Designation of Cholic Acid (TBA) as an Orphan drug - Orpharma Pty Ltd

Overview

The Therapeutic Goods (Orphan Drugs) Amendment Regulations 2013 (C2013G01147), which were gazetted on 22 July 2013, introduced a designation framework for orphan drugs in Australia. This regulation was enacted to address the gap in treatment options for patients suffering from rare diseases, which often lack adequate research and development due to the small patient populations and the associated high costs. This legislative measure was introduced by the Australian Government to incentivise the development and availability of orphan drugs by offering certain benefits, such as streamlined approval processes and market exclusivity. The policy objective was to ensure that patients with rare, life-threatening or seriously debilitating conditions have access to potentially life-saving treatments. Dr. Anthony Gill, as the Delegate of the Secretary, designated Cholic Acid (TBA) as an orphan drug for the treatment of inborn errors of bile acid synthesis responsive to treatment with cholic acid, highlighting the importance of this legislative framework in facilitating the development and approval of treatments for rare diseases.

Scope and Application

The designation of Cholic Acid (TBA) as an orphan drug under the Therapeutic Goods Regulations 1990 pertains specifically to the treatment of inborn errors of bile acid synthesis that are responsive to treatment with cholic acid. This designation applies to the sponsor of the drug, Orpharma Pty Ltd, and the drug itself, in the form of oral capsules of 50 mg and 250 mg. This legislative action is confined to the Commonwealth of Australia, reflecting a federal approach to regulating therapeutic goods. The Act does not explicitly outline any exclusions or exemptions, but the nature of orphan drug designation inherently limits its application to rare diseases or conditions, which by definition affect a small number of people. The scope of this legislation may be extended or clarified through subordinate instruments that provide further details or guidelines on the implementation and enforcement of the orphan drug designation. This ensures that the therapeutic needs of patients with rare conditions are met efficiently within the regulatory framework.

Key Provisions

The main sections of the legislation establish the designation of Cholic Acid (TBA) as an orphan drug for the treatment of inborn errors of bile acid synthesis. This designation is made under subregulation 16J(2) of the Therapeutic Goods Regulations 1990, as referenced in section 16J of the Regulations (section 16J(2)). This decision, made on 22nd July 2013 by Dr Anthony Gill, acting as the Delegate of the Secretary, identifies Cholic Acid (TBA) as an orphan drug specifically for treating conditions responsive to cholic acid treatment. The relevant dose forms specified in the legislation are oral capsules of 50 mg and 250 mg, and the sponsor for this orphan drug is Orpharma Pty Ltd. Under this legislation, the primary obligations fall on Orpharma Pty Ltd, the sponsor of Cholic Acid (TBA). The sponsor must ensure that the drug is produced and marketed in accordance with the therapeutic requirements outlined in the legislation. This includes ensuring that the drug meets the specified dose forms and indications, and that it is appropriately registered and approved by the relevant authorities. Additionally, the sponsor must adhere to all other regulatory requirements set out in the Therapeutic Goods Regulations 1990. Breach of the requirements outlined in this legislation may result in various civil and criminal consequences. While specific penalties are not detailed in the legislation, the Therapeutic Goods Regulations 1990 generally provide for substantial penalties for non-compliance. These penalties can include fines, imprisonment, or both, depending on the severity of the breach. For example, under section 41AB of the Therapeutic Goods Act 1989, the maximum penalty for supplying therapeutic goods that do not comply with the Regulations can be up to 500 penalty units for individuals, or 5,000 penalty units for bodies corporate, plus additional penalties for ongoing breaches. These consequences underscore the importance of adhering to the legislative requirements to avoid legal repercussions.