Brain cancer killed Nancy Goodman’s 10-year-old son Jacob, but no one was happier than Goodman when Regeneron Pharmaceuticals Inc. and Sanofi SA asked the Food and Drug Administration for speedy approval of a cholesterol-fighting drug.
Here’s why: Goodman, a lawyer by training, worked the halls of Congress following Jacob’s death in January 2009 to create a voucher program, aimed at spurring development of drugs aimed at rare childhood diseases.
BioMarin Pharmaceutical Inc. received the program’s first voucher after it won FDA approval in February for Vimizim, a drug for a rare and crippling childhood genetic condition known as Morquio A syndrome. Then last month the San Rafael company (NASDAQ: BMRN) became the first to sell a voucher, to Regeneron (NASDAQ: REGN) and Sanofi (NASDAQ: SNY) for $67.5 million.
That kind of price — fed by Regeneron’s and Sanofi’s desire to leapfrog Amgen Inc.’s (NASDAQ: AMGN) evolocumab in the race to bring a new type of cholesterol-lowering drug to market faster — is the incentive Goodman had in mind when she spearheaded creation of the voucher.
The voucher could cut four to six months off the typical 10-month FDA review of Regeneron and partner Sanofi’s drug, called alirocumab.
“What I told everyone would happen — it happened,” said Goodman, whose efforts fell under the banner of a Washington, D.C., nonprofit she created, called Kids v Cancer.
Another voucher program to promote development of tropical diseases, such as malaria and Chagas disease, had been in place for years, but it had shortcomings. For one, drug developers racked up more costs by creating clinical trials for the FDA as well as the countries with those tropical diseases, and a company had to give a year’s notice before exercising a voucher.
In all, the factors conspired to make the value of a tropical disease voucher practically nil, Goodman said.
That clearly was not the value of BioMarin’s voucher. The BioMarin voucher is more lucrative because voucher users only have to give 90-days notice before the voucher is exercised, so it comes at the tail end of an drug-approval submission. What’s more, there’s no limit on how many times a voucher can be transferred or sold.
“Nobody suffers. There’s nobody that’s paying for this,” Goodman said. “Sanofi and Regeneron get to market faster, BioMarin makes money and patients get a choice.”
So what, she said, if the drugs developed by BioMarin or Regeneron and Sanofi don’t treat the pediatric brain cancer that killed Jacob.
“Any sick kid is a sick kid to me,” she said.