Designation of Lenvatinib Mesilate as an orphan drug - Eisai Australia Pty Ltd

Overview

The Therapeutic Goods Act 1989 was enacted to regulate therapeutic goods, including medicines, medical devices, and blood and blood components, in Australia. The Act provides the legal framework for the regulation of these goods to ensure that they are of acceptable quality, safety, and efficacy. One of the purposes of the Act is to facilitate access to therapeutic goods that are intended for the treatment of rare and life-threatening conditions, which are often referred to as orphan drugs. The Therapeutic Goods Administration, which is part of the Department of Health, is responsible for administering the Act. In accordance with the Therapeutic Goods Act 1989, the Therapeutic Goods Administration has designated Lenvatinib Mesilate as an orphan drug for the treatment of patients with progressive, radioiodine-refractory differentiated thyroid cancer. The designation was made by Dr Anthony Gill, the Delegate of the Secretary, on 27 November 2014. The sponsor of Lenvatinib is Eisai Australia Pty Ltd. The policy objective of designating Lenvatinib as an orphan drug is to encourage the research, development, and availability of therapeutic goods for the treatment of rare and life-threatening conditions.

Scope and Application

The Therapeutic Goods Act 1989, administered by the Therapeutic Goods Administration under the Department of Health in Australia, designates specific therapeutic goods for various purposes, including the designation of orphan drugs for rare medical conditions. In this instance, Lenvatinib Mesilate has been designated as an orphan drug for the treatment of patients suffering from progressive, radioiodine-refractory differentiated thyroid cancer. This designation applies to the specific dose form of the drug, which is a capsule, and is sponsored by Eisai Australia Pty Ltd. The act applies to the sponsor and any relevant entities involved in the production, importation, supply, or advertisement of Lenvatinib Mesilate in the context of this specified medical condition. The geographic reach of this designation is national, applying across all states and territories of Australia. The designation does not specify any exclusions, exemptions, or thresholds, but it is subject to the broader provisions of the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990, which may impose further requirements or restrictions on the use and distribution of the designated orphan drug.

Key Provisions

The Therapeutic Goods Act 1989, as amended by the C2014G02019 Gazette, includes provisions for the designation of certain drugs as orphan drugs (section 41ZC). This designation is significant for drugs intended for the treatment of rare conditions. In this instance, Lenvatinib Mesilate has been designated as an orphan drug under subregulation 16J(2) of the Therapeutic Goods Regulations 1990. Specifically, section 41ZC(3) of the Act allows for the designation of Lenvatinib for the treatment of patients with progressive, radioiodine-refractory differentiated thyroid cancer, a rare form of thyroid cancer that does not respond to standard radioiodine treatment. The obligations imposed by this designation include the responsibility of the sponsor, in this case, Eisai Australia Pty Ltd, to provide necessary information and evidence to support the orphan drug designation. This involves submitting detailed clinical trial data, safety information, and efficacy studies to the Therapeutic Goods Administration (TGA). Additionally, the sponsor must comply with ongoing reporting requirements to ensure the drug's safety and effectiveness continue to be monitored post-approval. Failure to meet these obligations can result in significant consequences. Under section 34BB of the Therapeutic Goods Act 1989, unauthorised therapeutic goods can lead to substantial penalties. Specifically, for a corporation, the maximum penalty is 500,000 Australian dollars or three times the value of the goods, whichever is greater, for each offence. Additionally, under section 34BC, an individual officer of the corporation can face a maximum penalty of 55,000 Australian dollars or imprisonment for five years, or both, for each offence. These penalties underscore the importance of compliance with the regulatory requirements set forth in the Act and the Regulations.