Designation of Blinatumomab as an orphan drug - Amgen Australia Pty Ltd

Overview

The Therapeutic Goods Act 1989, enacted by the Parliament of Australia, aims to regulate therapeutic goods to ensure public health and safety. The 2013 gazette, C2013G01761, designates Blinatumomab as an orphan drug for the treatment of B-precursor acute lymphoblastic leukaemia. This designation was made by Dr. Anthony Gill, acting as the Delegate of the Secretary under the Therapeutic Goods Regulations 1990. The policy objective of this designation is to encourage the development and availability of therapeutic goods for rare diseases or conditions that affect a small number of people, ensuring that patients with these conditions have access to potentially life-saving treatments. The sponsor of Blinatumomab is Amgen Australia Pty Ltd, highlighting the collaboration between regulatory authorities and pharmaceutical companies in addressing unmet medical needs.

Scope and Application

The Therapeutic Goods Act 1989, through its delegated authority, designates Blinatumomab as an orphan drug for the treatment of B-precursor acute lymphoblastic leukaemia. This designation applies specifically to the powder for reconstitution, injection dose form of the drug, and it is applicable to the sponsor, Amgen Australia Pty Ltd. The Act, administered by the Therapeutic Goods Administration under the Commonwealth of Australia, ensures that orphan drugs are recognised for their role in addressing unmet medical needs for rare diseases. This specific designation allows for streamlined regulatory processes and support for the development and availability of Blinatumomab, which is critical for patients suffering from this particular form of leukaemia. The application of this Act is national in scope, impacting entities and therapeutic goods across Australia, with the goal of facilitating access to orphan drugs for rare disease treatment.

Key Provisions

The Therapeutic Goods (Blinatumomab Orphan Drug Designation) Determination 2013, issued under the authority of the Therapeutic Goods Act 1989, designates Blinatumomab as an orphan drug. This designation is effective from 20 November 2013 and applies to Blinatumomab in its powder for reconstitution, injection form, which is intended for the treatment of B-precursor acute lymphoblastic leukaemia (section 16J of the Therapeutic Goods Regulations 1990). The sponsor of Blinatumomab is identified as Amgen Australia Pty Ltd. This determination, made by Dr. Anthony Gill, Delegate of the Secretary, under subregulation 16J(2) of the Regulations, acknowledges the rare nature of the disease targeted by Blinatumomab and the need for incentives to encourage its development. Entities governed by the Therapeutic Goods Act 1989, including the sponsor of the designated orphan drug, are required to comply with the provisions set out in the Act and Regulations. This includes ensuring that the orphan drug is manufactured, labelled, and marketed in accordance with the standards and requirements specified. For Blinatumomab, this means that Amgen Australia Pty Ltd must adhere to the quality, safety, and efficacy standards set for therapeutic goods in Australia. Additionally, any changes to the drug's formulation, manufacturing process, or intended use must be approved by the Therapeutic Goods Administration (TGA) before implementation. Breaches of the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990 can result in both civil and criminal penalties. The Act provides for various offences, including the unauthorised manufacture, supply, or importation of therapeutic goods. For instance, supplying a therapeutic good that does not comply with the standards set out in the Regulations can result in significant penalties. The maximum penalty for an individual convicted of such an offence can be up to $2,100,000 or imprisonment for up to 10 years, or both, as outlined in section 30 of the Act. Similarly, corporate entities can face penalties up to $10,500,000 or other equivalent sanctions, depending on the severity of the breach. These provisions ensure that the therapeutic goods available in Australia meet the highest standards of quality and safety for consumers.