Designation of Elosulfase-Alpha as an Orphan drug - BioMarin Pharmaceutical Australia Pty Ltd

Overview

The Therapeutic Goods Act 1989, enacted by the Commonwealth Parliament, addresses the regulation of therapeutic goods in Australia to ensure their safety, quality, and efficacy. The Act establishes the framework for the oversight of therapeutic goods, including medicines, medical devices, and blood products. The Therapeutic Goods Regulations 1990 further refine these provisions by detailing specific requirements and processes, including the designation of orphan drugs. The designation of Elosulfase-alpha as an orphan drug on 3rd July 2013 under the Therapeutic Goods Regulations 1990, by Dr Anthony Gill, Delegate of the Secretary, aimed to address the treatment of rare diseases such as mucopolysaccharidosis IV type A, also known as Morquio A syndrome (MPS IVA). The policy objective behind this designation is to encourage the development of treatments for rare and neglected conditions that would otherwise lack sufficient commercial incentive for pharmaceutical companies.

Scope and Application

The Therapeutic Goods Act 1989, as amended and supplemented by the Therapeutic Goods Regulations 1990, provides the legislative framework for regulating therapeutic goods in Australia. Under this Act, the designation of specific drugs, such as Elosulfase-alpha, as orphan drugs is possible to encourage the development of treatments for rare diseases. This particular designation, made by Dr Anthony Gill, Delegate of the Secretary, on 3rd July 2013, recognises Elosulfase-alpha as an orphan drug for the treatment of mucopolysaccharidosis IV type A, also known as Morquio A syndrome or MPS IVA. The application of this Act extends to the regulation of therapeutic goods across Australia, encompassing all states and territories, thereby ensuring a unified approach to the oversight of medical treatments and their availability. This designation is significant as it facilitates benefits such as streamlined approval processes and potential financial incentives for the sponsor, BioMarin Pharmaceutical Australia Pty Ltd, in developing and marketing the drug for this rare condition.

Key Provisions

The Therapeutic Goods Act 1989, as amended by the Therapeutic Goods (Orphan Drugs) Amendment Regulations 2003, provides the framework for the regulation of therapeutic goods in Australia. Section 16J of the Therapeutic Goods Regulations 1990 specifically pertains to the designation of orphan drugs. In this context, section 16J(2) empowers a Delegate of the Secretary to designate a therapeutic good as an orphan drug if it meets certain criteria, including the treatment of a rare disease and the lack of alternative therapies. On 3rd July 2013, Dr Anthony Gill, as the Delegate of the Secretary, designated Elosulfase-alpha as an orphan drug for the treatment of mucopolysaccharidosis IV type A, also known as Morquio A syndrome (MPS IVA), under this provision. The designation of Elosulfase-alpha as an orphan drug brings with it specific obligations for the sponsor, in this case, BioMarin Pharmaceutical Australia Pty Ltd. Under the Therapeutic Goods Regulations 1990, the sponsor must ensure that the therapeutic good meets the regulatory requirements for orphan drugs. This includes providing sufficient evidence that the drug is intended for a rare disease, demonstrating the lack of alternative treatments, and ensuring that the drug’s safety and efficacy are appropriately established through clinical trials and other means. The sponsor must also comply with any further requirements or conditions stipulated by the Therapeutic Goods Administration (TGA) as part of the approval process. Failure to comply with the provisions of the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990 can result in significant consequences. Section 33 of the Therapeutic Goods Act 1989 provides for civil and criminal penalties for non-compliance. For instance, individuals and entities found in breach of the Act may face substantial fines, with penalties escalating based on the severity and frequency of the breach. Additionally, under section 34, the TGA has the authority to take enforcement actions such as issuing infringement notices, seizing non-compliant goods, and prosecuting cases in the Federal Court. The maximum penalties can include fines of up to $2,200,000 for corporations and $440,000 for individuals, reflecting the seriousness with which the legislation treats non-compliance.