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FDA Orphan Drug Designations: 2014 9-Month Approval Rate

October 21, 2014



The chart below compares the number of FDA Orphan Drug Marketing Approvals in the first 9 months of 2013 versus the number of FDA ODD Marketing Approvals in the first 9 months of 2014. The FDA’s Orphan Drug Product Designation Database Application is the source of the information.

FDA Orphan Drug 9-Month Approval Rate (2013 vs. 2014)

# of Jan-Sept   2013 FDA ODD Approvals # of Jan-Sept 2014   FDA ODD Approvals % Change From 2013 To 2014
23 31 34.8 %


Thus, there is approximately a 34.8 % increase in the # of FDA Orphan Drug Marketing Approvals in the first 9 months of 2014 compared to the # of FDA Orphan Drug Marketing Approvals in the first 9 months of 2013.

The chart below shows the 31 FDA ODD Marketing Approvals in the first 9 months of 2014, in ascending chronological order.

FDA Orpan Drug Approvals for 2014 (January-September)

Brand/Generic    Name Orphan   Designation Marketing   Approval   Date Sponsor
Mekinist (Trametinib) and Tafinlar (Dabrafenib) Melanoma 01.08.14/ 01.09.14 GSK
Hetlioz (Tasimelteon) Non 24-hour sleepwake disorder in blind individuals without light perception 01.31.14 Vanda Pharmaceuticals
Imbruvica (Ibrutinib) Chronic Lymphocytic Leukemia (CLL) 02.12.14 Pharmacyclics
Vimizim (Elosulfase Alfa) Mucopolysaccharidosis (MPS) Type IV A (Morquio A syndrome) 02.14.14 BioMarin Pharmaceutical
Northera (Droxidopa) Symptomatic neurogenic orthostatic hypotension 02.18.14 Lundbeck LLC
Myalept (Metreleptin) Lipodystrophy 02.24.14 Amylin Pharmaceuticals
Hemangeol (Propranolol) Proliferating infantile hemangioma 03.14.14 Pierre Fabre Dermatologie
Impavido (Miltefosine) Leishmaniasis 03.19.14 Knight Therapeutics
Alprolix (Coagulation factor IX (recombinant), Fc fusion protein) Hemophilia B 03.28.14 Biogen Idec
Kalbitor (Ecallantide) Acute attacks of hereditary angioedema (HAE) 03.28.14 Dyax Corp
Lipiodol (Ethiodized Oil Injection) Imaging tumors in adults with known hepatocellular carcinoma (HCC) 04.04.14 Guerbet LLC
Arzerra (Ofatumumab) In combination with Chlorambucil for CLL whom fludarabine-based therapy is considered inappropriate 04.17.14 Glaxo Group Limited c/b/a GSK
Cyramza (Ramucirumab) Advanced gastric cancer or gastro-esophageal junction adenocarcinoma 04.21.14 Eli Lilly
Sylvant (Siltuximab) Castleman’s Disease 04.23.14 Janssen Biotech
Purixan (Mercaptopurine Oral Solution) Acute lymphoblastic leukemia as part of combo regimen 04.28.14 Nova Laboratories Limited
Zykadia (Ceritinib) ALK+ NSCLC 04.29.14 Novartis Pharmaceuticals
Eloctate (antihemophilic factor (recombinant), Fc fusion protein) Hemophilia A 06.06.14 Biogen Idec
Lymphoseek Guiding sentinel lymph node biopsy 06.13.14 Navidea Biopharmaceuticals
Novoseven Rt (coagulation factor VIIa (recombinant)) Bleeding episodes and peri-operative management in adults and children with Glanzmann’s thrombasthenia 07.02.14 Novo Nordisk
Beleodaq (Belinostat) Peripheral T-cell lymphoma (PTCL) 07.03.14 Spectrum Pharmaceuticals
Ruconest (C1-esterase inhibitor (recombinant)) Acute attacks of hereditary angioedema (HAE) 07.16.14 Santarus
Ryanodex Malignant hyperthermia 07.22.14 Eagle Pharmaceuticals
Zydelig (Idelalisib) CLL & Small lymphocytic lymphoma (SLL)/ Follicular B-cell non-Hodgkin lymphoma (FL) 07.23.14/ 07.23.14 Gilead Sciences
Imbruvica (Ibrutinib) CLL with 17p deletion 07.28.14 Pharmacyclics
Myozyme/Lumizyme Pompe Disease 08.01.14 Genzyme
Cerdelga (Eliglustat) Gaucher Disease 08.19.14 Genzyme
Promacta (Eltrombopag) Severe Aplastic Anemia 08.26.14 GSK
Keytruda (Pembrolizumab) Metastatic Melanoma if BRAF V600 mutation + 09.04.14 Merck
Humira (Adalimumab) Crohn’s Disease 09.23.14 AbbVie
Relistor (Methylnaltrexone) Opioid-induced constipation (OIC) 09.29.14 Progenics Pharmaceuticals
Humira (Adalimumab) Juvenile idiopathic arthritis 09.30.14 AbbVie


Please Note: “Interferon” courtesy of Linda Bartlett (Photographer) from the National Cancer Institute (NCI). [Public domain in the US].

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