David Mott, ex-CEO of MedImmune and a high-profile biotech partner at New Enterprise Associates, has led the way toward an orphan drug accelerator also backed by Pfizer Ventures.
I've got a busy schedule of events coming up over the next few months. First, if you're headed to Chicago for BIO later this month, I hope you'll come by my executive breakfast. The big challenge in drug development now is figuring out how to do much faster, more efficient clinical drug programs. -- John Carroll, Editor-in-Chief. Follow me on Twitter .
David Mott at New Enterprise Associates has spearheaded the development of an orphan drug accelerator that plans to hunt down the best in vivo-stage assets in academia, biopharma and the nonprofits, assemble them in a portfolio of rare disease therapies and mold them into pipeline programs that can be spun out into a slate of new biotech companies.
Everyone who's anyone in Big Pharma wants to amp up in orphan drugs. Treatments for rare disorders, many of them deadly, aren't easily copied, and competitors are few, if not completely absent. Plus, payers are willing to pay top dollar for drugs aimed at very small groups of patients. Even if they are costly.
Edison Pharmaceuticals shed its stealth mode and went public with a $545 million-plus collaboration deal with Japan's Dainippon Sumitomo that centers on its lead late-stage drug for rare diseases.
The FDA has issued a warning letter for the plant where Alexion Pharmaceuticals manufactures its pricey rare-disease drug Soliris. The letter has yet to make it to the FDA's website, but the company got ahead of the news by reporting to the SEC that the warning was delivered yesterday.
The clock is ticking for Sarepta, a multitude of biotech investors and the boys who suffer from Duchenne muscular dystrophy.
Astellas Pharma has struck research agreements with two Japanese universities--Tokyo Institute of Technology and Nagasaki University--to find new treatments for neglected tropical diseases caused by dengue virus, such as dengue fever.
A few days ago the European Commission mapped out a plan to help spur development of 200 new treatments for rare diseases by the year 2020. And the Commission put its money where its mouth is, committing $187 million for 26 research projects.
With several developers now crowding into the same field, some frustrated parents are urging developers to start sharing their data, removing some of the burden associated with having their children journey far and wide to receive a hoped-for therapeutic intervention