What is medicine repurposing?
Some medicinal products have been found to have therapeutic uses outside of their licensed indication(s) and/or instructions for use. While this undoubtedly provides a benefit to patients, a legal issue remains over the off-licence use of medicines.
Repurposing moves the newly discovered off-label use to be on-label, so the medicine becomes authorised in patients in this new way. A new therapeutic indication that delivers “significant clinical benefit” can extend the products market protection by an additional year (the EU “8+2+1” framework).
Some well-known success stories of medicine repurposing include:
- Dexamethasone: a potent glucocorticoid, repurposed for use in COVID-19 patients to improve the survival chances of ventilated patients
- Propranolol: a beta-blocker, repurposed for complicated infantile haemangioma, reducing reliance on steroids and surgery in children
- Thalidomide: although its original intended use led to an international health disaster, this notorious chemical has been successfully repurposed to prolong the survival of patients with newly diagnosed multiple myeloma.
The NHS England Medicines Repurposing Programme
The Medicines Repurposing Programme was launched in March 2021 and was an NHS England initiative aimed to identify opportunities to license and implement existing medicines for new indications to benefit patients and the NHS. This programme was suspended in April 2025 due to an unforeseen lack of repurposing opportunities in comparison to original expectations, as well as the UK government’s announcement to dissolve NHS England during this parliament. There are no current plans to relaunch this scheme, although it has not yet been formally permanently retired.
How to repurpose a medicine
Assuming your medicine has been reported to have a potentially clinically significant off-licence use case, a new indication can be added by submitting a major Type II C.6.a variation. As part of this process, it may be helpful to initially confirm:
- What exactly is changing? New indication only, or also dose/route/formulation, target population, etc.
- What evidence package is available (and what is missing)? Ensure strong enough data and a clear clinical rationale to support the change is available to support the change to the label.
- What is the correct regulatory route? Check if the change fits the classification of a major Type II variation
- Would an Orphan designation be applicable? Orphan designation incentives such as up to 10 years’ orphan market exclusivity and potential fee refunds/waivers. The MHRA can also provide valuable regulatory support to make the development path more viable.
- Do I need scientific advice? This can be requested from the MHRA/EMA as appropriate. See our blog.
- Are there paediatric implications? A new indication/population may require updates to an agreed PIP or other paediatric obligations.
What is a Line Extension and how does this differ from repurposing?
Line Extensions are defined as a change listed in Article 2(4) of the European Commission Regulation 1234/2008 that is considered to “fundamentally alter the terms” of an MA. To assess such large changes to an MA, Line Extensions are processed as a new initial Marketing Authorisation Application (MAA).
While repurposing is usually implemented via a major Type II variation (depending on the change and procedure), adding to or expanding the clinical use (e.g. new indication) within the existing MA, a line extension is used for specific, fundamental changes (such as new strength, route or pharmaceutical form) that are treated as a new application and so a new MA number is created.
Annex I of 1234/2008 is a list of possible changes made during a Line Extension, and include (but are not limited to):
- Replacement of an active with a different salt or derivative, where the efficacy/safety characteristics are not significantly different
- Change of pharmacokinetics, such as change in rate of release
- Addition of a new strength, pharmaceutical form or route of administration.
Line Extensions do not cover new indications, which is the main difference compared to repurposing. Repurposing is also assessed via a major variation, not as an MAA; however, if the changes brought about by repurposing cannot be implemented into the existing MA, a Line Extension can arise as a result (for example, if a new indication can only be granted for a completely new pharmaceutical form).
If you would like to discuss how Fusion Pharma can support you with repurposing your medicines or extending your marketing authorisations, please email us at hello@fusion-pharma-limited.com or phone us on +44 (0) 203 1379 799.
While the information in this article is considered to be true and correct at the date of publication, changes in guidance and/or experience may impact on the accuracy of the information. Please refer to the relevant guidance for the latest information.
