EU/3/14/1397 - orphan designation for treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis

amikacin sulfate

Overview

On 16 December 2014, orphan designation (EU/3/14/1397) was granted by the European Commission to PlumeStars s.r.l., Italy, for amikacin sulfate for the treatment of Pseudomonas aeuriginosa lung infections in cystic fibrosis.

What is Pseudomonas aeuriginosa lung infection in cystic fibrosis?

Cystic fibrosis is a genetic disease that affects the production of secretions such as mucus in the body. In patients with cystic fibrosis, there is an overproduction of thick mucus in the lungs, which leads to inflammation and a high risk of the lungs becoming infected with bacteria. Pseudomonas aeruginosa (P. aeruginosa) is one of the most common types of bacteria that tend to infect the lungs of patients with cystic fibrosis.

P. aeruginosa lung infection in cystic fibrosis is a long-term debilitating disease and may be life threatening because it severely damages the lung tissue and does not allow the patient to breathe normally.

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

t the time of designation, P. aeruginosa lung infection in cystic fibrosis affected approximately 0.8 in 10,000 people in the European Union (EU). This was equivalent to a total of around 41,000 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 511,100,000 (Eurostat 2014).

What treatments are available?

At the time of designation, P. aeruginosa lung infection in patients with cystic fibrosis was mainly treated with antibiotics (medicines that kill bacteria). These were available as tablets, infusions (drips into a vein) and solutions for inhalation. Other medicines used to treat the lung symptoms of cystic fibrosis included bronchodilators that help open up the airways in the lungs and mucolytics that help to dissolve the mucus.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with the condition because it will be available as a new formulation (powder for inhalation) which would allow patients to use amikacin sulfate outside of the hospital. This could be of major advantage for patients with the condition. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

Amikacin is an antibiotic that is already available in the EU as a solution for infusion for the treatment of P. aeruginosa infection in patients with cystic fibrosis. It belongs to the group 'aminoglycosides' and works by disrupting the production of proteins that bacteria need to build their cell walls, thereby damaging the bacteria and eventually killing them. This will prevent P. aeruginosa infection from developing in the lungs and prevent the inflammation caused by the infection.

What is the stage of development of this medicine?

The effects of amikacin sulfate have not been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with amikacin sulfate in patients with the condition had been started.

At the time of submission, amikacin sulfate was not authorised anywhere in the EU for P. aeruginosa lung infection in patients with cystic fibrosis 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 13 November 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/3/14/1397: Public summary of opinion on orphan designation: Amikacin sulfate for the treatment of Pseudomonas aeuriginosa lung infection in cystic fibrosis

Adopted Reference Number: EMA/COMP/736592/2014

Key facts

• Active substance:
  amikacin sulfate
• Intended use:
  Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/14/1397
• Date of designation:
  16/12/2014
• Sponsor:

  PlumeStars s.r.l.; Strada Inzani 1; 43125 Parma; Italy; 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.