Designation of Eliglustat (TBA) as an Orphan drug - Sanofi-Aventis Australia Pty Ltd

Overview

The Therapeutic Goods Act 1989 was enacted by the Parliament of Australia to regulate therapeutic goods, including medicines, medical devices, and blood, within the country. This Act was introduced to address the need for a comprehensive regulatory framework that ensures the safety, quality, and efficacy of therapeutic goods available to the public. In line with this objective, the Act establishes the Therapeutic Goods Administration (TGA) as the regulatory body responsible for overseeing the regulation of therapeutic goods. The 2013 Gazette C2013G01206 issued under the Therapeutic Goods Act 1989 designates eliglustat (TBA) as an orphan drug for the long-term treatment of adult patients with Gaucher disease type 1 (GD1). This designation is granted to encourage the development of treatments for rare diseases, addressing a policy objective to support the availability of therapeutic options for patients suffering from less common conditions. The Delegate of the Secretary, Dr Anthony Gill, made this designation on 2 August 2013, with the sponsor of the drug being Sanofi-Aventis Australia Pty Ltd.

Scope and Application

The Therapeutic Goods Act 1989, administered by the Therapeutic Goods Administration (TGA) under the Commonwealth of Australia's Department of Health and Ageing, provides the legislative framework for the regulation of therapeutic goods in Australia. Under this Act, the designation of a drug as an orphan drug can significantly affect its development and availability, particularly for rare diseases. In this instance, eliglustat (TBA) has been designated as an orphan drug for the long-term treatment of adult patients with Gaucher disease type 1 (GD1), effective from 2 August 2013. This designation applies to the specific dose form of eliglustat (TBA), which is in capsule form, and is sponsored by Sanofi-Aventis Australia Pty Ltd. The scope of the Act encompasses the regulation of therapeutic goods, including the designation of orphan drugs intended to treat rare medical conditions, thereby facilitating access to potentially life-saving treatments for patients with unmet medical needs. The Act’s jurisdictional reach is national, applying across all states and territories of Australia. This legislative action does not specify exclusions or exemptions but is subject to the broader provisions and subordinate instruments of the Therapeutic Goods Act 1989 and its Regulations.

Key Provisions

The Therapeutic Goods Act 1989, under the authority of the Therapeutic Goods Administration (TGA), includes provisions that allow for the designation of certain drugs as orphan drugs through the Therapeutic Goods Regulations 1990. Specifically, section 16J of the Regulations outlines the criteria and process for designating orphan drugs, which are intended for the treatment of rare medical conditions that affect a small number of people. In this instance, eliglustat (TBA) has been designated as an orphan drug for the treatment of adult patients with Gaucher disease type 1 (GD1) (section 16J(2)). Under the Act, the designation of eliglustat (TBA) as an orphan drug imposes several obligations on the parties involved. Firstly, the sponsor of the drug, in this case, Sanofi-Aventis Australia Pty Ltd, must ensure that the drug meets the necessary safety, efficacy, and quality standards as stipulated by the TGA. The sponsor is also required to provide comprehensive data supporting the drug’s therapeutic benefits for the specified condition and ensure that it is manufactured and distributed in compliance with regulatory requirements. Additionally, the sponsor must maintain records and documentation that demonstrate adherence to these obligations. Failure to comply with the provisions of the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990 can result in significant consequences. Violations of the Act may lead to civil or criminal penalties, including fines and imprisonment. For instance, under section 33HA of the Act, any person who contravenes certain provisions related to therapeutic goods can face penalties. The maximum penalties vary depending on the nature of the offence, but can include substantial fines for corporations and imprisonment for individuals. Furthermore, the TGA has the authority to take enforcement actions such as recalling non-compliant products, issuing public warnings, or revoking the registration of the therapeutic good in severe cases. These measures are designed to protect public health and ensure that therapeutic goods available in the market are safe, effective, and of high quality.