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Pfizer explores rare disease path

Nature Biotechnology volume 28, pages 881–882 (2010)Cite this article

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An Erratum to this article was published on 13 October 2010

This article has been updated

“[Pharma] companies are realizing that for niche diseases, you can charge a significant premium,” says Simos Simeonidis, managing director senior biotechnology analyst Rodman & Renshaw. Patient numbers are small by pharma standards, but because the drugs are lifesaving, even though they are expensive, insurers must pay up. Investors and corporate decision makers are slowly waking up to the potential value of these drugs that some have started calling 'minibusters'.

In February, GSK announced plans to form a new stand-alone rare diseases unit. The new unit, described in a company release as operating under a “lean structure,” will work with the company's existing capabilities and seek strategic collaborations with other companies. Analysts, however, have been reserved in their assessments of these initiatives, noting that many of them are very small, resembling almost a token effort, rather than a full commitment to rare disease research. Indeed, Pfizer's new initiative, still in its very earliest stages, consists of two employees and a few laboratory benches.

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Change history

  • 22 September 2010

    In the version of this article initially published, it was reported that GlaxoSmithKline’s (GSK) EpiNova was one of several “biotech-like ideas” that “have been known to fizzle in pharma hands”; in fact, EpiNova has not “fizzled” but is in its second year of operation as a discovery performance unit of GSK focusing on epigenetic approaches to autoimmune disease. The error has been corrected in the HTML and PDF versions of the article.

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    Catherine Shaffer

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Shaffer, C. Pfizer explores rare disease path. Nat Biotechnol 28, 881–882 (2010). https://doi.org/10.1038/nbt0910-881

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