Designation of Normal Immunoglobulin (Human) (Kiovig) as an Orphan Drug - Baxter Healthcare Pty Ltd

Overview

The Designation of Normal Immunoglobulin (Human) (KIOVIG) as an Orphan Drug Act 2012, enacted in 2012, was introduced to address the need for incentivising the development and availability of orphan drugs, which are medications designed for the treatment of rare diseases or conditions that affect a small number of people. This legislation was enacted by the Australian Parliament to provide a legal framework that encourages pharmaceutical companies to invest in the research and development of treatments for rare diseases by offering certain benefits, including the designation of orphan drugs. The policy objective of the Act is to improve access to life-saving and life-enhancing therapies for patients suffering from rare and debilitating conditions, thereby enhancing public health outcomes. The designation of KIOVIG as an orphan drug for the treatment of immunomodulatory therapy in multifocal motor neuropathy (MMN) exemplifies the Act's purpose, recognising the critical need for specialised treatments in underserved patient populations.

Scope and Application

The designation of normal immunoglobulin (human) (KIOVIG) as an orphan drug under the Therapeutic Goods Regulations 1990 applies to the specific therapeutic use of this drug for the treatment of Immunomodulatory therapy in multifocal motor neuropathy (MMN). This designation is effective from 29 November 2012 and pertains to the intravenous use injection form of KIOVIG. The sponsor for this orphan drug designation is Baxter Healthcare Pty Ltd, indicating their responsibility and involvement in the development and supply of KIOVIG for this particular medical condition. The geographic and jurisdictional reach of this designation is governed by the Commonwealth of Australia, as per the Therapeutic Goods Regulations 1990, and it does not specify any exclusions, exemptions, or thresholds within the text provided. The application of this legislation may be extended or restricted through subordinate instruments, which are not elaborated upon in the given text.

Key Provisions

The legislation C2012G00389 designates normal immunoglobulin (human) (KIOVIG) as an orphan drug for the treatment of immunomodulatory therapy in multifocal motor neuropathy (MMN), effective from 29 November 2012. This designation (Section 16J(2) of the Therapeutic Goods Regulations 1990) is made by Dr Anthony Gill, the Delegate of the Secretary, and specifies that the drug is intended for intravenous use as an injection. Baxter Healthcare Pty Ltd is identified as the sponsor of the drug. Entities governed by this legislation, primarily Baxter Healthcare Pty Ltd as the sponsor, are required to ensure that KIOVIG is used strictly for the designated indication of MMN treatment. They must also comply with all relevant regulatory requirements to maintain the orphan drug status, including providing necessary documentation and data to support the drug's efficacy and safety for the specified use. The sponsor must also ensure that the manufacturing and distribution processes adhere to the therapeutic standards set by the Therapeutic Goods Administration. Failure to comply with the provisions of the Therapeutic Goods Regulations 1990 or to maintain the designated use of KIOVIG for MMN could result in enforcement actions by the Therapeutic Goods Administration. While the specific offences and penalties are not detailed in the provided text, breaches of the Regulations can generally lead to civil and criminal penalties. These can include fines, product recalls, and potential legal action against the entities involved. The maximum penalties for such breaches can vary widely depending on the severity and nature of the offence, but they may include substantial financial penalties and imprisonment for serious violations.