Tag:
orphan drug status
Latest Headlines
Latest Headlines
Raptor's orphan drug-win at FDA overshadowed by debate over steep price
Raptor Pharmaceutical has scored an FDA approval of its new drug for a rare metabolic disorder. But the steep, 6-figure price tag being put on the drug, Procysbi, is already drawing flak from a growing contingent of analysts who wonders just how much drug developers can command from payers for rare-disease drugs before a backlash occurs.
AbbVie spins out a PhII orphan drug to Receptos--with a hook attached
San Diego-based Receptos has snagged a deal to put one of AbbVie's respiratory drugs through a Phase II study, taking on the risk of mid-stage work while securing an option deal that allows AbbVie the right to step back in if the therapy looks promising enough to take it forward into Phase III.
Analysts track a spike in mid-cap biotechs' R&D spend
Analysts at GlobalData took a quarterly snapshot of the R&D expenses for a big batch of mid-cap biotechs and concluded that rising research costs were eroding corporate profitability. And they pinned the blame squarely on the pricey hunt for new cancer drugs.
Roche grabs rights to Chiasma's PhIII rare disease drug in $595M deal
Roche has tied up the worldwide commercial rights to Chiasma's Phase III drug for acromegaly, a rare and progressive disease caused by the over-production of growth hormone.
Santhera Pharma slammed with EU denial of orphan drug
Santhera Pharmaceuticals suffered a setback today with the rejection of its key experimental drug for a genetic cause of blindness. European Union reviewers denied the Swiss specialty pharma their support for approval of Raxone over concerns about the small number of patients studied during clinical development.
Shire gambles on one-man biotech startup to gain ultra-orphan drug
The deal bolsters the pipeline of Shire's fast-growing Human Genetic Therapies, while providing Lotus investor Third Rock Ventures with its second exit in rare diseases.
Tufts: Cancer drugs win a speedier race to the FDA goal line
Anyone trying to explain the onrush of new cancer therapies in the pipeline should check out a new report from the Tufts Center for the Study of Drug Development, which found that cancer drugs have enjoyed faster approval times at the FDA than all other disease categories combined.
Analysts show why orphan drug development is going gangbusters
Small patient populations represent a huge market opportunity. Orphan drugs represent one of the pharma industry's hottest growth markets, and new figures from Thomson Reuters underscores that the blistering rise in revenue will drive the development of the next generation of experimental products.
Shire's CEO, business model win accolades
A few key buyouts has helped transform Shire Therapeutics over the past decade from a "one-trick pony" to a diversified pharma group with a string of marketed products and a leadership role in several strategic areas.
Bluebird adds $60M venture haul to fuel pivotal gene therapy study
Bluebird bio is singing a sweet song today, to the tune of $60 million. That's the amount its lineup of investors--including the newly arrived Shire--is sinking into the Cambridge, MA-based biotech as it prepares a pivotal Phase II/III study for its lead gene therapy and brings along the next two programs in the pipeline.
