphocytic

Leukemia

(CLL)

14 Sebelipase Alfa Synageva

BioPharma

Lysosomal

Acid   Lipase

Deficiency

15 Asfotase Alfa Alexion

Pharmaceuticals

Hypophosphatasia
16 Serelaxin Novartis Acute Heart

Failure

(AHF)

17 Drisapersen GlaxoSmithKline Duchenne

Muscular

Dystrophy

18 Bimagrumab Novartis Sporadic

Inclusion

Body Myositis

19 Sofosbuvir +Ledipasvir Gilead Sciences Hepatitis C
20 Firdapse Catalyst

Pharmaceutical

Partners

Lambert-Eaton

Myasthenic

Syndrome

21 Entinostat Syndax Pharmaceuticals Metastatic

ER+ Breast

Cancer

22 Ofatumumab Genmab/GSK Chronic

Lymphocytic

Leukemia (CLL)

23 Volasertib Boehringer

Ingelheim

Acute Myeloid

Leukemia

(AML)

24 Alectinib Roche ALK+

Non-Small

Cell Lung

Cancer

(NSCLC)

25 MK-5172/MK-8742 Merck Hepatitis C
26 Cyclic Pyranopterin     Monophosphate (cPMP) Alexion

Pharmaceuticals

Molybdenum

Cofactor

Deficiency

(MoCD) Type A

27 Andexanet Alfa Portola Pharmaceuticals Reversal of Factor Xa

Inhibitor

Anticoagulation

28 Tafenoquine GSK/Medicines for

Malaria Venture

Plasmodium

Vivax

Malaria

29 Idelalisib Gilead Sciences Relapsed

Chronic Lymphocytic

Leukemia

30 Dabrafenib GSK Non-Small

Cell Lung

Cancer With

BRAF Mutation

31 Kalydeco + VX-661 Vertex  Pharmaceuticals Cystic Fibrosis
32 Eltrombopag GSK Severe

Aplastic

Anemia

33 Sofosbuvir Gilead Sciences Hepatitis C
34 Daclatasvir + Asunaprevir Bristol-Myers Squibb Hepatitis C
35 Bivalent rLP2086 Vaccine Pfizer Meningococcal B

Disease

36 Bexsero Vaccine Novartis Meningococcal B

Disease

37 Mydicar Celladon Corporation Reducing hospitalizations for heart failure in NYHA class III or IV chronic heart failure patients who are NAb negative
38 AZD9291 AstraZeneca Metastatic EGFR T790M mutation-positive NSCLC
39 Nivolumab Bristol-Myers Squibb Hodgkin Lymphoma
40 Elotuzumab Bristol-Myers Squibb/AbbVie Multiple Myeloma
41 CO-1686 Clovis Oncology 2nd Line EGFR Mutant NSCLC with T790M Mutation
42 MPDL3280A (anti-PDL1) Genentech Urothelial Bladder Cancer (UBC)
43 Arikayce Insmed Treatment-Refractory Nontuberculous Mycobacterial Lung Disease
44 Idarucizumab Boehringer Ingelheim Antidote for Pradaxa
45 Blinatumomab Amgen Acute Lymphoblastic Leukemia
46 Personalized Cell Therapy CTL019 Novartis Relapsed/refractory acute

lymphoblastic

leukemia (r/r ALL)

47 Nintedanib Boehringer Ingelheim Idiopathic Pulmonary Fibrosis (IPF)
48 Pirfenidone InterMune Idiopathic Pulmonary Fibrosis (IPF)
49 CRS-207 + GVAX Pancreas Aduro BioTech Pancreatic Cancer
50 Nuplazid (Pimavanserin) ACADIA Pharmaceuticals Parkinson’s Disease Psychosis
51 Eylea (Aflibercept) Regeneron Pharmaceuticals Diabetic Retinopathy in Patients with Diabetic Macular Edema
52 AP26113 Ariad Pharmaceuticals ALK+ Non-Small Cell Lung Cancer (NSCLC) Resistant to Crizotinib
53 Keytruda (Pembrolizumab) Merck EGFR-Negative & ALK Rearrangement-Negative NSCLC
54 NBI-98854 Neurocrine Biosciences Tardive Dyskinesia
55 SPK–‐RPE65 (Gene Therapy) Spark Therapeutics Nyctalopia (night blindness) for Leber’s congenital amaurosis
56 Dupilumab Regeneron Pharmaceuticals/ Sanofi Atopic Dermatitis
57 Immunotherapy JCAR015 Juno Therapeutics Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia (r/r ALL)
58 Ixazomib Takeda Pharmaceutical Relapsed/Refractory Systemic Light-chain Amyloidosis
59 Lucentis Genentech (Roche) Diabetic Retinopathy
60 Obeticholic Acid (OCA) Intercept Pharmaceuticals Nonalcoholic Steatohepatitis (NASH)
61 MPDL3280A (anti-PDL1) Genentech PD-L1+ NSCLC
62 LeniGlobin BB305 (Gene Therapy) Bluebird bio Beta-Thalassemia Major
63 Nivolumab Bristol-Myers Squibb Melanoma
64 Rindopepimut (Rintega) Celldex Therapeutics EGFRvIII-positive Glioblastoma
65 Ibalizumab TaiMed Biologics HIV/AIDS
66 Epstein-Barr Virus (EBV) Targeted T-Cells Memorial Sloan Kettering Cancer Center/ Atara Biotherapeutics EBV-Associated Lymphoproliferative Disease
67 Rucaparib Clovis Oncology Advanced Ovarian Cancer with BRCA-Mutated Tumors
68 Crizotinib (Xalkori) Pfizer ROS1+ NSCLC
69 Venetoclax AbbVie CLL with 17p Deletion Gene Mutation
70 Sirolimus (Rapamune) Pfizer LAM
71 Olipudase Alfa Genzyme Nonneurological Manifestations of Niemann-Pick Disease Type B

.

The Column in the Chart titled “Drug Name” contains the URL for the link to the Press Release announcing the FDA Breakthrough Therapy Designation for each drug.

The FDA doesn’t disclose information regarding sponsors who submitted requests for or who have been granted or denied breakthrough therapy designations. Per the FDA website,  “Breakthrough therapy designation requests are typically submitted to an IND, and the FDA cannot disclose the existence of an IND, or any submissions that have been submitted to the IND, unless it has previously been publicly disclosed or acknowledged per 21 CFR 312.130(a).”

References

FDA BTD Chart

FDA BTD Approval Chart

FDA BTD Statistics Chart.

Please Note: FDA Official Logo from FDA website.

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

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