It's fairly routine for the U.K.'s cost-effectiveness watchdogs to ask companies for more data to support approval for use by the country's National Health Service. Clinical data, price modeling, the works. But asking for information on R&D costs? That's something different altogether.
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.
Alexion, maker of orphan drug Soliris, last year said it was opening a center in Dublin, Ireland, to expand its supply chain operations in Europe. Now the drugmaker is buying a vial-filling facility there, a move that is providing a boost to a manufacturing expansion project by another drugmaker.
Cambridge, MA's Moderna Therapeutics is marching on with another high-dollar deal, getting $125 million up front to lend its messenger RNA platform to rare disease luminary Alexion Pharmaceuticals.
Alexion is again having problems with a contract filler, forcing it to recall two more lots of its rare-disease drug Soliris because of visible particles found in vials. It is the second time in about three months that the company has been in this situation.
Alexion Pharmaceuticals has racked up its second breakthrough therapy designation from the FDA, securing a faster track for an early-phase enzyme-replacement treatment targeting an ultra-rare metabolic disorder.
So is Roche going to do some rare disease deals or not? Rumors of a possible buyout of Alexion and BioMarin--alternatively dissed and touted--helped make this one of the big questions for deal watchers. Roche CEO Severin Schwan's answer can now be summarized in one word: Maybe.
Maybe Roche isn't as interested in Alexion Pharmaceuticals as some recent news reports suggest. Roche pharma chief Daniel O'Day told Reuters ' Ben Hirschler that ultra-rare diseases--an Alexion specialty--just doesn't fit its business model.
Amid a major hot streak for biotech stocks, New York Times scribe Andrew Pollack caught up with David Blech, who is heading to prison later this month after making fraudulent trades on drug company stocks. His story has become a cautionary tale in the biotech industry, which once regarded him as a major player.
Biotech companies have set out to keep U.S. tax credits pinned to costs of developing drugs for rare or "orphan" diseases, seeking to escape the mammoth budgetary ax of Congress on Capitol Hill.