恩瑞格的获批适应症有什么呢？

Thalassemia is a rare genetic disease. In addition to iron removal and blood transfusion, most patients in China use oral dispersible tablets as treatment. Oral deferasirox treatment is far safer and more convenient than blood transfusion, and has fewer side effects. Today let’s take a look at what are the approved indications of Enrig?

Deferasirox (Enrige) was previously approved for the treatment of iron overload due to blood transfusion in patients 2 years of age and older, and this approval allows deferasirox to be used in the treatment of iron overload in patients with NTDT. Deferasirox should be used in patients with NTDT who have at least 5 mg of iron per 1 g of desiccated liver tissue. The new indication for deferasirox was approved by the FDA through the accelerated approval process, which allows patients to have early access to this promising new drug to treat serious or life-threatening diseases, while the company conducts additional studies to ensure the clinical effectiveness of deferasirox.

Approved indications of Enrig: In November 2005, the US FDA approved Enrig for the treatment of patients 2 years old and older with chronic iron overload caused by blood transfusion. In December 2012, Deferasirox was approved by the European Commission for the treatment of chronic iron overload in patients aged 10 years and older with non-transfusion-dependent thalassemia (NTDT) syndrome who require chelation therapy due to contraindications or insufficiency of deferoxamine mesylate therapy. On January 23, 2013, the FDA approved a new indication for deferasirox for the treatment of chronic iron overload in patients aged 10 years and older with non-transfusion-dependent thalassemia (NTDT).

It is recommended for patients with iron accumulation, such as those who require long-term blood transfusions (blood transfusions up to 100 ml/kg) and whose serum ferritin exceeds 1000 μg/L. According to the body weight, it is initially recommended to take a daily dose of 20 mg/kg of deferasirox. Depending on the improvement of serum ferritin indicators, patients may need to adjust or increase the dose of deferasirox, which is usually 5 mg/kg or 10 mg/kg. The dose is adjusted in units of kilograms, but the total dose should not exceed 30 mg/kg per day. 

The above is the content of the indications, I hope it can help you!