Alnylam's third RNAi drug, Oxlumo, has been approved by the FDA

On November 23rd the FDA approved the listing of Oxlumo, Anylam's rare disease drug, and on November 19th the European Commission approved the listing of the drug, making it the third time the company has introduced RNA Interference (RNAi).
With FDA and EC approval, Oxlumo has thus become the first drug to treat primary high oxalic acid uremia type 1 (PH1), a serious autosomal recessive genetic disease, due to the lack of liver peroxidation alanine-acetaldehyde salt amino transferase, resulting in excessive oxalic acid production, clinical manifestations of oxalic acid urinary disease and repeated urinary stones.
studies have shown that Oxlumo works by targeting mutations that lead to excessive oxalic acid production, which can lead to the recurrence of kidney stones and lead to end-stage kidney disease and serious complications, while increasing patient mortality.
over time, PH1 patients' kidney function gradually declines, and many patients eventually need a double liver and kidney transplant.
until Oxlumo is approved, the only treatment option is to use a method called "excessive hydration", which uses excessive liquid to dilute oxalate.
and Oxlumo's use requires only subsestrapic injections every three months, which can greatly reduce the pain of treatment.
But according to Dr. John Maraganore, the company's chief executive, in an interview, Alnylam intends to set the average price of the drug at $493,000 per patient per year, and expects a net price of about $380,000 per patient per year.
, Alnylam enters into a three-way agreement with the patient based on the therapeutic value so that fees are charged only if the drug is effective.
these agreements, the company will reach a "success criterion" with the payer if the patient reacts to the treatment and collect the payment after one year.
, if the diagnosis increases over time as awareness of the disease increases, the company is prepared to lower its pricing based on market conditions.
However, because the drug is distributed according to the patient's own weight and the patient's weight changes dynamically throughout his or her life, Allenylam will agree to cap the total number of bottles paid per patient to provide them with a reliable medication budget.
alnylam's point of view is to have a stable introduction to the market, rather than the initial large-term use, mainly because of the current lack of patient diagnosis and disease awareness.
as doctors' awareness deepens and the market expands aggressively, Oxlumo is likely to become an ultra-rare orphan product in the future, generating annual sales of up to $500 million at peak times.
Alnylam began offering new RNAi drugs nearly 20 years ago to treat diseases with inadequate clinical options.
is the third RNAi drug approved by Allenylam, after the FDA approved Onpattro in August 2018 and Givlaari in November 2019.
, Onpattro is mainly used to treat amyloid degeneration disease (hATTR), which is mediated by hereditary thyroxine, which is used to treat nerve damage around adults. The world's first RNAi drug has been approved in the UK.
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