Alnylam is in active talks with major payers and has agreed in principle with Express Scripts, Harvard Pilgrim and Highmark to follow the VBAs for OXLUMO. VBAs build on Alnylam`s patient access philosophy announced last year, which focuses the company`s business objectives on proactively responding to patient access while providing added value to patients, physicians and insurers. This philosophy obliges Alnylam to act urgently for patients, to pursue value-based agreements and not to increase the annualized price of its medicines through the Consumer Price Index (CPI-U), unless valuable new innovations have been obtained. “Express Scripts, Accredo and Cigna are committed to providing patients with rare diseases with simpler, affordable and more predictable ways to receive appropriate treatment and treatment,” said Steve Miller, Executive Vice President and Chief Clinical Officer at Express Scripts. “The kind of agreement proposed by Alnylam helps ensure that people living with acute liver porphyria have access to revolutionary drugs and that the plans have value for every dollar they pay for these therapies.” Under this innovative framework for VBA, participating government and commercial payers will only pay the full value of GIVLAARI if it provides patient outcomes in the real environment, similar to the results demonstrated in clinical trials. An additional and newly developed prevalence-based adjustment function (PBA) will trigger rebates for participating payers if the number of diagnosed patients covering them exceeds current epidemiological estimates for AHP. There are often uncertainties about diagnostic rates and disease prevalence assessments for extremely rare diseases, making it difficult for payers to predict the number of patients who will be covered by their plans. This innovative approach allows payers to better ensure that their overall financial risk is adjusted when a much larger number of patients than currently estimated with GIVLAARI is identified, diagnosed and treated. The company is developing value-based pricing agreements with “major commercial insurers,” she said, noting that it had already agreed “in principle” on the structure of a pact with Harvard Pilgrim Health Care and other payers. “The partnership with the payers of these agreements…

Help speed up coverage decisions for patients,” Alnylam said. OXLUMO is an RNAi therapeutic drug that targets hydroxy-oxidase 1 (HAO1) in the treatment of primary hyperoxaluria type 1 (PH1) to reduce harnoxalate levels in pediatric and adult patients. HAO1 encodes glycolate oxidase (GO), an enzyme before the disease defect in PH1. OXLUMO works by debolizing HAO1 messenger RNA and reducing THE synthesis of GO, which inhibits liver production of oxalate, the toxic metabolite responsible for clinical manifestations of PH1. The crucial illuminate-A study showed that OXLUMO significantly reduced harnoxalate compared to placebo, with the majority of patients reaching normal or near-normal levels. Injection site reactions (SRIs) were the most common drug-related side effects. In the Phase 3 pediatric study, ILLUMINATE-B, OXLUMO presented an efficacy and safety profile consistent with that observed in ILLUMINATE-A. OXLUMO uses enhanced Stabilization Chemistry (ESC) technology -Alnylam`s GalNAc combined technology that increases power and durability. OXLUMO is given once a month for three months by subcutaneous injection, and then once a quarter at a dose based on actual body weight. In patients weighing less than 10 kg, the continuous dose remains monthly. OXLUMO must be administered by a health professional.

For more information about OXLUMO, see OXLUMO.com. Based on the Alnylam Patient Access Philosophy and Alnylam`s continued commitment to delivering fair value to payers and suppliers, the company worked with payers on a new and improved VBA framework.