MHRA grants early access to lumasiran

IMage of a pen signing on a blue background to show MHRA grants early access to lumasiran

Alnylam has announced that the Medicines and Healthcare Products Regulatory Agency (MHRA) has granted early access to lumasiran.

Alnylam Pharmaceuticals, Inc, the leading RNAi therapeutics company, announced that the MHRA has granted lumasiran, an investigational RNAi therapeutic in development for the treatment of primary hyperoxaluria type 1 (PH1), a positive scientific opinion through the Early Access to Medicines Scheme (EAMS). With this decision, eligible PH1 patients in the UK, many of whom are children, can gain access to lumasiran before the drug is granted marketing authorization by the European Commission (EC).

The aim of EAMS is to provide early availability of innovative, unlicensed medicines
to UK patients who have a high degree of unmet clinical need. The medicines included in the scheme are those that are intended to treat, diagnose or prevent seriously debilitating or life-threatening conditions where there are no adequate treatment options.

PH1 is an ultra-rare orphan disease characterized by excessive oxalate production, which can lead to end-stage kidney disease (ESKD) and other systemic complications.1,2 PH1 affects 1-3 individuals per million in Europe and the United States.3 In the United Kingdom, it is estimated that there are around 90 patients diagnosed with PH1.4

Current treatment approaches do not prevent oxalate overproduction and aim to lessen damage to the kidneys and delay progression to ESKD.1,2,5 PH1 patients with advanced kidney disease require dialysis to help filter waste products from their blood, until they are able and eligible to receive a dual or sequential liver/kidney transplant.1,2 However, long-term dialysis and post-transplant complications can severely impact patients’ quality of life.6,7

“This positive scientific opinion to make lumasiran available through the EAMS is wonderful news for PH1 patients and their families, who currently have limited treatment options,” said Brendan Martin, Country Manager, UK & Ireland at Alnylam. “New medicines that address the underlying cause of this ultra-rare condition, and have the potential for a favorable impact on disease manifestations, are urgently needed. This decision will allow eligible PH1 patients in the UK to have access to lumasiran at the earliest opportunity.”

The MHRA’s decision is based on the evaluation of the effects of lumasiran in PH1 patients and its safety profile, including data from the ILLUMINATE-A Phase 3 study. The results of the ILLUMINATE-A study were presented at the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) International Congress, held June 6-9, 2020 as a virtual event.

A marketing authorization application (MAA) for lumasiran has been submitted to the European Medicines Agency (EMA) in April 2020. In 2018, lumasiran was granted access to the Priority Medicines (PRIME) scheme which allows for Accelerated Assessment by the EMA. The EC decision, which will apply to the UK, is expected in late 2020. In addition, Alnylam filed a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA). The FDA has granted a Priority Review for the NDA and has set an action date of December 3, 2020 under the Prescription Drug User Fee Act (PDUFA).

1. Cochat P & Rumsby G. Primary hyperoxaluria. N Engl J Med. 2013;369:649-658. doi:
2. Milliner DS et al. Primary Hyperoxaluria Type 1. 2017. In: Adam MP, Ardinger HH, Pagon RA, et al., editors. GeneReviews® . Seattle (WA): University of Washington, Seattle; 1993-2020
3. Bhasin B, Urekli HM & Atta MG. Primary and Secondary Hyperoxaluria: Understanding the Enigma. World J Neprhol. 2015;4(2):235-244. doi: 10.5527/wjn.v4.i2.235
4. RaDaR Rare Disease Group Annual Report. April 2018 – March 2019. The Renal Association. Accessed June 2020. Available at:
5. Hoppe B, Beck BD & Milliner DS. The primary hyperoxalurias. Kidney Int. 2009;75:1264-1271. doi:10.1038/ki.2009.32
6. Dialysis side effects. National Health Service (NHS). Accessed February 2020. Available at:
7. Silkensen JR. Long-term Complications in Renal Transplantation. J Am Soc Nephrol.