Brineura Promises To Prolong Quality Of Life In CLN2 Patients
Brineura® (cerliponase alfa), the only FDA-approved enzyme replacement therapy for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2 disease).
continues to demonstrate significant clinical benefits in slowing disease progression, even as access barriers persist for families dependent on government-funded treatment programs.
CLN2 disease is a rare paediatric neurodegenerative disorder.
Symptoms usually appear between the ages of 2 and 4 years and include seizures, delayed speech development, and subsequent rapidly progressive loss of motor and language function.
Breakthrough Therapy With Proven Efficacy
- Mechanism & Benefits:
- Brineura delivers tripeptidyl peptidase-1 (TPP1) directly to the brain via intraventricular infusion, breaking down toxic lipofuscin deposits that cause neurodegeneration.
- Clinical trials showed it preserves motor function, with treated children maintaining walking ability for ~2 years longer than untreated peers.
- Real-world data from patients like 9-year-old Charleigh Pollock in Canada reveal stabilized seizures and improved quality of life, though the drug is not a cure.
- Global Adoption & Sales:
- Approved in the US (2017), EU (2017), and Canada (2018), Brineura is projected to generate $358 million in global sales by 2025.
- BioMarin’s patented delivery system requires specialized administration, limiting rapid market uptake.
Funding Battles Highlight Systemic Gaps
- Case Study: Charleigh Pollock
- British Columbia’s health ministry initially withdrew funding for Charleigh’s $1M/year treatment, citing outdated clinical criteria, but extended coverage through June 2025 after public outcry.
- Her family argues that real-world outcomes—like seizure reduction—should override rigid trial benchmarks.
- Broader Challenges:
- Cost: At ~$1M annually, payer resistance persists despite NICE’s 2020 recommendation for UK coverage.
- Access: Only ~20 CLN2 patients exist in Canada, complicating cost-benefit analyses.
Safety & Future Directions
- Risks: Device-related infections (e.g., meningitis), seizures, and hypersensitivity reactions require close monitoring.
- Innovations: BioMarin is exploring next-gen delivery systems to reduce infusion complications.
Expert Voices
Dr. Ineka Whiteman (Batten Disease Research Association)
“Brineura is transformative—it buys time for children who once had none. But equitable access demands updated reimbursement frameworks.
Brineura Looking Ahead
BioMarin plans to submit long-term survival data to regulators in 2026, potentially expanding eligibility.
However, HK DengYue’s efforts to increase the affordability of the medicine are critical to ensuring that more patients benefit from it.
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