BioMarin has slapped a price tag on its new drug Vimizim: $380,000 a year. That's hefty even for a rare disease treatment. But even at such nosebleed rates, Vimizim ranks in just third place on the list of the world's most expensive drugs.
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.
Jazz Pharmaceuticals, the Ireland-based company known for its narcolepsy drug Xyrem, is often the subject of takeover talk. Because of its specialty drug portfolio and low-tax domicile, analysts often see it as a potential target. But the drugmaker has kept up a steady drumbeat of mostly smaller deals itself to expand its line of specialty drugs. Today it jumped the $1 billion deal fence with plans to buy Italy-base, Gentium and get its EU-approved orphan drug Defitelio.
In an effort to better understand diseases that have long eluded diagnosis, the New York Stem Cell Foundation Research Institute is partnering with the National Institutes of Health's Undiagnosed Diseases Program to create cell models of rare medical conditions.
Shire is one of those companies that's always rumored to be a buyer or a seller. Not so long ago, it was setting up poison-pill protections against an unwanted suitor. Now, it has beat out Big Pharma with a deal for ViroPharma, the maker of rare disease drugs.
The buyout--at $50 a share--will add Cinryze for hereditary angioedema to Shire's product lineup along with the experimental drug maribavir for cytomegalovirus, one of 8 clinical-stage programs in the pipeline.
While bioinformatics lies at the leading edge of biological research, it is still underpinned by one of the oldest tools available to humanity: communication. Recognizing this, researchers are trying to standardize terminology through the creation of a rare genetic disease dictionary.
Fidelity Biosciences is placing a big bet on gene therapy, funding the launch of startup Dimension Therapeutics with some licensed patents and a plan to develop novel treatments for hemophilia.
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.
Eli Lilly has found a new path into the hot rare-disease field. An NIH program has backed the Indianapolis-based drug giant's preclinical-stage research of a potential treatment for a hormone deficiency often caused by a genetic defect.