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FDA Orphan Drug Approvals: 2014 In Review

January 3, 2015




Updated 01/04/15 – Deletion of Relistor (withdrawn by OPD) & addition of Opdivo (Nivolumab)

The chart below compares the number of FDA Orphan Drug Marketing Approvals in 2013 versus the number of FDA ODD Marketing Approvals in 2014. The approvals include new label indication extensions. The FDA’s Orphan Drug Product Designation Database Application is the source of the information.

FDA Orphan Drug 2014 Approval Rate (2013 vs. 2014)

# of 2013 FDA ODD Approvals # of 2014 FDA ODD Approvals % Change From 2013 To 2014
32 41 22 %



•   Approximately 22 % increase in the # of FDA Orphan Drug Marketing Approvals in 2014 compared to 2013

•   Orphan approvals for rare diseases:

1.Vimizim for Mucopolysaccharidosis (Morquio A syndrome)

2.   Sylvant for Castleman’s Disease

3.   Ofev & Esbriet for Idiopathic Pulmonary Fibrosis (IPF)

•    3 orphan approvals for Hemophilia A/B:

1.   Alprolix from Biogen Idec

2.   Eloctate from Biogen Idec

3.   Obizur   from Baxter.

The chart below shows the 41 FDA ODD Marketing Approvals for 2014, in ascending chronological order.

FDA Orpan Drug Approvals for 2014

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
Humira (Adalimumab) Juvenile idiopathic arthritis 09.30.14 AbbVie
Velcade (Bortezomib) Mantle Cell Lymphoma 10.08.14 Millennium Pharmaceuticals
Ofev (Nintedanib) Idiopathic Pulmonary Fibrosis 10.15.14 Boehringer Ingelheim Pharmaceuticals
Esbriet (Pirfenidone) Idiopathic Pulmonary Fibrosis 10.15.14 InterMune
Obizur (antihemophilic factor (recombinant), porcine sequence) Bleeding episodes in adults with acquired Hemophilia A 10.23.14 Baxter
Cyramza (Ramucirumab) Advanced gastric or gastro-esophageal junction adenocarcinoma 11.05.14 Eli Lilly & Co
Avastin (Bevacizumab) Ovarian/ Fallopian Tube/ Primary Peritoneal Cancers 11.14.14 Genentech
Sensipar (Cinacalcet) Hypercalcemia in adult patients with primary hyperparathyroidism 11.21.14 Amgen
Blinatumomab Philadelphia chromosome-negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) 12.03.14 Amgen
Jakafi (Ruxolitinib) Polycythemia vera 12.04.14 Incyte Corp
Xgeva (Denosumab) Hypercalcemia of malignancy refractory to biphosphonate therapy 12.05.14 Amgen
Opdivo (Nivolumab) Unresectable/ Metastatic Melanoma 12.22.14 Bristol-Myers Squibb


Please Note: “Abstract pills” by Robson# (Flickr: Pills here) [CC-BY-2.0] | Wikimedia Commons..

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