Amgen ($AMGN) has taken another big step in its head-to-head race with Sanofi ($SNY) and Regeneron ($REGN), filing its application for the prospective cholesterol blockbuster evolocumab and backing it up with more positive data from the latest in a long string of major clinical studies.
Altogether the biologics application comes with safety and efficacy results covering 6,800 patients who were enrolled in 10 late-stage trials. Investigators have consistently racked up evidence that the drug can lower levels of bad cholesterol in an impressive way for patients who cannot tolerate statins, patients with heterozygous familial hypercholesterolemia, and patients with homozygous familial hypercholesterolemia, a rare genetic disorder.
Until recently, Amgen appeared to be in the lead in the race for the first PCSK9 approval. Then Regeneron and its Big Pharma partner Sanofi struck back in late July, rolling out a stack of positive data from its own slate of Phase III studies. Just hours later, the partners issued a surprise announcement that they had nabbed a priority review voucher that BioMarin had won for one of its in-house programs, cutting 4 months off of what is routinely a 10-month process. The partners paid $67 million for that voucher.
Regeneron and Sanofi are expected to file alirocumab in the fourth quarter, which is coming up fast.
Amgen, meanwhile, also posted top-line results for its Phase III study in Japan, hitting the co-primary endpoints for lowering bad cholesterol among the 404 patients in the study. All the leaders in the field are planning a global rollout, with multiple applications in markets around the world. The U.S., though, represents their biggest single market opportunity.
"This BLA submission to the FDA marks the first of several submissions to regulatory authorities around the world for our lipid-lowering program and represents a critical milestone in our overall global development program for evolocumab," said Dr. Sean Harper, executive vice president of R&D at Amgen. "We look forward to working closely withregulatory authorities to bring this new treatment option to patients with high cholesterol who, despite currently available therapies, are unable to adequately reduce their LDL cholesterol levels."
- here's the release on the application
- here's the release on the study in Japan