EU/3/15/1447 - orphan designation for treatment of soft tissue sarcoma

Olaratumab

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products by the European Commission in July 2019 at the time of withdrawal of the marketing authorisation.

On 12 February 2015, orphan designation (EU/3/15/1447) was granted by the European Commission to Elli Lilly Nederland B.V., the Netherlands, for olaratumab for the treatment of soft tissue sarcoma.

Olaratumab was authorised in the EU as Lartruvo since 9 November 2016.

The marketing authorisation was withdrawn on 23 July 2019.

What is soft tissue sarcoma?

Soft tissue sarcoma is a type of cancer that affects the soft, supportive tissues of the body. It can occur in muscles, blood vessels, fat tissue or in other tissues that support, surround and protect organs. Patients with soft tissue sarcoma do not usually have symptoms in the early stages of the disease. First symptoms appear when the tumour grows large enough to cause swelling and pain.

Soft tissue sarcoma is a long-term debilitating and life-threatening disease, particularly when the cancer has spread to other parts of the body.

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

At the time of designation, soft tissue sarcoma affected not more than 3 in 10,000 people in the European Union (EU). This was equivalent to a total of not more than 154,000 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 512,900,000 (Eurostat 2015).

What treatments are available?

At the time of designation, the main treatment for early-stage soft tissue sarcoma was surgery. For large sarcomas, surgery was usually followed by radiotherapy (treatment with radiation) and chemotherapy (medicines to treat cancer) to kill any cancerous cells that were left behind. Several medicines were authorised in the EU for the treatment of soft tissue sarcoma including doxorubicin.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with soft tissue sarcoma because early studies indicate that it might improve the outcome of patients when combined with doxorubicin. 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?

Olaratumab is a monoclonal antibody, a type of protein that has been designed to recognise and attach to a protein called platelet-derived growth factor receptor alpha (PDGFR?). This protein is often found on the surface of cells where it is thought to play a role in regulating cell multiplication. In cancers such as soft tissue sarcoma these proteins are present in high levels or overactive, causing cells to become cancerous. When olaratumab attaches to PDGFR? on sarcoma cells, it is expected to block its activity, thereby slowing down the growth of the cancer.

What is the stage of development of this medicine?

The effects of olaratumab have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with olaratumab in patients with soft tissue sarcoma were ongoing.

At the time of submission, olaratumab was not authorised anywhere in the EU for soft tissue sarcoma. Orphan designation of olaratumab had been granted in the United States of America for the treatment of soft tissue sarcoma.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 9 January 2015 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/3/15/1447: Public summary of opinion on orphan designation: Olaratumab for the treatment of soft tissue sarcoma

AdoptedReference Number: EMA/COMP/25527/2015

Key facts

• Active substance:
  Olaratumab
• Medicine name:
  Lartruvo
• Intended use:
  Treatment of soft tissue sarcoma
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/15/1447
• Date of designation:
  12/02/2015
• Sponsor:

  Eli Lilly Nederland B.V.; Papendorpseweg 83; Utrecht 3528 BJ; The Netherlands; Tel. [phone]; E-mail: [e-mail]

Review of designation

During its meeting of 4-6 October 2016, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/15/1447 for Lartruvo (olaratumab) as an orphan medicinal product for the treatment of soft tissue sarcoma. The COMP assessed whether, at the time of 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 soft tissue sarcoma. The COMP recommended that the orphan designation of the medicine be maintained¹.

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¹The 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 the same therapeutic indication cannot be placed on the market.