US FDA approves Novo Nordisk's therapy for rare genetic condition

[October 03, 2023]  (Reuters) -Novo Nordisk said on Monday the U.S. Food and Drug Administration (FDA) had approved its therapy to treat a rare genetic condition that affects the kidneys.

The once-monthly injection, to be sold under the brand name Rivfloza, is approved for use in people nine years and older with a type of primary hyperoxaluria and relatively preserved kidney function.

The Danish drugmaker had gained access to the therapy through its $3.3 billion buyout of Dicerna Pharmaceuticals in 2021.

The therapy, which helps to lower urinary oxalate levels, uses the RNA interference technology, or RNAi, where genes that contribute to disease are silenced or rendered ineffective.

Novo Nordisk plans to make the therapy available for eligible patients early next year and said it plans to price the therapy according to "demonstrated clinical value and benefit to those living with PH1 (primary hyperoxaluria type 1)".

Primary hyperoxaluria is a rare condition characterized by recurrent kidney and bladder stones that prevents the liver to make enough of a certain protein leading to the over production of a natural chemical known as oxalate in the urine.

A general view of the offices of Danish drugmaker Novo Nordisk in Copenhagen, Denmark, September 26, 2023. REUTERS/Tom Little/File photo

Alnylam Pharmaceuticals Inc's Oxlumo was the first to receive the health regulator's nod for the treatment of the condition.

The approval for Novo's therapy is based on studies in which the drug reduced the levels of 24 hour-urinary oxalate excretion measured against a pre-decided baseline value and when compared to placebo.

The company estimates that more than 2,000 people are living with the condition in the United States.

(Reporting by Sriparna Roy in Bengaluru; Editing by Sriraj Kalluvila and Maju Samuel)

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