Forbes' Matthew Herper brings us up close to Vertex Pharmaceuticals' ($VRTX) scientist Fred Van Goor and his part in advancing the firm's cystic fibrosis drug VX-770, which is a game-changing therapy for patients with the rare disease--or at least for the 4% of those with the illness that the treatment is supposed to benefit.

Indeed, the story drills down to some of the unique dynamics of developing targeted drugs--treatments that are intended for specific patients based on the molecular drivers of their disease. Developers have shown that the treatments can do wonders for those with the right genetics in diseases like cancer and relatively rare conditions such as CF. With some cancers, for example, there's a big push toward developing combinations of the targeted treatments, yet the cost of treating patients with multiple expensive drugs will likely be steep.

Developing niche drugs for small pools of patients is nothing new, as Sanofi's ($SNY) Genzyme made providing ultra-expensive treatments for people with rare diseases into a multibillion-dollar business. The Genzyme story has motivated other companies, including Big Pharma outfits, to get in on the act.

"Pharmaceutical firms have shown new willingness to develop drugs for very rare diseases," Herper wrote, "partly because they have found they can charge a small fortune for the ones that work."

- read Herper's article

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