EU/3/18/2113 - orphan designation for treatment of pseudomyxoma peritonei

bromelain

Overview

On 14 December 2018, orphan designation (EU/3/18/2113) was granted by the European Commission to MUCPharm Pty Ltd, United Kingdom, for bromelain for the treatment of pseudomyxoma peritonei.

The sponsorship was transferred to Mucpharm Europe Limited in February 2020.

What is pseudomyxoma peritonei?

Pseudomyxoma peritonei is a disease where tumours that produce mucus (a thick slimy fluid) develop and spread throughout the abdomen (belly). If left untreated, the tumours and the mucus they produce fill the abdominal cavity causing pain and swelling, and even blockage of the bowel.

Pseudomyxoma peritonei is a long-term debilitating and life-threatening disease because it can cause pain, pressure on internal organs, malnutrition, bowel obstruction and internal bleeding.

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

At the time of designation, pseudomyxoma peritonei affected approximately 0.2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 10,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based 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 517,400,000 (Eurostat 2018).

What treatments are available?

At the time of designation, the main treatments for the condition were surgery and chemotherapy (medicines to treat cancer) given directly into the peritoneum (the lining around the abdomen).

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with pseudomyxoma peritonei. Preliminary data show that treatment with bromelain in combination with another medicine called acetylcysteine can dissolve the mucus and lead to tumour reduction in patients who are not suitable for surgery and chemotherapy or in whom these methods have not worked.

How is this medicine expected to work?

Bromelain is the name for a mixture of enzymes extracted from the stem of the pineapple plant. The enzymes are expected to work by dissolving the mucus so that it can be more easily cleared out. Bromelain is also thought to stop the growth of tumour cells in ways that are not clearly understood.

Bromelain is expected to be injected into the tumours together with acetylcysteine, which also breaks down mucus.

What is the stage of development of this medicine?

The effects of bromelain have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with bromelain in combination with acetylcysteine in patients with pseudomyxoma peritonei were ongoing.

At the time of submission, a medicine containing bromelain was authorised in the EU for the removal of scar tissue.

At the time of submission, bromelain was not authorised anywhere in the EU for pseudomyxoma peritonei or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 8 November 2018 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/3/18/2113: Public summary of opinion on orphan designation: Bromelain for the treatment of pseudomyxoma peritonei

Adopted Reference Number: EMA/818577/2018

Key facts

• Active substance:
  bromelain
• Intended use:
  Treatment of pseudomyxoma peritonei
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/18/2113
• Date of designation:
  14/12/2018
• Sponsor:

  Mucpharm Europe Limited; 2 Bella Vista Lodge; Rathfarnham; Dublin 14 D14 K2T9; Co. Dublin, Ireland; Tel: [phone]; 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.