rare diseases

Latest Headlines

Latest Headlines

Edison's orphan drug misses the mark in Phase II

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 sets sights on approval as rare disease pill clears Phase III

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 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.

$9.9M 'golden hello' awaits Shire chief Ornskov at new AbbVie post

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 inks a $225M rare disease deal on the eve of life with AbbVie

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.

Mount Sinai, Neurotrope partner to develop bryostatins for rare metabolic disease

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.

AbbVie and Shire come to terms on a $55B union

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.

Orphan drug approvals are up, but prices are up more

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.

Jazz gambles up to $250M on a long-delayed rare-disease drug

When Jazz Pharmaceuticals bought out the rare-disease drug developer Gentium for a billion dollars late last year, the company picked up a new therapy for severe veno-occlusive disease that it went on to launch in Europe last April. Now it's buying back the U.S. rights to the drug--which Gentium had bargained away to Sigma-Tau--for $75 million in cash and up to $175 million in milestones. And that extra cash will be staked entirely on its ability to shove the long-delayed drug through the FDA approval process.

FDA smacks Mexican facility that makes scorpion antivenom for U.S.

The FDA has found problems at the Mexican plant where Anascorp, the only scorpion antivenom available in the U.S., is manufactured, listing more than a dozen observations in a recent warning letter.