Roche gets a 'breakthrough' at the FDA for a top PhIII development effort

Roche CMO Sandra Horning

Roche ($RHHBY) got a boost for one of its top late-stage prospects from the FDA today. The agency has handed out a breakthrough drug designation for its hemophilia A treatment ACE910 among patients with factor VIII inhibitors.

A couple of months ago, investigators for Roche had heralded long-term follow-up results from ACE910's Phase I study, showing its success in controlling needing episodes among hemophilia patients with or without Factor VIII inhibitors. About one in four patients develop the inhibitors, according to Roche, making the disease particularly hard to control.

"People with hemophilia A may require regular and frequent infusions of replacement clotting factor to reduce the risk of dangerous bleeding, and they can develop inhibitors that make replacement ineffective," said Roche Chief Medical Officer Sandra Horning in a statement.

The antibody--an anti-factor IXa/X bispecific which mimics the function of FVIII, even in the presence of FVIII inhibitors--was originally developed at Roche subsidiary Chugai.

Over the last couple of years the FDA has handed out dozens of BTD designations, promising to make regulators more easily available to speed through the development process. The program has helped change the structure and timeline of cancer drugs, in particular. And Roche has already laid out plans to start a Phase III study for this treatment among patients with inhibitors before the end of the year, with another late stage study for patients without inhibitors launching next year.

Roche pharma chief Daniel O'Day has repeatedly tapped ACE910 as a top prospect for the company, after its lead effort on the PD-L1 checkpoint inhibitor atezolizumab. Other top contenders are ocrelizumab for MS, lebrikizumab for asthma and crenezumab, an experimental Alzheimer's drug partnered with AC Immune. Roche isn't letting go of its failed Alzheimer's drug gantenerumab, but it also hasn't outlined a new Phase III effort. And the pharma giant has sided with the more cautious wing of the pharma industry, which believes that the current prices for experimental products are too steep to encourage dealmaking.

- here's the release

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