61
Views
5
CrossRef citations to date
0
Altmetric
Orphan Drug Designations

Fresh from the designation pipeline: orphan drugs recently designated in the European Union (November 2012 – January 2013)

, MD PhD (Lecturer)
Pages 499-505 | Published online: 08 May 2013

Bibliography

  • Antoniu S. Fresh from the designation pipeline: orphan drugs recently designated in the European Union. Exp Opin Orphan Drugs 2013;1(3):249-53
  • Turner MD, Chaudhry A, Nedjai B. Tumour necrosis factor receptor trafficking dysfunction opens the TRAPS door to pro-inflammatory cytokine secretion. Biosci Rep 2012;32(2):105-12. Available from: www.ema.europa.eu
  • Dhimolea E. Canakinumab. MAbs 2010;2(1):3-13. Available from: www.ema.europa.eu
  • European, M, Agency. Canakinumab for the treatment of tumour necrosis factor receptor-associated periodic syndrome. 2012
  • Novartis Phase III study showed 62% of patients with most severe form of childhood arthritis were symptom-free with ACZ885 treatment. 2012. Available from: http://www.novartis.com/newsroom/media-releases/en/2012/1617524.shtml
  • Comenzo RL. How i treat amyloidosis. Blood 2009;114(15):3147-57. Available from: www.ema.europa.eu
  • Bulawa CE, Connelly S, DeVit M, et al. Tafamidis, a potent and selective transthyretin kinetic stabilizer that inhibits the amyloid cascade. Proc Natl Acad Sci USA 2012;109(24):9629-34. Available from: www.ema.europa.eu
  • Coelho T, Maia LF, Martins da Silva A, et al. Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial. Neurology 2012;79(8):785-92. Available from: www.ema.europa.eu
  • European, M, Agency. Tafamidis for treatment of senile systemic amyloidosis. 2012
  • Rossmiller B, Mao H, Lewin AS. Gene therapy in animal models of autosomal dominant retinitis pigmentosa. Mol Vis 2012;18:2479-96. Available from: www.ema.europa.eu
  • European, M, Agency. Adeno-associated viral vector encoding an inducible short hairpin RNA targeting claudin-5 (prior to administration of 17-dimethylaminoethylamino-17-demethocygeldanamycin) for the treatment of retinitis pigmentosa; 2012
  • Lipinski DM, Singh MS, MacLaren RE. Assessment of cone survival in response to CNTF, GDNF, and VEGF165b in a novel ex vivo model of end-stage retinitis pigmentosa. Invest Ophthalmol Vis Sci 2011;52(10):7340-6. Available from: www.ema.europa.eu
  • Kauper K, McGovern C, Sherman S, et al. Two-year intraocular delivery of ciliary neurotrophic factor by encapsulated cell technology implants in patients with chronic retinal degenerative diseases. Invest Ophthalmol Vis Sci 2012;53(12):7484-91. Available from: www.ema.europa.eu
  • European, M, Agency. Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotrophic factor for the treatment of retinitis pigmentosa. 2012. Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Orphan_designation/2013/03/WC500139544.pdf
  • Gourronc FA, Robertson MM, Herrig AK, et al. Proliferative defects in dyskeratosis congenita skin keratinocytes are corrected by expression of the telomerase reverse transcriptase, TERT, or by activation of endogenous telomerase through expression of papillomavirus E6/E7 or the telomerase RNA component, TERC. Exp Dermatol 2010;19(3):279-88. Available from: www.ema.europa.eu
  • European, M, Agency. Public summary of opinion on orphan designation-Recombinant human dyskerin for the treatment of dyskeratosis congenita. 2012. Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Orphan_designation/2012/12/WC500136191.pdf
  • Newton KF, Mallinson EK, Bowen J, et al. Genotype-phenotype correlation in colorectal polyposis. Clin Genet 2012;81(6):521-31. Available from: www.ema.europa.eu
  • Tonelli F, Valanzano R, Dolara P. Sulindac therapy of colorectal polyps in familial adenomatous polyposis. Dig Dis 1994;12(5):259-64. Available from: www.ema.europa.eu
  • Mackenzie GG, Ouyang N, Xie G, et al. Phospho-sulindac (OXT-328) combined with difluoromethylornithine prevents colon cancer in mice. Cancer Prev Res 2011;4(7):1052-60. Available from: www.ema.europa.eu
  • European,M, Agency. Public summary of opinion on orphan designation-Eflornithine in combination with sulindac for the treatment of familial adenomatous polyposis; 2012
  • Hinchcliff M, Varga J. Systemic sclerosis/scleroderma: a treatable multisystem disease. Am Fam Physician 2008;78(8):961-8. Available from: www.ema.europa.eu
  • European, M, Agency. Tracleer (bosentan); 2002
  • Antoniu SA. Terguride for pulmonary arterial hypertension. Expert Opin Ther Targets 2011;15(11):1333-5. Available from: www.ema.europa.eu
  • Kekewska A, Görnemann T, Jantschak F, et al. Antiserotonergic properties of terguride in blood vessels, platelets, and valvular interstitial cells. J Pharmacol Exp Ther 2012;340(2):369-76. Available from: www.ema.europa.eu
  • European,M, Agency. Terguride for the treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension. 2007. Available from: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/orphans/2009/11/human_orphan_000308.jsp&mid=WC0b01ac058001d12b
  • European, M, Agency. Public summary of opinion on orphan designation-Terguride for the treatment of systemic sclerosis. 2013
  • European, M, Agency. Public summary of opinion on orphan designation-Terguride for the treatment of systemic sclerosis; 2013
  • Van Den Berg-Vos RM, Van Den Berg LH, Visser J, et al. The spectrum of lower motor neuron syndromes. J Neurol 2003;250(11):1279-92
  • Meyer K, Miranda CJ, Kaspar BK. Transplantation of gene-corrected motor neurons as a therapeutic strategy for spinal muscular atrophy. Mol Ther 2013;21(3):502-3
  • European,M, Agency. Public summary of opinion on orphan designation-Allogeneic motor neuron progenitor cells derived from human embryonic stem cells for the treatment of 5q spinal muscular atrophy; 2013
  • Richette P, Bardin T, Stheneur C. Achondroplasia: from genotype to phenotype. Joint Bone Spine 2008;75(2):125-30. Available from: www.ema.europa.eu
  • Lorget F, Kaci N, Peng J, et al. Evaluation of the therapeutic potential of a CNP analog in a Fgfr3 mouse model recapitulating achondroplasia. Am J Hum Genet 2012;91(6):1108-14. Available from: www.ema.europa.eu
  • European, M, Agency. Public summary of opinion on orphan designation-Modified recombinant human C-type natriuretic peptide for the treatment of achondroplasia; 2013
  • Valstar MJ, Bruggenwirth HT, Olmer R, et al. Mucopolysaccharidosis type IIIB may predominantly present with an attenuated clinical phenotype. J Inherit Metab Dis 2010;33(6):759-67. Available from: www.ema.europa.eu
  • Fu H, Dirosario J, Killedar S, et al. Correction of Neurological Disease of Mucopolysaccharidosis IIIB in Adult Mice by rAAV9 Trans-Blood-Brain Barrier Gene Delivery. Mol Ther 2011;19(6):1025-33. Available from: www.ema.europa.eu
  • European, M, Agency. Public summary of opinion on orphan designation-Adeno-associated viral vector serotype 9 containing the human N-acetylglucosaminidase alpha gene for the treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome); 2013
  • International Rare Disease Research Consortium. Available from: http://www.irdirc.org/
  • Applied Clinical Research on Very Rare Diseases – call for proposals. Available from: http://www.nihr.ac.uk/research/Pages/VeryRareDiseasesResearch.aspx

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.