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Orphan designation

On 21 March 2018, orphan designation (EU/3/18/1995) was granted by the European Commission to Loxo Oncology Limited, United Kingdom, for larotrectinib for the treatment of salivary gland cancer.

The sponsorship was transferred to Bayer AG - Germany, in June 2018.

What is salivary gland cancer?

Salivary gland cancer is a cancer of the glands that make saliva. Tumours often start off as painless lumps and can form in the mouth, tongue, nose, ear and neck. Salivary gland cancer is a life-threatening disease that can spread and worsen quickly.

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

At the time of designation, salivary gland cancer affected approximately 0.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 26,000 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 28), Norway, Iceland and Liechtenstein. This represents a population of 517,400,000 (Eurostat 2018).

What treatments are available?

At the time of designation, no satisfactory methods were authorised in the EU for the treatment of salivary gland cancer. Patients underwent surgery to remove the cancer if possible.

How is this medicine expected to work?

Some patients with salivary gland cancer have genetic mutations (changes) called NTRK-fusion mutations which result in the production of altered TRK proteins that can cause cancer.

This medicine blocks the activity of the altered TRK proteins, thus preventing or slowing down the growth of salivary gland cancer.

What is the stage of development of this medicine?

The effects of larotrectinib have been evaluated in experimental models.

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

At the time of submission, larotrectinib was not authorised anywhere in the EU for salivary gland cancer. Orphan designation of larotrectinib had been granted in the US for treatment of solid tumors with NTRK-fusion proteins.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 15 February 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.

Key facts

Product details for <p>Larotrectinib</p>
Active substanceLarotrectinib
Medicine Name
Disease/conditionTreatment of salivary gland cancer
Date of decision21/03/2018
Orphan decision numberEU/3/18/1995

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor's contact details

Bayer AG
Kaiser-Wilhelm-Allee 3
51368 Leverkusen

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.