EU/3/16/1783 - orphan designation for treatment of acute pancreatitis

N-(5-(6-chloro-2,2-difluorobenzo[d][1,3]dioxol-5-yl)pyrazin-2-yl)-2-fluoro-6-methylbenzamide

Overview

On 18 November 2016, orphan designation (EU/3/16/1783) was granted by the European Commission to EMAS Pharma Limited, United Kingdom, for N-(5-(6-chloro-2,2-difluorobenzo[d][1,3]dioxol-5-yl)pyrazin-2-yl)-2-fluoro-6-methylbenzamide (also called CM4620) for the treatment of acute pancreatitis.

The sponsorship was transferred:
•    to Cogas Pharma Limited, Ireland in April 2017;
•    to Reglntel Ltd, Ireland in July 2018 and 
•    to Turnkey Pharmaconsulting Ireland Limited, Ireland in July 2020.

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. 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 multiple organ failure.

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

At the time of designation, acute pancreatitis affected approximately 4.1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 211,000 people¹, 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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¹Disclaimer: 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 513,700,000 (Eurostat 2016).

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 medicine blocks the action of CRAC channels, tiny pores in the outer surface of cells that help to regulate the movement of calcium into the cell. Evidence suggests that this movement of calcium is involved in the development of the damage and cell death that occur in the pancreas in acute pancreatitis. By blocking these channels, the medicine is expected to help reduce this damage and so limit the symptoms of the condition.

What is the stage of development of this medicine?

The effects of this medicine have been evaluated in experimental models.

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

At the time of submission, the medicine 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 6 October 2016 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.

EU/3/16/1783: Public summary of positive opinion for orphan designation of N-(5-(6-chloro-2,2-difluorobenzo[d][1,3]dioxol-5-yl)pyrazin-2-yl)-2-fluoro-6-methylbenzamide for the treatment of acute pancreatitis

Key facts

• Active substance:
  N-(5-(6-chloro-2,2-difluorobenzo[d][1,3]dioxol-5-yl)pyrazin-2-yl)-2-fluoro-6-methylbenzamide
• Intended use:
  Treatment of acute pancreatitis
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/16/1783
• Date of designation:
  18/11/2016
• Sponsor:

  Turnkey PharmaConsulting Ireland Limited; Ellerman House ; Cratloe Wood ; Cratloe V95 X925; County Clare ; Ireland ; Tel: [phone] ; E-mail: [e-mail]