Pfizer's innovative drug has been approved by FDA to fill the gap in the treatment of rare heart disease

May 10, 2019 / AP / -- PFE (NYSE: PFE) recently announced that the U.S Food and Drug Administration (FDA) has approved vyndaqel (meglumine chlorobenzoate) and vyndamax Gamma (chlorobenzoic acid) is used to treat adult wild-type or hereditary ttr-cm to reduce cardiovascular related mortality and hospitalization Vyndaqel and vyndamax are the first two oral preparations of transthyroxine protein stabilizer, chlorobenzoic acid They are also the first and only drug approved by FDA to treat attr-cm Transthyroxine amyloid cardiomyopathy is a rare and fatal disease It is characterized by the deposition of misfolded protein forming abnormal substances (amyloid) in the heart, which is characterized by restrictive cardiomyopathy and progressive heart failure There was no drug approved for the treatment of attr-cm in the past; the available treatment only included symptomatic treatment and heart (or heart and liver) transplantation in individual cases At present, there are about 100000 attr-cm patients in the United States, of which only 1-2% are diagnosed "The approval of vyndaqel and vyndamax is based on the experimental results of attr-act, our innovative drug development project to improve cardiovascular prognosis We are very proud to be able to benefit those attr-cm patients who need treatment " Dr Brenda cooperstone, senior vice president and chief R & D officer of Pfizer's global product R & D rare diseases business unit, said, "vyndaqel and vyndamax can reduce the cardiovascular related mortality and hospitalization rate of patients with this wild-type or genetic rare disease, so that these patients have more time to accompany their close relatives." "Pfizer's goal is to bring breakthrough innovations to patients that change their lives This approval of vyndaqel and vyndamax reflects our commitment to attr-cm patients " Paul Levesque, global president of Pfizer's rare diseases division, said, "for the vast number of patients suffering from this rare and fatal disease without approved drugs to be treated, this milestone will undoubtedly bring a breakthrough improvement in their lives We will continue to strengthen the cooperation of academic organizations, improve people's awareness of the disease, and ultimately promote the detection and diagnosis of the disease " "Attr-cm is not only lethal, but also has a very low diagnostic rate Some patients have seen many doctors and received many examinations in several years, but their condition is still deteriorating." "For attr-cm, a rare disease, education and disease awareness need to be strengthened," said CEO Isabelle lousada, founder of the amyloidosis research alliance The approval of the drug has brought great benefits to our patients; it is also important that we, as a whole, fully recognize the importance of early diagnosis " The FDA approval is based on data from the critical phase III clinical trial (attr-act) of transthyroxine amyloidosis, the first global, double-blind, randomized, and placebo-controlled clinical study to evaluate the efficacy of the drug in the treatment of this disease In the attr-act trial, vynaqel significantly reduced all-cause mortality and cardiovascular related hospitalization rates (P = 0.0006) after 30 months of treatment compared with placebo In addition, a single evaluation endpoint of the primary endpoint demonstrated a 30% (P = 0.026) and 32% (P < 0.0001) reduction in all-cause mortality and cardiovascular related hospitalization rates in the vynaqel group compared to the placebo group In both treatment groups, about 80% of deaths were cardiovascular related From the 6-month follow-up and the subsequent 30-month follow-up, it was found that compared with the placebo group, the vynaqel group also showed significant and consistent treatment effect in terms of activity and health status In particular, vynaqel reduced the 6-minute walk test score (P < 0.0001) and the health status measured by the total score of the Kansas City Cardiomyopathy Questionnaire (P < 0.0001) Safety evaluation showed that patients in this study were well tolerated to vynaqel compared with placebo group The frequency of adverse events in patients treated with vyndaqel was the same as that in placebo group; the proportion of patients interrupted due to adverse events in vyndaqel group was the same as that in placebo group As for ttr-cm, ttr-cm is a rare and fatal disease, which is caused by the instability of a transporter, ttr-cm The transthyroxine protein consists of four identical parts (tetramer) When the unstable tetramer of thyrotropin is dissociated, the misfolded protein will form amyloid fiber and deposit in the heart, which will make the heart become stiff and eventually lead to heart failure There are two subtypes of attr-cm: one is genetic type, also known as mutant type, which is caused by the mutation of thyroxine transforming protein gene The age of disease is earlier, ranging from 50 to 60 years old; the other is non mutant, which is related to aging, known as wild type, which is a more common type, generally involving men over 60 years old Patients with attr-cm are often diagnosed after their symptoms become severe Once confirmed, the median life expectancy of patients with attr-cm is about 2 to 3.5 years, depending on the subtype About vyndaqel (tafamidis meglumine) and vyndamax (tafamidis meglumine) and vyndaax (chlorobenzoic acid meglumine) are oral transthyroxine protein stabilizers, which can selectively bind to transthyroxine protein, stabilize the tetramer of transthyroxine protein, and slow down the formation of starch like substances leading to attr-cm Vyndaqel was recognized as an orphan drug for treating attr-cm in the European Union and the United States in 2012, and as an orphan drug in Japan in 2018 In June 2017 and may 2018, the U.S FDA successively awarded vynaqel quick review qualification, and identified attr-cm as a breakthrough therapy In November 2018, FDA approved the priority review qualification of vyndaqel new drug application (NDA) In March 2019, the Ministry of health, labor and welfare of Japan approved vyndaqel as sakigake (leading, pioneering) drug for the treatment of wild-type and variant attr-cm patients Applications for registration of patients with attr-cm using vynaqel have been submitted to the European Drug Administration (EMA) and are currently under review Vyndaqel was first approved in the European Union in 2011 for the treatment of attr-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, it has been approved to treat attr-pn in 40 countries, including Japan, some European countries, Brazil, Mexico, Argentina, Israel, Russia and South Korea Vyndaqel and vyndamax have not been approved for treatment of attr-pn in the United States Pfizer's rare diseases division includes many serious diseases, involving millions of patients around the world Pfizer is committed to using our knowledge and expertise to help address critical medical needs Our focus on the field of rare diseases is based on more than 20 years of experience in this field, research departments focusing on rare diseases, and drugs in multiple treatment fields (including hematology, neurology and genetic metabolic diseases) Pfizer's rare diseases business department has integrated advanced technology and in-depth understanding of disease mechanism, and integrated insights gained from innovative strategic cooperation with a large number of academic research / scientific researchers, patients and other companies, forming a variety of innovative treatment approaches and solutions Every day, we are making use of the company's global influence to carry out innovation, accelerate the product development process, and strive to bring breakthrough drugs and healing hope to patients as soon as possible Pfizer: work together to create a healthy world? At Pfizer, we are committed to using science and our global resources to provide treatments that can extend and significantly improve human life In the process of exploration, R & D and production of health products, we have established quality, safety and value standards Our global product line includes drugs and vaccines, as well as many world-famous health drug products Every day, Pfizer employees in developed and emerging markets are committed to promoting health and prevention and treatment programs that can cope with the most difficult diseases of our time Pfizer also works with healthcare professionals, governments and communities around the world to support access to more reliable and affordable healthcare services for people around the world This is consistent with Pfizer's responsibility as a world-wide innovative biopharmaceutical company.