EU/3/17/1891 - orphan designation for treatment of primary hyperoxaluria

Bacillus subtilis oxalate decarboxylase

Overview

On 17 July 2017, orphan designation (EU/3/17/1891) was granted by the European Commission to Allena Pharmaceuticals Ireland Limited, Ireland, for Bacillus subtilis oxalate decarboxylase (also known as ALLN-177) for the treatment of primary hyperoxaluria.

What is primary hyperoxaluria?

Primary hyperoxaluria is an inherited disease in which patients suffer from recurring kidney and bladder stones which lead to pain, blood in the urine and frequent urinary tract infections. The disease is caused by the lack of certain enzymes produced by the liver that are needed to breakdown a substance called glyoxylate in the body. Instead of being converted into the amino acid glycine, glyoxalate is therefore converted into excess oxalate. This can form calcium oxalate deposits, which cause kidney and bladder stones and may damage the kidneys and other organs.

Primary hyperoxaluria is long-term debilitating and life threatening because of the high rate of kidney failure seen in patients with the condition.

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

At the time of designation, primary hyperoxaluria affected approximately 0.1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 5,000 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 515,700,000 (Eurostat 2017).

What treatments are available?

At the time of designation, no satisfactory methods were authorised in the EU for treating primary hyperoxaluria. Different treatments were used to prevent the accumulation of calcium oxalate such as dietary changes, drinking plenty of fluids and taking vitamin B6. Kidney and liver transplantation have been possible options in patients with kidney failure.

How is this medicine expected to work?

The medicine contains an enzyme capable of breaking down oxalate that was originally derived from the bacterium Bacillus subtilis. When the medicine is swallowed the enzyme passes through the gut and breaks down oxalate present in food that would otherwise be absorbed, as well as oxalate that passes from the body into the gut. This action is expected to lower levels of oxalate in the body and so help reduce the formation of kidney and bladder stones.

What is the stage of development of this medicine?

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

At the time of submission of the application for orphan designation, no clinical trials with Bacillus subtilis oxalate decarboxylase in patients with primary hyperoxaluria had yet started.

At the time of submission, this medicine was not authorised anywhere in the EU for primary hyperoxaluria. Orphan designation of the medicine had been granted in the United States for both primary and paediatric hyperoxaluria.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 15 June 2017 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/3/17/1891: Public summary of opinion on orphan designation: Bacillus subtilis oxalate decarboxylase for the treatment of primary hyperoxaluria

Reference Number: EMA/402771/2017

Key facts

• Active substance:
  Bacillus subtilis oxalate decarboxylase
• Intended use:
  Treatment of primary hyperoxaluria
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/17/1891
• Date of designation:
  17/07/2017
• Sponsor:

  Allena Pharmaceuticals Ireland Limited; Arthur Cox Building; Earlsfort Terrace; Dublin 2; DOC CK83; Ireland; Tel. [phone]; E-mail: [e-mail]

Review of designation

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