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FDAnews Drug Daily Bulletin
July 16, 2008 | Vol. 5 No. 138
FDA Moves Forward With Exempting Phase I Drugs From GMPs
More than two years after withdrawing a final rule that would have exempted investigational drugs in Phase I testing from certain good manufacturing practice (GMP) regulations, the FDA is issuing a final rule to do just that.
The new rule, which amends the GMP regulation with the exact same language as the withdrawn rule, was published in Tuesday’s Federal Register. Slated to take effect Sept. 15, it will apply to small-molecule drugs and biologics, including vaccines and gene therapy products.
“FDA’s position is that the United States’ [GMP] regulations were written primarily to address commercial manufacturing and do not consider the differences between early clinical supply manufacture and commercial manufacture,” the agency says.
For example, the requirements for a fully validated manufacturing process, rotation of stock for drug product containers, repackaging and relabeling of drugs and separate packaging and production areas need not apply to investigational drug products made for use in Phase I trials, the agency says.
In connection with the final rule on Phase I drug GMPs, the FDA issued a guidance recommending approaches to satisfy statutory GMP requirements for such drugs.
“During product development, the quality and safety of Phase I investigational drugs are maintained, in part, by having appropriate [quality control (QC)] procedures in effect,” the guidance states. “Using established or standardized QC procedures and following appropriate cGMP will also facilitate the manufacture of equivalent or comparable IND product for future clinical trials as needed.”
More information on the final rule can be accessed at www.fda.gov/OHRMS/DOCKETS/98fr/oc07114.pdf.
A copy of the guidance, “CGMP for Phase 1 Investigational Drugs,” can be viewed at
If you feel like reading an unputdownable novel while collaborating with a just and solidary cause, "The Legacy of Marie Schlau" is your book! 100% of all funds raised will be dedicated to medical research to find a cure for Friedreich's Ataxia, a neurodegenerative disease that affects mostly young people, shortening their life expectancy and confining them to a wheelchair.
The life of Marie Schlau, a German Jewish girl born in 1833 hides great unsolved mysteries: accidents, disappearances, enigmas, unknown diagnoses, disturbing murders, love, tenderness, greed, lies, death ... alternatively a different story unfolds every time and takes us closer to the present. Thus, there are two parallel stories unravelling, each in a different age and place, which surprisingly converge in a revelatory chapter.
Paperback and Kindle versions for "The legacy of Marie Schlau" available for sale at Amazon now!
Currently, BabelFAmily is financing two promising research projects aimed at finding a cure for Friedreich's Ataxia. Whenever you make a donation to us or purchase a copy of "The legacy of Marie Schlau", this is where all funds raised will be devoted to:
1) Gene Therapy for Friedreich's Ataxia research project:
The project is the result of an initiative of Spanish people affected by this rare disease who are grouped in GENEFA in collaboration with the Spanish Federation of Ataxias and the BabelFAmily. The Friedreich’s Ataxia Research Alliance (FARA), one of the main patients’ associations in the United States now joins the endeavour.
2) Frataxin delivery research project:
The associations of patients and families Babel Family and the Asociación Granadina de la Ataxia de Friedreich (ASOGAF) channel 80,000 euros of their donations (50% from each organisation) into a new 18-month project at the Institute for Research in Biomedicine (IRB Barcelona). The project specifically aims to complete a step necessary in order to move towards a future frataxin replacement therapy for the brain, where the reduction of this protein causes the most damage in patients with Friedreich’s Ataxia.
The study is headed by Ernest Giralt, head of the Peptides and Proteins Lab, who has many years of experience and is a recognised expert in peptide chemistry and new systems of through which to delivery drugs to the brain, such as peptide shuttles—molecules that have the capacity to carry the drug across the barrier that surrounds and protects the brain. Since the lab started its relation with these patients’ associations in 2013*, it has been developing another two projects into Friedrich’s Ataxia.