Designation of Eculizumab (SOLIRIS) as an Orphan Drug - Alexion Pharmaceuticals Australasia Pty Ltd

Overview

The Designation of eculizumab (SOLIRIS) as an Orphan Drug, Gazetted on 26 October 2012, represents a significant step in the management of rare diseases in Australia. This legislation, enacted by Dr. Anthony Gill, the Delegate of the Secretary under the Therapeutic Goods Regulations 1990, aims to address the gap in treatment options for patients suffering from infection-associated haemolytic uraemic syndrome. This rare condition, which can have severe and life-threatening consequences, previously lacked adequate therapeutic interventions. By designating eculizumab (SOLIRIS) as an orphan drug, the Australian government is facilitating the development, approval, and availability of this treatment, ultimately providing a crucial medical advancement for those affected by this debilitating disease. The policy objective underpinning this designation is to incentivise the research and development of orphan drugs, ensuring that patients with rare conditions receive the necessary treatments to improve their quality of life.

Scope and Application

The designation of eculizumab (SOLIRIS) as an orphan drug under the Therapeutic Goods Regulations 1990 applies to the pharmaceutical product eculizumab, marketed as SOLIRIS, specifically for the treatment of infection-associated haemolytic uraemic syndrome. This designation is issued by Dr Anthony Gill, who acts as the Delegate of the Secretary under the regulations, thereby highlighting the commitment to addressing rare diseases with significant unmet medical needs. The scope of this legislation is confined to the therapeutic application of eculizumab for the specified medical condition, with the intended use being the administration of the concentrated solution for intravenous infusion. The sponsor responsible for this drug is Alexion Pharmaceuticals Australasia Pty Ltd, which bears the responsibility for ensuring compliance with the regulatory requirements for orphan drugs. This legislation, however, does not extend to other uses of eculizumab or to other pharmaceutical products unless specifically included through further regulation or amendment.

Key Provisions

The key operative sections of this legislation (C2012G00219) include the designation of eculizumab (SOLIRIS) as an orphan drug, as outlined in section 16J of the Therapeutic Goods Regulations 1990. Specifically, section 16J(2) allows for the designation of a drug as an orphan drug if it is intended for the treatment of a rare disease or condition. In this case, eculizumab (SOLIRIS) has been designated for the treatment of infection-associated haemolytic uraemic syndrome, which is recognised as a rare disease. The designation, effective from 26 October 2012, applies to the specific dose form of eculizumab (SOLIRIS), which is a concentrated solution for IV infusion Vial. This legislation imposes several obligations and requirements on the parties involved. The sponsor of the drug, Alexion Pharmaceuticals Australasia Pty Ltd, must ensure that eculizumab (SOLIRIS) is manufactured, stored, and distributed in compliance with all relevant regulations. Furthermore, they must provide evidence to support the orphan drug designation and any subsequent changes to the drug's status. The sponsor must also comply with all labelling, advertising, and other promotional activities that are consistent with the therapeutic claims of the drug as designated. In terms of legal consequences, breaches of the Therapeutic Goods Regulations 1990 can lead to both civil and criminal penalties. Under the Act, penalties for non-compliance can include fines, imprisonment, or both. For example, section 34 of the Therapeutic Goods Act 1989 provides for fines of up to $220,000 for individuals and $1,100,000 for bodies corporate for breaches of the regulations. Additionally, section 35A allows for imprisonment of up to five years for serious or repeated offences. These penalties underscore the importance of adhering to the regulations and the potential consequences of non-compliance.