Amgen's drug meets main goal in late-stage study for rare disease
By Sriparna Roy
(Reuters) -Amgen said on Wednesday its drug helped to reduce the risk of flares in patients with an immune system-related condition, meeting the main goal of a late-stage study.
The drug, Uplizna, was being studied for the treatment of Immunoglobulin G4-related disease (IgG4-RD), which is characterized by periods of remission and unpredictable disease flares.
The condition can affect almost any organ in the body, often involving multiple organs at a time, and can result in irreversible organ damage.
The company said the drug showed a statistically significant 87% reduction in the risk of flares, compared to placebo.
There are currently no therapies approved for the condition, which is usually managed with steroids such as glucocorticoids.
It is estimated to affect one to five patients in 100,000, although the number of IgG4-RD patients is difficult to determine based on limited epidemiology data, the company said.
The late-stage trial, which studied the safety and efficacy of the drug in reducing the risk of flares, enrolled 135 adults with IgG4-RD, who received 300 milligram intravenous dose of the drug or placebo.
Amgen had gained access to the drug through its $27.8 billion acquisition of Horizon Therapeutics, which closed last year, and recorded sales of $80 million from it in the first quarter.
Full data from the trial will be presented at a future medical meeting, Amgen said.
Based on the results, the drugmaker is planning to file for an approval for the condition in the United States.
Approval in IgG4-RD could represent a significant market expansion from Uplizna's only approved indication, said Leerink Partners analyst David Risinger.
Uplizna is currently approved in the U.S., Europe and Canada, among others, for a rare, severe, neuroinflammatory disease called neuromyelitis optica spectrum disorder, which attacks the optic nerve spinal cord and brain stem.
(Reporting by Sriparna Roy in Bengaluru; Editing by Shilpi Majumdar)
