October 7, 2022 02:22 PM EDT Pharma
Alnylam touts label expansion for rare oxalate disease
Almost two years after securing its third drug approval, Alnylam is touting an expanded label for Oxlumo.
The company put out word on Thursday night that the FDA approved an sNDA for its treatment lumasiran for primary hyperoxaluria type 1, also known as Ph1. The rare genetic disease, in which the body is unable to regulate the overproduction of oxalate, can spur the development of kidney stones and play a role in later-stage kidney disease.
While the drug was initially approved to lower levels of urinary oxalate, it’s now also cleared to treat Ph1 by reducing the levels of plasma oxalate.
Reducing levels of oxalate is imperative in treating the disease, as excess oxalate can result in calcium oxalate crystals forming in the kidneys and urinary tract — leading to recurrent kidney stones and later kidney failure. What’s more, oxalate can get deposited into other organs beyond the kidneys, affecting the rest of the body in a condition called systemic oxalosis.
What makes plasma oxalate levels important, per Alnylam, is that higher levels of plasma oxalate play a direct role in oxalosis. That could lead to vision loss, skin ulcers, cardiomyopathy and bone fractures.
Alnylam backed its sNDA up with six-month results from a Phase III study called Illuminate-C, where lumasiran (Oxlumo) showed reductions in levels of plasma oxalate, plus “encouraging” safety and tolerability results.
When Oxlumo won approval back in November 2020, former CEO John Maraganore estimated only 1,000 to 1,700 patients in the US and Europe had the disease at any given time.
Alnylam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll
Alnylam did not respond to multiple requests for comment from Endpoints News before publication.
In the meantime, lumasiran is being assessed in a Phase II study as a treatment for patients suffering from recurrent kidney stone disease and increased levels of urinary oxalate.
Alnylam hasn’t been the only company going after primary hyperoxaluria — Dicerna has been a competitor since before Alnylam secured the FDA win in 2020. However, after a trial where it succeeded in Ph1 but not another type it was going for in 2021, shares tanked and the company was later bought by Novo Nordisk for $3.3 billion.