EU/3/17/1875

On 22 May 2017, orphan designation (EU/3/17/1875) was granted by the European Commission to NeoImmuneTech, Inc, Poland, for recombinant human interleukin-7 fused to a hybrid crystallisable fragment region of a human antibody for treatment of idiopathic CD4 lymphocytopenia.

What is idiopathic CD4 lymphocytopenia?

Idiopathic CD4 lymphocytopenia is a condition in which patients have very low levels of a type of white blood cell called CD4 T cell for no apparent reason. To diagnose this condition, doctors have to rule out other causes of low CD4 T cell levels, including HIV infection and treatments that weaken the immune system.

Because their immune system is weakened, many patients may have recurring, sometimes severe, infections. Patients also commonly have auto-immune diseases, where the immune system attacks the patients’ own tissues, but the relationship between the two conditions is unclear. Patients may also have cancers linked to viral infections such as EBV-associated lymphomas and Kaposi sarcoma.

Idiopathic CD4 lymphocytopenia is life threatening and long-term debilitating due to the recurring infections.

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

At the time of designation, idiopathic CD4 lymphocytopenia affected less than 0.01 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 500 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 515,700,000 (Eurostat 2017).

What treatments are available?

At the time of the orphan designation, there were no satisfactory treatments for idiopathic CD4 lymphocytopenia authorised in the EU. CD4 T cell levels were carefully monitored and infections detected and treated promptly. Patients also received treatments to prevent infections.

How is this medicine expected to work?

The medicine is made of interleukin-7, a natural substance in the blood which is involved in the development of CD4 T cells. It is expected to increase the patient’s CD4 T cell levels and by doing so help fight and prevent infection.

The interleukin-7 in this medicine has been made by a method known as ‘recombinant DNA technology’: it is made by cells into which a gene (DNA) has been introduced that enables them to produce interleukin-7. In this medicine the interleukin-7 is attached to part of a protein, which is expected to make it stay in the body for longer.

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 the medicine in patients with idiopathic CD4 lymphocytopenia had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for idiopathic CD4 lymphocytopenia 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 11 April 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.

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