With FDA nod for rare disease drug Rivfloza, Novo Nordisk eyes showdown with Alnylam

Novo Nordisk has spent the last few years grappling with the demands stemming from the launch of its popular obesity therapy Wegovy. Now, with a new FDA approval, the drugmaker is making a splash in a much rarer disease. 

Novo’s Rivfloza, a treatment for the rare disease primary hyperoxaluria type 1 (PH1), has gained approval from the FDA, the company said Monday. A monthly, subcutaneous treatment, Rivfloza belongs to the of ribonucleic acid interference (RNAi) class of drugs.

It’s the second FDA-approved option for the disease after Alnylam’s rival RNAi therapy Oxlumo won its FDA approval in November 2020. 

PH1 is a rare, severe disease characterized by an overproduction of oxalate, which causes the formation of kidney stones and leads to progressive kidney damage. 

It’s estimated about 2,000 patients live with the disease in the U.S., Novo said. Many are believed to be undiagnosed. 

The Danish drugmaker got its hands on the therapy with its 2021 purchase of Dicerna Pharmaceuticals for $3.3 billion. At the time, the drug was Dicerna’s lead pipeline candidate. 

Dicerna previously studied the drug in other forms of primary hyperoxaluria (PH2 and 3), but ultimately narrowed its focus to PH1 after seeing early data. 

The FDA endorsed Rivfloza to lower urinary oxalate levels in PH1 patients 9 and older who have relatively preserved kidney function. In a phase 2 study, patients on the medicine experienced a reduction in urinary oxalate excretion compared with placebo, according to Novo.

As for rival Alnylam, the company has been busy with launch efforts for Oxlumo since its 2020 approval. By the end of June, more than 350 commercial patients were taking the drug worldwide. In the second quarter, the drug generated $24.2 million.  

Oxlumo is given subcutaneously every three months, while Novo’s Rivfloza is a monthly therapy. Novo plans to launch its drug in early 2024.