EU/3/18/2100: Orphan designation for the treatment of focal segmental glomerulosclerosis

Focal segmental glomerulosclerosis is a disease where the glomeruli (small filtering units in the kidneys which filter waste products from the blood and start urine production) are damaged and become scarred. This stops them from working properly, so that substances needed by the body, such as proteins, pass into the urine. Focal segmental glomerulosclerosis can affect both adults and children. Symptoms include swelling, low blood protein levels and high blood pressure. It is called 'focal segmental' because only parts of some glomeruli are affected.

Focal segmental glomerulosclerosis is a long-term debilitating and life-threatening disease because it may lead to kidney failure.

At the time of designation, focal segmental glomerulosclerosis affected approximately 2.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 135,000 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).

At the time of designation, ciclosporin was authorised in some EU countries specifically for the treatment of focal segmental glomerulosclerosis, while other medicines were authorised to treat the symptoms.

The sponsor has provided sufficient information to show that propagermanium might be of significant benefit for patients with focal segmental glomerulosclerosis. Laboratory studies showed that the medicine could be an alternative for patients in whom cyclosporin is not effective. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

Inflammation is an important aspect of focal segmental glomerulosclerosis. Propagermanium is expected to work by blocking a receptor (target) on the surface of kidney cells called CCR2, which is involved in the inflammatory response in the kidney. By blocking CCR2, the medicine is expected to reduce the inflammation associated with the condition, therefore reducing damage and scarring to the glomeruli and improving symptoms of the condition.

The effects of propagermanium 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 focal segmental glomerulosclerosis had been started.

At the time of submission, propagermanium was not authorised anywhere in the EU for focal segmental glomerulosclerosis. Orphan designation of the medicine had been granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 11 October 2018 recommending the granting of this designation.

• 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.