AHP treatment drugs were approved in advance for the first time, and the second RNAi treatment was launched

Photo source: today, the U.S Food and Drug Administration (FDA) approved givlaari for the treatment of adult acute Porphyra (AHP), the second RNAi drug approved by the FDA after last year's onpattro (paisiran) Givlaari is also the first drug approved for the treatment of AHP Photo source: the website of Alnylam FDA approved the application for breakthrough treatment and priority review Givlaari has also been assigned drugs for rare diseases, which provides an incentive to help and encourage the development of drugs for rare diseases FDA approved the use of givlaari by Alnylam pharmaceutical company In addition, givlaari is undergoing an accelerated evaluation by the European Drug Administration (EMA) to treat patients with AHP and rare disease drugs designated by EMA after receiving the priority drug (prime) designation Photo source: givlaari, the official website of Alnylam, is a RNAi treatment method for acute porphyria (AHP) with ALAS1 as the target In this key study, givlari was shown to significantly reduce the incidence of porphyria requiring hospitalization, emergency medical care, or intravenous heme at home compared to placebo Givlaari is Alnylam's first commercial therapeutic drug, based on its enhanced stable chemical ESC GalNAc conjugation technology to improve efficacy and durability It is administered by subcutaneous injection once a month, and the dosage is determined according to the actual weight It should be administered by medical professionals Givlaari reduces the level of alanine synthetase 1 (ALAS1) messenger RNA (mRNA) specifically, thereby reducing toxins associated with the attack of AHP and other disease manifestations Givlaari Gamma (givesiran) packaging and product bottles (picture source: businesswire) acute porphyria hepatica (AHP) is a very rare genetic disease, which is characterized by a potential life-threatening attack, for some patients, it is a chronic manifestation of negative impact on daily life There are four types of AHP: acute intermittent porphyria (AIP), hereditary fecal porphyrin (HCP), variegated porphyria (VP) and ALA dehydratase deficient porphyria (ADP) Each type of AHP is caused by a lack of an enzyme in the heme biosynthesis pathway in the liver due to genetic defects The majority of patients with AHP are women, and the symptoms of the disease vary greatly Severe, unexplained abdominal pain is the most common symptom, which can be accompanied by limb, back, chest pain, nausea, vomiting, confusion, anxiety, epilepsy, limb weakness, constipation, diarrhea or dark red urine Nonspecific signs and symptoms of AHP often lead to misdiagnosis of other more common diseases such as viral gastroenteritis, irritable bowel syndrome (IBS), detoxification and appendicitis As a result, AHP patients sometimes have a diagnosis of up to 15 years In addition, long-term complications and complications of AHP can include hypertension, chronic kidney disease or liver disease, including HCC Richard pazdur, director of the FDA cancer center and acting director of the FDA Office of drug evaluation and research, said: "acute attack of porphyria can lead to severe pain and paralysis, respiratory failure, epilepsy and mental state change These lesions are sudden and can cause permanent nerve damage and death Until givlaari's approval today, previous treatments have only partially alleviated the persistent severe pain caused by these lesions Today's approved drugs can treat the disease by helping to reduce the number of episodes that disrupt a patient's life " Givlaari was approved by FDA less than four months after receiving NDA Based on the results of phase 3 study of vision, phase 3 study of vision is a randomized, double-blind, placebo-controlled transnational study 94 patients with acute porphyria were studied in 36 research sites in 18 countries, which is the largest AHP intervention study ever Patients were given placebo or givlaari The performance of Givlaari is measured by incidence rate of porphyria The incidence rate of porphyria requires hospitalization, emergency medical care or intravenous hemoglobin at home Compared with patients taking placebo, patients taking givlaari had a 70% reduction in the number of porphyrias But some of the side effects of givlaari are nausea and injection site reactions It is suggested that medical staff should monitor the allergic reaction and renal function of patients Before and during the treatment, patients should have regular liver function examination Previously, FDA approved the infusion of onpattro (paisiran), which was also developed by Alnylam, for adult patients with peripheral neuropathy (polyneuropathy) caused by hereditary tattr RNA interference (RNAi) is a natural process of gene silencing cells, which represents one of the most promising and rapidly developing frontier fields in biology and drug development Its discovery is known as "a major scientific breakthrough once in ten years", and won the Nobel Prize in physiology or medicine in 2006 By using the natural biological process of intracellular RNAi, a new drug called RNAi therapy has now become a reality Small interfering RNA (siRNA) is a molecule that mediates RNAi and is an integral part of the RNAi treatment platform of Alnylam Through potential silencing of messenger RNA (mRNA), which is a genetic precursor encoding pathogenic proteins, it can prevent their production This is a revolutionary approach that has the potential to change care for patients with genetic and other diseases Photo source: Alnylam official website Now there are good news about RNAi drug research and development For example, the medicine company (TMC) company's lipid-lowering drug inclisiran; arrowhead and its partner Johnson & Johnson's Janssen drug jnj-3989, which is developed to treat chronic hepatitis B virus, etc RNAi therapy was rated as one of the top ten scientific developments in 2001 by science magazine, and ranked first among the top ten scientific developments in 2002 Andrew in 2006 ·Faye and Craig Mello won the Nobel Prize in physiology and medicine for their contributions to the study of RNAi mechanism Now with the approval of drugs, RNAi research has made a breakthrough Refer to source 1.http://suo.im/54msld 2.https://www.businesswire.com/news/home/20191120005849/en/jienigui statement: this point of view only represents the author, not the position of yaozhiwang, welcome to exchange and supplement in the message area; if you need to reprint, please be sure to indicate the author and source of the article.