Rare disease drugs can cost as much as $400,000 a year, and those hefty price tags have sparked a lively debate about the cost of treating rare diseases over the long term.
Some orphan drugs to treat rare diseases can cost as much as $400,000 a year. Those hefty price tags have sparked a lively debate in recent years about whether the American health care system can shoulder the burden of treating rare diseases over the long term.
Edison Pharmaceuticals' in-development rare disease treatment missed its primary endpoint in a mid-stage trial, denting the company's plans for an expansive take on CNS R&D.
Amicus Therapeutics has come a long way in two years, shaking off a clinical misstep on the way to a second Phase III success for its rare disease treatment. Now the biotech is preparing to make its case to regulators, hoping to win approval for a new approach to Fabry 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.
When AbbVie buys out Shire for nearly $55 billion in a deal the two companies agreed on last week, there will be no golden parachute awaiting Shire chief Flemming Ornskov. Instead, there's a signing bonus in order: The helmsman will pocket just under $10 million for staying on with the combined company in a new role.
Shire has signed a $225 million agreement to brighten its future in rare diseases, bolstering a major selling point for AbbVie as the two prepare for a $54.7 billion merger.
Startup Neurotrope is teaming up with the Icahn School of Medicine at Mount Sinai in New York City to further develop its bryostatins for the treatment of Niemann-Pick disease type C, a rare disease that mostly affects children.
After months of semi-clandestine courtship, AbbVie and Shire have settled on a price: For $54.7 billion in cash and stock, the U.S. drugmaker will absorb its Irish target, securing a pipeline of promising rare disease treatments and a new address that should slash its tax rate.
More orphan drugs are hitting the market than ever before. But as their numbers increase, their prices do, too. That makes them harder for patients to access--and pharma companies may have to do something about it, if they don't want to back themselves into a corner.