New PH1 Treatment Clears Phase 3 Study

Big development for those in the primary hyperoxaluria type 1 (PH1) community!

Alnylam Pharmaceuticals just announced that its Phase 3 study of lumasiran, an investigational therapy for the treatment of primary hyperoxaluria type 1 (PH1), has met its primary efficacy endpoint.

In other word — the results pointing to the treatment’s effects were statistically significant!

What is Primary Hyperoxaluria Type 1?

Primary hyperoxaluria type 1 (PH1) is a rare disorder that affects the kidneys. PH1 results from buildup of oxalate, which normally is filtered through the kidneys and excreted in the urine.

For those with PH1, the oxalate accumulates in the kidneys and urinary tract and combines with calcium to form the main component of kidney and bladder stones (calcium oxalate).

To read more about PH1, click here.

Positive Topline Results

We previously covered when Alnylam announced its plan for clinical trial back in April — so we’re pleased in the progress!

Below is from a statement made by Akshay Vaishnaw., president of R&D at Alnylam:

“We are very pleased to report positive topline Phase 3 results for lumasiran… Patients living with PH1 and their families are faced with the burden of recurrent and painful stone events and a progressive and unpredictable decline in kidney function that ultimately results in end-stage renal disease and the need for intensive dialysis as a bridge to dual liver/kidney transplantation.”

Vaishnaw went on to say that the results demonstrate lumasiran can significantly reduce the underlying pathophysiology of PH1 — and just as important,  it has shown to be safe and tolerable for patients.

“We extend our deepest gratitude to the patients, caregivers, investigators, and study staff who participated… and contributed to what we believe is an important medical advance for the treatment of PH1.”

Next Steps

Based on these results, the Company plans to submit a New Drug Application (NDA) and file a Marketing Authorisation Application (MAA) for lumasiran in early 2020.

We will keep a close eye on this as it enters this crucial next phase!

Are you or someone you know diagnosed with PH1? Share your stories, thoughts, and hopes with the Patient Worthy community!

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