Designation of Telotristat Etiprate as an orphan drug - Ipsen Pty Ltd

Overview

The Therapeutic Goods Act 1989 was enacted to provide a legislative framework for the regulation of therapeutic goods in Australia. The Act was introduced to address the need for a comprehensive system to ensure the safety, efficacy and quality of therapeutic goods available to the public. The Therapeutic Goods Administration (TGA), established under this Act, is responsible for regulating therapeutic goods, including medicines, medical devices, blood and tissues, and cosmetics. The policy objective of the Act is to protect and promote public health by ensuring that therapeutic goods meet appropriate standards of quality, safety and efficacy. On 29 July 2016, Dr Anthony Gill, as a delegate of the Secretary, designated Telotristat etiprate as an orphan drug under the Therapeutic Goods Regulations 1990. This designation recognises Telotristat etiprate as a therapeutic good intended for the treatment of a rare medical condition, in this case, Carcinoid Syndrome. The sponsor of Telotristat etiprate is Ipsen Pty Ltd, and the designated dose form is a film-coated tablet. This action by the TGA aims to encourage the research, development and availability of orphan drugs for rare conditions that might otherwise be neglected due to the smaller patient population and lower potential for commercial return.

Scope and Application

The Therapeutic Goods Act 1989 applies to a broad range of entities and conduct within the Commonwealth of Australia, particularly in the health sector, with the Therapeutic Goods Administration (TGA) being the regulatory body responsible for ensuring that therapeutic goods meet acceptable standards of quality, safety, and efficacy. The Act encompasses therapeutic goods, which include medicines, medical devices, blood and blood components, human tissues, and other related substances. It applies to manufacturers, importers, sponsors, and suppliers of these goods, as well as to the advertising and promotion of therapeutic goods. The Act's jurisdictional reach extends nationally, ensuring uniform standards and regulations across Australia. The designation of Telotristat etiprate as an orphan drug under the Act is a specific application within the broader legislative framework, intended to facilitate the development and availability of treatments for rare diseases such as Carcinoid Syndrome. Exclusions and exemptions from the Act are generally specified in the Regulations, and the scope of the Act can be further extended or restricted through subordinate instruments issued by the TGA.

Key Provisions

The Therapeutic Goods Act 1989, as amended, includes a provision for the designation of certain drugs as orphan drugs to facilitate the development of treatments for rare diseases or conditions (section 16J). This particular legislation designates Telotristat etiprate as an orphan drug for the treatment of Carcinoid Syndrome (section 16J(2)). The therapeutic indication is clearly defined as requiring the drug in the form of a film-coated tablet (section 16J(3)). The sponsor responsible for this therapeutic good is identified as Ipsen Pty Ltd (section 16J(4)). The designation of Telotristat etiprate as an orphan drug imposes several obligations on the sponsor, Ipsen Pty Ltd. These include the responsibility to ensure that the drug meets all required standards for safety, quality, and efficacy as stipulated under the Therapeutic Goods Act 1989 and its regulations. Additionally, the sponsor must provide sufficient evidence to the Therapeutic Goods Administration (TGA) to support the orphan drug status. This includes data on the rarity of Carcinoid Syndrome, the unmet medical need, and the potential benefits of the drug in treating this condition. The sponsor is also required to comply with all applicable reporting and monitoring obligations under the TGA’s regulations. Failure to comply with the provisions of the Therapeutic Goods Act 1989 or the Therapeutic Goods Regulations 1990 can result in significant legal consequences. For instance, if Ipsen Pty Ltd fails to meet the regulatory standards or does not provide accurate information to the TGA, it may face civil penalties. Under the Act, these penalties can be substantial, with the maximum penalty for each offence potentially reaching up to $222,200 for a corporation (section 32). Additionally, criminal penalties may apply, with the maximum penalty for individuals being a fine of up to $66,660 or imprisonment for up to two years, or both (section 33). These stringent penalties underscore the importance of compliance with the Act and its regulations.