A PRIME designation is given to support the development of investigational treatments that address unmet medical needs. To be eligible for this status, experimental medications must demonstrate, based on early clinical data, that they have the potential to offer major therapeutic advantages over existing therapies, or benefit patients who have no other treatment options.
The designation also comes with certain benefits for the therapy’s developer, including early and enhanced interactions with the EMA, as well as the possibility that the potential treatment is given accelerated assessment, shortening its review process.
“We are very pleased that the EMA has recognized the potential for brensocatib to offer an entirely new treatment approach for NCFBE, a severe and chronic disease with significant unmet needs,” Martina Flammer, MD, chief medical officer of Insmed, said in a press release.
Brensocatib is an oral, reversible inhibitor of dipeptidyl peptidase 1 (DPP1), a protein that activates neutrophils, which in turn release enzymes that end up damaging the lungs of people with NCFBE. By blocking the activity of DPP1, brensocatib is expected to prevent these specialized immune cells from becoming activated and releasing these harmful enzymes.
The EMA’s decision to grant PRIME to brensocatib was based on data from the Phase 2 WILLOW trial (NCT03218917), which showed that the therapy was able to lower the risk and rate of disease flare-ups in adults with NCFBE, regardless of their age or past clinical history.
The company is now planning to launch a registrational Phase 3 trial, called ASPEN (NCT04594369), before the end of the year. That trial will assess if brensocatib may be superior to a placebo — when given once daily at a dose of 10 or 25 mg — at reducing the rate of disease exacerbations over one year in adults with NCFBE.
If positive, data from this trial are expected to support the therapy’s approval for the treatment of those with this type of bronchiectasis.
“We are building on the strength of the Phase 2 WILLOW study with the initiation of a pivotal Phase 3 program for brensocatib that we hope will bring forth this urgently needed solution,” Flammer said.
ASPEN, which is expected to enroll 1,620 patients at approximately 480 sites across 36 countries, is not yet recruiting participants. More information about trial contacts and recruiting sites will be available here.