New drugs for rare diseases in China! Pfizer's world's first oral preparation of transthyroxine protein stabilizer, vidacon (meglumine chlorobenzoate) was approved!

February 13, 2020 / BIOON / -- Pfizer The company recently announced that the world's first oral preparation of transthyroxine protein stabilizer, vdaqel ® (20mg), was approved by China National Drug Administration on February 5 for the treatment of adult patients with stage I symptoms of transthyroxine amyloid polyneuropathy (attr-pn), so as to delay the damage of peripheral nerve function Andreas PENK, President of Pfizer biopharmaceutical group in China, said: "Pfizer has always been aiming to" bring breakthrough innovation for patients to change their lives ", and the new rare disease drug of Pfizer, vidaquan ® The approval of listing in China will fill the gap that there is no effective drug in the treatment field of adult patients with stage I symptoms of attr-pn, and actively help clinicians to bring effective treatment programs for such rare diseases " Attr-pn is a rare and fatal neurodegenerative disease Attr-pn is a rare and fatal neurodegenerative disease The pathogenesis of attr-pn is the abnormal folding of monomers caused by the instability of TTR tetramer, which forms the deposition of amyloid The disease is a multi-system disease, mainly manifested as symmetrical nerve dysfunction from distal to proximal limb, involving motor, sensory and autonomic nerves, as well as damage to heart, kidney, eye and other organs Attr-pn is an adult patient onset, about 10 years after the onset of the disease, gradually progressing to the end At present, the disease has been reported in neurology, ophthalmology and cardiology in different regions of China Because the clinical characteristics of attr-pn are easy to be confused with other common clinical diseases, and the rare cases make clinicians lack of understanding of it, and the chance of obtaining pathological and genetic diagnosis is limited, leading to the delay of diagnosis of the disease Pfizer's innovative drug to assist the treatment of Chinese attr-pn patients before the emergence of Vader, there was no internationally approved drug to treat attr-pn, the available treatment scheme was only symptomatic treatment, and the prognosis of patients was not ideal Vadaquan ® fills this gap and improves patients' confidence in disease treatment Andreas PENK, President of Pfizer biopharmaceutical group in China, said: "Pfizer is willing to continue to expand and develop in the field of rare diseases in China to meet the unmet needs of more Chinese patients The approval of vedazron ® will provide a new treatment scheme for these attr-pn patients who suffer from rare fatal diseases but have no approved drugs to be treated In the future, Pfizer will continue to introduce innovative drugs into China, so that more patients with rare diseases can get better treatment " Vadaquan ® was first approved in the European Union in 2011 for the treatment of ttr-pn in adult patients with early symptomatic polyneuropathy to delay peripheral nerve injury At present, vidacon has been approved to treat attr-pn in more than 45 countries and regions, including the UK, Germany, France, Japan, South Korea, Brazil, Mexico, Argentina and Russia About tafamidis meglumine, tafamidis meglumine is an oral transthyroxin protein stabilizer Vadaquan ® was first approved in the European Union in 2011 for the treatment of ttr-pn in adult patients with early symptomatic polyneuropathy to delay peripheral nerve injury Attr-pn is a neurodegenerative disease of amyloidosis, which can cause sensory loss, pain and weakness of lower limbs, and damage of autonomic nervous system At present, vidacon ® has been approved to treat attr-pn in more than 45 countries and regions around the world, such as the United Kingdom, Germany, France, Japan, South Korea, Brazil, Mexico, Argentina and Russia Original source: Pfizer pharmaceutical