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Ruzurgi
Sponsor
Device/Product name
Ruzurgi
Active Ingredient
Amifampridine
Date of decision
Published
Submission type
New chemical entity
ATC codes
N07XX05
Decision
Approved
What was the decision based on
The decision was based on quality (chemistry and manufacturing), nonclinical (pharmacology and toxicology), clinical (pharmacology, safety and efficacy) and risk management plan information submitted by the sponsor. The benefit-risk profile of Ruzurgi was considered favourable for the therapeutic use approved.
What steps were involved in the decision process
Registration timeline
The following table summarises the key steps and dates for this application, evaluated through Priority Review.
| Description | Date |
|---|---|
| Positive Designation (Orphan) | 15 October 2020 |
| Submission dossier accepted and first round evaluation commenced | 1 February 2021 |
| Evaluation completed | 10 June 2021 |
| Delegate's overall benefit-risk assessment and request for Advisory Committee advice | 5 July 2021 |
| Sponsor's pre-Advisory Committee response | 20 July 2021 |
| Advisory Committee meeting | 5 and 6 August 2021 |
| Registration decision (Outcome) | 10 September 2021 |
| Completion of administrative activities and registration on ARTG | 14 September 2021 |
| Number of working days from submission dossier acceptance to registration decision* | 127 |
*Target timeframe for priority applications is 150 working days from acceptance for evaluation to the decision.
Date of entry onto ARTG
Original publication date
Black triangle scheme
Yes. This product will remain in the scheme for 5 years, starting on the date the product is first supplied in Australia
Dose forms
Tablet
Strength
10 mg
Other ingredients
Colloidal anhydrous silica, calcium hydrogen phosphate dihydrate, magnesium stearate, microcrystalline cellulose, and sodium starch glycollate.
Containers
Bottle
Pack sizes
100
Routes of administration
Oral
Dosage
Dosing should be individualised based on clinical circumstances, patient response, and patient population. The dose should be gradually titrated to the optimal effective dose with the minimum of side effects. Once achieved, this optimal dose should be maintained, and dosing frequency should be adjusted, as needed.
The recommended oral dose is based on body weight.
Safety and effectiveness in paediatric patients below the age of 6 years have not been established.
For further information refer to the Product Information.
Pregnancy category
CDrugs which, owing to their pharmacological effects, have caused or may be suspected of causing, harmful effects on the human fetus or neonate without causing malformations. These effects may be reversible. Accompanying texts should be consulted for further details.The use of any medicine during pregnancy requires careful consideration of both risks and benefits by the treating health professional. This must not be used as the sole basis of decision making in the use of medicines during pregnancy. The TGA does not provide advice on the use of medicines in pregnancy for specific cases. More information is available from obstetric drug information services in your State or Territory.
What was approved
Ruzurgi (amifampridine) was approved for the following therapeutic use:
Ruzurgi is indicated for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults and children aged 6 years and above.
What is this medicine and how does it work
The mechanism by which amifampridine exerts its therapeutic effect in Lambert-Eaton myasthenic syndrome (LEMS) patients has not been fully elucidated. Amifampridine is a broad-spectrum potassium channel blocker.
What post-market commitments will the sponsor undertake
- Ruzurgi (amifampridine) is to be included in the Black Triangle Scheme. The Product Information (PI) and Consumer Medicines Information (CMI) for Ruzurgi must include the black triangle symbol and mandatory accompanying text for five years, which starts from the date that the sponsor notifies the TGA of supply of the product.
- The Ruzurgi Australia (AU)-risk management plan (RMP) (version 0.3, dated 24 June 2021, data lock point 20 November 2020), included with submission PM-2021-00020-1-1, and any subsequent revisions, as agreed with the TGA will be implemented in Australia.
An obligatory component of risk management plans is routine pharmacovigilance. Routine pharmacovigilance includes the submission of periodic safety update reports (PSURs).
Unless agreed separately between the supplier who is the recipient of the approval and the TGA, the first report must be submitted to TGA no later than 15 calendar months after the date of the approval letter. The subsequent reports must be submitted no less frequently than annually from the date of the first submitted report until the period covered by such reports is not less than three years from the date of the approval letter. The annual submission may be made up of two PSURs each covering six months. If the sponsor wishes, the six monthly reports may be submitted separately as they become available.
If the product is approved in the European Union (EU) during the three years period, reports can be provided in line with the published list of EU reference dates no less frequently than annually from the date of the first submitted report until the period covered by such reports is not less than three years from the date of the approval letter.
The reports are to at least meet the requirements for PSURs as described in the European Medicines Agency's Guideline on Good Pharmacovigilance Practices (GVP) Module VII-periodic safety update report (Rev 1), Part VII.B Structures and processes. Note that submission of a PSUR does not constitute an application to vary the registration. Each report must have been prepared within ninety calendar days of the data lock point for that report.
- The sponsor should confirm with the TGA when testing of the proposed container/closure system for child resistance is complete.
- The sponsor should provide to the TGA the reports of carcinogenicity, reproductive and developmental toxicity and juvenile toxicity studies formally requested by the Food and Drug Administration (United States) (FDA) (Studies 3612-1 to 3612-7), as soon as available.
- The sponsor should provide to the TGA the reports of studies in humans with renal impairment and in humans with hepatic impairment (Studies 3612-9, 3612-10), as soon as available.