orphan drug status

Latest Headlines

Latest Headlines

Epizyme picks up FDA orphan nod for lead cancer drug as going public pays off

The drug is in a Phase I clinical trial that began in September 2012. Its shares rose nearly 1% this morning to $31.44 per share, which is more than double the IPO price of $15 per share set on May 31.

Acceleron pitches $75M IPO as Celgene pushes partnership through PhII

Acceleron has jumped into the crowded IPO ring, looking to raise about $75 million at a time when a long lineup of biotech companies have tapped public markets for more than $1.5 billion from maiden offerings.

Alnylam Pharma spotlights more upbeat human data for gene-silencing drugs

The top-line results from a Phase I study show that its therapy ALN-TTRsc reduced levels of Transthyretin (TTR)-mediated amyloidosis protein by more than 80%, setting the stage for a mid-stage study planned for late this year in patients with a genetic heart disease called familial amyloidotic cardiomyopathy (FAC).

GlaxoSmithKline fuels VC fund as part of rare diseases blitz

GlaxoSmithKline is backing another venture fund. The London-based drug giant has committed 17.5 million euros for a VC vehicle focused on rare diseases startups in Europe, building on its previous investments in venture firms on both sides of the Atlantic.

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.