The European Medicines Agency's Committee for Orphan Medicinal Products has issued 15 positive opinions for orphan drug designation in the European Union.
IHS Global Insight perspective | |
Significance | The Committee for Orphan Medicinal Products (COMP) has issued recommendations for orphan drug designation for 15 products at its 152th meeting. |
Implications | Within the decisions adopted by the COMP, recommendation for orphan drug designation was granted to Novartis (Switzerland)'s polycythaemia vera treatment Jakavi (ruxolitinib). |
Outlook | The recommendations adopted by the COMP are now presented to the European Commission for final approval. If the European Commission gives the green light, the drugs will receive orphan designation in the European Union. The designation enables manufacturers to receive incentives for research and development of the compounds. |
The European Medicines Agency (EMA) has published 15 recommendations for orphan designation adopted by the Committee for Orphan Medicinal Products (COMP) adopted during its 152th meeting on 7–9 January 2014. Within the 15 opinions, six decisions were reached at the committee's second discussion, and nine at the first. In particular, according to EMA rules, decisions taken at the second discussion are adopted following the sponsors' responses to a list of questions from the COMP. The full decision document is available here.
COMP's recommendations | ||
Opinions adopted at second discussion | ||
Drug | Company/organisation | Indication |
11-(4-Dimethylamino-3-hydroxy-6-methyl-tetrahydro-pyran-2-yloxy)-2-ethyl-3,4,10-trihydroxy-3,5,6,8,10,12,14-heptamethyl-1-oxa-6-aza-cyclopentadecane-13,15-dione | Synovo (US) | Treatment of cystic fibrosis |
Cysteamine | Istituto Europeo per la Ricerca sulla Fibrosi Cistica (Italy) | Treatment of cystic fibrosis |
Diacerein | Prof. Johann W Bauer (Austria) | Treatment of epidermolysis bullosa |
Eculizumab | Alexion (US) | Prevention of delayed graft function after solid organ transplantation |
Gallium [Ga-68]-N-[(4,7,10-tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl-Lcysteinyl-L-threonine-cyclic(2-7)disulfide | Advanced Accelerator Applications (France) | Diagnosis of gastro-entero-pancreatic neuroendocrine tumour |
Mixture of recombinant human IgG1 monoclonal antibodies against human cytomegalovirus envelope glycoproteins | Roche (Switzerland) | Prevention of congenital cytomegalovirus infection following primary cytomegalovirus infection |
Opinions adopted at first discussion | ||
3-Chloro-4-fluorophenyl-[4-fluoro-4-{[(5-methylpyrimidin-2-ylmethyl) amino]methyl}piperidin-1-yl]methanone | Neurolixis (US) | Treatment of Rett syndrome |
68Ga-2,2'-(7-(4-((S)-1-((4S,7S,10S,13R,16S,19R)-4-((R)-1-amino-3-(4-hydroxyphenyl)- 1-oxopropan-2-ylcarbamoyl)-10-(4-aminobutyl)-16-(4-((S)-2,6- dioxohexahydropyrimidine-4-carboxamido)benzyl)-7-((R)-1-hydroxyethyl)- 6,9,12,15,18-pentaoxo-13-(4-ureidobenzyl)-1,2-dithia-5,8,11,14,17- pentaazacycloicosan-19-ylamino)-3-(4-chlorophenyl)-1-oxopropan-2-ylamino)-1- carboxy-4-oxobutyl)-1,4,7-triazonane-1,4-diyl)diacetic acid | OctreoPharm Sciences (Germany) | Diagnosis of gastro-entero-pancreatic neuroendocrine tumours |
Asp-Arg-Val-Tyr-Ile-His-Pro | Gregory Fryer Associates (UK) | Treatment of Duchenne muscular dystrophy |
Autologous dendritic cells pulsed with tumour antigen-derived synthetic peptides (MAGE-1, HER-2, AIM-2, TRP-2, gp-100, and interleukin-13 receptor alpha) | Diamond BioPharm (UK) | Treatment of glioma |
N-({Carbamoylmethyl-[3-(2-oxo-pyrrolidin-1-yl)-propyl]-carbamoyl}-methyl)-2-[2-(2- fluoro-phenyl)-ethylamino]-N-isobutyl-acetamide | Bionure Farma (Spain) | Treatment of optic neuritis |
Phosphorothioate oligonucleotide targeted to apolipoprotein C-III | Isis (US) | Treatment of familial chylomicronaemia syndrome |
Pioglitazone | Minoryx Therapeutics (Spain) | Treatment of adrenoleukodystrophy |
Recombinant human acid ceramidase | QOL Therapeutics (UK) | Treatment of Farber disease |
Ruxolitinib | Novartis (Switzerland) | Treatment of polycythaemia vera |
Source: COMP | ||
The COMP has recommended the European Commission to keep Cometriq (cabozantinib; TMC Pharma Services, UK) and Sirturo (bedaquiline; Janssen-Cilag, subsidiary of Johnson & Johnson, US) in the European Union registry of orphan medicinal products. Conversely, the COMP has issued two initial opinions recommending the removal of Deltyba (delamanid; Otsuka, Japan) and para-aminosalicylic acid Lucane (Lucane Pharma, France).
Outlook and implications
Among the decisions adopted by the COMP, Novartis's Jakavi (ruxolitinib) received a positive opinion for orphan drug designation. The product is already approved for commercialisation in the EU markets for the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis, post-polycythaemia vera myelofibrosis or post-essential thrombocythaemia myelofibrosis (see Europe: 28 August 2012: Novartis Obtains EU Approval for Blood Cancer Drug Jakavi).
The orphan drug designation represents positive news for the applicants. In addition it represents a significant opportunity to address unmet medical needs. Prevalence of the disease should be no more than five in 10,000 or it is unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development, and no other satisfactory treatment is available in the EU. Therefore, the designation represents an incentive aimed at prompting investments into research and development of products that target life-threatening or chronically debilitating and rare diseases.
Furthermore, orphan drug designation provides applicants with 10 years of marketing exclusivity upon authorisation which can be extended by an additional two years, provided that the treatment has also complied with an agreed paediatric investigation plan. Through the designation, manufacturers are entitled to receive the EMA's scientific advice at reduced fees.