Orphan Drug Designation Portal Launched

FDA has launched its Orphan Drug Designation Portal to support therapy development for rare diseases by enhancing sponsor communication with the agency and to simplify orphan drug designation submissions. “This important initiative is part of broader technology modernization efforts at the FDA to develop a more connected information technology system, advanced analytics and improvements in knowledge management,” FDA principal deputy commissioner Amy Abernethy says in a release. “This is especially key during the current public health emergency when the ease of electronic submission options is more important than ever before.”

The portal will now allow electronic submission of new orphan drug designation requests through a cloud-based online submission. “The portal system will help ensure questions are answered and will allow for feedback from sponsors,” the agency says. Additional detailed information is provided on the agency’s portal landing page.