Orphan Druganaut Blog

June 13, 2015

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

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-(1â€™,1â€™-dimethylheptyl)-Î”8-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

** 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.

June 7, 2015

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

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

** 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.

June 5, 2015

Patient Advocacy: Top Grant-Giving Disease Foundations

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

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

June 4, 2015

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

Genzyme, a Sanofi company, announces June 4^th 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 20^th 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