FDA Hosts Drug Development Meeting for Neurologic Manifestations of Inborn Metabolism Errors

The FDA will listen to real-world experiences of patients living with metabolic diseases such as phenylketonuria, Wilson’s disease and lysosomal storage disorders at a June 10 meeting on patient-focused drug development for neurologic manifestations of inborn metabolism errors.

The meeting, which will be held at the agency’s White Oak, Md., campus, will open with a patient panel about effects of the diseases on daily activities such as sleeping or climbing stairs, whether symptoms change over time and whether or not current treatments meet their needs. The panel will be followed by a facilitated discussion designed to help inform the FDA’s drug approval process, according to a Friday Federal Register notice.

The meeting is open to patients, patient advocates, caregivers, FDA review divisions and other interested parties, with registration closing on May 27.  Comments are due to regulations.gov by August 11.

Read the notice at www.fdanews.com/ext/resources/files/04/04-25-14-Metabolism.pdf.—Lena Freund

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