Designation of (ASFOTASE ALFA) as an Orphan Drug - Alexion Pharmaceuticals Australasia Pty Ltd

Overview

The Designation of (Asfotase Alfa) as an Orphan Drug Act, enacted in 2012, was introduced to address the need for incentivising the development of treatments for rare and life-threatening conditions, specifically hypophosphatasia (HPP), where there is a significant unmet medical need. This legislation was enacted by the Australian Parliament to amend the Therapeutic Goods Act 1989 and provide a pathway for the designation of orphan drugs, thereby encouraging the pharmaceutical industry to invest in research and development for conditions that affect a small number of people. The policy objective of this Act is to ensure that there are effective treatments available for rare diseases, thereby improving the quality of life for those affected and providing a regulatory framework that supports innovation in the medical field.

Scope and Application

The designation of Asfotase Alfa as an orphan drug under C2012G00120 applies specifically to this therapeutic product for the treatment of hypophosphatasia (HPP) as of 15 October 2012. This legislation pertains to the therapeutic agent Asfotase Alfa, which is administered via subcutaneous injection, and is sponsored by Alexion Pharmaceuticals Australasia Pty Ltd. The designation as an orphan drug is made by Dr. Anthony Gill, acting as the Delegate of the Secretary under subregulation 16J(2) of the Therapeutic Goods Regulations 1990, for the purpose of providing incentives and support for the development and availability of treatments for rare diseases. The scope of the Act is limited to the specific indication of HPP and the particular formulation of Asfotase Alfa. While the Act does not explicitly state any exclusions, exemptions, or thresholds, it implicitly applies to the regulatory processes concerning the approval, manufacturing, and marketing of orphan drugs within Australia. The application of this Act is subject to the overarching Therapeutic Goods Regulations 1990, which may be extended or restricted through subordinate instruments.

Key Provisions

The main operative sections of this legislation pertain to the designation of Asfotase Alfa as an orphan drug for the treatment of hypophosphatasia (HPP). Section 16J of the Therapeutic Goods Regulations 1990 provides the authority for such a designation, and section 16J(2) outlines the process by which this is done. The Delegate of the Secretary, Dr Anthony Gill, has exercised this authority to designate Asfotase Alfa for the specified indication on 15 October 2012. The legislation specifies the form of the drug, which is subcutaneous injection, and identifies the sponsor, Alexion Pharmaceuticals Australasia Pty Ltd. Under the Therapeutic Goods Regulations 1990, the designation of Asfotase Alfa as an orphan drug imposes specific obligations on the parties involved. The sponsor, Alexion Pharmaceuticals Australasia Pty Ltd, must ensure that the drug is manufactured and supplied in accordance with the Therapeutic Goods Act 1989 and the Regulations. This includes compliance with quality, safety, and efficacy standards. The sponsor is also responsible for providing the Therapeutic Goods Administration (TGA) with any required information, updates, and reports regarding the drug. Furthermore, the sponsor must ensure that the drug is used in accordance with the approved indication and dosage form, as designated by the Delegate of the Secretary. The legislation also sets out the potential consequences for breaches of the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990. Offences under the Act may result in both civil and criminal penalties. For example, section 35 of the Act provides for civil penalties, including fines up to $222,200 for individuals and $1,111,000 for bodies corporate, for breaches of certain provisions. Additionally, section 42 of the Act outlines criminal offences, with maximum penalties including fines and imprisonment. For example, section 42(1) provides for a maximum penalty of $222,200 and/or two years imprisonment for individuals, and $1,111,000 and/or five years imprisonment for bodies corporate, for knowingly making a false or misleading statement in relation to therapeutic goods. The specific penalties applicable to breaches related to the orphan drug designation would be determined based on the nature and severity of the breach, in accordance with the relevant provisions of the Act and Regulations.