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

Overview

The Therapeutic Goods Act 1989, enacted by the Parliament of Australia, is a comprehensive piece of legislation aimed at regulating therapeutic goods within the country. This Act provides a framework for the control of therapeutic goods, including medicines, medical devices, and blood products, to ensure their safety, quality, and efficacy. One of the significant objectives of the Act is to address the gap in the provision of treatments for rare diseases by facilitating the availability of orphan drugs. Orphan drugs are those intended for the treatment of rare medical conditions that affect a small number of people. By designating certain drugs as orphan drugs, the Act aims to encourage the research, development, and availability of treatments for these underserved conditions. On 26 November 2015, Dr. Anthony Gill, as the Delegate of the Secretary, exercised powers under the Therapeutic Goods Regulations 1990 to designate Lonoctocog alfa (AFSTYLA) as an orphan drug for the treatment of haemophilia A in both adults and children. This designation was made to support the control and prevention of bleeding episodes, routine prophylaxis, and perioperative management in individuals with this rare congenital condition. The designation was carried out under subregulation 16J(2) of the Regulations, aligning with the policy objective of the Act to facilitate access to essential therapeutic products for rare diseases. The sponsor for Lonoctocog alfa (AFSTYLA) is CSL Behring Australia Pty Ltd.

Scope and Application

The Therapeutic Goods Act 1989, under the Therapeutic Goods Regulations 1990, applies to the designation of therapeutic goods, including the classification and regulation of drugs, medical devices, and other related products within the Commonwealth of Australia. In this instance, the Act and its regulations are used to designate Lonoctocog alfa (AFSTYLA) as an orphan drug for the treatment of haemophilia A in both adults and children. This designation is specific to the therapeutic use of Lonoctocog alfa for controlling and preventing bleeding episodes, routine prophylaxis, and perioperative prophylaxis, applicable to the powder for reconstitution and injection dose form. The Act provides the legal framework for the Therapeutic Goods Administration (TGA) to oversee the quality, safety, and efficacy of therapeutic goods, ensuring that they meet the necessary standards before being made available to the public. The geographic reach of this Act is national, applying to all entities and persons involved in the manufacture, importation, supply, and advertising of therapeutic goods across Australia. The Act does not specify any exclusions, exemptions, or thresholds for this particular designation, and its application can be extended or restricted through subordinate instruments issued by the TGA.

Key Provisions

The Therapeutic Goods Act 1989, through the Therapeutic Goods Administration, designates Lonoctocog alfa (marketed as AFSTYLA) as an orphan drug under section 16J of the Therapeutic Goods Regulations 1990 (subregulation 16J(2)). This designation, made on 26 November 2015 by Dr Anthony Gill, acting as the Delegate of the Secretary, recognises AFSTYLA as a treatment for haemophilia A, a rare condition involving congenital FVIII deficiency. This orphan drug designation aims to support the development and availability of treatments for rare diseases that affect fewer than 5,000 people in Australia. The therapeutic use of AFSTYLA is specified for three primary purposes: controlling and preventing bleeding episodes, routine prophylaxis to minimise bleeding frequency, and perioperative prophylaxis during surgery (section 16J(2)). The drug is available in a powder form intended for reconstitution and injection, highlighting its intended use in medical settings. The designation imposes specific obligations on the parties involved, particularly the sponsor, CSL Behring Australia Pty Ltd. The sponsor must ensure that AFSTYLA meets the required standards for safety, efficacy, and quality as stipulated by the Therapeutic Goods Administration. This includes providing comprehensive data and evidence to support the drug's benefits and risks. Additionally, the sponsor must comply with ongoing reporting requirements to maintain the orphan drug status and ensure that AFSTYLA remains available for its designated use. These obligations are critical for maintaining the integrity and availability of treatments for rare diseases in Australia. In the event of a breach of the Therapeutic Goods Act 1989 or the Therapeutic Goods Regulations 1990, various civil and criminal penalties may apply. For example, offences under section 28 of the Act can result in substantial fines, with penalties varying according to the severity and intent of the offence. Specifically, section 35 of the Act outlines maximum penalties for breaches, which can include fines up to $1,260,000 for corporations and $252,000 for individuals, along with potential imprisonment terms. These stringent measures underscore the importance of compliance with the regulatory framework to protect public health and ensure the safety and efficacy of therapeutic goods in Australia.