Food and Drug Administration (FDA) is on track to set a record for orphan drugs approved this year. At last count, 42 orphan drugs have been approved between January 1, 2017 and August 9, 2017.
An orphan drug designation gives companies numerous tax breaks and filing fee exemptions during the review process. In addition, if a drug gets approved, the company receives 7 years of market exclusivity for that drug. With the addition of the pediatric rare disease priority review voucher, it is likely that even more companies will understand the financial and altruistic rewards that come from helping those in the rare disease community.
Below is a list of orphan drugs approved this year (till August 11 2017)
An orphan drug designation gives companies numerous tax breaks and filing fee exemptions during the review process. In addition, if a drug gets approved, the company receives 7 years of market exclusivity for that drug. With the addition of the pediatric rare disease priority review voucher, it is likely that even more companies will understand the financial and altruistic rewards that come from helping those in the rare disease community.
Below is a list of orphan drugs approved this year (till August 11 2017)
| Generic Name | Orphan Designation | Designation Date | Marketing Approval Date | Exclusivity End |
| 5-aminolevulinic acid | Visualization of malignant tissue during surgery for malignant glioma (WHO grades III and IV) | 1/15/2013 | 6/6/2017 | 6/6/2024 |
| avelumab (Recombinant human monoclonal IgG1 antibody against programmed death ligand-1) | Treatment of merkel cell carcinoma. | 9/21/2015 | 3/23/2017 | 3/23/2024 |
| brigatinib | Treatment of anaplastic lymphoma kinase-positive (ALK+), c-ros 1 oncogene positive (ROS1+), or epidermal growth factor receptor positive (EGFR+) non-small cell lung cancer (NSCLC). | 4/28/2016 | 4/28/2017 | 4/28/2024 |
| C1-esterase-inhibitor, human, pasteurized | Prevention and/or treatment of acute attacks of hereditary angioedema. | 10/16/1992 | 6/22/2017 | 6/22/2024 |
| ceritinib | Treatment of patients with non-small cell lung cancer (NSCLC) that is anaplastic lymphoma kinase(ALK)-positive | 9/27/2013 | 5/26/2017 | 5/26/2024 |
| cerliponase alfa | Treatment of neuronal ceroid lipofuscinosis type 2 | 4/1/2013 | 4/27/2017 | 4/27/2024 |
| Cytarabine:daunorubicin liposome injection | Treatment of acute myeloid leukemia | 8/22/2008 | 8/3/2017 | TBD |
| d6-tetrabenazine, deutetrabenazine | Treatment of Huntington's Disease | 11/5/2014 | 4/3/2017 | 4/3/2024 |
| dabrafenib and trametinib | Treatment of patients with BRAF mutation positive non-small cell lung cancer. | 10/29/2015 | 6/22/2017 | 6/22/2024 |
| daratumumab | Treatment of multiple myeloma | 5/6/2013 | 6/16/2017 | TBD |
| Deferasirox | Treatment of chronic iron overload in patients with transfusion-dependent anemias | 11/21/2002 | 5/18/2017 | TBD |
| deferasirox | Treatment of chronic iron overload in alpha-thalassemia | 2/24/2015 | 5/18/2017 | TBD |
| deflazacort | Treatment of Duchenne muscular dystrophy | 8/16/2013 | 2/9/2017 | 2/9/2024 |
| edaravone | Treatment of amyotrophic lateral sclerosis | 5/12/2015 | 5/5/2017 | 5/5/2024 |
| enasidenib | Treatment of acute myelogenous leukemia | 6/12/2014 | 8/1/2017 | 8/1/2024 |
| glycerol phenylbutyrate | Maintenance treatment of patients with deficiencies in enzymes of the urea cycle | 4/27/2009 | 4/28/2017 | 4/28/2024 |
| ibrutinib | Treatment of nodal marginal zone lymphoma | 2/5/2015 | 1/18/2017 | 1/18/2024 |
| ibrutinib | Treatment of chronic Graft versus Host disease. | 6/23/2016 | 8/2/2017 | TBD |
| ibrutinib | Treatment of splenic marginal zone lymphoma | 2/5/2015 | 1/18/2017 | 1/18/2024 |
| ibrutinib | Treatment of patients with extranodal marginal zone lymphoma (mucosa associated lymphoid tissue [MALT type] lymphoma) | 2/2/2016 | 1/18/2017 | 1/18/2024 |
| ipilimumab | Treatment of high risk Stage II, Stage III, and Stage IV melanoma | 6/3/2004 | 7/21/2017 | 7/21/2024 |
| L-glutamine | Treatment of sickle cell disease | 8/1/2001 | 7/7/2017 | 7/7/2024 |
| ledipasvir/sofosbuvir | Treatment of chronic hepatitis C virus (HCV) infection in pediatric patients | 10/12/2016 | 4/7/2017 | 4/7/2024 |
| lenalidomide | Treatment of multiple myeloma | 9/20/2001 | 2/22/2017 | 2/22/2024 |
| methotrexate oral solution | Treatment of acute lymphoblastic leukemia in pediatric patients (0 through 16 years of age) | 5/28/2015 | 4/25/2017 | 4/25/2024 |
| methotrexate oral solution | Management of pediatric patients with active polyarticular juvenile idiopathic arthritis (pJIA) who are intolerant of or had an inadequate response to first-line therapy. | 8/27/2015 | 4/25/2017 | 4/25/2024 |
| midostaurin | Treatment of acute myeloid leukemia | 7/7/2009 | 4/28/2017 | 4/28/2024 |
| midostaurin | Treatment of mastocytosis | 4/30/2010 | 4/28/2017 | 4/28/2024 |
| niraparib | Treatment of ovarian cancer | 4/30/2010 | 3/27/2017 | 3/27/2024 |
| nivolumab | Treatment of Hodgkin lymphoma | 8/7/2014 | 4/25/2017 | 4/25/2024 |
| pembrolizumab | Treatment of Hodgkin lymphoma. | 12/30/2015 | 3/14/2017 | 3/14/2024 |
| regorafenib | Treatment of hepatocellular carcinoma | 6/4/2015 | 4/27/2017 | 4/27/2024 |
| rituximab and recombinant human hyaluronidase | Treatment of diffuse large B-cell lymphoma | 9/7/2016 | 6/22/2017 | 6/22/2024 |
| rituximab and recombinant human hyaluronidase | Treatment of chronic lymphocytic leukemia (CLL) | 8/22/2016 | 6/22/2017 | 6/22/2024 |
| rituximab and recombinant human hyaluronidase | Treatment of follicular lymphoma | 8/22/2016 | 6/22/2017 | 6/22/2024 |
| sofosbuvir | Treatment of pediatric chronic hepatitis C virus infection | 10/25/2016 | 4/7/2017 | 4/7/2024 |
| Sterile talc | Treatment of malignant pleural effusion | 12/8/1997 | 5/1/2017 | TBD |
| Sterile talc | Treatment of pneumothorax | 12/8/1997 | 5/1/2017 | 5/1/2024 |
| telotristat etiprate | Treatment of carcinoid syndrome in patients with neuroendocrine tumors | 3/9/2012 | 2/28/2017 | 2/28/2024 |
Thiotepa |
Conditioning treatment prior to hematopoietic stem cell transplantation |
4/2/2007 |
1/26/2017 |
1/26/2024 |
triptorelin pamoate |
Treatment of central precocious puberty |
8/20/2012 |
6/29/2017 |
6/29/2024 |
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