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siRNA targeting glycolate oxidase (HAO1) Pregnancy: US label §8.1: 'There are no available data with the use of OXLUMO in pregnant women to evaluate a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes.' No adverse effects on pregnancy or embryo-fetal development were observed in rats at 45 times, or in rabbits at 90 times, the maximum recommended human dose. No formal pregnancy category is stated; no UK SPC §4.6 was fetched.

Lumasiran

Brand names: Oxlumo

Lumasiran is a subcutaneous small interfering RNA (siRNA) therapy used to treat primary hyperoxaluria type 1 in adults and children.

Auto-extracted from the source labelling — not yet independently clinician-verified. These values were distilled from the UK SPC (or the US label where noted) but have not had a clinician sign-off. Confirm against the current SmPC before prescribing.

Adult dose

Dose: Body weight 20 kg and above: loading dose 3 mg/kg once monthly for 3 doses, then maintenance 3 mg/kg once every 3 months (quarterly), beginning 1 month after the last loading dose. 'Dosing is based on actual body weight.'
Route: Subcutaneous injection into the abdomen, thigh, or the side or back of the upper arms; rotate injection sites and do not inject into scar tissue or reddened areas. To be administered by a healthcare professional. 'Divide injection volumes greater than 1.5 mL equally into multiple syringes.' Supplied as a ready-to-use solution requiring no reconstitution or dilution
Frequency: Loading: once monthly for 3 doses. Maintenance: once every 3 months (quarterly), beginning 1 month after the last loading dose
SOURCE: no UK SPC (eMC) record was fetched for this drug (providers.emc is null). The dose above is from the US prescribing information for OXLUMO (lumasiran), Alnylam Pharmaceuticals, label date 2025-12-01 (https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=6ef869cb-8ccd-4339-9991-0fab45da1fbd) — verify against UK labelling before use. FULL WEIGHT-BASED TABLE (§2.1, verbatim): body weight less than 10 kg — loading 6 mg/kg once monthly for 3 doses, maintenance 3 mg/kg once monthly beginning 1 month after the last loading dose; 10 kg to less than 20 kg — loading 6 mg/kg once monthly for 3 doses, maintenance 6 mg/kg once every 3 months (quarterly) beginning 1 month after the last loading dose; 20 kg and above — loading 3 mg/kg once monthly for 3 doses, maintenance 3 mg/kg once every 3 months (quarterly) beginning 1 month after the last loading dose. Adults fall in the '20 kg and above' band. MISSED DOSE: 'If a dose is delayed or missed, administer OXLUMO as soon as possible. Resume prescribed monthly or quarterly dosing, from the most recently administered dose.' STRENGTH (§3): injection 94.5 mg/0.5 mL in a single-dose vial. PRACTICAL: for volumes less than 0.3 mL a sterile 0.3-mL syringe is recommended (on a 0.3 mL / 30-unit insulin syringe, 1-unit markings indicate 0.01 mL). No maximum dose is stated in the fetched label. §6.1 and §8.1 text was truncated at the source-fetch limit.

Paediatric dose

Route: Subcutaneous injection (abdomen, thigh, or side or back of the upper arms), administered by a healthcare professional
Frequency: Loading doses once monthly for 3 doses, then maintenance monthly or quarterly depending on body weight (see notes)
dosePerKg is left null because paediatric dosing in this label is banded by body weight AND differs between loading and maintenance — carrying a single mg/kg number would risk it being applied to the wrong band or phase. VERBATIM §2.1 (Table 1, OXLUMO Weight-Based Dosing Regimen): body weight LESS THAN 10 kg — loading '6 mg/kg once monthly for 3 doses', maintenance '3 mg/kg once monthly, beginning 1 month after the last loading dose'; 10 kg TO LESS THAN 20 kg — loading '6 mg/kg once monthly for 3 doses', maintenance '6 mg/kg once every 3 months (quarterly), beginning 1 month after the last loading dose'; 20 kg AND ABOVE — loading '3 mg/kg once monthly for 3 doses', maintenance '3 mg/kg once every 3 months (quarterly), beginning 1 month after the last loading dose'. 'Dosing is based on actual body weight.' AGE: §8.4 states safety and effectiveness 'have been established in pediatric patients aged birth and older', supported by ILLUMINATE-A (patients 6 years or older and adults), ILLUMINATE-B (patients less than 6 years of age) and ILLUMINATE-C (patients with advanced chronic kidney disease including patients on haemodialysis). No paediatric maximum dose is stated. Verify all under-18 dosing against a children's formulary before prescribing.

Dose adjustments

Renal

No dose adjustment is stated for renal impairment in the fetched label. Administration timing only: 'For Patients on Hemodialysis — Administer OXLUMO after hemodialysis if administered on dialysis days.' The label notes ILLUMINATE-C included patients with moderately or severely reduced GFR (eGFR ≤45 mL/min/1.73 m2) including patients on haemodialysis.

Dose auto-extracted from US FDA prescribing information (openFDA / DailyMed) — cross-check; US labelling may differ from UK — not yet clinician-verified. Always confirm against the product SmPC and your local formulary before prescribing.

Contraindications

  • None stated — the US label §4 Contraindications reads 'None.'

Side effects

  • Injection site reactions — 'The most common adverse reaction (reported in ≥20% of patients) is injection site reactions'
  • NOTE: the §6.1 adverse reactions table was truncated at the source-fetch limit in this bundle — the clinician must verify the full adverse-effect list before publishing

Clinical monograph

How it works

It is an RNA interference agent that reduces hepatic glycolate oxidase by degrading its messenger RNA, lowering the production of oxalate and thereby reducing urinary and plasma oxalate.

Prescribing in practice

  • Treatment should be initiated and supervised by specialists experienced in managing primary hyperoxaluria, with monitoring of oxalate response.
  • Injection-site reactions are among the most frequently reported adverse effects.
  • Dosing in children is by body weight and should follow specialist protocols and a children's formulary.

Monitoring

Monitor urinary and/or plasma oxalate to assess response to treatment under specialist supervision.

Counselling the patient

  • This medicine is given as an injection under the skin, usually by a healthcare professional.
  • Maintaining good fluid intake remains important alongside this treatment.
  • Report marked or persistent injection-site reactions.

Evidence & guidelines

Lumasiran is licensed for primary hyperoxaluria type 1, with the ILLUMINATE trials showing substantial reductions in urinary oxalate versus placebo.

Reference: NICE HST; SmPC; Drug verified in RxNorm (NLM); confirm dosing against the manufacturer SPC (eMC). Verify against your local formulary and current prescribing references before prescribing. The structured dose values shown have been reviewed by a clinician. Monograph status: clinician-reviewed (2026-07-04).