Designation of Albutrepenonacog Alfa as an orphan drug - CSL Behring Australia

Overview

The Therapeutic Goods Act 1989 (TGA Act) was enacted to regulate the import, supply, sale, and manufacture of therapeutic goods in Australia, aiming to ensure their quality, safety, and efficacy. This legislation was introduced to address the need for a robust regulatory framework that safeguards public health by controlling the therapeutic goods market. The Act is administered by the Therapeutic Goods Administration (TGA), an agency within the Department of Health. The policy objective behind the Act is to protect and promote public health by ensuring that therapeutic goods available in Australia are of acceptable quality, performance, and safety. On 27 October 2014, Dr. Anthony Gill, as the Delegate of the Secretary, exercised powers under the Therapeutic Goods Regulations 1990 to designate albutrepenonacog alfa as an orphan drug for the treatment and prophylaxis of bleeding associated with congenital factor IX deficiency, also known as haemophilia B. This designation recognises the rarity of the condition and aims to encourage the development of treatments for such rare diseases by providing certain incentives and regulatory support. The sponsor of albutrepenonacog alfa is CSL Behring Australia Pty Ltd.

Scope and Application

The Therapeutic Goods Act 1989 applies to the designation of therapeutic goods within the Commonwealth of Australia. Specifically, this designation involves the Therapeutic Goods Administration, which is a department within the Commonwealth of Australia responsible for regulating therapeutic goods. The Act applies to the designation of albutrepenonacog alfa as an orphan drug for the treatment and prophylaxis of bleeding associated with congenital factor IX deficiency, also known as haemophilia B. The scope of this Act pertains to the identification and regulation of therapeutic goods, including the designation of certain drugs as orphan drugs for rare medical conditions. This particular Act designates albutrepenonacog alfa as an orphan drug, thereby potentially providing benefits such as financial incentives or streamlined regulatory processes for the treatment of haemophilia B. The Act applies to the sponsor of the drug, CSL Behring Australia Pty Ltd, and the drug itself, albutrepenonacog alfa, for the specified medical condition. The geographic reach of this Act is national, applying across the Commonwealth of Australia. There are no stated exclusions, exemptions, or thresholds in this particular gazette. The Therapeutic Goods Regulations 1990 may extend or restrict application through subordinate instruments, but no such extensions or restrictions are mentioned in this specific gazette.

Key Provisions

The Therapeutic Goods Act 1989, through the Therapeutic Goods Regulations 1990, includes provisions for the designation of certain drugs as orphan drugs (sections 16J(1) and 16J(2)). In this context, albutrepenonacog alfa has been designated as an orphan drug for the treatment and prophylaxis of bleeding associated with congenital factor IX deficiency, also known as haemophilia B (section 16J(2)). This designation is specific to the injection form of the drug and is applicable as of 27 October 2014. The sponsor of albutrepenonacog alfa is CSL Behring Australia Pty Ltd. Entities governed by the Therapeutic Goods Act and Regulations, including CSL Behring Australia Pty Ltd, are required to comply with the specific provisions that pertain to orphan drug designation. This includes ensuring that the drug meets the criteria for orphan drug status, which typically involves the treatment of rare diseases or conditions that affect a small number of people. Additionally, these entities must adhere to any other regulatory requirements outlined in the Act and Regulations, such as ensuring the drug's safety, efficacy, and quality. Failure to comply with the requirements of the Therapeutic Goods Act and Regulations can result in various consequences. For instance, any entity found to be in breach of the Act may face civil or criminal penalties. The specific penalties can include fines and, in some cases, imprisonment, depending on the severity of the breach. The maximum penalties are stipulated within the Act and can vary based on the specific offence committed. It is imperative for entities like CSL Behring Australia Pty Ltd to ensure full compliance to avoid these potential repercussions.