Biotech CEO campaigns to speed drugs for rare diseases

Emil Kakkis, who runs the biotech company Ultragenyx as well as the Kakkis EveryLife Foundation, has launched a campaign to persuade the FDA to provide clear guidelines on how developers can meet its standard for determining whether a surrogate endpoint in a rare disease program meets its standard for being "reasonably likely" to indicate a clear patient benefit. It's a big deal for drug developers working on a new drug for a rare disease. They can gain accelerated approval for their products if they can measure up to the agency's expectations.

Kakkis co-authored a paper with Brigitta Miyamoto in the Orphanet Journal of Rare Diseases saying that clearer guidelines would mean more accelerated approvals. And he's got a personal stake in the issue. Ultragenyx just announced a $45 million venture round to advance new treatments for rare diseases. And he's planning to host a workshop this fall to study whether some rare diseases warrant a specific set of criteria.

To underscore their point, the authors highlighted 15 drug programs that foundered after showing promise in animals, explains the Wall Street Journal. Some were considered too expensive to develop, some could never make the leap from academia to commercial development and others are getting a second look. All of them, says Kakkis, would be helped by new criteria and better guidelines.

Kakkis is likely to find a rather large audience of experts interested in his regulatory program. Big Pharma companies have been diving into rare diseases over the last two years, drawn by the big paybacks that Genzyme and others have earned. Faster development times would make this field even more attractive.

- here's the story from the Wall Street Journal's Health Blog

Special Report: Rare diseases: All the rage in Big Pharma