- Roche may have hit a setback in an attempt to penetrate the nearly $10 billion hemophilia market — one currently dominated by Danish drugmaker Novo Nordisk.
- The Swiss drugmaker has reported positive results so far for emicizumab, also known as ACE910, threatening the long-standing market leader from Novo Nordisk NovoSeven.
- Yet, reports surfaced in Reuters this week noting that there have been some serious adverse events (SAEs) in the ongoing clinical trial of the drug that could impact its safety profile.
Roche confirmed in an email there had been four spontaneous SAEs during the trial, which is expected to report out by the end of the year.
The company said the safety issues "were seen with the concomitant use of multiple doses of a bypassing agent with emicizumab while treating a breakthrough bleed; in some cases the bypassing agent at doses exceeding the recommended labeled doses."
Roche has put a risk mitigation strategy in place and expects to reveal all of the data at the end of the year when it will evaluate the overall risk-benefit profile of the drug.
"The current recommendation is to avoid the use of one specific bypassing agent and if necessary, to use it starting with the lower range of the recommended labeled dose and perform dosing under monitoring. Importantly, all of these adverse events have resolved and two of the patients have since restarted emicizumab and have not experienced a recurrence of the adverse event to date," Roche said.
Jefferies analyst Jeffrey Holford wrote in a November 2 note to investors that the readout of the Phase 3 Roche results of emicizumab could jeopardize up to 50% of NovoSeven's revenues.
Hemophilia, while only serving a small patient population, is a chronic disease that needs constant monitoring and life-long medication. Patients with the bleeding disorder are often reluctant to switch therapies and risk a breakthrough bleeding episode.
Yet, technological advances in the space in recent years are making it a much more valuable and crowded market. Companies are developing long-acting treatments that allow patients to administer infusions every three to five days instead of daily, and some companies in the space are even working on gene therapies that could mean annual treatments.