Key Highlights:
- AGAMREE® (vamorolone) has been recommended by NICE for use in the UK’s National Health Service (NHS) for Duchenne muscular dystrophy (DMD) patients aged 4 and older.
- Santhera has begun launch preparations in the UK, with first sales expected this quarter.
- AGAMREE is the first and only fully approved DMD treatment in the EU, US, and UK, offering a clinically validated alternative to standard corticosteroids.
- The drug’s unique mechanism of action provides anti-inflammatory benefits while maintaining bone health, density, and normal growth.
Santhera Pharmaceuticals (SIX: SANN) has received a major regulatory milestone as the National Institute for Health and Care Excellence (NICE) has issued positive Final Guidance for AGAMREE® (vamorolone). This recommendation paves the way for AGAMREE’s availability in the National Health Service (NHS) across England, Wales, and Northern Ireland, offering a transformative treatment option for patients with Duchenne muscular dystrophy (DMD).
This decision follows the December 10, 2024, Final Draft Guidance (FDG) announcement, during which no appeals were filed. As a result, Santhera has expedited launch preparations, ensuring that AGAMREE will reach patients in the UK within the current quarter.
Why This Matters for DMD Patients
Duchenne muscular dystrophy (DMD) is a severe and progressive genetic disorder characterized by muscle degeneration, loss of mobility, and life-threatening complications. Historically, corticosteroids have been the standard of care; however, their long-term use is associated with severe side effects, including growth suppression and bone density loss.
AGAMREE® is a next-generation, dissociative steroid therapy that maintains efficacy while reducing common steroid-related side effects. The European Medicines Agency (EMA) and the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) have validated its clinical benefits, particularly in preserving bone metabolism and normal growth patterns.
How AGAMREE® Works: A Breakthrough in DMD Therapy
Unlike traditional corticosteroids, AGAMREE® works by binding to glucocorticoid receptors while modifying downstream activity, leading to Potent anti-inflammatory effects without the amplified local tissue toxicity seen with corticosteroids, No significant impact on growth or bone metabolism, addressing key safety concerns of long-term steroid use, Improved tolerability in children and adolescents, making it a viable alternative to corticosteroids and AGAMREE® has been designed as a dissociative anti-inflammatory drug, offering a novel, safer approach to managing DMD.
The VISION-DMD study, which served as the basis for regulatory approvals in the UK, EU, and US, demonstrated Significant improvement in Time to Stand (TTSTAND) velocity compared to placebo (p=0.002) at 24 weeks, A favorable safety profile, with mild to moderate side effects such as weight gain, irritability, and vomiting and No observed negative impact on bone health or growth, a stark contrast to conventional corticosteroids.
Furthermore, data presented at major scientific conferences (WMS 2022, MDA 2022) confirmed that AGAMREE preserves bone integrity while effectively managing DMD symptoms.
Santhera’s Global Expansion & Market Strategy
AGAMREE® has received full approval in the US (FDA), EU (EMA), UK (MHRA), China (NMPA), and Hong Kong (DoH). To maximize patient access, Santhera has strategically out-licensed commercialization rights, North America, Partnered with Catalyst Pharmaceuticals, China & Southeast Asia, Licensed to Sperogenix Therapeutics.
The company is actively working on expanding market access while ensuring a swift commercial launch in the UK, reinforcing its commitment to neuromuscular disease patients worldwide.
What Healthcare Professionals Should Know
AGAMREE® is subject to additional safety monitoring, allowing for quick identification of new safety insights. Healthcare providers are encouraged to report any suspected adverse reactions to facilitate ongoing safety evaluation.
For prescribing details in Great Britain (including Scotland), refer to the Summary of Product Characteristics on the Santhera website. The NICE approval of AGAMREE® represents a significant step forward in the treatment of DMD, providing an innovative alternative to corticosteroids with a superior safety profile.
Santhera’s commitment to rare neuromuscular diseases positions it as a leader in the specialty pharmaceutical space. The company remains focused on driving innovation, expanding market access, and ensuring AGAMREE’s successful adoption across global healthcare systems.
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