EU/3/09/645 - orphan designation for treatment of acromegaly

octreotide hydrochloride

Overview

On 12 June 2009, orphan designation (EU/3/09/645) was granted by the European Commission to Camurus AB, Sweden, for octreotide hydrochloride (lipid depot solution) for the treatment of acromegaly.

The sponsorship was transferred to Novartis Europharm Limited, United Kingdom, in April 2014, then to Novartis Europharm Limited, Ireland, in May 2018 and finally back to Camurus AB, Sweden, in July 2018.

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 in middle 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 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).

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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 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). These medicines were available as solutions for injection into a muscle or under the skin. Other treatments included surgery and, in rare cases, radiotherapy (treatment with radiation).

The sponsor has provided sufficient information to show that octreotide hydrochloride (lipid depot solution) might be of significant benefit for patients with acromegaly because the medicine will be available as a ready-to-use prefilled syringe that will enable the patients to inject themselves. It will also be injected using a thinner needle than with other available medicines. These two factors might improve the patients' quality of life. 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?

Octreotide has been available as the 'acetate salt' for the treatment of acromegaly since the 1980s. Octreotide is an analogue (a copy) of the natural hormone somatostatin. Like somatostatin, octreotide blocks the release of growth hormone. This results in the reduction of the symptoms and complications of acromegaly. The acetate salt is available as two forms, one that releases octreotide immediately and is injected once a day under the skin, and another that releases it more slowly and is injected once a month into a muscle.

In octreotide hydrochloride (lipid depot solution), octreotide is to be available as the 'chloride salt' in a new formulation that is expected to release octreotide slowly and consistently. The new formulation is to be injected under the skin through a thin needle.

What is the stage of development of this medicine?

The effects of octreotide chloride (lipid depot solution) 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, octreotide chloride (lipid depot solution) 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 5 May 2009 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 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.

EU/3/09/645: Public summary of positive opinion for orphan designation of octreotide hydrochloride (lipid depot solution) for the treatment of acromegaly

AdoptedReference Number: EMEA/COMP/296368/2009 Rev.2 Corr.

Key facts

• Active substance:
  octreotide hydrochloride
• Intended use:
  Treatment of acromegaly
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/09/645
• Date of designation:
  12/06/2009
• Sponsor:

  Camurus AB