Takeda Ripja (Aga glycoenzyme alpha injection with a thick solution) was approved to enter China.

Takeda China announced today that its innovative drug Ripja ® (a strong solution for injection of aga glycosin alpha) has been approved by the State Drug Administration for long-term enzyme replacement therapy in patients diagnosed with Fabre disease (alpha-semi-lactose glycoside a deficiency).
Repja® (Aga glycosin alpha injection thick solution) is the only human-origin enzyme replacement therapy currently introduced in China, which can bring long-term heart and kidney protection and delay the progression of the disease, improving the quality of life of patients and their families.
the first ® to be licensed in the European Union in 2001 with the Rypjama (Aga glycosin alpha injection solution).
to April 2019, it has been listed in more than 63 countries, accumulating up to 20 years of real-world experience worldwide, benefiting more than 31,000 patients.
In March 2018, Ripja ® (Aga glycosin 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, and the international innovative drugs were rapidly benefiting Chinese patients through accelerated review and approval through specialized channels.
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.
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.
"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 cause serious functional damage and lesions, which are life-threatening," said Professor Chen Nan, director of nephrology at Ruijin Hospital, affiliated with Shanghai Jiaofu University School of Medicine.
real-world data ® 20 years of real-world data validation of the efficacy of Ripja ® (Aga glycosin alpha injection) will lead to more treatment options for patients in Fabre, China.
past, patients were diagnosed for decades due to low awareness of Fabre disease and lack of diagnostic techniques and referrals.
At present, the majority of Fabre patients in China 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, so it is urgent to break-through therapy to alleviate the survival difficulties.
2018, Fabre disease is listed in the First Rare Diseases Directory, jointly developed by five departments, including the National Health Andy Council, which aims to accelerate the review and listing of rare disease therapies and improve the quality of life of patients. Mr. Shan Guohong, President of
Takeda China, said, "Thank you very much for the priority review and approval of the relevant government departments, so that Ripja ® (Aga glycosin alpha injection solution) can quickly benefit Chinese patients, fully demonstrating the Government's determination and strength to accelerate the introduction of innovative drugs for high-quality rare diseases."
Takata has always adhered to the concept of 'patient first', working with all parties to accelerate the introduction of more of the world's first and most urgently needed drugs, so that patients with rare diseases in China can synchronize with patients in other parts of the world to obtain the most innovative drugs, to change the current patient 'no cure' dilemma.
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