Designation of Stiripentol (Diacomit) As An Orphan Drug

Overview

The Therapeutic Goods Act 1989, enacted by the Parliament of Australia, aims to regulate therapeutic goods to protect public health by ensuring the safety, quality and efficacy of these goods. The Act was introduced to address the need for a comprehensive regulatory framework governing the provision and use of therapeutic goods, including medicines, medical devices, and other related products. One of the significant policy objectives of the Act is to facilitate access to therapeutic goods that are essential for addressing rare diseases and unmet medical needs. In this context, the designation of stiripentol (DIACOMIT) as an orphan drug under the Therapeutic Goods Regulations 1990 seeks to encourage the research, development and availability of treatments for rare conditions such as severe myoclonic epilepsy in infancy (SMEI or Dravet syndrome). This initiative, signed by Dr Anthony Gill, Delegate of the Secretary on 23 June 2016, is a reflection of the Australian Government's commitment to addressing the healthcare needs of patients with rare diseases and supporting the therapeutic goods industry in Australia.

Scope and Application

The Therapeutic Goods Act 1989, as applied through the Therapeutic Goods Regulations 1990, designates stiripentol (DIACOMIT) as an orphan drug for the treatment of severe myoclonic epilepsy in infancy, also known as Dravet syndrome. This designation applies to individuals suffering from this specific medical condition and encompasses the therapeutic goods in question, namely the capsule and oral powder formulations of stiripentol (DIACOMIT). The sponsor for this orphan drug designation is Emerge Health Pty Ltd. The legislative action is taken under the authority of the Therapeutic Goods Administration (TGA), which is part of the Department of Health in the Commonwealth of Australia. This designation extends nationally across Australia, impacting the availability and regulation of therapeutic goods for this orphan indication within the country. The scope of the designation is limited to the specified medical condition and formulation, and no exclusions or exemptions are explicitly stated in this particular legislative action. The broader regulatory framework, however, may provide for additional controls and requirements through subordinate instruments or other legislative provisions.

Key Provisions

The Therapeutic Goods Act 1989 designates specific therapeutic goods to be considered orphan drugs for particular medical conditions. In this instance, section 41FL of the Act enables the designation of drugs that treat rare medical conditions, including severe myoclonic epilepsy in infancy (SMEI or Dravet syndrome). The designation is meant to encourage the development and availability of treatments for conditions that affect a small number of people in Australia. Section 16J of the Therapeutic Goods Regulations 1990 further outlines the process and criteria for such designations. The designation of stiripentol (DIACOMIT) as an orphan drug places certain obligations on the sponsor, Emerge Health Pty Ltd. This includes ensuring the drug is available for the specified condition, complying with all relevant regulations regarding the manufacture, importation, and supply of the drug, and providing any necessary information to the Therapeutic Goods Administration (TGA) to support the orphan drug designation. The sponsor must also ensure that the drug is safe and effective for its intended use, and that it meets the standards set out in the Therapeutic Goods Regulations. Breaches of the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990 can result in significant consequences. For example, under section 33 of the Act, manufacturing, supplying, or advertising a therapeutic good in a way that contravenes the Act or Regulations can lead to criminal charges. Section 34 of the Act imposes civil penalties for such contraventions, including fines of up to $1,215,000 for corporations and $243,000 for individuals. Additionally, section 35 allows for the issuance of infringement notices for certain offences, with penalties of up to $2,430 for individuals and $12,150 for corporations. The Act also provides for the seizure and destruction of therapeutic goods that do not comply with the regulations, as stipulated in section 85.