Designation of Riluzole (Teglutik) as an orphan drug - Seqirus Pty Ltd

Overview

The Therapeutic Goods Act 1989, enacted by the Parliament of Australia, serves as the foundational piece of legislation for regulating therapeutic goods in the country. One of its key purposes is to provide mechanisms for the regulation of therapeutic goods, including medicines, medical devices, and other related products, to ensure their safety, quality, and efficacy. The Act was introduced to address the need for a comprehensive regulatory framework that would protect public health by ensuring that therapeutic goods available in Australia meet stringent standards. The 2017 designation of Riluzole (Teglutik) as an orphan drug by the Therapeutic Goods Administration under the Act exemplifies the legislation's objective to facilitate access to treatments for rare diseases, in this case, amyotrophic lateral sclerosis (ALS), by providing incentives to sponsors for the development of such therapies.

Scope and Application

The Therapeutic Goods Act 1989, through the Therapeutic Goods Regulations 1990, designates Riluzole (Teglutik) as an orphan drug for the treatment of amyotrophic lateral sclerosis (ALS) as of 13 February 2017. This designation applies specifically to the liquid - oral suspension form of Riluzole (Teglutik), and is issued by Dr Anthony Gill, acting as the Delegate of the Secretary under subregulation 16J(2) of the Regulations. The Act pertains to therapeutic goods within the Commonwealth of Australia, thereby affecting the pharmaceutical industry, particularly the sponsor Seqirus Pty Ltd, and those involved in the distribution and administration of therapeutic goods to patients with ALS. The scope of this legislation is national, with its provisions extending throughout Australia, impacting entities and persons engaged in the production, supply, and regulation of therapeutic goods. There are no explicit exclusions or exemptions mentioned in this specific designation, though the broader Therapeutic Goods Act and Regulations may encompass such provisions. This legislative action is an extension of the Therapeutic Goods Administration's role in ensuring the availability of orphan drugs for rare conditions and is supported by subordinate instruments within the Act.

Key Provisions

The Therapeutic Goods Act 1989, under section 16J of the Therapeutic Goods Regulations 1990, empowers the Delegate of the Secretary to designate certain drugs as orphan drugs. In this instance, Riluzole (Teglutik) has been designated as an orphan drug for the treatment of patients with amyotrophic lateral sclerosis (ALS) (section 16J(2)). The designation recognises the drug's use for a rare condition that affects a small number of people, and it is in the interest of public health to encourage its development and availability. The specific dose form designated is a liquid oral suspension. Entities governed by this Act, particularly the sponsor of the drug, Seqirus Pty Ltd, have specific obligations. They must ensure that the therapeutic goods comply with the standards set out in the regulations, including quality, safety, and efficacy. The sponsor is also responsible for maintaining records and providing information to the Therapeutic Goods Administration (TGA) as required. This includes any adverse event reports and updates on the drug's safety and efficacy data. Failure to comply with the provisions of the Therapeutic Goods Act 1989 or the Therapeutic Goods Regulations 1990 can result in various civil or criminal penalties. For instance, supplying a therapeutic good that does not comply with the standards could lead to substantial fines, with the maximum penalty being $1,110,000 for a corporation and $222,000 for an individual, as outlined in section 33DA of the Act. Additionally, in serious cases, criminal prosecution could result, leading to imprisonment for up to five years for individuals, as stipulated in section 33DC. The TGA retains the authority to take enforcement actions, including product recalls and legal proceedings, to ensure compliance and protect public health.