The gospel of patients with the rare disease Fabre disease! The new drug Ripja has been approved in China

On August 28th, Takeda China announced that its innovative drug Agagase α injected with a thick solution (the commodity name "Ripja") has been approved by china's Drug Administration for long-term enzyme replacement therapy for patients diagnosed with Fabre disease (α-semi-lactose glycosidease A deficiency).
it is worth mentioning that this is also the first successful introduction of human-origin enzyme replacement therapy, can be described as the domestic Fabre disease patients and their families of the gospel.
is a α of X chromosome companion genetic lysosome storage caused by mutations in the X-semi-lactose glycosidease (GLA) gene.
the disease originated in the fetus and gradually worsened with the patient's life, in children and adolescents, patients will appear neuropathic pain, proteinuria and other symptoms.
as the disease progresses, patients can be life-threatening due to kidney failure or severe complications such as hypertrophic heart muscle and moderate stroke.
data show that there are more than 300 people diagnosed with Fabre disease in China.
delayed diagnosis can be as long as 15-20 years due to the variety of clinical manifestations, lack of specificity of early symptoms, lack of clinical awareness and other reasons.
Currently, most Fabre patients in China mainly receive pain relief, kidney dialysis, the installation of pacemakers and other interventions, resulting in most patients can not control the condition, the quality of life of patients and their families are worrying.
In 2018, Fabre disease was included in the list of the first rare diseases jointly developed by the five departments, which is regarded by the industry as a historic breakthrough in the protection of rare diseases, meaning that the pace of national regulation of rare diseases diagnosis and treatment, to protect the use of rare diseases patients is accelerating.
On June 6 this year, Fabzan China announced that The first Fabre disease special effects drug, Fabuzan (injected with agaglyzyme β), was launched in China for long-term enzyme replacement therapy for patients diagnosed with Fabre disease (α-semi-lactose glycosidease A deficiency), suitable for adults, children and adolescents 8 years and older.
same time, the China Primary Health Care Foundation, in cooperation with Sanofi, launched the "Life Salute" Fabre Disease Patient Assistance Program, which provides drug assistance to eligible patients and reduces the financial burden on patients.
the start of the project, for eligible low-income patients over the age of 8 years, for every 2 months of continuous use of Fabzan, one month of free treatment is provided to them.
and enzyme replacement therapy are recommended by foreign guidelines/consensus as a first-line treatment for Fabre disease.
The 2013 edition of China's Fabry Disease Specialist Consensus mentioned that: a number of randomized control and open expansion clinical trial results have shown that enzyme replacement treatment Of Fabre disease can reduce the deposition of GL3 in patients' cells, reduce patients' limb pain, gastrointestinal symptoms, improve myocardial hypertrophicity, stabilize kidney function, improve patients' quality of life and prognosis.
, after Fabzan, Ripja has been successfully approved at home or brought new treatment options to Fabre patients.
patients will be provided with an effective opportunity to intervene before irreversible organ damage.
, Fabre disease will also become one of the few rare diseases in the country that can be prevented and treated.
is understood to have been licensed to list in the European Union in 2001 and as of April 2019, it has been listed in more than 63 countries, with a cumulative global real-world experience of up to 20 years, benefiting more than 31,000 patients.
March 2018, Ripja entered the second batch of clinically in urgent need of new drugs from abroad, through the accelerated review and approval of special channels.