EU/3/10/778

About

Cysteamine bitartrate (gastroresistant) has been authorised in the EU as Procysbi since 6 September 2013.

On 20 September 2010, orphan designation (EU/3/10/778) was granted by the European Commission to Raptor Pharmaceuticals Europe BV, the Netherlands, for cysteamine bitartrate (gastroresistant) for the treatment of cystinosis.

This medicine is now known as mercaptamine.

In February 2017, Raptor Pharmaceuticals Europe B.V changed name to Horizon Pharma Europe B.V.

In November 2017, Horizon Phama Europe B.V. changed name to Chiesi Orphan B.V.

The sponsorship was transferred to Chiesi Farmaceutici S.p.A., Italy, in May 2018.

Key facts

Active substance
Cysteamine bitartrate (gastroresistant) (mercaptamine).
Medicine name
Procysbi
Disease / condition
Treatment of cystinosis
Date of decision
20/09/2010
Outcome
Positive
Orphan decision number
EU/3/10/778

Review of designation

During its meeting of 9-11 July 2013, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/10/778 for Procysbi (mercaptamine1) as an orphan medicinal product for the treatment of cystinosis. 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 satisfactory methods of treatment. As other methods of treatment for patients with this condition are authorised in the European Union (EU), the COMP also looked at the significant benefit of the product over existing treatments. The COMP recommended that the orphan designation of the medicine be maintained2.


1Previously known as cysteamine bitartrate (gastroresistant).
2The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with a comparable therapeutic indication cannot be placed on the market.

Sponsor's contact details

Chiesi Farmaceutici S.p.A.
Via Palermo 26/A
43122 Parma
Italy
Tel. +39 0521 279 793
E-mail: info@chiesigroup.com

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

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