Drugs in the new PCSK9 inhibitor class of cholesterol-lowering drugs with blockbuster sales potential could be approved on both sides of the Atlantic within weeks.
Later this week, Sanofi and Regeneron’s Praluent (alirocumab) will come in front of an FDA advisory committee, armed with an FDA reviewer document, which states it lowers low-density lipoprotein cholesterol (LDL-c) and is well tolerated.
Meanwhile, rival Repatha (evolocumab) from Amgen already has a recommendation for approval in the EU and is on track for a green light from the EMA in a few weeks’ time, and will also be reviewed by the FDA panel this week.
Both drugs have been developed to lower high levels of cholesterol in the blood of patients in whom statins are ineffective or for those cannot take statins.
The big question how the panel will respond to the FDA reviewer’s key question: given that LDL-c is a surrogate marker for efficacy and statins are already effective and widely used – should a new class be approved without cardiovascular outcomes data?
Analysts believe that despite the discussion on outcomes, the panel will likely give the go-ahead to both drugs, particularly as they have proved to be effective in statin-intolerant or resistant patients.
Moreover, both Sanofi/Regeneron and Amgen already have large-scale cardiovascular outcomes trials in hand, with data coming within the next couple of years, so panellists should be comfortable recommending approval in the interim.
Amgen said late last week that it has completed enrolment in its 27,500-patient FOURIER trial, which is seeing if adding evolocumab to a statin can reduce the risk of recurrent cardiovascular events in patients with high cholesterol and clinically evident cardiovascular disease. Data are due in 2017, according to Amgen.
Meanwhile, Sanofi and Regeneron are carrying out the ODYSSEY OUTCOMES trial in patients with acute coronary syndrome (ACS) receiving “evidence-based medical therapy and dietary management” for high cholesterol, which in most cases will be statins. The primary outcome measure is a composite of coronary heart disease death, nonfatal heart attack, fatal and nonfatal ischaemic stroke and unstable angina requiring hospitalization. Results are expected in 2018.
GlobalData recently said the PCSK9 inhibitor class – which also includes a late stage candidates from Pfizer – could capture a 40% share of the $17.5bn acute coronary syndromes market by 2023.
The FDA is due to deliver a verdict on Praluent in July and Repatha in August, with Sanofi clawing back Amgen’s earlier lead by purchasing a priority review voucher from BioMarin last year.
The rivalry has also spilled into the courts, with Amgen filing a patent infringement lawsuit against Sanofi and Regeneron last October.