Tag:

rare diseases

Latest Headlines

Latest Headlines

$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.

BioMarin's $380K price tag on Vimizim is high, but far from the highest

BioMarin has slapped a price tag on its new drug Vimizim: $380,000 a year. That's hefty even for a rare disease treatment. But even at such nosebleed rates, Vimizim ranks in just third place on the list of the world's most expensive drugs.

Rare-disease powerhouse Alexion grabs an option on muscular dystrophy buyout

The biotech has struck a deal with little Prothelia and the University of Nevada, Reno, where it will step in and conduct development research on a protein replacement therapy for merosin-deficient congenital muscular dystrophy, or MDC1A.

Jazz will pay $1B for Italy's Gentium, sweeping up another specialty drugmaker

Jazz Pharmaceuticals, the Ireland-based company known for its narcolepsy drug Xyrem, is often the subject of takeover talk. Because of its specialty drug portfolio and low-tax domicile, analysts often see it as a potential target. But the drugmaker has kept up a steady drumbeat of mostly smaller deals itself to expand its line of specialty drugs. Today it jumped the $1 billion deal fence with plans to buy Italy-base, Gentium and get its EU-approved orphan drug Defitelio.