Designation of Mepolizumab (Nucala) as an orphan drug - GlaxoSmithKline Australia

Overview

The Therapeutic Goods Act 1989, enacted by the Parliament of Australia, establishes a legislative framework for the regulation of therapeutic goods within Australia. This Act was introduced to address the need for a comprehensive and cohesive system to ensure that therapeutic goods available in Australia are of an acceptable quality, safety and efficacy. One of the key mechanisms under this Act is the ability to designate orphan drugs, which are intended for the diagnosis, prevention or treatment of a rare disease or condition. The designation of mepolizumab (Nucala) as an orphan drug on 11 May 2017 by the Delegate of the Secretary, Dr Anthony Gill, exemplifies the Act's objective to support the development of treatments for rare conditions that might otherwise be neglected due to their limited market size. The policy objective behind the orphan drug designation is to encourage the research, development and availability of therapeutic goods for rare diseases, ultimately improving health outcomes for patients with such conditions.

Scope and Application

The Therapeutic Goods Act 1989, administered by the Therapeutic Goods Administration (TGA), designates mepolizumab (Nucala) as an orphan drug for the treatment of patients with Eosinophilic Granulomatosis with Polyangiitis (EGPA). This designation applies specifically to the powder for solution for injection dose form of mepolizumab (Nucala). The sponsor for this therapeutic good is GlaxoSmithKline Australia Pty Ltd. The scope of this Act pertains to the regulation of therapeutic goods within Australia, ensuring their safety, quality, and efficacy. The designation of an orphan drug under this Act provides certain incentives to sponsors for the development of treatments for rare diseases, thereby addressing a specific medical need with limited market potential. The Act's jurisdictional reach is national, applying to all entities and persons involved in the supply, importation, and advertising of therapeutic goods across Australia. The Act does not specify exclusions or exemptions for orphan drug designations but does provide mechanisms through subordinate instruments to extend or restrict application as necessary.

Key Provisions

The Therapeutic Goods Act 1989, through its amendment by the Therapeutic Goods Regulations 1990, facilitates the designation of certain drugs as orphan drugs for rare diseases, as seen in the recent designation of mepolizumab (Nucala) for the treatment of Eosinophilic Granulomatosis with Polyangiitis (EGPA) on 11 May 2017. Under section 16J of the Regulations, the Delegate of the Secretary, Dr Anthony Gill, has designated mepolizumab (Nucala) as an orphan drug for this specific medical condition, recognising the rarity and unmet medical need associated with EGPA. This designation (Regulation 16J(2)) permits special considerations and potential benefits for the development, marketing, and availability of mepolizumab (Nucala) for this indication. The drug in question, mepolizumab (Nucala), is administered as a powder for solution for injection, and the sponsor responsible for its development and marketing is GlaxoSmithKline Australia Pty Ltd. Entities involved with the designated orphan drug must comply with specific obligations and requirements outlined under the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990. The sponsor, in this case GlaxoSmithKline Australia Pty Ltd, must ensure that the drug is safe, of high quality, and effective for its intended use. This involves rigorous clinical trials, manufacturing processes, and adherence to Good Manufacturing Practice (GMP) standards. Additionally, the sponsor must provide comprehensive data to support the orphan drug designation, including information on the disease prevalence, the therapeutic benefits of mepolizumab (Nucala), and the lack of alternative treatments. Accurate and timely reporting to the Therapeutic Goods Administration (TGA) is also required, ensuring that any changes in the drug's status, manufacturing processes, or clinical trial outcomes are communicated promptly. Failure to comply with the provisions of the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990 can result in serious consequences. Breaches of these laws can lead to civil and criminal penalties. Under section 33DA of the Act, misleading or deceptive conduct in relation to therapeutic goods can result in substantial fines, up to $2.1 million for corporations and $210,000 for individuals, or imprisonment for up to five years. Additionally, offences related to the unauthorised supply of therapeutic goods can attract penalties of up to $1.05 million for corporations and $105,000 for individuals, or imprisonment for up to three years. These penalties underscore the importance of strict adherence to the legislative framework governing the development, approval, and marketing of therapeutic goods in Australia.