It's like a bamboo! Alnylam's another RNAi treatment is FDA Fast Track!

Alnylam has announced that the U.SFDA has granted the vutrisiran fast-track eligibility (FTD), a research RNAi therapy used to treat multiple neuropathy caused by hereditary thyroxine protein (hATTR) amyloid degeneration in adultsFTD aims to accelerate drug development and rapid review of serious diseases to address critically unmet medical needsAccess to FTD for research drugs means that pharmaceutical companies can interact more frequently with the FDA during the development phase, are eligible for accelerated approval and priority review if they meet the relevant criteria after filing a listing application, and are also eligible for rolling reviewin addition to being granted FTD,, vutrisiran was also granted the Orphan Drug (ODD) for the treatment of ATTR amyloid degeneration in the United States and the European UnionCurrently, Alnylam is conducting two Phase III clinical trials (HELIOS-A, HELIOS-B) to assess the safety and effectiveness of vutrisiranTogether, these studies form a comprehensive clinical development program designed to demonstrate the widespread impact of vutrisiran on the multi-system manifestations of the disease and the full spectrum of ATTR amyloid degeneration patientsin theHELIOS-A study, 164 patients were randomly assigned to receive vutrisiran or Onpattro to demonstrate whether vutrisiran is superior to Onpattro in the treatment of nerve damageIn the HELIOS-B study, 600 patients with hATTR amyloid amyloid with cardiomyopathy were randomly assigned to vutrisiran or placebo treatment to assess the effect of vutrisiran on all-cause mortality and hospitalization rates associated with cardiovascular diseaseData from both studies are expected to be available in early 2021Vutrisiran is a research, subcutaneous ritphinology for atTR amyloid degeneration, including hereditary and wild amyloid degenerationThe drug is designed to target and silence specific messenger RNA, which is blocked before wild and mutant transthyroxine (TTR) proteins are producedVutrisiran injections quarterly will help reduce the deposition of TTR amyloid protein in tissues, promote its removal, and restore the function of these tissuesVutrisiran took advantage of The Next Generation Delivery Platform, Enhanced Stability Chemistry (ESC) -GalNAc Conjugate Delivery Platform, developed by AlnylamIf approved, it will further strengthen Alnylam's leading position in the hATTR amyloid therapy market Alnylam is a leader in RNAi therapy development, and its first product, Onpattro,approved by the FDA in August 2018, became the first RNAi drug to be approved for market in 20 years In November 2019, the company's other drug, givlaari,approved by the FDA for adult patients with acute hepatic rickets (AHP), became the world's second RNAi drug , two more RNAi treatments are under review by regulators and are expected to be approved for listing this year One is lumasiran, which is used to treat primary high uremia type 1 (PH1), and the other is inclisiran, which is used to treat hypercholesterolemia In addition, the company has six RNAi therapies in the pipeline in the late stages of clinical development prior to the signing of a licensing and cooperation agreement with Alnylam, TMC acquired the global development and commercialization rights of inclisiran In November 2019, Novartis bought TMC for $9.7 billion, putting it in the pocket Based on the agreement, Alnylam is eligible to receive tiered royalties of up to 20% of the global sales from Novartis there are currently two cholesterol-lowering drugs on the market targeting PCSK9, but they need to be delivered every 2 weeks or once a month Inclisiran is given once every six months and only twice a year, giving it very advantageously in terms of ease of treatment Pharma, a pharmaceutical market research firm, predicts that sales will peak at $2.6 billion a year after the listing Before the patent expires in 2035, Alnylam will receive more than $2.2 billion in royalties from Novartis it's worth noting that just two days ago, Blackstone struck a strategic partnership with Alnylam In this partnership, Blackstone will provide $2 billion to help Alnylam advance technological innovation and commercialization Alnylam said the partnership would help Alnylam "be financially sustainable without refinance" reference source: 1, Alnylam's vutrisiran gets fast-track treatment at the FDA 2, Inclisiran keeps on giving