Tag:
rare diseases
Latest Headlines
Latest Headlines
UPDATED: In rare slap, FDA review rips up Pfizer's case for tafamidis
After considering the trial's failure to meet its co-primary endpoints and questioning the data gathered at 8 sites, the agency is recommending that the experts gathering to discuss the drug's fate on Thursday reject the therapy.
UPDATED: Upstart Ra sheds stealth status, targets new drug class
Two years after Ra snagged an initial $10 million in venture cash, the company has hauled in a second tranche of $8.6 million and picked its disease target: hereditary angioedema, a rare and lethal immune system disorder.
Novartis passes Phase III test for rare-disorder drug
The Swiss drug giant says its experimental drug pasireotide LAR beat the standard of care in the largest Phase III study involving patients with acromegaly.
Rare-disease drugs won't help out-of-work pharma reps
As drugmakers turn their focus to rare-disease drugs, armies of sales reps become unnecessary.
Conflicting signals cloud Chelsea's chances on D-Day for Northera
It's D-Day for Chelsea Therapeutics and its rare disease drug Northera .
Sanofi secures computational help for rare disease research
Sanofi ( $SNY ) has made another move to tap external partners for new science and technology, this time in a tie-up that enables the French drug giant to benefit from computational approaches to...
FDA nudged to treat antibiotics like rare disease drugs
A research group wants the FDA to give applications for new antibiotics some of the same treatment now lavished on rare disease drugs.
Aegerion application for lead rare disease drug lands at FDA
Cambridge, MA-based Aegerion Pharmaceuticals has cleared an important milestone.
Fledgling 4s3 garners $20M for rare disease work
The fledgling, Medford, MA-based biotech 4s3 Bioscience has rounded up $20 million in Series A cash to advance its antibody strategy for delivering macromolecules to skeletal tissue--taking a new...
GSK lassos rare disease program in Angiochem tie-up
GlaxoSmithKline ( $GSK ) has grabbed rights to biotech startup Angiochem's early-stage program for treating a lysosomal storage disease. The deal comes as the London-based drug giant and its large...
