Designation of Panobinostat (Farydak) as an orphan drug - Novartis Pharmaceuticals Australia Pty Ltd

Overview

The Therapeutic Goods Act 1989 was enacted to provide a regulatory framework for the approval, quality, and safety of therapeutic goods in Australia. This legislation was introduced to address the need for stringent control and regulation of therapeutic goods to protect public health, including ensuring that medicines are safe, of high quality, and effective. The Therapeutic Goods Administration (TGA), operating under the auspices of the Commonwealth of Australia's Department of Health, is the body responsible for administering the Act. The policy objective underlying the designation of Panobinostat (Farydak) as an orphan drug under the Act is to encourage the development of treatments for rare and life-threatening conditions by providing incentives such as expedited review processes and potential financial benefits to the sponsor. In this instance, Panobinostat (Farydak) has been designated as an orphan drug for the treatment of patients with relapsed multiple myeloma, or relapsed-and-refractory multiple myeloma, highlighting the Act's focus on addressing significant unmet medical needs.

Scope and Application

The Therapeutic Goods Act 1989 designates Panobinostat (Farydak) as an orphan drug specifically for the treatment of patients suffering from relapsed multiple myeloma or relapsed-and-refractory multiple myeloma. This designation is effective from 21 January 2014 and applies to the particular dose form of Panobinostat, which is in capsule form. The designation is made under subregulation 16J(2) of the Therapeutic Goods Regulations 1990 by Dr Anthony Gill, who is acting as the Delegate of the Secretary, on behalf of the Commonwealth of Australia's Therapeutic Goods Administration. The sponsor of Panobinostat (Farydak) is Novartis Pharmaceuticals Australia Pty Ltd. This legislation extends to the entire Commonwealth of Australia and applies to the pharmaceutical product in question and its sponsor. The scope of this Act includes the regulation of therapeutic goods, with specific attention to the designation and approval processes for orphan drugs, which are intended to address unmet medical needs in rare diseases. No exclusions or exemptions are explicitly stated in this particular designation, though broader provisions of the Act and Regulations may apply to other aspects of therapeutic goods regulation.

Key Provisions

The Therapeutic Goods Act 1989 (Cth), through the Therapeutic Goods Regulations 1990, includes provisions for the designation of orphan drugs, which are intended for the treatment of rare medical conditions. Under section 16J of the Regulations, the Delegate of the Secretary can designate a drug as an orphan drug when specific criteria are met, such as the drug being intended for a condition affecting fewer than 5,000 people in Australia. In this instance, section 16J(2) was invoked to designate Panobinostat (FARYDAK) as an orphan drug on 21 January 2014 for the treatment of patients with relapsed multiple myeloma (MM), or relapsed-and-refractory MM. This designation recognises the rarity of these conditions and the importance of developing treatments tailored to these specific patient groups. Entities involved in the development, manufacture, or supply of therapeutic goods must comply with the provisions of the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990. For instance, the sponsor of Panobinostat (FARYDAK), Novartis Pharmaceuticals Australia Pty Ltd, must ensure that the drug meets the quality and safety standards set forth by the Therapeutic Goods Administration (TGA). This includes providing adequate data on the drug's efficacy and safety for the designated orphan indication, as well as ensuring that manufacturing practices adhere to Good Manufacturing Practice (GMP) standards. Additionally, the sponsor must maintain records and documentation that demonstrate compliance with these regulatory requirements. Failure to comply with the Therapeutic Goods Act 1989 or the Therapeutic Goods Regulations 1990 can result in significant legal consequences. For instance, section 32 of the Act allows the TGA to take enforcement action against entities that fail to comply with the regulatory requirements. This can include the issuance of infringement notices, prosecution in the Federal Court of Australia, or the imposition of civil penalties. The maximum civil penalty for each contravened provision can be up to $222,000 for individuals and $1,110,000 for bodies corporate. Furthermore, section 33A of the Act provides for the imposition of criminal penalties, including fines of up to $2,220,000 for individuals and $11,100,000 for bodies corporate, and potential imprisonment for up to five years. These penalties underscore the importance of adhering to the regulatory requirements set forth by the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990.