The rare disease drug agaga sugarase alpha injection with a strong solution was approved to enter China.

Medicine Network August 31 - Takeda China announced on the 28th that its innovative drug Ripja (Aga glycosin alpha injection with a thick solution) approved by the State Drug Administration, for the diagnosis of Fabre disease (alpha-semi-lactose glycosidease A deficiency) patients with long-term enzyme replacement treatment.
, Ripja (Aga glycoenzyme alpha injection with a thick solution) is currently the only human-origin enzyme replacement therapy introduced in China, which can bring long-term heart and kidney protection and delay the progression of the disease.
Fabre disease is a rare X-chain hereditary lysosome storage disease, the patient's body metabolite triamtrogyl alcohol (GL3) can not be cleavage, in various tissues and organs a large amount of storage, resulting in the patient's hands and feet usually have burning-like pain, accompanied by less sweat, no sweat and no heat symptoms.
With the progression of the course of the disease, symptoms will gradually worsen and appear kidney, heart and other organ lesions, such as severe renal failure and even uremia, or stroke, myocardial infarction and other cardiovascular complications, if not effective treatment will be seriously life-threatening.
alternative therapy is recommended by foreign guidelines/consensus as a first-line treatment for Fabre disease.
The 2013 edition of China's Fabry Disease Specialist Consensus also mentioned: a number of randomized control and open expansion clinical trial results have shown that enzyme replacement treatment 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.
Professor Chen Nan, Director of Nephrology at Ruijin Hospital, affiliated with Shanghai Jiaofu University School of Medicine, said: "The organ damage caused by Fabre disease is almost irreversible, and if not treated promptly and effectively, the patient's kidneys, heart and other important organs will produce serious functional damage and lesions, life-threatening."
20 years of real-world data validation of the efficacy of Ripja (aga glycosin alpha injection solution) will lead to more treatment options for patients in Fabre, China.
past, patients were diagnosed for decades due to low awareness of Fabre's disease and lack of diagnostic techniques and referrals.
present, the majority of Fabre patients in the country are still receiving intervention, such as pain relief, kidney dialysis, the installation of pacemakers, resulting in the majority of patients can not control the disease, seriously affecting the quality of life of patients and families.
2018, Fabre disease is listed in the "First Rare Diseases List" jointly formulated by the National Health Andy Commission and five other departments.
(Aga glycosin alpha injection solution) was the first to be licensed in the European Union in 2001.
has been listed in more than 63 countries as of April 2019.
In March 2018, Ripja (Aga glycoenzyme alpha injection with a thick solution) was selected to enter the second batch of clinically urgently needed new drugs from abroad issued by the Drug Review Center of the State Drug Administration, so that the international innovative drug benefits Patients in China through accelerated review and approval through a special channel.
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