FDA grants orphan drug designation to ADV7103 for cystinuria

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ADV7103 is currently being studied in the phase 2/3 CORAL-1 study, enrolling patients with cystinuria across centers in France and Belgium.

The FDA has granted an orphan drug designation (ODD) to ADV7103 for the treatment of patients with cystinuria, according to a news release from Advicenne, the developer of the therapy.¹

Advicenne is planning on signing a partnership to advance the development and marketing of ADV7103 in the US.

ADV7103 was also granted an ODD status by the FDA in 2022 for the treatment of patients with distal renal tubular acidosis (dRTA).² According to Advicenne, ADV7103 is a combination of potassium citrate and potassium hydrogen carbonate, formulated as prolonged release coated granules.

“After receiving an initial orphan designation of ADV7103 in dRTA in 2022, we are absolutely delighted to announce today that we have obtained orphan drug status in cystinuria. After months of intense discussions with the FDA, this is fantastic news for Advicenne and a major element of value creation,” said Didier Laurens, Advicenne CEO, in the news release.¹ “I am very proud of the teams who worked hard for this success, especially in the USA. I am also very proud that the first clinical data obtained in France have convinced the FDA of the benefit of ADV7103 in the treatment of cystinuria. The treatment of this condition with simplified alkalinization is a major unmet medical need with a very significant market potential with more than 30,000 patients in the USA. Obtaining this ODD in cystinuria is the powerful lever we have been waiting for to accelerate and finalize discussions with potential partners in the United States”.

Takeaways

US FDA grants orphan drug designation to ADV7103 for cysturia
Advicenne plans to sign a partnership for development and marketing of the drug in the US
ADV7103 is currently being assessed in the phase 2/3 CORAL-1 study, with primary results expected in May 2025

Following the FDA designation, Advicenne is planning on signing a partnership to advance the development and marketing of ADV7103 in the US.

ADV7103 is currently being studied in the phase 2/3 CORAL-1 study, enrolling patients with cystinuria across centers in France and Belgium.³

In the first phase of the study (B12CS, NCT04147871), investigators will explore the efficacy, safety, tolerability, and acceptability of repeated doses of ADV7103 following 7 days of treatment. This phase of the study will include 60 patients who will be randomly assigned 1:1:1:1 to either a low dose, medium dose, or high dose of ADV7103 or to placebo.

The primary end point for this phase of the study is the percentage of urinary pH values of 7.0 or higher during repeated measures 24 hours following 7 days of treatment among patients who received ADV7103 compared with those who received placebo. Secondary outcome measures include the effect and dose-response relationship of ADV7103 on urinary pH, cystine crystalluria (including Vcys), and cystinuria parameters following 7 days of treatment.

The second phase of the study (B13CS) is a pre-planned exploratory sub-study to assess the safety and efficacy of ADV7103 in patients who are under 6 years of age or are urinary incontinent. The B13CS study will not include a placebo arm. In total, the B12CS and B13CS studies plans to enroll 72 patients. Primary completion of the studies is expected in May 2025.⁴

A follow-up study (B14CS, NCT04137978) will then be conducted over 2 years to assess ADV7103 long-term in patients who completed the previous B12CS or B13CS studies.

References

1. FDA grants orphan drug designation to ADV7103 for the treatment of cystinuria. News release. Advicenne. Published online and accessed March 25, 2024. https://www.businesswire.com/news/home/20240324280382/en/FDA-Grants-Orphan-Drug-Designation-to-ADV7103-for-the-Treatment-of-Cystinuria

2. Advicenne obtains orphan drug designation for ADV7103 in the United States for the treatment of distal renal tubular acidosis (dRTA). News release. Advicenne. December 13, 2022. Accessed March 25, 2024. https://www.businesswire.com/news/home/20221212005598/en/Advicenne-Obtains-Orphan-Drug-Designation-for-ADV7103-in-the-United-States-for-the-Treatment-of-Distal-Renal-Tubular-Acidosis-dRTA

3. Evaluation of ADV7103 efficacy and safety in cystinuria. https://www.asn-online.org/education/kidneyweek/2019/program-abstract.aspx?controlId=3258415

4. Study evaluating patients with cystinuria and efficacy and safety exploratory study in the youngest children. ClinicalTrials.gov. Last updated May 9, 2023. https://clinicaltrials.gov/study/NCT04147871