EU/3/14/1414 - orphan designation for treatment for calciphylaxis

Sodium thiosulfate

Overview

On 15 January 2015, orphan designation (EU/3/14/1414) was granted by the European Commission to Hope Pharmaceuticals, Ltd, United Kingdom, for sodium thiosulfate for the treatment of calciphylaxis.

The sponsor’s address was updated in March 2019.

The sponsorship was transferred to Hope Pharmaceuticals Limited, Ireland, in August 2019.

What is calciphylaxis?

Calciphylaxis, also known as calcific uraemic arteriolopathy, is a severe and progressive disease mainly seen in patients with end-stage kidney disease (when the kidneys have stopped working). It involves the build-up of calcium in very small arteries resulting in a restricted blood supply and small clots. The skin develops ulcers (sores) that do not heal and usually cause severe pain.

Calciphylaxis is a long-term debilitating and life-threatening condition, particularly due to the deep, painful, non-healing ulcers and the risk of infection.

What is the estimated number of patients affected by the condition?

At the time of designation, calciphylaxis affected less than 0.5 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 26,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

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*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 28), Norway, Iceland and Liechtenstein. This represents a population of 511,100,000 (Eurostat 2014).

What treatments are available?

At the time of designation, no satisfactory methods were authorised in the EU for the treatment of calciphylaxis. Treatments included medicines to reduce the build-up of calcium in the arteries, skin wound management and surgery.

How is this medicine expected to work?

Sodium thiosulfate is a medicine that has been used for many years in the EU for the treatment of poisoning. In patients with calciphylaxis it is expected to act as a 'calcium chelator'. This means that it is expected to attach to calcium to form a compound which is easily eliminated in the urine, thereby reducing the build-up of calcium in the arteries seen in patients with calciphylaxis. It is also thought to act as an antioxidant (a molecule that may prevent damage to cells caused by other molecules known as 'free radicals'), which may help restore the healthy functioning of cells lining the interior walls of the arteries.

What is the stage of development of this medicine?

The effects of sodium thiosulfate have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with sodium thiosulfate in patients with calciphylaxis were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for calciphylaxis. Orphan designation of the medicine had been granted in the United States of America for the treatment of uremic and non-uremic calciphylaxis.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 11 December 2014 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:

• the seriousness of the condition;
• the existence of alternative methods of diagnosis, prevention or treatment;
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

EU/3/14/1414: Public summary of opinion on orphan designation: Sodium thiosulfate for the treatment for calciphylaxis

AdoptedReference Number: EMA/COMP/786341/2014

Key facts

• Active substance:
  Sodium thiosulfate
• Intended use:
  Treatment for calciphylaxis
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/14/1414
• Date of designation:
  15/01/2015
• Sponsor:

  Hope Pharmaceuticals Limited; Kyletelogue House; 9 Cherrywood Park; Tallanstown; Dundalk A91 WV74; Co. Louth; Ireland ; E-mail: [e-mail]

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.