Tag:

rare diseases

Latest Headlines

Latest Headlines

UPDATED: Biotech upstart Sage sees early efficacy for rare brain seizure drug

Sage Therapeutics has successfully resolved a rare seizure disorder among the majority of patients in its small but closely watched Phase I/II study, adding more promising outcomes to the early evidence that helped inspire one of this year's most successful biotech IPOs. In the data update out Monday morning investigators say the 73% overall response rate in their study is also being mirrored among a small group of patients who are receiving the drug on an emergency-use basis.

Survey: No need to worry about sky-high prices for orphan drugs

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.

Orphan drugs may be pricey, but payers are barely balking

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'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.