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Msg  34977 of 34981  at  5/3/2021 11:43:03 AM  by


Endpoints article - Oxlumo

May 3, 2021 10:28 AM EDT R&D
Alnylam provides long-term look at Oxlumo, aiming to further carve out stake in PH1 market
Max Gelman
Associate Editor
Alnylam is already a leader in RNAi therapy, having secured three approvals in the field with a fourth potentially on the way. Now, the drugmaker is releasing new data on one of its OK’ed medicines that boost its long-term safety profile.

As part of continuing analyses from its randomized, placebo-controlled Phase III trial for Oxlumo (lumasiran), Alnylam reported positive 12-month follow-up data in trial participants with primary hyperoxaluria type 1. New results have shown the drug was associated with improving calcium deposit levels in one or both kidneys, or nephrocalcinosis, relative to baseline.

“Nephrocalcinosis is a key indicator of disease severity in PH1,” said trial investigator Jeffrey Saland in a statement. “Unilateral and bilateral improvements in nephrocalcinosis in some patients treated with Oxlumo are a welcome observation and are consistent with our expectation of the potential clinical impact of a sustained and substantial reduction in urinary oxalate levels.”


The Cambridge, MA-based company presented the data at the American Society of Pediatric Nephrology and Pediatric Academic Societies (PAS) virtual meeting being held over the weekend.

Monday’s results come from 24 patients who had been treated with Oxlumo for 12 months that had valid renal ultrasounds at baseline. Among that group, 11 of 24 patients saw an improvement in their nephrocalcinosis grades relative to baseline, four remained stable and three had declined grades. The remaining six didn’t have available ultrasounds at the follow-up.

Moreover, 14 patients with ultrasounds had been diagnosed with nephrocalcinosis at their baselines. Eleven of these patients showed improvement, and of that group, eight saw improvements in both kidneys.

Alnylam also reported that after 12 months, patients’ estimated glomerular filtration rate (eGFR) remained stable with lumasiran. Kidney stone events, meanwhile, decreased sharply after the first six months of treatment but then leveled off in the following six months among patients in the drug arm of the trial.

In patients who started in the placebo group but then crossed over to Oxlumo after six months, the rate of kidney stones similarly decreased in the following half-year period.

All of Monday’s results came specifically from their randomized, double-blinded Phase III trial in 39 patients, age six and older. Alnylam had also run an open-label Phase III study in parallel, but is yet to report follow-up data from this study.

Oxlumo was approved to treat PH1 back in November, and though the exact price tag is tricky because it’s dosed according to weight, estimates range from $380,000 to $480,000 per year. Alnylam pegged peak sales at about $500 million per year, and it entered the market with an initial showing of $9 million in the first quarter of 2021.

The results come a little less than a month after Dicerna, who had previously feuded with Alnylam over RNAi intellectual property rights, sold off its Oxlumo royalty stake to Royalty Pharma for up to $240 million. Alnylam and Dicerna put aside their legal fight and came together for a collaboration agreement in April 2020, though the latter continues work on a rival product.

Max Gelman
Associate Editor

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