Designation of Nitisinone (Disanit) as an orphan drug - Emerge Health Pty Ltd

Overview

The Therapeutic Goods Act 1989, enacted by the Parliament of Australia, provides a comprehensive framework for regulating therapeutic goods in the country. One of the significant amendments to this Act is the designation of nitisinone (DISANIT) as an orphan drug, effective from 20 July 2017, through the Therapeutic Goods Administration. This specific designation addresses the gap in treatment options for patients with hereditary tyrosinemia type 1 (HT-1), a rare metabolic disorder. By recognising nitisinone as an orphan drug, the legislation aims to incentivise the development and availability of treatments for rare diseases, thereby improving health outcomes for affected individuals. The policy objective is to ensure that medications for rare conditions receive the necessary support and attention to facilitate their development and accessibility.

Scope and Application

The Therapeutic Goods Act 1989, under which this designation operates, applies to therapeutic goods, which are defined broadly to include medicines, medical devices, and other health-related products within the Australian jurisdiction. The Act facilitates the regulation of therapeutic goods to ensure their quality, safety, and efficacy. Specifically, the designation of nitisinone (DISANIT) as an orphan drug applies to this medicinal product intended for the treatment of hereditary tyrosinemia type 1 (HT-1) in patients of all ages. This designation is made pursuant to the Therapeutic Goods Regulations 1990 and acknowledges the rarity of the condition and the need for targeted treatments. The application of this Act is national, encompassing the entire Commonwealth of Australia. The Act does not specify exclusions or exemptions for orphan drug designations, but it does allow for the regulation of therapeutic goods through subordinate instruments, which may provide further details or conditions for the use and approval of such drugs. The sponsor of the designated orphan drug, Emerge Health Pty Ltd, is responsible for ensuring compliance with relevant regulatory requirements.

Key Provisions

The Therapeutic Goods Act 1989, as amended by the Gazette C2017G00830, includes a significant designation of nitisinone (DISANIT) as an orphan drug (s16J of the Therapeutic Goods Regulations 1990). Specifically, on 20 July 2017, Dr Tim Greenaway, acting as the Delegate of the Secretary, designated nitisinone (DISANIT) for the treatment of hereditary tyrosinemia type 1 (HT-1) in patients of all ages, in conjunction with dietary restrictions of tyrosine and phenylalanine. This designation acknowledges the drug's importance in treating a rare disease, thereby facilitating its development and availability. The approved dose forms for nitisinone (DISANIT) in this context are 2mg, 5mg, and 10mg capsules, which must be stable capsule formulations. The obligations imposed by this designation are primarily on the sponsor, Emerge Health Pty Ltd. As the sponsor, Emerge Health Pty Ltd is responsible for ensuring that nitisinone (DISANIT) meets the standards set out in the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990. This includes providing adequate evidence of the drug’s safety, efficacy, and quality for the designated orphan indication. Additionally, the sponsor must comply with any further requirements or conditions that the Delegate of the Secretary may impose to facilitate the drug’s availability for patients with HT-1. Failure to comply with the provisions of the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990 can result in significant penalties and consequences. For instance, any contravention of the Act can lead to civil or criminal penalties. Civil penalties may include fines up to a maximum of $2,220,000 for corporations and $444,000 for individuals, depending on the severity and frequency of the breach. Additionally, criminal penalties may be imposed, which can include imprisonment for up to five years for individuals and fines for corporations that can be substantial. These consequences underscore the importance of strict compliance with the legislative requirements governing the designation and use of orphan drugs like nitisinone (DISANIT).