Designation of Ponatinib (Tradename TBA) as an Orphan Drug - ARIAD Pharmaceutical (Australia) Pty Ltd

Overview

The Designation of Ponatinib as an Orphan Drug, gazetted as C2013G00752, was enacted on 14 May 2013. This legislative action addresses the need for therapeutic options for patients suffering from rare and life-threatening conditions, particularly acute lymphoblastic leukaemia and various phases of chronic myeloid leukaemia (CML) that are resistant or intolerant to prior tyrosine kinase inhibitor therapy. This Act was introduced by Dr. Anthony Gill, the Delegate of the Secretary, under the authority of subregulation 16J(2) of the Therapeutic Goods Regulations 1990. The policy objective behind this designation is to provide a pathway for the development and availability of treatments for conditions that affect a small number of people in Australia, thereby ensuring that these patients have access to potentially life-saving drugs. The sponsor of the drug is ARIAD Pharmaceutical (Australia) Pty Ltd, which is responsible for the development and distribution of Ponatinib under the specified conditions.

Scope and Application

The designation of Ponatinib (tradename TBA) as an orphan drug under the Therapeutic Goods Regulations 1990 applies to this specific pharmaceutical product intended for the treatment of certain leukaemias in patients who have shown resistance or intolerance to prior tyrosine kinase inhibitor therapy. The legislation, executed by Dr Anthony Gill, Delegate of the Secretary, identifies the drug for use in acute lymphoblastic leukaemia and in adult patients with chronic phase, accelerated phase, or blast phase chronic myeloid leukaemia. The scope of the Act is narrowly focused on this particular drug and its specified indications, with the application limited to the sponsor, ARIAD Pharmaceutical (Australia) Pty Ltd. The geographic reach of this legislation is inherently tied to Australia, as it is enacted under the Commonwealth’s authority through the Therapeutic Goods Administration. While the Act itself does not specify exclusions or exemptions, the orphan drug designation may come with certain benefits under the Regulations, such as reduced fees or priority review, which are not explicitly detailed in this particular gazette. Any further details or conditions regarding the application and enforcement of this legislation would be subject to the broader provisions of the Therapeutic Goods Act 1989 and its subordinate instruments.

Key Provisions

The legislation (C2013G00752) designates Ponatinib as an orphan drug under section 16J of the Therapeutic Goods Regulations 1990. This designation was made by Dr Anthony Gill, acting as the Delegate of the Secretary on 14 May 2013. Ponatinib is designated for the treatment of acute lymphoblastic leukaemia and for adult patients with chronic phase, accelerated phase, or blast phase chronic myeloid leukaemia (CML) who are resistant or intolerant to prior tyrosine kinase inhibitor (TKI) therapy. The drug is available in two dose forms: 15 mg and 45 mg immediate film-coated tablets. The designation of Ponatinib as an orphan drug imposes certain obligations on the parties involved. Firstly, the sponsor, ARIAD Pharmaceutical (Australia) Pty Ltd, must ensure that the drug is manufactured, packaged, and labelled in accordance with the requirements of the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990. This includes adhering to good manufacturing practice standards and obtaining the necessary approvals for the drug’s use. Secondly, the sponsor is responsible for providing information and data to support the orphan drug designation, such as clinical trial results and evidence of the drug’s efficacy and safety. Breach of the obligations imposed by the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990 can result in significant penalties. For example, under section 32 of the Therapeutic Goods Act 1989, a person who contravenes a provision of the Act or the Regulations may be liable to a civil penalty not exceeding $1,650,000 for a corporation and $330,000 for an individual. Additionally, under section 34 of the Act, a person who commits an offence against the Act may be liable to a criminal penalty, which may include imprisonment for up to five years, a fine of up to $330,000, or both, for an individual, and a fine of up to $1,650,000 for a corporation. The penalties are intended to ensure compliance with the regulatory requirements and to protect public health and safety.