FDA Orphan Drug Designations: 2014 9-Month Approval Rate
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 % |
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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 |
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Please Note: “Interferon” courtesy of Linda Bartlett (Photographer) from the National Cancer Institute (NCI). [Public domain in the US].
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