2 October 2017 EMA/568817/2017

Recommendation for removal of orphan designation at the time of marketing authorisation Cuprior (trientine tetrahydrochloride) for the treatment of Wilson's disease

On 20 July 2017, the Committee for Orphan Medicinal Products (COMP) completed a review of the designation EU/3/15/1471 for Cuprior (trientine tetrahydrochloride) as an orphan medicinal product in the treatment of Wilson's disease. The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other methods of treatment. As other methods of treatment are authorised in the European Union (EU), the COMP also considered whether the medicine is of significant benefit to patients with Wilson's disease. As one of the criteria for orphan designation is no longer met, the COMP recommended that the orphan designation of the product should not be maintained 1.

Life-threatening or chronically debilitating nature of the condition The Committee for Medicinal Products for Human Use (CHMP) recommended the authorisation of Cuprior for: ‘treatment of Wilson’s disease in adults, adolescents and children ≥ 5 years intolerant to D-penicillamine therapy’. This falls within the scope of the product’s designated orphan indication, which is: ‘treatment of Wilson's disease’. The COMP concluded that there had been no change in the seriousness of the condition since the orphan designation in 2015. Wilson's disease remains a chronically debilitating disease that can be life threatening if not treated, due to the toxicity of excess copper in the liver and brain.

Prevalence of the condition The sponsor provided updated information on the prevalence of Wilson's disease based on data from the published literature.

1

The removal of the orphan designation at time of marketing authorisation means that the product cannot benefit from 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with the same therapeutic indication can be placed on the market. 30 Churchill Place ● Canary Wharf ● London E14 5EU ● United Kingdom Telephone +44 (0)20 3660 6000 Facsimile +44 (0)20 3660 5555 Send a question via our website www.ema.europa.eu/contact

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On the basis of the information provided by the sponsor and the knowledge of the COMP, the COMP concluded that the prevalence of Wilson's disease remains below the ceiling for orphan designation, which is 5 people in 10,000. At the time of the review of the orphan designation, the prevalence was still estimated to be approximately 0.6 people in 10,000. This is equivalent to a total of around 31,000 people in the EU.

Existence of other methods of treatment At the time of the review of the orphan designation, penicillamine, trientine dihydrochloride and zinc acetate were authorised in the EU for the treatment Wilson’s disease.

Significant benefit of Cuprior The COMP concluded that the claim of a significant benefit of Cuprior over penicillamine is justified because Cuprior is for use in patients who cannot take penicillamine. Cuprior was also considered of significant benefit over zinc acetate since the latter is for use only when the symptoms have been brought under control and it is not recommended for use at the beginning of treatment. Therefore Cuprior can be used in a wider range of patients with Wilson’s disease. However, the COMP did not conclude that Cuprior is of significant benefit over trientine dihydrochloride. The company for Cuprior had claimed that Cuprior would significantly improve availability of trientine throughout the EU; however, the company did not provide sufficient evidence to demonstrate lack of availability of trientine in the EU. Therefore, the COMP concluded that the assumption that Cuprior may be of significant benefit for patients affected by Wilson’s disease is no longer valid.

Conclusions Based on the data submitted and the scientific discussion within the COMP, the COMP concluded that Cuprior no longer meets one of the criteria for designation as an orphan medicinal product. Therefore, the COMP recommended that the product should be removed from the Community Register of Orphan Medicinal Products.

More information on the COMP assessment can be found in the September 2017 COMP minutes. Further information on Cuprior can be found in the European public assessment report (EPAR) on the Agency’s website ema.europa.eu/Find medicine/Human medicines/European public assessment reports.

Recommendation for removal of orphan designation at the time of marketing authorisation EMA/568817/2017

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Recommendation for removal of orphan designation at the time of ...

Oct 2, 2017 - 30 Churchill Place ○ Canary Wharf ○ London E14 5EU ○ United Kingdom. An agency of the ... The sponsor provided updated information on the prevalence of Wilson's disease based on data from ... On the basis of the information provided by the sponsor and the knowledge of the COMP, the COMP.

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