Here's why Big Pharma wants to be in rare diseases--and why Sanofi made its $20.1 billion play for Genzyme.
More than a couple of markets have been shaken up lately by new competition in oral form. And now, Sanofi's Genzyme is wondering if it can't make a splash of its own with Cerdelga, its newly approved pill for Gaucher disease.
Sanofi's rare ailment-focused Genzyme unit won FDA approval for a new Gaucher disease treatment, providing an oral alternative to its own trailblazing intravenous therapy.
Sanofi's Genzyme snagged a 6-month priority review from the FDA for what could become the first oral treatment for Gaucher disease, a rare genetic disorder that causes abnormal fat buildup in cells, enlarging organs in the body.
urrent therapies for Gaucher rely on an injected enzyme replacement approach, but Sanofi's oral strategy for eliglustat could seriously undercut those drugs.
With pharma interest in rare-disease drugs becoming quite common, Protalix BioTherapeutics, maker of a rare disease drug partnered with Pfizer ($PFE), said today that it has hired Citigroup to help review "a broad array of product partnering, technology sharing and other strategic alternatives" for the Israel-based biotech company.
Pfizer ($PFE) and Protalix ($PLX) are near a coup for their new Gaucher's disease drug Elelyso. According to Globes, the Israel-based company and its behemoth partner are negotiating a supply deal with the Brazilian government. If the deal is signed within a few days, as Globes ' sources say, it would be worth "hundreds of millions" to the companies.
The company announced trial results showing its Gaucher disease drug Vpriv improved bone density in patients' spines, but Sanofi's Cerezyme didn't.
Just weeks after winning U.S. approval for their new Gaucher drug taliglucerase alfa, the EMA's Committee for Medicinal Products for Human Use says Pfizer and Protalix BioTherapeutics should be shut out of the EU in order to honor Shire's exclusive status for its currently used Vpriv.
Pfizer followed GlaxoSmithKline and other pharma companies that journeyed into the rare disease arena, paying $60 million upfront to partner with Protalix and agreeing to pay out up to $55 million in milestones.