FDA accepts BLA for review of what could be first new antiretroviral in 10 years
The US Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for ibalizumab as a treatment for multidrug resistant human immunodeficiency virus-1 (MDR HIV-1).
If approved, ibalizumab will be the first antiretroviral treatment (ART) with a new mechanism of action to be introduced in nearly 10 years and the only treatment that does not require daily dosing.Ibalizumab is the first-ever antibody HIV treatment, according to Canadian biotech firm Theratechnologies (TSX: TH), which has licensed right to the drug in the USA and Canada from Taiwan-based TaiMed Biologicals (4147: TT).
Its shares advanced 4.4% to C$8.58 on the news by close of trading Friday, but many investors and traders were already heading off for a long ‘Canada Day’ weekend holiday, so reaction was mute.
"We are excited to be one step closer to potentially bringing an important new treatment, with a new mechanism of action, to patients whose virus has become resistant to therapies in multiple classes and have limited treatment options for the long-term management of their condition," said Luc Tanguay, president and chief executive of Theratechnologies. “The granting of Priority Review status is important since it confirms that, if approved, ibalizumab would represent a significant improvement in the treatment of this serious condition,” added Mr Tanguay.
January 2018 PDUFA date set
The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of January 3, 2018, for the ibalizumab application. Priority Review status accelerates FDA review time from 10 months to a goal of six months from the day of acceptance.In addition, ibalizumab received Breakthrough Therapy designation from the FDA in 2015, which is given if a therapy may provide a substantial improvement over what is currently available to address a serious and life-threatening condition. The FDA also granted Orphan Drug designation in 2014.
The BLA, submitted on May 3, 2017, is based on data from the Phase III TMB-301 study, a single arm, 24-week study of ibalizumab plus an optimized background regimen (OBR) in treatment-experienced patients who had high pre-existing levels of drug resistance and advanced clinical disease.
The ibalizumab Expanded Access Program (EAP), or study TMB-311, is ongoing and enrolling patients. The drug has already gained Breakthrough Therapy and orphan designations from the FDA.
As HIV multiplies in the body, the virus may mutate to produce drug-resistant strains. Viral mutations may mean that HIV medicines that previously controlled a person's virus are no longer effective, causing treatment to fail. There are approximately 20,000 to 25,000 Americans with HIV-1 that are resistant to at least one drug out of the three different classes of antiretroviral therapies. Up to 12,000 of these patients experience a virological failure over a period of 48 weeks of treatment, requiring their physician to modify their treatment.