Senators advocate blockbuster remedy for rare drug woes

Lawmakers will soon get a chance to start pondering a bill that would dangle some blockbuster cash as an incentive to developers to take on the risk of developing new therapies for rare children's diseases. The new law, which was proffered last week, would give pharma companies a shot at quicker approvals of blockbusters if they take on new drug programs that tackle rare pediatric diseases. Adding even a few months to a drugmaker's window of market opportunity could add hundreds of millions of...

Feds reviewing new R&D incentives for rare diseases

Charged with spurring greater R&D efforts on new drugs for rare diseases, the FDA's newly formed rare disease review group plans to meet today and tomorrow, when it will hear about a host of new ideas on a richer set of research incentives. Earlier this year the FDA and the NIH announced one initiative aimed at encouraging early studies on new therapies for rare diseases, which has been the subject of growing industry interest with the launch of special units devoted to the field at...

EXCLUSIVE: Pfizer plans to move fast on rare disease pacts

Pfizer went public with its new rare disease unit earlier this week, but one of its top execs has been working away for the past few months setting up a division that is preparing to team up with a range of biotechs on new therapeutics. "This morning at 7 a.m. I was at breakfast, talking to a company," Jose Carlos Gutierrez-Ramos, Pfizer's Cambridge-based senior vice president for biotherapeutics research and development, tells FierceBiotech. "We are actively talking to people and have been for...

Pfizer creates a new R&D unit for rare diseases

Following in the footsteps of GlaxoSmithKline, Pfizer has set up a new research unit that will be devoted to developing new biologics to treat rare diseases. And the global pharma company has put Edward Mascioli, M.D., the founder and CEO of life sciences private equity firm Dapis Capital, in charge. The Rare Diseases Research Unit will be based in Cambridge, MA, home to one of the world's largest clusters of biotech companies. The new unit has been given a broad mission: "pursue treatments...

Sarcoma Foundation calls for radical change in FDA review process

Mark Thornton, a former medical officer at the FDA and current president of the Sarcoma Foundation of America, uses the recent Provenge approval to illustrate why the FDA needs to reverse its position on the cancer drug Mepact. Back in 2007 the FDA decided that both Provenge and Mepact should go back to the clinic for a new study. Dendreon was able to afford that because it was pursuing a mega-blockbuster approval for prostate cancer. But the developers of Mepact were essentially...