CRED ERP 25

Case Study 1

TOPRA CRED EU Procedures

TOPRA CRED EU PROCEDURES

Case Study 1

Introduction

Your company, EsraBio Ltd, is developing a new endothelin receptor antagonist drug for use in the orphan indication Primary Arterial Hypertension (PAH). The new product has less side-effects when compared with two other drugs already authorised in the same indication and there are early indications of improved efficacy. This makes it a very attractive option for use in patients in this disease setting. The new product is called Pulmolax.

EsraBio is a multinational company with affiliates across 10 member states across Europe and has its headquarters in the US.

You are responsible for developing the regulatory strategy and for filing the submission for this product in Europe.

Relevant information relating to Pulmolax

The drug substance (DS) in Pulmolax is a New Chemical Entity (NCE).

Route of administration is IV.

Pulmolax has Orphan Drug Designation (ODD) based on its potential for improved safety and/or efficacy (the COMP has granted an orphan designation for this product which has a different active substance from a previously approved product for this orphan indication; the indication itself already has orphan status).

The DS is manufactured in China by a manufacturing site which has not been inspected by any EU competent authority.

Pulmolax appears to have a very strong competitor advantage from a side-effects and efficacy point of view.

There are two competitor products on the market for treatment of the same condition.

Competitor 1 was authorised centrally for PAH 3 years ago and has Orphan Medicinal product status.

Competitor 2 was authorised nationally for PAH in one member state 8 years ago, and does not have Orphan Drug status.

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