Research & Development
Alnylam Launches Value-Based Agreement Framework for Oxlumo to Accelerate Access for Patients
25 November 2020 - - US-based RNAi therapeutics company Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) has launched a new framework for value-based agreements designed to help people with primary hyperoxaluria type 1 gain access to Oxlumo (lumasiran), the company said.

Now approved by the US Food and Drug Administration for the treatment of PH1 to lower urinary oxalate levels in pediatric and adult patients, Oxlumo is the first-ever approved targeted therapeutic that substantially curbs oxalate production in patients living with PH1, an ultra-rare genetic disease characterized by oxalate overproduction.

Alnylam is in active discussions with leading payers and has reached an agreement in principle with Express Scripts, Harvard Pilgrim, and Highmark to pursue VBAs for Oxlumo.

Oxalate overproduction in PH1 results in elevated urinary oxalate and the deposition of calcium oxalate crystals in the kidneys and urinary tract. People with PH1 typically endure intensive management of debilitating, painful and recurrent kidney stones.

With limited treatment options previously available, the disease would inevitably progress to kidney failure, nephrocalcinosis (calcification of the kidneys), and multi-organ dysfunction as a consequence of systemic oxalosis (the spread of oxalate to organs and tissues outside of the kidneys).

People with PH1 often present with kidney failure at the time of diagnosis.

PH1 patients with renal failure undergo dialysis almost daily, for up to 10-12 hours per day and night.

A dual or sequential liver/kidney transplant is then typically performed to resolve the underlying metabolic defect in the liver and restore kidney function, but these interventions are associated with life-long immunosuppression and a high risk of morbidity and mortality.

Until now, there have been no FDA-approved pharmaceutical therapies for PH1.

There are approximately one to three individuals per m across the US and Europe with a confirmed PH1 diagnosis, of those it is estimated that 1,000 - 1,700 individuals have not yet received a liver transplant, making them potentially eligible for treatment.

Building on the Alnylam Patient Access Philosophy and Alnylam's ongoing commitment to deliver fair value to payers and providers, the company has worked with payers on a new and enhanced VBA framework.

Since Oxlumo is indicated for both pediatric and adult patients, and is dosed by weight, related costs can vary relative to each patient and use over time.

As such, Alnylam has structured a new VBA component that specifically addresses many payers' concerns for budget predictability and value, particularly for ultra-rare orphan disease therapies that are administered across a wide spectrum of ages from infants to adults.

The new VBA component, called a Patient Need Adjustment, is now being added to Alnylam's overall VBA offering for Oxlumo.

Participating payers will qualify for the PNA rebate if the average number of vials utilized by a plan member exceeds an established threshold, providing payers with greater short-term and long-term predictability.

The PNA was designed to mitigate the risk of escalating or varying costs associated with dosing requirements, thereby accelerating access for people diagnosed with PH1.

To further address budget predictability, Alnylam is also making available its Prevalence Based Adjustment component, which was first introduced last year for Alnylam's second-approved therapy, GIVLAARI (givosiran).

There are often uncertainties in diagnosis rates and disease prevalence estimates in ultra-rare, poorly diagnosed orphan diseases, making it challenging for payers to predict the number of patients who will be covered within their plans.

This feature triggers a rebate to participating payers if the number of diagnosed patients they cover exceeds current epidemiologic estimates for PH1.
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