Designation of C1 Esterase Inhibitor, Human (Berinert) as an orphan drug - CSL Behring Australia Pty Ltd

Overview

The Therapeutic Goods Act 1989, enacted by the Australian Parliament, serves to regulate therapeutic goods within Australia, ensuring their safety, quality, and efficacy. This Act addresses the need for a comprehensive legal framework governing the availability, registration, and oversight of therapeutic goods, thereby protecting public health. One of its key policy objectives is to facilitate access to life-saving and therapeutic innovations while maintaining stringent quality and safety standards. As part of this legislative framework, the Therapeutic Goods Administration, a department within the Commonwealth of Australia, has the authority to designate specific therapeutic substances as orphan drugs. This designation, as illustrated by the 2017 designation of C1 esterase inhibitor, human (Berinert), for the prophylaxis of hereditary angioedema attacks, aims to encourage the development and availability of treatments for rare diseases by offering certain incentives to sponsors, thereby addressing a significant gap in the provision of medical treatments for uncommon conditions.

Scope and Application

The Therapeutic Goods Act 1989, as amended and in conjunction with the Therapeutic Goods Regulations 1990, applies to the designation of therapeutic goods within Australia, including their classification, evaluation, and regulation. In this instance, the Act designates C1 esterase inhibitor, human (Berinert), as an orphan drug for the specific therapeutic use of subcutaneous administration for the routine prevention of hereditary angioedema (HAE) attacks. This designation, made by Dr Anthony Gill, a Delegate of the Secretary, specifically targets the pharmaceutical product Berinert, manufactured by CSL Behring Australia Pty Ltd, and is intended to encourage the development of treatments for rare and previously neglected diseases. The geographic and jurisdictional reach of this designation is within the Commonwealth of Australia, and it is executed under the authority granted by the Therapeutic Goods Act and Regulations. The designation does not specify any exclusions, exemptions, or thresholds other than the orphan drug criteria for rare diseases, and the application of this Act may be further extended or restricted by any subordinate instruments issued under its authority.

Key Provisions

The main operative section of this legislation is the designation of C1 esterase inhibitor, human (Berinert), as an orphan drug (section 16J of the Therapeutic Goods Regulations 1990). This designation was made on 10 April 2017 by Dr. Anthony Gill, who is acting as the Delegate of the Secretary under the Regulations. Specifically, C1 esterase inhibitor, human (Berinert), has been designated for subcutaneous administration for the prophylaxis (routine prevention) of hereditary angioedema (HAE) attacks. The form of this therapeutic good is powder for injection, subcutaneous, and the sponsor of this product is CSL Behring Australia Pty Ltd. The designation of C1 esterase inhibitor, human (Berinert) as an orphan drug imposes several obligations and requirements on the parties involved. The sponsor, CSL Behring Australia Pty Ltd, must ensure that the product meets the necessary safety, efficacy, and quality standards set forth by the Therapeutic Goods Administration (TGA). This includes providing the TGA with all necessary data and documentation to support the therapeutic claims and the safety profile of the product. Additionally, the sponsor must comply with any other requirements or directions issued by the TGA regarding the manufacture, supply, or advertising of the orphan drug. Failure to comply with the provisions of the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990 can result in serious consequences. Under the Act, breaches can lead to both civil and criminal penalties. For instance, individuals or companies found guilty of manufacturing, supplying, or advertising a therapeutic good that does not meet the required standards can face substantial fines. The maximum penalties vary depending on the severity of the offence, but they can include fines of up to $1.65 million for corporations and up to $330,000 for individuals, as well as potential imprisonment terms. Additionally, the TGA has the authority to issue infringement notices for less severe breaches, which can result in fines of up to $5,520 for individuals and $27,600 for corporations. It is crucial for all parties involved to adhere to the regulatory requirements to avoid these penalties and ensure the safety and efficacy of therapeutic goods in the Australian market.