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

Overview

The Therapeutic Goods Act 1989, enacted by the Commonwealth Parliament, serves as the primary legislative framework for regulating therapeutic goods in Australia, aiming to ensure the safety, quality, and efficacy of such goods. The 1989 Act provides the necessary powers and mechanisms to govern the therapeutic goods industry, addressing gaps and problems in the regulation of medicines, medical devices, and other related products. The Delegate of the Secretary, Dr Anthony Gill, exercised authority under the Therapeutic Goods Regulations 1990 to designate Sodium Benzoate (Prohippur) as an orphan drug for the treatment of Nonketotic hyperglycinaemia (NKH), highlighting the policy objective to support the development of treatments for rare diseases that affect a small number of people in Australia. On 6 December 2016, Sodium Benzoate (Prohippur) was officially designated as an orphan drug for the treatment of NKH, with granules being the approved dose form. This designation was made by Dr Anthony Gill, the Delegate of the Secretary, under subregulation 16J(2) of the Therapeutic Goods Regulations 1990. The sponsor of Sodium Benzoate (Prohippur) is Orpharma Pty Ltd, which has taken on the responsibility of developing and supplying this therapeutic good to address the specific needs of patients suffering from NKH, a rare and debilitating condition.

Scope and Application

The Therapeutic Goods Act 1989, through its regulations, applies to entities involved in the supply of therapeutic goods within Australia, including drugs such as Sodium Benzoate (Prohippur) designated as orphan drugs. This designation pertains specifically to Sodium Benzoate (Prohippur) for the treatment of Nonketotic hyperglycinaemia (NKH), a rare condition, and is issued under the authority of the Therapeutic Goods Administration (TGA). The Act extends its reach to the Commonwealth level, regulating the importation, supply, and advertising of therapeutic goods to ensure public health and safety. The designation of Sodium Benzoate (Prohippur) as an orphan drug is a specific measure aimed at facilitating the availability of treatments for rare diseases by providing certain incentives and streamlined regulatory processes to sponsors like Orpharma Pty Ltd. The Act and its regulations may also provide for exemptions or thresholds in certain circumstances, although this particular gazette does not detail such provisions explicitly. The application and scope of the Therapeutic Goods Act 1989 can be further defined and modified through subordinate instruments, which may include additional regulations or guidelines issued by the TGA.

Key Provisions

The key operative sections of this legislation are found in the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990. Specifically, section 16J of the Regulations is the provision under which Sodium Benzoate (Prohippur) is designated as an orphan drug for the treatment of Nonketotic hyperglycinaemia (NKH). This designation (section 16J(2)) is made by a Delegate of the Secretary, in this case Dr Anthony Gill, on 6 December 2016. The document specifies that the dose form of Sodium Benzoate (Prohippur) for this condition is granules, and the sponsor of the drug is Orpharma Pty Ltd. The obligations and requirements imposed by this Act pertain primarily to the sponsor, Orpharma Pty Ltd, and the Delegate of the Secretary, Dr Anthony Gill. Under section 16J of the Regulations, the sponsor must ensure that the drug is manufactured and supplied in compliance with the therapeutic goods legislative framework. This includes adhering to quality standards and ensuring that the drug is safe and effective for its intended use. The Delegate of the Secretary, in this instance Dr Anthony Gill, is tasked with making the designation of Sodium Benzoate (Prohippur) as an orphan drug, which involves assessing whether the drug meets the criteria for orphan drug designation as set out in the Regulations. Breaches of the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990 can result in various offences and penalties. For instance, supplying therapeutic goods that do not comply with the Act or Regulations can be an offence. The penalties for such breaches can be substantial. For example, under section 32DA of the Act, a corporation can be fined up to 500,000 Australian dollars for each offence if the contravention is serious. For less serious contraventions, the maximum penalty is generally 10,000 Australian dollars per offence. Additionally, individuals responsible for the contravention can also be subject to penalties, including fines and imprisonment. The specific penalties depend on the nature and seriousness of the contravention, as well as any previous convictions.