Inside the Orphan Drug Revolution: The Promise of Patient-Centered Biotechnology

Published

February 29, 2024

Inside the Orphan Drug Revolution: The Promise of Patient-Centered Biotechnology

Thousands of rare diseases, most of genetic origin, were long ignored by a pharmaceutical industry that judged them too small to provide a return on the investment needed to develop an effective remedy. Yet these “orphaned” diseases collectively caused misery and expense, often far greater than did more common ailments, for in the aggregate tens of millions of individuals and their families.

Forty years ago, a revolution that transformed the prospects of patients with rare diseases was lit by three sparks. The passage of the 1983 U.S. Orphan Drug Act resulted from public pressure brought by rare disease patients, their families, and advocates. The AIDS epidemic then triggered rare disease activism, when patients with the rare disease hemophilia became HIV-positive after infusion of tainted blood products. The third spark was the emergence in the early 1980s of biotechnology companies like Genentech, Amgen, and Biogen. Soon after, Genzyme became the first company to develop a treatment for a rare genetic disorder, Gaucher disease, which would come to transform the industry and spawn many efforts toward other diseases.

OMass’s work in applying a world-class novel mass spec platform to developing a therapy for Congenital Adrenal Hyperplasia (CAH) is in the finest tradition of establishing the highest possible standard of care for a debilitating rare disease. I’m honored to support them in that mission.

 

Biotech pioneer, Genzyme veteran Jim Geraghty on the 'orphan drug  revolution' - Boston Business Journal

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Inside the Orphan Drug Revolution: The Promise of Patient-Centered Biotechnology
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