Designation of Cholic acid (Orphacol) as an orphan drug - Emerge Health

Overview

The Therapeutic Goods Act 1989 was enacted to regulate the importation, supply, and other uses of therapeutic goods in Australia, ensuring they are of acceptable quality, safety, and efficacy. This legislation was introduced to address the need for a comprehensive framework governing the therapeutic goods market, aiming to protect public health by ensuring that only products meeting stringent safety and quality standards are available. The Act was enacted by the Commonwealth Parliament, with the Therapeutic Goods Administration (TGA) established as the regulatory body under the auspices of the Department of Health. The policy objective underpinning this legislation is to safeguard the health and wellbeing of Australians by effectively regulating therapeutic goods and fostering innovation in the health sector. On 4 September 2014, Dr Anthony Gill, as the Delegate of the Secretary for the purposes of section 16J of the Therapeutic Goods Regulations 1990, designated Cholic acid (ORPHACOL) as an orphan drug for the treatment of inborn errors of primary bile acid synthesis due to specific enzymatic deficiencies. This designation acknowledges the rarity of these conditions and aims to incentivise the development of treatments for such rare diseases, addressing a gap in the availability of therapeutic options for affected individuals. The sponsor for Cholic acid (ORPHACOL) is Emerge Health, and the designated dose form for this indication is a capsule.

Scope and Application

The Therapeutic Goods Act 1989, administered by the Therapeutic Goods Administration (TGA) under the Commonwealth of Australia, designates Cholic acid (ORPHACOL) as an orphan drug for the treatment of specific inborn errors of primary bile acid synthesis. This legislation applies specifically to Cholic acid (ORPHACOL) and its use in treating conditions caused by 3β-hydroxy-Δ5-C27-steroid oxidoreductase (3β-HSD) deficiency or Δ4-3-oxosteroid 5β-reductase (Δ4-3-oxoR) deficiency, targeting a particular medical need within a defined patient population. The orphan drug designation is applicable to the sponsor, Emerge Health, and the specific formulation of Cholic acid (ORPHACOL) in capsule form. The scope of the Act extends to the regulation and oversight of therapeutic goods within the Australian jurisdiction, ensuring the safety, quality, and efficacy of these products for public health. There are no stated exclusions or exemptions in this specific designation, and the application of the Act may be further refined or extended through subordinate instruments issued by the TGA.

Key Provisions

The main operative section of this legislation is the designation of Cholic acid (ORPHACOL) as an orphan drug under subregulation 16J(2) of the Therapeutic Goods Regulations 1990 (Regulations). According to section 16J of the Regulations, an orphan drug is a drug intended for the treatment of a rare disease or condition. In this case, Cholic acid (ORPHACOL) is designated for the treatment of inborn errors of primary bile acid synthesis due to specific deficiencies (section 16J(2)(a)). The specific dose form for this treatment is a capsule, and the sponsor of this drug is Emerge Health (section 16J(2)(b)). The date of this designation is 4 September 2014. The Therapeutic Goods Act 1989 (the Act) and the Therapeutic Goods Regulations 1990 impose several obligations and requirements on parties and entities involved in the therapeutic goods industry. For the purposes of this legislation, the Delegate of the Secretary has the authority to designate a drug as an orphan drug. The sponsor, in this case Emerge Health, must ensure that the drug meets the necessary standards and requirements for the treatment of the specified condition. The Act and Regulations also require that the drug is appropriately labelled, and that any advertising or promotion of the drug complies with the relevant provisions of the Act and Regulations. Failure to comply with the obligations and requirements of the Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990 can result in a range of civil and criminal penalties. For example, a person who contravenes a provision of the Act may be liable for a civil penalty of up to $222,200 for a corporation and $44,440 for an individual (section 15(2) of the Act). In addition, a person who is found guilty of an offence against the Act may be subject to criminal penalties, including fines and imprisonment. The maximum penalties for offences under the Act depend on the specific offence and can range from a fine of up to $1,111,000 for a corporation and $222,200 for an individual, to imprisonment for up to five years (section 15(3) of the Act). In summary, this legislation designates Cholic acid (ORPHACOL) as an orphan drug for the treatment of specific inborn errors of primary bile acid synthesis. The Therapeutic Goods Act 1989 and the Therapeutic Goods Regulations 1990 impose obligations and requirements on parties and entities involved in the therapeutic goods industry, including the sponsor of the drug. Failure to comply with these obligations and requirements can result in civil and criminal penalties, including fines and imprisonment.