Sanofi ($SNY) and Regeneron ($REGN) just stole a march on Amgen ($AMGN) in the race to get a PCSK9 cardio drug through the FDA and onto the market. The two companies revealed Wednesday evening that they had picked up a priority review voucher BioMarin ($BMRN) had won for a recent rare disease drug approval, paying $67.5 million for the regulatory shortcut. They'll split the cost and share in the benefit, shaving four months off the regulatory review time for alirocumab.
The deal offers Sanofi and Renegeron a shortcut at the FDA that cuts the regulatory review period from 10 months down to 6 months. That could make a big difference, given that Amgen is looking to file evolocumab in the third quarter, with Sanofi and Regeneron now looking toward the fourth quarter.
Earlier today, Sanofi and Regeneron offered late-stage data from a slew of Phase III studies for their drug. The data created a foundation for a regulatory submission at the FDA, while suggesting that the therapy may also ultimately help prevent cardiovascular events such as heart attacks and strokes. Clear evidence of cardiovascular benefits, however, will take considerably longer to nail down as investigators focus for now on cholesterol. Amgen, meanwhile, has been racking up one Phase III success after the next on evolocumab, a PCSK9 blocker that has been clearing LDL cholesterol from the blood.
If both drugs are approved, you can expect to see a big marketing showdown among a large patient group that includes people at risk of a cardio event, especially those patients whose cholesterol levels remain stubbornly resistant to the cheap, commercially available therapies already on the market.
EvaluatePharma expects this to pan out as a near draw on the sales side between Amgen and Sanofi, which in particular needs to see some new approvals after experiencing setbacks for its own in-house pipeline work. Both PCSK9 drugs are expected to earn a little more than a billion dollars a year, putting them in the blockbuster club, though some analysts have speculated that peak sales could go much higher.
Technically, a subsidiary, Regeneron Ireland, is buying the voucher from BioMarin, which obtained it through the FDA's Rare Pediatric Disease Priority Review Voucher Program after winning an OK for Vimizim, a new biological product for patients with Mucopolysaccharidosis type IVA, also known as Morquio A syndrome. BioMarin CEO Jean-Jacques Bienaimé says he expects to use the cash to reinvest in rare disease drugs.
Depending on who you talk to, this could be a $10 billion drug class in its own right. But the FDA has made it abundantly clear that it expects to see a mountain of human data on efficacy and safety before it agrees to provide an OK for marketing the therapy to a large audience. Pfizer, meanwhile, is bringing up the rear with a trademark case of slow-motion R&D syndrome.
One potential stumbling block: neurocognitive impairment. Investors experienced a bad case of vertigo after Sanofi recently revealed that the FDA wants everyone in the field to track cases of "fuzzy thinking" among patients. Cholesterol drugs have been linked to confusion before, but generally it's been quickly treated by getting the patients off the drug.
"The priority review voucher program was established to provide incentives that would enhance innovation in key areas of unmet patient need," said Dr. Ned Braunstein, vice president of regulatory affairs at Regeneron. "Our decision to acquire and leverage the voucher is clear evidence that this program is a valuable incentive for biopharmaceutical companies."
- here's the release