EU/3/09/670

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Orphan designation

On 8 October 2009, orphan designation (EU/3/09/670) was granted by the European Commission to Novartis Europharm Limited, United Kingdom, for pasireotide for the treatment of acromegaly.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

What is acromegaly?

Acromegaly is a disease in which the pituitary gland, a small gland located at the base of the brain, produces too much growth hormone. Acromegaly usually affects adults between 30 and 50 years of age. In over 90% of patients, it is caused by a non-cancerous tumour of the pituitary gland called a pituitary adenoma. One of the most common symptoms of the disease is the abnormal growth of the hands and feet. The disease can result in serious complications, such as severe damage to the joints and problems affecting the cardiovascular (heart and blood vessels) and respiratory (lungs and airways) systems.

Acromegaly is a long-term debilitating disease that can be life threatening because of its cardiovascular and respiratory complications, and the increased risk of developing cancer.

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

At the time of designation, acromegaly affected approximately 1.2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 61,000 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).


*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 27), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 504,800,000 (Eurostat 2009).

What treatments are available?

At the time of submission of the application for orphan drug designation, several medicines were authorised in the EU to treat acromegaly, including ‘somatostatin analogues’ (medicines that block the release of growth hormone) such as octreotide and lanreotide, and pegvisomant (a medicine that blocks the effects of growth hormone). Other treatments included surgery and, in rare cases, radiotherapy (treatment with radiation).

The sponsor has provided sufficient information to show that pasireotide might be of significant benefit for patients with acromegaly because early studies indicate that it might improve the treatment of patients with this condition, and it might be used in patients who do not respond to treatment with octreotide. These assumptions 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?

Pasireotide is a somatostatin analogue, a copy of the natural hormone somatostatin. Like somatostatin, pasireotide is expected to attach to somatostatin receptors, thus blocking the release of growth hormone. This may result in the reduction of the symptoms and complications of acromegaly.

What is the stage of development of this medicine?

The effects of pasireotide have been evaluated in experimental models.

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

At the time of submission, pasireotide was not authorised anywhere in the EU for acromegaly or designated as 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 July 2009 recommending the granting of this designation.

Update: Pasireotide (Signifor) has been authorised in the EU since 19 November 2014 for the treatment of adult patients with acromegaly for whom surgery is not an option or has not been curative and who are inadequately controlled on treatment with another somatostatin analogue.

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 Community) 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.

Name Language First published Last updated
EU/3/09/670: Public summary of positive opinion for orphan designation of pasireotide for the treatment of acromegaly (English only) 20/10/2009 12/03/2015

Key facts

Product details for <p>Pasireotide</p>
Active substancePasireotide
Medicine NameSignifor
Disease/conditionTreatment of acromegaly
Date of decision08/10/2009
OutcomePositive
Orphan decision numberEU/3/09/670

Review of designation

During its meeting of 7 to 9 October 2014, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/09/670 for Signifor (pasireotide) as an orphan medicinal product for the treatment of acromegaly. The COMP assessed whether, at the time of addition of a new indication to the 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 acromegaly. The COMP recommended that the orphan designation of the medicine be maintained*.


*The 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.

Life-threatening or long-term debilitating nature of the condition

The Committee for Medicinal Products for Human Use (CHMP) recommended extending the approved therapeutic indication for Signifor to include the following indication:

‘treatment of adult patients with acromegaly for whom surgery is not an option or has not been curative and who are inadequately controlled on treatment with another somatostatin analogue’.

This falls within the scope of the product’s designated orphan indication, which is: ‘treatment of acromegaly’.

The COMP concluded that there had been no change in the seriousness of the condition since the orphan designation in 2009. Acromegaly remains a condition that is debilitating in the long term and life threatening, particularly because of its cardiovascular (heart and blood vessels) and respiratory (lungs and airways) complications and the increased risk of developing cancer.

Prevalence of the condition

On the basis of the information provided by the sponsor and the knowledge of the COMP, the COMP concluded that the prevalence of acromegaly 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 estimated to be approximately 2 people in 10,000. This is equivalent to a total of around 102,000 people in the EU.

Existence of other methods of treatment

At the time of the review of the orphan designation, other treatments were authorised in the EU for the treatment of acromegaly. These included ‘somatostatin analogues’ (medicines that block the release of growth hormone) such as octreotide and lanreotide, and pegvisomant (a medicine that blocks the effects of growth hormone). Other treatments included surgery and, in rare cases, radiotherapy (treatment with radiation).

Significant benefit of Signifor

The COMP concluded that the claim of a significant benefit of Signifor in acromegaly is justified on the basis of relevant data from a study which shows improved reduction of growth hormone levels in patients inadequately controlled with the currently authorized somatostatin analogues.

Therefore, although other methods for the treatment of this condition have been authorised in the EU, the COMP concluded that Signifor is of significant benefit to patients affected by acromegaly.

Conclusions

Based on the data submitted and the scientific discussion within the COMP, the COMP considered that Signifor still meets the criteria for designation as an orphan medicinal product and that it should remain in the Community Register of Orphan Medicinal Products.

Sponsor’s contact details

Novartis Europharm Limited
Frimley Business Park
Camberley GU16 7SR
United Kingdom
Tel. +41 6132 41111 (Switzerland)
E-mail: orphan.enquiries@novartis.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.