EU/3/14/1318

On 22 August 2014, orphan designation (EU/3/14/1318) was granted by the European Commission to BSV BioScience GmbH, Germany, for ulinastatin for the treatment of acute pancreatitis.

What is acute pancreatitis?

Acute pancreatitis is a disease characterised by sudden, painful inflammation of the pancreas, an organ of the digestive system. The pancreas produces various enzymes in an inactive form, which are secreted into the gut where they become active and help break down components of the diet, especially proteins. In acute pancreatitis, some of these enzymes become active in the pancreas itself and start to break down the tissues of the pancreas and surrounding organs, causing inflammation and pain and killing the cells. Complications may include infection, internal bleeding and organ failure. Pancreatitis can have various causes but is often triggered by gallstone disease or excess alcohol consumption.

Acute pancreatitis is potentially life threatening due to complications such as internal bleeding and organ failure.

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

At the time of designation, acute pancreatitis affected approximately 4.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 230,000 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 511,100,000 (Eurostat 2014).

What treatments are available?

At the time of designation, no satisfactory methods were authorised in the EU for the treatment of acute pancreatitis. Patients were generally managed with supportive care, including fluid replacement, oxygen, feeding via a tube or into a vein, antibiotics and painkillers. Severely ill patients with organ failure needed to be managed in intensive-care units.

How is this medicine expected to work?

The active substance in this medicine, ulinastatin, is a protease inhibitor, a substance which blocks the actions of enzymes that break down protein. By blocking the effect of these enzymes in the pancreas it is expected to reduce the tissue damage and inflammation of acute pancreatitis, relieving the symptoms of the condition and reducing its complications.

What is the stage of development of this medicine?

The effects of ulinastatin have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with ulinastatin in patients with acute pancreatitis had been completed.

Ulinastatin was authorised in China, India, Japan and South Korea for acute pancreatitis. Ulinastatin was not authorised anywhere in the EU for acute pancreatitis or 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 10 July 2014 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.