EU/3/01/035

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Orphan designation

On 10 May 2001, orphan designation (EU/3/01/035) was granted by the European Commission to NeoPharma Production AB, Sweden, for levodopa / carbidopa (gastroenteral use) for the treatment of advanced idiopathic Parkinson’s disease with severe motor fluctuations and not responding to oral treatment.

The sponsorship was transferred to Solvay Pharmaceuticals GmbH, Germany, in March 2006.

The sponsor changed name to Abbott Products GmbH in November 2010.

The sponsorship was transferred to AbbVie Ltd, United Kingdom, in February 2013.

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

What is advanced idiopathic Parkinson’s disease with severe motor fluctuations and not responding to oral treatment?

Parkinson’s disease results from progressive damage to the nerve cells in the area of the brain responsible for controlling muscle tone and movement. The damaged cells are those needed to produce a neurotransmitter (chemical messenger in the brain that transmits information from one nerve cell to another) called dopamine. Patients with Parkinson’s disease have low levels of dopamine. Parkinson’s disease occurs primarily, but not exclusively, in the elderly. The symptoms of Parkinson’s disease are bradykinesia (slowness and poverty of movement), muscular rigidity, resting tremor and an impairment of postural balance. The cause of Parkinson’s disease is not known in the vast majority of the cases (idiopathic).

Oral treatment with a dopamine precursor called levodopa is effective in the early stages of the disease. The brain still has the ability to store dopamine precursor and transform it into dopamine. As the disease progresses, the brain loses this ability to store or use its reserves of dopamine precursor. In practice, hours after taking the oral treatment, the characteristic symptoms such as motor fluctuations will re-appear ('off periods'). Therefore, as the disease progresses, the action of oral treatment with dopamine precursors will gradually shorten. Advanced idiopathic Parkinson’s disease with severe motor fluctuations and not responding to oral treatment is progressive and chronically debilitating.

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

At the time of designation, advanced idiopathic Parkinson’s disease with severe motor fluctuations and not responding to oral treatment affected approximately 2.4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 91,000 people*, and is below the threshold for orphan designation, which is 5 people in 10,000. This is based 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. At the time of designation, this represented a population of 378,800,000 (Eurostat 2001).

What treatments are available?

At the time of submission of the application for the orphan-drug designation, several medicinal products, such as levodopa and apomorphine, were authorised for the treatment of Parkinson’s disease. However, in spite of these authorised oral treatments, there was a subset of advanced Parkinson’s disease patients who did not respond satisfactorily to the available treatments and there were no satisfactory authorised methods available for these patients, at the time of designation.

How is this medicine expected to work?

Levodopa is the naturally occurring precursor of dopamine. It is converted to dopamine in the cells. Dopamine is a neurotransmitter, a chemical used for transmitting signals from the nervous system to the body. Levodopa is given to increase the production of dopamine in the brain to reduce the symptoms of Parkinson’s disease. In order to maintain high levels of levodopa in the brain, levodopa is given with another substance called carbidopa which prevents the breakdown of levodopa. This particular medicinal product will be administered as a suspension into the duodenum (the first section of the small intestine) using a surgically placed permanent tube. This way, the product is expected to be delivered at a steady rate, resulting in stable plasma concentrations and decreasing the symptoms of advanced idiopathic Parkinson’s disease with severe motor fluctuations and not responding to oral treatment.

What is the stage of development of this medicinal product?

At the time of submission of the application for orphan designation, clinical trials in patients with advanced idiopathic Parkinson’s disease with severe motor fluctuations and not responding to oral treatment were ongoing.

Levodopa / carbidopa was not authorised anywhere in the world for advanced idiopathic Parkinson’s disease with severe motor fluctuations and not responding to oral treatment, at the time of submission. Orphan designation of levodopa / carbidopa was granted in the United States in January 2000 for the treatment of late-stage Parkinson’s disease.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 20 March 2001 recommending the granting of this designation.

Update: Levodopa / carbidopa (Duodopa) was designated as an orphan drug in Australia in April 2006. Levodopa / carbidopa (gastroenteral use) (Duodopa intestinal gel) is authorised via mutual recognition in the European Union for the treatment of advanced levodopa-responsive Parkinson's disease with severe motor fluctuations and hyper- / dyskinesia when available combinations of Parkinson medicinal products have not given satisfactory results.

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;
  • and either the rarity of the condition (affecting not more than five in 10,000 people in the Community) or the 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 the quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

Key facts

Product details for <p>Levodopa / carbidopa (gastroenteral use)</p>
Active substanceLevodopa / carbidopa (gastroenteral use)
Medicine Name
Disease/conditionTreatment of advanced idiopathic Parkinson’s disease with severe motor fluctuations and not...
Date of decision10/05/2001
OutcomePositive
Orphan decision numberEU/3/01/035

Review of designation

Sponsor’s contact details:

AbbVie Ltd
Vanwall Business Park
Vanwall Road
Maidenhead
Berkshire
SL6 4XE
United Kingdom
Tel. +44 (0)1628 644501
Fax +44 (0)1628 672624
E-mail: orphandrugs.enquiries@abbvie.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.