Gaucher disease

Latest Headlines

Latest Headlines

Sanofi nears European Gaucher market shakeup with EMA recommendation

In August, Sanofi's Genzyme won the FDA's blessing to take brand-new orphan drug Cerdelga into the U.S. Gaucher disease market, where the company hoped the pill could show up older, intravenous treatments. And now, it could be on its way to the same opportunity in the EU.

What price for an oral Gaucher drug? $310,250, says Genzyme

Here's why Big Pharma wants to be in rare diseases--and why Sanofi made its $20.1 billion play for Genzyme.

Price could be key for Sanofi's new Gaucher pill as it sets out against IV rivals

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 wins FDA's blessing for an oral Gaucher drug

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 nets FDA priority review for its oral Gaucher pill

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.

Sanofi reports positive PhIII data on oral Gaucher drug

urrent therapies for Gaucher rely on an injected enzyme replacement approach, but Sanofi's oral strategy for eliglustat could seriously undercut those drugs.

Pfizer partner Protalix soars on sale speculation

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, Protalix near Elelyso supply deal in Brazil

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.

Shire's Gaucher drug beats Cerezyme in bone study

The company announced trial results showing its Gaucher disease drug Vpriv improved bone density in patients' spines, but Sanofi's Cerezyme didn't.

EU regulators shut out new Gaucher drug from Pfizer, Protalix

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.