Thought Leadership

Accelerated Approval Offers a Promising Roadmap for Rare Neurological Diseases

By: Emil D. Kakkis is the CEO, president, and founder of Ultragenyx Pharmaceutical, a company member of the Rare Disease Company Coalition

The FDA’s decision to grant accelerated approval to Biogen’s aducanumab (Aduhelm) for the treatment of Alzheimer’s disease was a difficult and bold one that people with the disease, their families, and other drug developers should be applauding.

When it comes to making new therapies for complex, difficult-to-treat diseases, history has shown that progress can’t be made without taking a first — often controversial — step. Without the FDA’s accelerated approval program and novel first treatments based on new and imperfect biomarker endpoints, HIV would not be a controllable disease today, and we might not have such a flourishing clinical research ecosystem in oncology.

Continue reading at STAT News.