Designation of Sodium Benzoate (Prohippur) as an orphan drug - Orpharma Pty Ltd

Overview

The Therapeutic Goods Act 1989, enacted by the Parliament of Australia, was introduced to regulate therapeutic goods, including medicines, medical devices, and related products, to ensure their quality, safety, and efficacy. The Act aims to protect public health by controlling the manufacturing, importation, advertising, and distribution of therapeutic goods. One of the policy objectives of the Act is to facilitate access to orphan drugs, which are medicines intended for the treatment of rare medical conditions that affect a small number of people. The Act allows for the designation of certain drugs as orphan drugs, providing incentives for their development and ensuring they are available to patients who need them. In line with this objective, the Therapeutic Goods Administration, under the authority of the Therapeutic Goods Act 1989, has designated Sodium Benzoate (Prohippur) as an orphan drug on 6 December 2016 for the treatment of specific rare metabolic disorders, thereby recognising the importance of making such treatments accessible to those in need.

Scope and Application

The Therapeutic Goods Act 1989, as amended and applied through the Therapeutic Goods Regulations 1990, designates specific substances as therapeutic goods and regulates their use within Australia. In this instance, Sodium Benzoate (Prohippur) has been designated as an orphan drug for the treatment of several rare metabolic disorders, including Argininaemia, Arginosuccinic aciduria, Citrullinaemia type I, Ornithine translocase deficiency (HHH syndrome), Lysinuric protein intolerance, N-acetylglutamate synthetase deficiency, and Ornithine transcarbamylase deficiency. This designation applies to Sodium Benzoate (Prohippur) in the form of granules, and the sponsor of this therapeutic good is Orpharma Pty Ltd. The legislation applies across the Commonwealth of Australia, ensuring a national standard for the use and regulation of therapeutic goods. Exclusions or exemptions are not specified in this particular gazette; however, the scope of application can be extended or restricted through subordinate instruments issued under the authority of the Act. The primary objective of this designation is to ensure that therapeutic goods for rare diseases are available and regulated appropriately, thereby addressing the specific needs of patients suffering from these rare conditions.

Key Provisions

The Therapeutic Goods Act 1989, as amended, includes specific provisions that allow for the designation of certain therapeutic goods under particular conditions. In this case, Section 16J of the Therapeutic Goods Regulations 1990 provides the framework for designating orphan drugs. According to this section, a therapeutic good can be designated as an orphan drug if it is intended for the treatment, prevention, or diagnosis of a rare disease or condition. The designation under Section 16J(2) specifically identifies Sodium Benzoate (Prohippur) as an orphan drug for the treatment of certain rare metabolic disorders such as Argininaemia, Arginosuccinic aciduria, Citrullinaemia type I, Ornithine translocase deficiency (HHH syndrome), Lysinuric protein intolerance, N-acetylglutamate synthetase deficiency, and Ornithine transcarbamylase deficiency. Under the Act and the Regulations, the designation of Sodium Benzoate (Prohippur) as an orphan drug entails several obligations and requirements for the parties involved. Firstly, the sponsor of the drug, in this instance, Orpharma Pty Ltd, must ensure that the drug is manufactured and supplied in compliance with the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990. The sponsor is also required to provide sufficient evidence and data to support the orphan drug designation, including information on the drug's efficacy, safety, and the nature of the rare conditions it is intended to treat. Additionally, the sponsor must comply with any post-market surveillance and reporting requirements to monitor the drug's performance and safety profile in the market. Failure to comply with the provisions of the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990 can lead to various civil and criminal consequences. For example, under Section 33 of the Act, manufacturing, supplying, or advertising a therapeutic good in a way that contravenes the Act or the Regulations can result in civil penalties. The maximum penalty for corporations can be up to $2,100,000 for each offence, while individuals may face penalties up to $210,000 for each offence. Additionally, under Section 34, supplying a therapeutic good that does not comply with the Act or the Regulations can result in criminal penalties. For corporations, the maximum penalty is up to $10,500,000 for each offence, and for individuals, it can be up to $1,050,000 for each offence. These penalties underscore the importance of adherence to the regulatory requirements governing the designation and use of orphan drugs in Australia.