Amgen Seeks Expanded US Approval For Autoimmune Disease Drug Acquired Via $28B Horizon Deal

On Wednesday, Amgen Inc AMGN announced topline results from its Phase 3 trial evaluating the efficacy and safety of Uplizna (inebilizumab-cdon) for Immunoglobulin G4-related disease, a systemic autoimmune fibroinflammatory multiorgan disease, usually involving the pancreas.

Amgen added Uplizna via a $27.8 billion acquisition of Horizon Therapeutics and recorded sales of $80 million in the first quarter of 2024.

The trial met its primary endpoint, showing a statistically significant 87% reduction in the risk of IgG4-RD flare compared to placebo (Hazard Ratio 0.13, p<0.0001) during the 52-week placebo-controlled period.

All key secondary endpoints were also met, which were annualized flare rate, flare-free, treatment-free complete remission, and flare-free, corticosteroid-free complete remission.

No new safety signals were identified. The overall safety results during the placebo-controlled trial period were consistent with Uplizna’s known safety profile.

MITIGATE was conducted at 80 sites in 22 countries. It is the first placebo-controlled trial providing class 1 evidence for treating IgG4-RD, a chronic, systemic, immune-mediated, fibroinflammatory disease that can affect almost any organ in the body, often involving multiple organs at a time.

The novel, steroid-sparing study design paves the way for a reduced toxicity treatment approach.

Uplizna is currently approved for Neuromyelitis Optica Spectrum Disorder (NMOSD) by several regulatory bodies, including the FDA, the European Medicines Agency, Health Canada, and the Brazilian Health Regulatory Agency (ANVISA).

NMOSD is an inflammatory and demyelinating disorder of the central nervous system. NMOSD primarily targets the optic nerves, brainstem, and spinal cord.

Based on the MITIGATE primary analysis results, Amgen plans to file for approval in the U.S., followed by other key markets.

The trial was conducted with the support of Mitsubishi Tanabe Pharma and Hansoh Pharma.

Mitsubishi Tanabe Pharma holds marketing authorization for Uplizna in Japan, Thailand, South Korea, Indonesia, Vietnam, Malaysia, Philippines, Singapore, and Taiwan.

Hansoh Pharma is the exclusive licensee, local regulatory, and commercial agent for China’s mainland, Hong Kong, and Macau.

Read Next: Amgen Secures FDA Nod For Rare-Disease Treatment Biosimilar Based On AstraZeneca’s Drug.

Price Action: AMGN shares are up 0.02% at $307.38 at the last check on Wednesday.

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