Designation of Anakinra (Kineret) as an Orphan Drug - Menarini Australia Pty Ltd

Overview

The Designation of Anakinra (KINERET) as an Orphan Drug 2013 was enacted on 17th April 2013 to address the issue of inadequate treatment options for rare and debilitating conditions such as Cryopyrin Associated Periodic Syndromes (CAPS), which include Muckle-Wells Syndrome, Familial Cold-induced Autoinflammatory Syndrome, and Neonatal-Onset Multisystem Inflammatory Disease. This legislation was introduced by Dr Anthony Gill, acting as the Delegate of the Secretary under the Therapeutic Goods Regulations 1990, to facilitate the availability of orphan drugs for these rare conditions. The policy objective of this designation is to encourage the research, development, and availability of treatments for rare diseases that otherwise might not be commercially viable due to their small patient populations. This was achieved by granting certain incentives to the sponsor, A. Menarini Australia Pty Ltd, for the development and supply of Anakinra (KINERET) in the specified dose form for the treatment of these rare syndromes.

Scope and Application

The designation of Anakinra (KINERET) as an orphan drug under the Therapeutic Goods Regulations 1990 applies specifically to this therapeutic agent for the treatment of a group of rare and debilitating conditions known as Cryopyrin Associated Periodic Syndromes (CAPS). These include Muckle-Wells Syndrome (MWS), Familial Cold-induced Autoinflammatory Syndrome (FCAS) / Familial Cold Urticaria (FUC), and Neonatal-Onset Multisystem Inflammatory Disease (NOMID) / Chronic Infantile Neurological Cutaneous and Articular syndrome (CINCA). This designation is intended to support the development and availability of treatments for rare diseases that affect fewer than 5,000 individuals in Australia. The Act applies to the sponsor of the drug, in this case, A. Menarini Australia Pty Ltd, and ensures that they receive certain benefits under the regulations, such as priority assessment and potential incentives for research and development. The scope of the legislation is limited to the specified orphan drug indication and its associated dose form, a solution in a pre-filled syringe of 100 mg /0.67 mL. There are no stated exclusions or exemptions in the text, and any further application or restrictions would be detailed in subordinate instruments.

Key Provisions

The main operative sections of the legislation, specifically section 16J of the Therapeutic Goods Regulations 1990, empower the Delegate of the Secretary to designate certain drugs as orphan drugs. This designation was exercised on 17th April 2013 for Anakinra (KINERET), a solution provided in a pre-filled syringe with a dosage of 100 mg/0.67 mL, for the treatment of various conditions under Cryopyrin Associated Periodic Syndromes (CAPS). These conditions include Muckle-Wells Syndrome (MWS), Familial Cold-induced Autoinflammatory Syndrome (FCAS) / Familial Cold Urticaria (FUC), and Neonatal-Onset Multisystem Inflammatory Disease (NOMID) / Chronic Infantile Neurological Cutaneous and Articular syndrome (CINCA) in both adults and children. The sponsor of this drug is A. Menarini Australia Pty Ltd. The obligations imposed by this Act on the parties involved primarily revolve around ensuring the appropriate use and regulation of Anakinra (KINERET) for the specified conditions. The designation of Anakinra as an orphan drug facilitates certain regulatory advantages, such as priority review and potential financial incentives, aimed at encouraging the development and availability of treatments for rare diseases. The sponsor, A. Menarini Australia Pty Ltd, must ensure that the drug is manufactured, labelled, and distributed in accordance with the Therapeutic Goods Regulations 1990. Additionally, they are required to provide any necessary updates or additional information to the relevant authorities to maintain the orphan drug designation and ensure ongoing compliance with regulatory standards. Under the Therapeutic Goods Regulations 1990, any breach of the provisions governing the designation and use of orphan drugs could result in significant civil or criminal consequences. While specific offences and penalties are not detailed within this particular piece of legislation, breaches of the Therapeutic Goods Act 1989 and its associated regulations can lead to substantial penalties. For example, unauthorised therapeutic goods can result in fines of up to $1.1 million for individuals and $5.5 million for corporations, along with potential imprisonment terms. Additionally, misleading or deceptive conduct related to therapeutic goods can incur similar penalties, reinforcing the importance of compliance with the legislative framework. These potential penalties underscore the seriousness with which the Australian government treats the regulation of therapeutic goods and the protection of public health.