PDUFA Date Set for October 6, 2022

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the U.S. Food and Drug Administration (FDA) has accepted the Company’s supplemental New Drug Application (sNDA) for lumasiran, an investigational RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – for the reduction of plasma oxalate in the treatment of patients with advanced primary hyperoxaluria type 1 (PH1). The FDA has set an action date of October 6, 2022, under the Prescription Drug User Fee Act (PDUFA).

“We are pleased that the FDA has accepted our sNDA for lumasiran based on the positive six-month results of the ILLUMINATE-C study showing that lumasiran can substantially reduce plasma oxalate levels in patients with compromised renal function due to PH1, including those on hemodialysis,” said Pushkal Garg, MD., Chief Medical Officer and EVP, Clinical Development and Medical Affairs at Alnylam. “This filing acceptance is a positive step for patients with advanced PH1, who are at risk for the devastating complications of systemic oxalosis.”

Additionally, a Type II Variation for lumasiran to amend the label to further inform on the use of lumasiran in patients with advanced PH1 was submitted to and validated by the European Medicines Agency (EMA) in December 2021. The application is undergoing review by the Committee for Medicinal Products for Human Use (CHMP), which will then issue an opinion to the European Commission.

In 2020, OXLUMO was approved by the FDA for the treatment of PH1 to lower urinary oxalate levels in pediatric and adult patients and by the EMA for the treatment of PH1 in all age groups.

OXLUMO® (lumasiran) INDICATION AND IMPORTANT SAFETY INFORMATION

Indication

OXLUMO is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.

Important Safety Information

Adverse Reactions

The most common adverse reaction that occurred in patients treated with OXLUMO was injection site reaction (38%). Symptoms included erythema, pain, pruritus, and swelling.

Pregnancy and Lactation

No data are available on the use of OXLUMO in pregnant women. No data are available on the presence of OXLUMO in human milk or its effects on breastfed infants or milk production. Consider the developmental and health benefits of breastfeeding along with the mother’s clinical need for OXLUMO and any potential adverse effects on the breastfed child from OXLUMO or the underlying maternal condition.

For additional information about OXLUMO, please see the full U.S.Prescribing Information.

About Lumasiran

Lumasiran is a subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in development for the treatment of advanced primary hyperoxaluria type 1 (PH1). HAO1 encodes glycolate oxidase (GO). Thus, by silencing HAO1 and depleting the GO enzyme, lumasiran inhibits production of oxalate – the metabolite that directly contributes to the pathophysiology of PH1. Lumasiran utilizes Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index. Lumasiran has received regulatory approvals from the U.S. Food and Drug Administration (FDA) for the treatment of PH1 to lower urinary oxalate levels in pediatric and adult patients and from the European Medicines Agency (EMA) for the treatment of PH1 in all age groups under the brand name OXLUMO®.

About ILLUMINATE-C Phase 3 Study

ILLUMINATE-C (NCT04152200) is a single arm, open-label, multinational Phase 3 study evaluating the safety and efficacy of lumasiran in PH1 patients of all ages with severe renal impairment (eGFR ≤ 45 mL/min/1.73m2 or elevated serum creatinine for patients