Designation of Momelotinib Dihydrochloride Monohydrate as an orphan drug by Gilead Sciences

Overview

The Therapeutic Goods Act 1989, enacted by the Commonwealth Parliament, was introduced to regulate the provision of therapeutic goods in Australia, ensuring that these products are safe, of high quality, and effective for their intended use. The Act provides the legal framework for the Therapeutic Goods Administration (TGA) to control the import, export, manufacture, and supply of therapeutic goods, including medicines, medical devices, and blood and blood components. The TGA, as part of the Department of Health, administers the Act and its accompanying regulations to protect public health by mitigating the risks associated with therapeutic goods. In the context of orphan drugs, the Act allows for the designation of certain medicines that treat rare conditions, providing incentives for research and development of treatments that might otherwise not be commercially viable. In 2017, the Therapeutic Goods Administration designated momelotinib dihydrochloride monohydrate as an orphan drug for the treatment of specific types of myelofibrosis. This action recognises the rarity of these conditions and the limited availability of treatments, aiming to encourage the development and availability of effective therapies. The designation of momelotinib dihydrochloride monohydrate as an orphan drug is a practical application of the policy objectives outlined in the Therapeutic Goods Act 1989, which include ensuring that Australians have access to safe and effective therapeutic goods, and supporting the development of treatments for rare diseases where there is an unmet medical need.

Scope and Application

The Therapeutic Goods Act 1989, as amended and administered by the Therapeutic Goods Administration, designates momelotinib dihydrochloride monohydrate as an orphan drug for the treatment of specific types of myelofibrosis, namely primary myelofibrosis, post-polycythaemia vera myelofibrosis, and post-essential thrombocythaemia myelofibrosis. This designation applies to the drug in its specific dose form, which is film-coated tablets. The decision to designate momelotinib dihydrochloride monohydrate as an orphan drug was made by Dr. Anthony Gill, the Delegate of the Secretary, under the authority granted by subregulation 16J(2) of the Therapeutic Goods Regulations 1990. The drug is sponsored by Gilead Sciences and this designation is effective from 31 July 2017. The application of this designation is limited to the specified conditions and does not extend to other uses or forms of the drug unless otherwise defined by the Therapeutic Goods Administration.

Key Provisions

The Therapeutic Goods Act 1989, as amended by this Gazette, designates momelotinib dihydrochloride monohydrate as an orphan drug for the treatment of specific types of myelofibrosis. Section 16J of the Therapeutic Goods Regulations 1990 provides the authority for this designation, which was made by Dr. Anthony Gill, the Delegate of the Secretary, on 31 July 2017. This action recognises momelotinib dihydrochloride monohydrate as an orphan drug for the treatment of primary myelofibrosis, post-polycythaemia vera myelofibrosis, and post-essential thrombocythaemia myelofibrosis, with the drug presented in the form of film-coated tablets. The sponsor for this drug is Gilead Sciences. Under the Act and the Regulations, the designation of an orphan drug carries with it certain obligations and requirements. The sponsor of the drug, Gilead Sciences in this case, must ensure that the drug meets the criteria for orphan drug status, including demonstrating that the drug is intended to treat a condition that affects a small number of people in Australia. Additionally, the sponsor must provide evidence that there are no satisfactory methods of diagnosis, prevention, or treatment of the condition, or that the drug would be of significant benefit to those affected by the condition. The sponsor must also ensure that the drug is manufactured according to good manufacturing practice standards and that it is safe and effective for its intended use. Failure to comply with the obligations and requirements of the Act and the Regulations may result in civil or criminal penalties. For example, section 33 of the Act provides for civil penalty provisions, including fines of up to $222,200 for individuals and $1,111,000 for bodies corporate, for breaches of the Act or the Regulations. Additionally, section 34 of the Act provides for criminal penalties, including fines of up to $555,500 for individuals and $2,777,500 for bodies corporate, and/or imprisonment for up to five years, for serious or repeated breaches of the Act or the Regulations. The specific penalties for a particular breach will depend on the circumstances of the case and will be determined by a court.