RARE Daily

Regeneron Pauses Trial of Experimental FOP Therapy After Patient Deaths

November 2, 2020

Rare Daily Staff

Regeneron has paused dosing of its experimental therapy garetosmab in the ongoing phase 2 LUMINA-1 trial in patients with the ultra-rare genetic disorder fibrodysplasia ossificans progressiva.

The decision to pause dosing was based on reports of fatal serious adverse events during the open-label extension, in which all patients received active treatment. These deaths are being investigated to understand if they are related to garetosmab treatment.

Fibrodysplasia ossificans progressiva (FOP) is a progressive, ultra-rare genetic disorder in which muscles, tendons and ligaments are progressively replaced by bone, a process known as heterotopic ossification (HO). There are believed to be approximately 900 patients diagnosed with FOP worldwide. HO of the jaw, spine, hip and rib cage can make it difficult to speak, eat, walk or breathe, leading to weight loss and escalating loss of mobility and skeletal deformity.

Most people with FOP are wheelchair bound by 30 years old and the median age of survival is approximately 56 years. Death often results from complications, such as pneumonia, heart failure and aspiration stemming from HO and loss of mobility in the chest, neck, and jaw.

Regeneron shared its update with the trial’s Independent Data Monitoring Committee and regulatory authorities, and will conduct a review of the trial data to date, to better understand the benefit/risk profile of garetosmab in people with FOP.

There were no deaths in the trial during the 28-week double-blind treatment period, results of which were reported in early 2020 that showed garetosmab failed to meet the primary endpoint of reducing total lesion activity. However, Regeneron said that FOP patients in the trial saw an approximate 25 percent decrease in total bone lesions (both new and existing) driven by a nearly 90 percent reduction in formation of new lesions.


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