Summary of opinion: Loargys,Pegzilarginase, 12/10/2023, Positive
On 12 October 2023, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation under exceptional circumstances1 for the medicinal product Loargys2, intended for the treatment of hyperargininemia. The applicant for this medicinal product is Immedica Pharma AB.
Loargys will be available as a 5 mg/ml solution for injection/infusion. The active substance of Loargys is pegzilarginase, an enzyme (ATC code: A16AB24) which reduces plasma arginine levels.
The benefit of Loargys is that it alleviates the manifestations and clinical symptoms of arginase 1 deficiency (ARG1-D) when compared to placebo, as observed in a multicentre, double‑blind, placebo‑controlled trial. The most common side effect was hypersensitivity.
The full indication is:
Loargys is indicated for the treatment of arginase 1 deficiency (ARG1‑D), also known as hyperargininemia, in adults, adolescents and children aged 2 years and older.
Loargys should be prescribed by physicians experienced in the management of patients with inherited metabolic diseases.
Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published in the European public assessment report (EPAR) and made available in all official European Union languages after the marketing authorisation has been granted by the European Commission.
1In exceptional circumstances, an authorisation may be granted subject to certain specific obligations, to be reviewed annually. This happens when the applicant can show that they are unable to provide comprehensive data on the efficacy and safety of the medicinal product, due to the rarity of the condition it is intended for, limited scientific knowledge in the area concerned, or ethical considerations involved in the collection of such data
2This product was designated as an orphan medicine during its development. EMA will now review the information available to date to determine if the orphan designation can be maintained
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