EU/3/18/2038 - orphan designation for treatment of multiple osteochondromas
Palovarotene
OrphanHuman
Overview
Please note that this product was withdrawn from the Union Register of orphan medicinal products in October 2021 on request of the Sponsor.
On 27 June 2018, orphan designation (EU/3/18/2038) was granted by the European Commission to PPD Global Ltd, United Kingdom, for palovarotene for the treatment of multiple osteochondromas.
The sponsorship was transferred to PPD Bulgaria EOOD, Bulgaria, in April 2019 and subsequently to Ipsen Pharma, France, in May 2021.
Multiple osteochondromas is an inherited condition in which patients have abnormal non-cancerous growths at the growing points of bones in the arms, legs, ribs, hip and spine. The number of growths and where they occur vary. The growths can damage nerves, blood vessels and other tissue by pressing on them, and they cause pain and restrict movement. Deformities caused by the condition can lead to limbs of uneven lengths and to short stature. In rare cases, the growths can become cancerous.
Multiple osteochondromas is a debilitating disease that is long lasting and may be life threatening because the growths can become cancerous.
At the time of designation, multiple osteochondromas affected approximately 0.3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 16,000 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).
At the time of designation, no satisfactory methods were authorised for the treatment of multiple osteochondromas. Painkillers were used for treating pain caused by the disease and surgery was used for more severe forms, including procedures to treat uneven leg length or misalignment of the legs.
In the body, proteins called bone morphogenetic proteins (BMPs) are involved in bone growth. The activity of BMPs is increased in multiple osteochondromas, which causes the abnormal growth. Palovarotene is expected to reduce the growth of cells that cause osteochondromas by blocking excess BMP activity. This is expected to reduce the sign and symptoms of the condition.
The effects of palovarotene have been evaluated in experimental models.
At the time of submission of the application for orphan designation, a clinical trial with the medicine in patients with multiple osteochondromas was ongoing.
At the time of submission, palovarotene was not authorised anywhere in the EU for multiple osteochondromas. Orphan designation of the medicine had been granted in the United States for multiple osteochondromas.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 24 May 2018 recommending the granting of this designation.
- 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
- Active substance
- Palovarotene
- Intended use
- Treatment of multiple osteochondromas
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/18/2038
- Date of designation
- Sponsor
Ipsen Pharma
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: