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10 New FDA Orphan Drug Designations: Week of 06/08/15

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The chart below identifies 10 new FDA Products Receiving Orphan Designation for the week of 06/08 – 06/12/15 as of 06/13/15 in ascending “Orphan Drug Designation Date” order.

FDA Products Receiving Orphan Drug Designation (Week of 06/08 – 06/12/15)

 # Generic Name/ODD Date Sponsor Company Indication
1 Adenoviral vector expressing the E. Coli purine nucleoside phosphorylase gene and fludarabine / 06.08 PNP Therapeutics Anatomically accessible oral & pharyngeal cancers
2 Fully human IgG2 monoclonal antibody that binds insulin receptors/ 06.09 XOMA Congenital hyperinsulinism
3 Double stranded oligomer AD00370 RNA interference-based liver targeted therapeutic/ 06.09 Arrowhead Research Corporation Alpha-1 Antitrypsin Deficiency
4 Recombinant human CD24Fclg (humanized fusion protein consisting of the extracellular domain of CD24 linked to IgG Fc domain/ 06.09 Oncolmmune Graft-versus-host Disease
5 Isocitrate dehydrogenase 1 (IDH1)-mutant inhibitor/ 06.09 Agios Pharmaceuticals Acute Myeloid Leukemia (AML)
6 Tumor-inflitrating lymphocytes; LN-144/ 06.09 Lion Biotechnologies Malignant melanoma Stages IIb to IV
7 Staphylococcal aureus protein A/ 06.10 Protalex Inc Immune thrombocytopenic purpura
8 (6aR, 10aR)-3-(1’,1’-dimethylheptyl)-Δ8-tetrahydro-cannabinol-9-carboxylic acid / 06.10 Corbus Pharmaceuticals Systemic sclerosis
9 Imetelstat/ 06.11 Janssen Research & Development Myelofibrosis
10 Obinutuzumab/ 06.11 Genentech Splenic marginal zone lymphoma

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** Generic Name/ODD Date” Column Link = Is the FDA Orphan Drug Product Designation Database Record.

Please Note : “Two small test tubes held in spring clamps” courtesy of  Amitchell125 at English Wikipedia [CC-BY-SA-30] | Wikimedia Commons.

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

6 New FDA Orphan Drug Designations: Week of 06/01/15

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The chart below identifies 6 new FDA Products Receiving Orphan Designation for the week of 06/01 – 06/05/15 as of 06/07/15 in ascending “Orphan Drug Designation Date” order.

FDA Products Receiving Orphan Drug Designation (Week of 06/01 – 06/05/15)

 # Generic Name/ODD Date Sponsor Company Indication
1 2-(5-fluoro-2-methyl-1H-indol-3-yl)-1H-imidazo[4,5-f][1,10] phenanthroline/ 06.01 Aptose Biosciences (Canada) Acute Myeloid Leukemia
2 17-a-hydroxyprogesterone caporate (oral formulation)/ 06.01 Lipocine Prevention of preterm birth in women with singleton pregnancy
3 Docosahexaenoic acid/ 06.01 Sancilio and Co Short Bowel Syndrome
4 Ibudilast/ 06.01 MediciNova Krabbe Disease
5 Immunologically active synthetic peptides/ 06.01 Apitope International NV (Belgium) Hemophilia A
6 Regorafenib/ 06.04 Bayer Hepatocellular carcinoma

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** Generic Name/ODD Date” Column Link = Is the FDA Orphan Drug Product Designation Database Record.

Please Note : “Two small test tubes held in spring clamps” courtesy of  Amitchell125 at English Wikipedia [CC-BY-SA-30] | Wikimedia Commons.

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

Patient Advocacy: Top Grant-Giving Disease Foundations

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This week, GEN (Genetic Engineering & Biotechnology News), publishes an online list of the top 35 Nonprofit Disease Foundations (2015 Edition) that give grants and awards for research. The Nonprofit Disease Foundations are ranked by the “percentage of total expenses spent on these grants and awards.”

Per the article:

“The list does not constitute a complete listing of all disease foundations. Also, the list does not include Section 501(c)(3) exempt private foundations (Bill & Melinda Gates, Clinton) or institutions that care for patients as well as carry out research (Dana-Farber, Mayo Clinic) or research institutes.”

The chart below identifies the top 10 Nonprofit Disease Foundations plus the rare disease foundations ranked among the 11-35. The data in the chart below is from the online article published in GEN.

Top Nonprofit Grant-Giving Disease Foundations

GEN Rank # Disease Foundation Name % of Total Expenses Awarded As  Grants Total Scientific Awards (Approx in Millions)
1 Cancer Research Fund of the Damon Runyon-Walter Winchell Foundation 80.2% $16.881
2 The Michael J. Fox Foundation for Parkinson’s Research 79.4% $67.429
3 Brain And Behavior Research Foundation 79.1% $22.682
4 Melanoma Research Alliance Foundation 78.7% $8.485
5 Rheumatology Research Foundation 76.7% $10.983
6 Alzheimer’s Drug Discovery Foundation 60.2% $7.137
7 Brain Research Foundation 53.4% $1.254
8 Parkinson’s Disease Foundation 49.6% $4.956
9 Cystic Fibrosis Foundation 46.4% $88.286
10 JDRF (Juvenile Diabetes Research Foundation) 45.8% $98.317
11 Foundation Fighting Blindness 42.2% $12.721
13 Hereditary Disease Foundation 32.1% $0.709
15 PKD Foundation 26.0% $1.894
16 Myelin Repair Foundation 25.6% $1.649
17 The ALS Foundation 25.4% $6.652
19 Chordoma Foundation 21.67% $0.250
22 Muscular Dystrophy Association 17.42% $25.783
29 March of Dimes Foundation 10.36% $22.392
34 National Hemophilia Foundation 3.7% $0.516

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Please Note: “Kid Hugging a Rainbow” by Marendo Müller, artwork (Own work) [Public domain] | Wikimedia Commons.

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

 

FDA Breakthrough Therapy And A Rare Disease: Genzyme’s ERT Olipudase Alfa For Niemann-Pick Disease

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Genzyme, a Sanofi company, announces June 4th that the FDA grants the Breakthrough Therapy Designation (BTD) to its investigational Enzyme Replacement Therapy (ERT), Olipudase Alfa. It is being developed for the treatment of the nonneurological manifestations of the rare disease, Niemann-Pick Disease (NPD) Type B. There are currently no approved treatment options for patients with Niemann-Pick disease type B. Genzyme has initiated enrollment of a Phase I/II pediatric study. In the second half of 2015, the company is getting ready for enrolling patients for a Phase II/III adult study.

NPDs are a subgroup of Lysosomal Storage Diseases (LSDs) that affect metabolism and are caused by genetic mutations. NPD is named after the two doctors who described the symptoms in the early part of the 20th century – Dr. Albert Niemann and Dr. Luddwick Pick. NPDs are characterized by the harmful accumulation of quantities of fatty substances, or lipids, in the spleen, lungs, bone marrow, liver, and brain. The three most commonly recognized forms of NPD are:

•   NPD Types A & B (ASMD or Acid Sphingomyelinase Deficiency)

•   NPD Type C (NPD Type C).

NPD types A and B are caused by a deficiency of an enzyme called acid sphingomyelinase. The enzyme deficiency leads to enlargement of the liver and spleen, lung disease, low blood cell counts, and in some cases a shortened lifespan. Mutations in the SMPD1 gene causes NPD Types A and B, while mutations in the NPC1 and NPC2 causes NPD Type C. NPD is an autosomal recessive disorder, which means it is not sex-linked and must be inherited from two parents who carry a mutation on one copy of the Niemann-Pick gene. The incidence of NPD:

•   Among Ashkenazi Jews is  approximately 1/40,000 for Type A

•   In all other populations, it is approximately 1/250,000 for Types A & B

•   It is approximately 1/150,000 for Type C.

FDA Orphan Drug Designations For NPD

The chart below identifies FDA Orphan Drug Designations (ODDs) for NPD:

  Generic Name/ODD  Date Sponsor Company Indication
1 Acid Sphingomyelinase/ 08.03.00 Genzyme Acid Sphingomyelinase Deficiency (NPD)
2 Miglustat (Zavesca)/ 11.12.08 Actelion Pharmaceuticals Neurological Manifestations of NPD Type C
3  Hydroxy-Propyl-Beta-Cyclodextrin (Trappsol)/   05.17.10 Children’s Hospital & Research Center Oakland NPD Type C
4  2-hydroxypropyl-B-cyclodextrin (Kleptose)/   02.18.13 National Institute of Health NPD Type C
5 Allopregnanolone/07.12.13 LaJolla Pharmaceutical NPD Type C
6 Arimoclomal Citrate/ 01.13.15 OrphazymeApS (Denmark) NPD Type C

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BTD References

FDA BTD Chart

FDA BTD Approval Chart

FDA BTD Statistics Chart

Niemann-Pick Disease Organizations

Ara Parseghian Medical Research Foundation (Type C)

International Niemann-Pick Disease Alliance (INPDA)

National Niemann-Pick Disease Foundation (US)

Niemann-Pick Disease Group (UK).

Please Note: “White Pocket Reflection” by John Fowler (Flickr: White Pocket Reflection) [CC BY 2.0] | Wikimedia Commons.

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

FDA Breakthrough Therapy Designation: Statistics Chart Updated (as of 05/31/15)

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The CBER BTD #s are current as of 05/31/15 and the CDER BTD #s are current as of 05/29/15.

The changes are as follows:

•   The total # of FDA CDER + CBER BTD Requests received by the FDA increases from 304 to 308

•   The total # of FDA CDER + CBER BTDs Granted by the FDA increases from 88 to 90

•   The total # of FDA CDER + CBER BTDs Denied by the FDA increases from 163 to 169

•   The total # of FDA CDER + CBER BTDs Pending by the FDA decreases from 53 to 49.

FDA CBER BTDs + CDER BTDs (as of 05/31/15) 

Breakthrough Therapy  Designation (BTD) Category Total # of CBER Designations (07/09/12-05/31/15) Total # of CDER Designations (07/09/12-05/29/15) Total # of CBER +   CDER BTD Designations (07/09/12-05/31/15)
Total # of BTD Requests Received 50 258 308
Total # of BTDs Granted 12 78 90
Total # of BTDs   Denied 37 132 169
Total # of BTDs   Pending 1 48 49

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OBSERVATIONS

•   29.2 % of the total # of BTD Requests Received results in the BTD being granted

•   54.9 % of the total # of BTD Requests Received results in the BTD being denied

•   15.9 % of the total # of BTD Requests Received results in the BTD pending.

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.

FDA May 2015 Products Receiving Orphan Designation

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The chart below identifies FDA May 2015 Products Receiving Orphan Designation as of 06/02/15 in ascending “Orphan Drug Designation Date” order.

FDA May 2015 Products Receiving Orphan Designation

# Generic Name/ODD Date Sponsor Company Indication
1 Tacrolimus/ 05.04 Stanford University School of Medicine Pulmonary Arterial Hypertension
2 Tadalafil/ 05.04 Eli Lilly & Co Duchenne Muscular Dystrophy
3 Pantothenate Phosphate / 05.04 Retrophin Pantothenate Kinase Associated Neurodegeneration
4 Pelareorep/ 05.04 Oncolytics Biotech Gastric Cancer
5 4-(pyrimidin-2-ylmethyl)-7-[4-(trifluoromethoxy)phenyl]-3,4-dihydro-1,4-benzoxazepin-5(2H)-one/ 05.04 Gilead Sciences Congenital Long QT Syndrome
6 LOAd703, a modified adenovirus 5/35 containing a CMV promoter-driven transgene cassette with the human transgenes for a membrane-bound CD40 ligand (TMZ-CD40L) and full length 4-1BBL/ 05.04 Lokon Pharma AB (Sweden) Pancreatic Cancer
7 Humanized monoclonal antibody that targets the a-subunit of the human IL-3 receptor also known as the CD123 antigen; (JNJ-56022473)/ 05.05 Janssen Research & Development Acute Myeloid Leukemia
8 Autologous tumor cells fused with polyethylene glycol to human allogeneic FO-1, DNA plasmid transfected tumor cell line/ 05.06 Sebastiano Gattoni-Celli, MD Glioblastoma
9 Edaravone/ 05.12 Mitsubishi Tanabe Pharma Corporation (Japan) Amyotrophic Lateral Sclerosis
10 N-t-butylhydroxylamine/ 05.12 Andrew Lim, California Institute of Technology Infantile neuronal ceroid lipofuscinosis
11 Recombinant human alkaline phosphatase/ 05.13 AM-Pharma BV Hypophosphatasia
12 Anti-eTau Humanized IgG4 Monoclonal Antibody / 05.13 Bristol-Myers Squibb Progressive Supranuclear Palsy
13 E-selectin antagonist/ 05.13 GlycoMimetrics Acute Myeloid Leukemia
14 Bruton’s Tyrosine Kinase (Btk) Inhibitor/ 05.13 Acerta Pharma BV (Netherlands) Chronic Lymphocytic Leukemia
15 Hydrocortisone oral granules/ 05.13 Diurnal Limited (UK) Adrenal insufficiency ( 0 – 16 Years of Age)
16 Adalimumab/ 05.13 AbbVie Moderate to severe hidradenitis suppurativa
17 Revusiran/ 05.18 Alnylam Pharmaceuticals Transthyretin amyloidosis
18 Xenon gas / 05.18 Neuroprotexeon (UK) To improve neurological outcome in hospitalized cardiac arrest patients
19 Chloroquine/ 05.20 DualTpharma (Netherlands) Glioblastoma multiforme
20 Lutetium (177Lu)-edotreotide/ 05.21 ITG Isotope Technologies Garching GmbH Gastro-entero-pancreatic neuroendocrine tumors
21 Poly-CD-PEG-camptothecin/ 05.21 Cerulean Pharma Ovarian cancer
22 Echinomycin/ 05.21 OncoImmune Acute Myeloid Leukemia
23 Prasugrel hydrochloride/ 05.26 Eli Lilly Sickle Cell Disease
24 Adult hemogenic endothelial cells/ 05.27 HemoGenyx Aplastic Anemia

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** Generic Name/ODD Date” Column Link = Is the FDA Orphan Drug Product Designation Database Record.

Please Note: “Nessler Cylinders” by Panek (Own work) [GFDL or CC-BY-3.0 | via Wikimedia Commons.

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

Friedreich’s Ataxia: Film Premiere

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I – Introduction

Friedreich’s Ataxia (FA) is a degenerative neuro-muscular disorder that affects approximately 1/50,000 people in the United States. FA can start anywhere from childhood to adulthood. There is loss of muscle strength and coordination. Those patients diagnosed with FA at a young age require mobility aids, such as a wheelchair, walker, or cane by their teens or early 20’s. There are currently no approved treatments for FA.

II – Documentary Premier: The Ataxian

Kyle Bryant, a spokesperson for the organization, Friedreich’s Ataxia Research Alliance (FARA), led a team of bicyclists in a race across America. Two Redwood Creek Entertainment filmmakers, Kevin Schlanser and Zack Bennett, decided to follow Kyle’s participation in the tough race. This resulted in a feature-length documentary titled, “The Ataxian”.

The World Premiere of the film is scheduled for this Saturday, June 6th, at the Dances With Films festival in Hollywood, California, at the TCL Chinese 6 Theaters.

The film’s social media information:

•   Facebook

•   Twitter.

III – FDA Orphan Drug Designations For FA

The chart below identifies FDA Orphan Drug Designations (ODDs) for FA in ascending ODD Date order:

  Generic Name/ODD Date Sponsor Company Indication
1 Physostigmine salicylatephysostigmine salicylate/ 01.16.85 Forest Pharmaceutical FA & other inherited ataxias
2 Idebenone/ 03.25.04 Santhera Pharmaceuticals FA
3 Deferiprone/ 07.31.08 ApoPharma FA
4 N-(6-2-aminophenylamino)-6-oxohexyl)-4-methylbenzamide/   05.17.10 Repligen Corporation FA
5 Coenzyme Q10 and d-alpha-tocopherol/ 03.14.11 NBI Pharmaceuticals FA
6 Interferon gamma/ 11.04.11 Roberto Testi, MD (Italy) FA
7 Dimethyl fumarate/ 09.11.13 Gino Cortopassi, USA FA
8 Vatiquinone/ 01.31.14 Edison Pharmaceuticals FA
9 Interferon Gamma-1b/ 10.01.14 Horizon Pharma (Ireland) FA

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References

Friedreich’s Ataxia Research Alliance (FARA)

National Ataxia Foundation

Ride Ataxia.

Please Note: Movie Logo courtesy of Redwood Creek Entertainment.

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

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