The U.S. FDA had granted GIVLAARI™ Priority Review which has been clearly used in approving the drug several months earlier than the agency announced. The agency has also granted GIVLAARI™ Breakthrough Therapy and Orphan Drug Designations.
In Europe, the product is being reviewed under accelerated assessment by the European Medicines Agency (EMA) for patients with AHP after being granted Priority Medicines (PRIME) Designation and Orphan Drug Designation from the EMA.
The FDA decision was based on positive results from the ENVISION Phase 3 study; a randomized, double-blind, placebo-controlled, multinational study of 94 patients with AHP at 36 study sites in 18 countries – the largest ever interventional study conducted in AHP.
In the ENVISION trial, AHP patients administered GIVLAARI™ experienced fewer porphyria attacks compared to placebo. The product has also caused a reduction in intravenous hemin use, as well as reductions in urinary aminolevulinic acid (ALA) and urinary porphobilinogen (PBG).
Prohost Observations
A few days ago the FDA approved Alnylam’s product inclisiran sodium which the firm has licensed to The Medicines Company (MDCO) for the lowering of Bad LDL cholesterol. Prior to the inclisiran approval, Alnylam was granted approval for its product Onpattro (patisiran) for the treatment of polyneuropathy in patients with hereditary transthyretin-mediated amyloidosis, a rare fatal disease affecting around 50,000 people worldwide.
The most recent approval is for Alnylam’s product GIVLAARI™ which is the second RNAi therapeutic to be approved in the last 16 months. It’s the first-ever GalNAc-conjugate RNA therapeutic to be approved and Alnylam’s third product to reach the market. This represents excellent news for ALNY and its holders. This news boosts investors’ trust in Alnylam’s capabilities and in the value of its pipeline products, especially the novel class of advanced and improved RNAi therapeutics. It also boosts investors’ conviction that Alnylam is on the road towards fulfilling its promised 2020 goals which comprise the approval of multiple RNAi therapeutics and other antisense products generating revenues and income while becoming a global commercial company driving growth and rewarding the shareholders.
The stock rallied and is now trading at about $110, exceeding the Prohost 2019 target by nearly $20.
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We are working on Prohost Letter #437 and it will be posted soon. This upcoming issue is important as it tackles all the products that excelled in clinical trials, all the firms that are outperforming and the reasons for their stocks’ rallies or their slow but sure climb.
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To read more about these firms please run a search from our website by clicking here.
Alnylam: Its Second RNAi Therapy Approval Boosted Savvy Investors’ Trust
Alnylam Granted FDA Early Approval of GIVLAARI™ for AHP
Alnylam’s (ALNY) stock outperformed as its subcutaneous injection GIVLAARI™ (givosiran) has been granted FDA early approval. GIVLAARI™ is created and developed for the treatment of adults with acute hepatic porphyria (AHP). AHP is a family of ultra-rare, genetic diseases characterized by debilitating, life-threatening attacks and, for some patients, chronic manifestations that demeans patients’ daily functioning and quality of life. Long-term complications of AHP can include chronic neuropathic pain, hypertension, chronic kidney disease and liver disease.
In a clinical trial, GIVLAARI™ demonstrated a significant reduction in the rate of porphyria attacks that required hospitalizations, urgent healthcare visits or IV hemin administration at home.
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