EU/3/13/1194

On 7 October 2013, orphan designation (EU/3/13/1194) was granted by the European Commission to Nicox, France, for naproxcinod for the treatment of Duchenne muscular dystrophy.

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

What is Duchenne muscular dystrophy?

Duchenne muscular dystrophy (DMD) is a genetic disease that gradually causes weakness and atrophy (wasting) of the muscles. It mainly affects boys, and usually starts before the age of six years. The muscle weakness usually starts in the hips and legs, before reaching the chest, arms, and in the late stage also the heart. Patients with DMD lack normal dystrophin, a protein found in muscles. Because this protein helps to strengthen and protect muscles from injury as muscles contract and relax, in patients with DMD the muscles become weak and eventually stop working.

DMD causes long-term disability and is life-threatening because of its effects on the heart and the respiratory muscles (muscles that are used to breathe). The disease usually leads to death in adolescence or early adulthood.

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

At the time of designation, DMD 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 is based 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 512,200,000 (Eurostat 2013).

What treatments are available?

At the time of designation, no satisfactory method had been authorised in the European Union to treat DMD. Treatment of patients with DMD primarily involved physiotherapy and other supportive treatments.

How is this medicine expected to work?

Naproxcinod is converted in the body into the medicine naproxen and a chemical that releases nitric oxide. Nitric oxide is a vasodilator, a substance that causes the widening of blood vessels and plays an important role at ensuring that sufficient oxygen reaches the muscle during exercise, thereby reducing or delaying muscle damage. Patients with DMD do not have enough nitric oxide which together with the absence of dystrophin leads to muscle damage and inflammation.

Naproxen is a non-steroidal anti-inflammatory drug (NSAID). It works by blocking an enzyme called cyclo-oxygenase, which produces prostaglandins, substances that are involved in the inflammation process. By reducing the production of prostaglandins, naproxen is expected to reduce the inflammation seen in DMD.

What is the stage of development of this medicine?

The effects of naproxcinod have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with naproxcinod in patients with DMD had been started.

At the time of submission, naproxcinod was not authorised anywhere in the EU for DMD designated as an 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 4 September 2013 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.

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