Rare diseases, by their very nature, don’t fit the mold—so neither should the trials for therapies designed to treat them nor the regulatory process to approve them. This was the tone set during a recent panel discussion where Peter Marks, director of the FDA’s Center...
Rare diseases impact a larger segment of the population than cancer and AIDS combined, encompassing over 7,000 unique conditions and affecting more than 300 million people worldwide. Roughly 1 in 10 individuals experience the challenges of rare diseases, and the...
Pictured: Gene therapy illustration showing an injection targeting a specific DNA site/iStock, Ilya Lukichev The FDA on Monday accepted Orchard Therapeutics’ Biologics License Application for OTL-200, an investigational gene therapy for rare disease metachromatic...
While FDA panelists supported Alnylam Pharmaceuticals’ medicine in transthyretin amyloidosis cardiomyopathy, they recommended a limited FDA approval that would severely restrict the med’s addressable population. (FDA) After Alnylam Pharmaceuticals faced...
New Jersey’s Rocket Pharmaceuticals has successfully aligned with the FDA on the design of a pivotal Phase 2 trial for a rare disease, potentially hastening the approval of their gene therapy. This open-label study will enlist 12 patients for treatment with...
