🧬FDA Draft Guidance on Clinical Trials for Gene Therapy Products
The Food and Drug Administration (FDA, the Agency, or we) is announcing the availability of a draft document entitled "Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations." The draft guidance document provides recommendations to sponsors who are planning clinical trials of cell and gene therapy (CGT) products intended for use in a disease or condition that affects a small population, generally one that meets the definition of a rare disease or condition under section 526(a)(2) of the FD&C Act (21 U.S.C. 360bb(a)(2)). It describes FDA requirements and provides considerations for the use of various clinical trial designs and endpoints to generate clinical evidence to support product licensure. The recommendations are intended for sponsors developing CGTs intended for use in small populations to leverage the use of innovative trial designs to simultaneously expedite drug development and generate data necessary to demonstrate substantial evidence of effectiveness.
Learn More💊DEA Notice on Controlled Substance Import Application by Biopharmaceutical Research Company
Biopharmaceutical Research Company has applied to be registered as an importer of basic class(es) of controlled substance(s). Refer to SUPPLEMENTARY INFORMATION listed below for further drug information.
Learn More💊DEA Notice on Biopharmaceutical Research Company’s Application
Biopharmaceutical Research Company has applied to be registered as a bulk manufacturer of basic class(es) of controlled substance(s). Refer to Supplementary Information listed below for further drug information.
Learn More💊FDA Regulatory Review Period for HEMGENIX Patent Extension
The Food and Drug Administration (FDA or the Agency) has determined the regulatory review period for HEMGENIX and is publishing this notice of that determination as required by law. FDA has made the determination because of the submission of an application to the Director of the U.S. Patent and Trademark Office (USPTO), Department of Commerce, for the extension of a patent which claims that human biological product.
Learn More💊Antitrust Notice
The Department of Justice's notice outlines changes in the BioMaP-Consortium’s membership, emphasizing compliance aspects associated with the National Cooperative Research and Production Act of 1993. Membership is open for future changes, promoting collaboration among the biopharmaceutical industry stakeholders to enhance manufacturing preparedness.
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