Designation of Fedratinib as an orphan drug - Sanofi-Aventis Australia Pty Ltd

Overview

The Therapeutic Goods Act 1989 was enacted to provide a legislative framework for regulating therapeutic goods in Australia, ensuring that they are of acceptable quality, safety, and efficacy. This Act was introduced to address the need for a unified regulatory approach to the manufacture, importation, supply, and advertising of therapeutic goods, thereby protecting public health. The Therapeutic Goods Administration (TGA) was established under this Act to administer and enforce the regulatory requirements. The policy objective underpinning this legislation is to ensure that therapeutic goods available in Australia meet rigorous standards that safeguard the health and wellbeing of consumers. In the context of the designation of Fedratinib as an orphan drug, the Act facilitates the recognition of treatments for rare diseases, which might otherwise not be commercially viable to develop without specific incentives. This particular designation, announced by the Delegate of the Secretary, Dr Anthony Gill, on 23 October 2013, aims to encourage the development of treatments for rare conditions such as primary myelofibrosis and its variants by providing certain benefits and regulatory support.

Scope and Application

The Therapeutic Goods Act 1989, administered by the Therapeutic Goods Administration (TGA) in the Commonwealth of Australia, provides a comprehensive regulatory framework for therapeutic goods, including medicines, medical devices, and blood products. The scope of this Act encompasses a wide range of activities and entities, including the designation of orphan drugs. Specifically, the Act applies to the sponsor, manufacturer, importer, and supplier of therapeutic goods, as well as to the advertising, representation, and sale of such goods. In this context, the Act applies to Fedratinib, which has been designated as an orphan drug for the treatment of primary myelofibrosis, as well as post-polycythaemia vera myelofibrosis and post-essential thrombocythaemia myelofibrosis. The designation of Fedratinib as an orphan drug is specifically for the indication of treating these rare blood disorders, and the dose form of Fedratinib for this indication is capsules. The sponsor of Fedratinib is Sanofi-Aventis Australia Pty Ltd. The geographic reach of the Therapeutic Goods Act extends across Australia, with the TGA having jurisdiction to regulate therapeutic goods nationwide. The Act does not specify exclusions, exemptions, or thresholds in this particular gazette, but it does provide for the extension and restriction of its application through subordinate instruments, such as regulations and guidelines, which may further define the scope and specifics of the legislation.

Key Provisions

The main operative sections of this legislation pertain to the designation of Fedratinib as an orphan drug under the Therapeutic Goods Regulations 1990. Specifically, Section 16J(2) of the Regulations empowers a delegate of the Secretary to designate a drug as an orphan drug if certain criteria are met (section 16J(2)). In this case, Dr Anthony Gill, acting as the Delegate of the Secretary, has designated Fedratinib as an orphan drug for the treatment of specific types of myelofibrosis. These include primary myelofibrosis, post-polycythaemia vera myelofibrosis, and post-essential thrombocythaemia myelofibrosis. The drug in question, Fedratinib, is provided in capsule form. The sponsor of Fedratinib is identified as Sanofi-Aventis Australia Pty Ltd. The Therapeutic Goods Act 1989, along with the Therapeutic Goods Regulations 1990, imposes certain obligations on parties involved in the designation and use of orphan drugs. The Act requires that drugs be designated as orphan drugs only when they meet specific criteria, such as treating a rare condition. The sponsor of the drug, in this case Sanofi-Aventis Australia Pty Ltd, must ensure that the drug is appropriately developed, tested, and marketed. The sponsor is also responsible for ensuring that the drug meets the necessary safety and efficacy standards before it can be designated as an orphan drug. Additionally, the Delegate of the Secretary, acting under the authority granted by the Act and Regulations, must follow due process in designating a drug as an orphan drug, including providing public notice of the designation. The Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990 establish various offences and penalties for non-compliance with the provisions of the Act and Regulations. While the specific document does not detail penalties for non-compliance in the context of orphan drug designation, the Act generally provides for both civil and criminal penalties for breaches. Civil penalties can include fines, while criminal penalties can include imprisonment. For example, under Section 26 of the Therapeutic Goods Act 1989, it is an offence to knowingly supply a therapeutic good that does not comply with the Act or Regulations, with potential penalties including fines of up to $222,000 for individuals and $1,110,000 for corporations, as well as imprisonment for up to five years. Furthermore, Section 33 of the Act allows for the imposition of fines for misleading or deceptive conduct in relation to therapeutic goods, with penalties up to $1,110,000 for individuals and $5,550,000 for corporations. These penalties underscore the importance of compliance with the regulatory requirements governing therapeutic goods in Australia.