EC Approves ADV7103 Marketing Authorization for dRTA

Recently, the European Commission (EC) approved a marketing authorization for ADV7103 (Sibnayal), the first approved treatment for patients with distal renal tubular acidosis (dRTA). In a news release, pharmaceutical company Advicenne shared that the treatment is available for all patients aged 1 or older. Altogether, the comprehensive marketing authorization is valid in the United Kingdom (UK), Iceland, Liechtenstein, Norway, and all countries within the European Union (EU).

ADV7103

Developed by Advicenne, ADV7103 is an investigational multi-particulate formulation (potassium citrate and potassium bicarbonate) in 2mm granules. According to Advicenne, the drug has:

a prolonged-release formulation designed to maintain a sustained release over a 12-hour period, thus allowing for twice-a-day treatment.

Altogether, ADV7103 has shown immense benefits for patients within clinical trials. For example, in the Phase3 B21CS trial, researchers evaluated the safety, efficacy, and tolerability of ADV7103 in patients spanning from infancy to adulthood. 37 patients enrolled. During the trial, researchers also assessed ADV7103 in comparison to the current standard of care. To begin, patients received the standard of care for 5 days. Next, patients received five days of ADV7103. After analyzing blood and urine biomarkers, researchers suggested that ADV7103 is both effective and shows clinical benefit for treating patients with dRTA. These findings were later confirmed in the B22CS extension study.

Overall, ADV7103 offers a more efficient treatment option, fills an unmet need, controls symptoms of dRTA, and improves quality of life (QOL).

Distal Renal Tubular Acidosis (dRTA)

Although distal renal tubular acidosis (dRTA) can be inherited, this rare kidney disease often occurs as a symptom of another disease or condition. For example, Fabry disease, lupus, hyperparathyroidism, or sickle cell anemia can all cause dRTA. Normally, during bodily functions, certain acids are produced. Healthy kidneys work to remove these acids from the blood and excrete them via urine. However, in dRTA, the kidneys are unable to do this. As a result, too much acid accumulates in the blood, causing the blood to acidify. An estimated 20,000 Americans have dRTA. Symptoms include:

  • Fatigue
  • Stunted growth (in children)
  • Failure to thrive (in children)
  • Kidney stones
  • Confusion
  • Nausea and vomiting
  • Abdominal, back, or bone pain
  • Rapid or irregular heart rate
  • Osteomalacia (bone softening)
  • Low potassium levels
  • Muscle weakness or pain
  • Progressive kidney and bone disease (in adults)
  • Low urine output
  • Paralysis
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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