EU/3/22/2611: Orphan designation for the treatment of focal segmental glomerulosclerosis

Fusion protein composed of the first 2 immunoglobulin-like domains of the human Roundabout guidance receptor 2 fused to a human IgG1 crystallised fragment

Overview
Key facts

Overview

This medicine was designated as an orphan medicine for the Treatment of focal segmental glomerulosclerosis in the European Union on 16 May 2022.

This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply for a marketing authorisation.

Orphan designation does not mean the medicine is available or authorised for use. All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.

During the medicine's development, doctors may be able to enrol patients in clinical trials investigating the medicine. For information on ongoing clinical trials in the EU, see:

EU Clinical Trials Register

ClinicalTrials.gov

: Focal segmental glomerulosclerosis (FSGS) is a rare kidney disease featuring scarring (sclerosis) of glomeruli, the filtering units of the kidney, and damage to podocytes which are cells in the filtering barrier. The damage results in protein loss in the urine (proteinuria) and progressive loss of kidney function. This medicine, also known as PF-06730512, is a fusion protein that binds to a protein called SLIT2. By attaching to SLIT2, the medicine prevents its interaction with a receptor (target) called ROBO2 on podocytes. This enhances podocyte structure and function in the filtering barrier and is expected to lead to reduced proteinuria and slowing of the loss of kidney function.
Based on description provided by sponsor

At the time of submission of the application for orphan designation:

The effects of the medicine had been evaluated in experimental models.
Clinical trials with the medicine in patients with focal segmental glomerulosclerosis were ongoing.

More information on how potential new medicines are tested during their development is available on Authorisation of medicines.

Medicines intended for rare diseases can be granted an orphan designation during their development.

The orphan designation allows the developer to benefit from:

scientific and regulatory support to advance their medicine to the stage where they can request marketing authorisation;

market exclusivity once the medicine is on the market.

To qualify for orphan designation, a medicine must meet a number of criteria:

it must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;

the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;

there are no satisfactory alternative methods for the diagnosis, prevention or treatment of the condition or, if such a method exists, the medicine is of significant benefit to those affected by the condition.

EMA's Committee for Orphan Medicinal Products (COMP) is responsible for issuing opinions on applications for orphan designations.

The Agency sends the COMP opinion to the European Commission, which is responsible for granting the orphan designation. The full list of orphan designations is available in the Community register of orphan medicinal products for human use.

For more information, see:

Orphan designation: Overview

Rare diseases, orphan medicines: Getting the facts straight

Key facts

Active substance	Fusion protein composed of the first 2 immunoglobulin-like domains of the human Roundabout guidance receptor 2 fused to a human IgG1 crystallised fragment
Intended use	Treatment of focal segmental glomerulosclerosis
Orphan designation status	Withdrawn
EU designation number	EU/3/22/2611
Date of designation	16/05/2022
Sponsor	Pfizer Europe MA EEIG Boulevard de la Plaine 17 1050 Bruxelles Brussels-Capital Region Belgium E-mail: orphan_enquiries@pfizer.com

Update history

Date	Update
July 2023	Please note that this product was withdrawn from the Union Register of orphan medicinal products in July 2023 on request of the Sponsor.

Documents related to this orphan designation evaluation

Minutes of the COMP meeting on 11-13 April 2022 (PDF/451.83 KB)

Adopted

First published: 16/06/2022
EMA/COMP/214405/2022

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

European Commission: Community Register of orphan medicinal products

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