Advicenne Receives Positive Feedback From US FDA on Pathway to Approval & Amended Phase 3 Study Protocol

Advicenne recemtly announced the US FDA acceptance of the amended protocol of the company’s US-based Phase 3 pivotal trial of ADV7103 (Sibnayal) for the treatment of primary distal renal tubular acidosis (dRTA). dRTA is an orphan disease of the kidney affecting an estimated 30,000 patients in Europe and 20,000 in the US for which no approved treatments currently exist.

Following the FDA’s written feedback, Advicenne is moving forward with the ongoing restart of its pivotal dRTA clinical Study (ARENA-2) in the US and Canada, with a clear plan for study execution and regulatory strategy to ensure a clear path for the market approval of ADV7103 in the US. The trial was halted last year due to challenges to its execution posed by COVID-19.

Advicenne’s lead product, ADV7103, is developed as a multiparticulate formulation in 2-mm microtablets, a novel pioneering delivery technology created by Advicenne that contains two active pharmaceutical ingredients to ease its administration and aid compliance and quality of life in patients of all ages.

Critically, the FDA has agreed that the proposed primary endpoint of its Phase 3 pivotal trial of ADV7103 (Sibnayal) for the treatment of dRTA in the US could serve as the basis for full approval in patients with hereditary dRTA.

Dr. David Horn Solomon, Chairman at Advicenne, said “We welcome the positive feedback by the FDA, which provides important clarity on the clinical roadmap for ADV7103 in the US and enables us to overcome the challenges to its execution posed by the COVID-19 pandemic. Following the recent approval by the European Commission to market ADV7103 for the treatment of dRTA in Europe, Advicenne is on course to commercialize a product with the potential of benefitting the lives of patients in an area of high unmet need.”

Dr. André Ulmann, Chief Medical Officer of Advicenne, added “The feedback by the FDA represents another very important milestone for Advicenne, providing key information as we ultimately intend to commercialize ADV7103 in the US. With the approval of the amended study protocol, which enables us to resume our pivotal Phase 3 trial, we are optimistic that thousands of patients in the US will also be able to benefit from this potentially life-changing treatment.”

Distal renal tubular acidosis (dRTA) is an orphan disease characterized by a failure in the renal excretion of acids generated through metabolism and for which there is no approved treatment. The excess of acids thus accumulated in the blood leads to an imbalance in pH (acidosis) as well as multiple other complications such as growth retardation and rickets (a disease affecting bone development) in children, and a series of metabolic disorders such as low potassium levels, elevated calcium in the urine resulting in kidney stones, the formation of calcium deposits in the kidneys (calcinosis), as well as possible kidney failure.

Whether genetic or acquired as a consequence of an immune disease, dRTA affects an estimated 30,000 patients in Europe and approximately 20,000 in the US.

Advicenne is a pharmaceutical company founded in 2007, specializing in the development of innovative treatments in Nephrology. Its lead drug candidate is currently in late-stage clinical trials for two kidney diseases: distal renal tubular acidosis and cystinuria. ADV7103 has just received a Marketing Approval (MAA) for the treatment of dRTA. Headquartered in Paris, Advicenne has been listed on the Euronext Paris stock exchange since 2017 and was cross-listed on the Euronext Brussels stock exchange in 2019. For additional info


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