FDA Breaks New Ground With First Approved Gene Therapy for Cancer

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When oncologist Dr. Carl June heard the Food and Drug Administration’s decision to bring the first gene therapy to market in the US, he pinched himself, hard.

“It was so improbable that this would ever be a commercially approved therapy,” he said, voice breaking with emotion.

June was referring to a revolutionary cancer therapy that he helped bring from lab bench to market. Co-developed with the drug giant Novartis, the therapy, CAR-T, genetically alters a patient’s own immune cells to target and destroy cancer cells.

Recently, in a historic decision, the FDA threw their support behind Kymriah (tisagenlecleucel), a “living drug” that is designed to treat blood and bone marrow cancer in children that, even with aggressive chemotherapy, is often lethal.

An entire process rather than a packaged pill, the therapy harvests a patient’s own immune cells—T cells that patrol and destroy abnormal cells—retrains them with extra bits of genetic code, and turns them into torpedoes aimed at cancerous cells once reintroduced into patients’ bodies.

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said FDA Commissioner Dr. Scott Gottlieb in a statement, adding that the therapy is “the first gene therapy available in the United States.”

Read More: http://snip.ly/bunjk#http://www.philly.com/philly/business/fda-advisors-give-a-thumbs-up-to-glaxosmithklines-shingles-vaccine-20170913.html

FDA advisors give a thumbs-up to GlaxoSmithKline’s shingles vaccine

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GlaxoSmithKline’s shingles Shingrix vaccine received an unanimous vote of support by a Food and Drug Administration advisory committee Wednesday on safety and effectiveness to be used in adults 50 and older.

A decision by the FDA to commercialize Shingrix is expected later this year. The agency usually follows the recommendations of its advisory panels.

GSK said in June that the vaccine produced a strong immune response in adults 65 and older who had previously been vaccinated against shingles with Merck’s vaccine, Zostavax. Scientific data published in the New England Journal of Medicine showed that the effectiveness of Merck’s vaccine wanes over time, while GSK’s vaccine appeared to have longer-lasting protection.

GSK said data show that people who received Merck’s vaccine, the only one approved now for the herpes zoster (shingles) vaccine, can later receive the Shingrix vaccine safely and effectively.

“The risk of developing shingles increases with age and it is estimated that up to one in three people in the United States will develop shingles,” said Emmanuel Hanon, GSK head of vaccines research and development. “Today’s vote brings us one step closer to approval of Shingrix, which is specifically designed to overcome age-related weakening of the immune system.”

 

Read More: http://snip.ly/bunjk#http://www.philly.com/philly/business/fda-advisors-give-a-thumbs-up-to-glaxosmithklines-shingles-vaccine-20170913.html

New FDA FSMA Rules on the Sanitary Transportation of Human and Animal Foods and adherence to them

The U.S. Food and Drug Administration (FDA) is in the process of finalising food safety rules. A major part of these rules is devoted to the ways by which to ensure scientific and safe transportation and logistics food transportation. The FDA is required to establish rules for that improve, audit and enforce new rules relating to food transportation. This is something required by the Food Safety Modernization Act (FSMA), under congressional instructions.

Many aspects of food transportation come under the ambit of these new FSMA Rules. These include:

–       Foods not completely enclosed by a container

–       Prevention and reduction in adulteration and risk

–       Personnel training and certification for this purpose

–       Inspecting food and collecting data

–       Maintaining compliance and reporting about its evidence.

How to do this right?

Comprehension of all the FDA FSMA Rules on the Sanitary Transportation of Human and Animal Foods is necessary and important for organizations that work in food transportation. They also need to know how to implement these rules. It is this understanding that GlobalCompliancePanel, a leading provider of professional trainings for all the areas of regulatory compliance, will offer at a seminar it is organizing.

The Director of this two-day seminar is John Ryan, President, TransCert, QualityInFoodSafety, RyanSystems. To gain insights into how to understand all the aspects of the FDA FSMA Rules on the Sanitary Transportation of Human and Animal Foods and to understand how to comply with them, just register for this session by visiting http://www.globalcompliancepanel.com/control/globalseminars/~product_id=900810?wordpress-SEO . This seminar has been pre-approved by RAPS as eligible for up to 12 credits towards a participant’s RAC recertification upon full completion

At this seminar, the Director will arm participants with an understanding of the legal requirements of food transportation and how to develop a company plan that meets the expectations and requirements of both its customers and the FDA. Also offered is total understanding and review of the ways of establishing the right temperature monitoring, sanitation, container test and traceability, training and data reporting procedures.

Technological aspects of FDA FSMA Rules on the Sanitary Transportation of Human and Animal Foods

Technological aspects of food transportation are gaining importance. These include new low cost GPS enabled traceability and temperature monitoring technology, EPA approved container sanitizers, washout technologies, temperature maintenance equipment, and food residue and bio-contaminant testing. FDA FSMA Rules on the Sanitary Transportation of Human and Animal Foods have a close relationship with these. The Director of this seminar will take these up for examination.

In reviewing the future of transportation food safety in the light of new and evolving technologies; the Director will dot the seminar with references to upcoming technology providers and provide Internet links to detailed information on the same.

This seminar is immensely useful for food supply chain logistics and food safety and security personnel whose primary responsibilities include management, sanitation, quality and operations. Those involved with handling incoming and outgoing food shipments, maintaining transportation equipment and tools, and purchasing or selling will also derive high value out of this seminar.

Making sense of the FDA’s GMP and Regulatory Expectations for Early IND Products

The FDA’s recent guidance documents covering GMP requirements for Phase I products have considerably reduced some of the complexities early phase products face. These guidance documents are in addition to those that cover the CMC sections for IND submissions at Phase I.

Although these new guidelines appear to remove the need to follow GMPs for Phase I products; the same still exists in the Food, Drug, and Cosmetic Act. As a result, the need for GMP requirements for Phase I products has not been done away with; rather, it has been altered. Now, the nature and extent of GMP-related activities will depend upon the nature of the investigational drug and the extent of the study that is planned.

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Get trained to understand these aspects

GlobalCompliancePanel, a leading provider of professional trainings for the areas of regulatory compliance, will be organizing a two-day seminar to help Directors, Managers and Supervisors in Regulatory Affairs, Quality Assurance and Quality Control get a grasp of these aspects. To enroll for this highly valuable training, please log on to http://www.globalcompliancepanel.com/control/globalseminars/~product_id=900695SEMINAR?GMP-regulatory-expectations-San-Diego. Steven S. Kuwahara, Founder and Principal, GXP BioTechnology LLC, will be the Director of this webinar.

Advice on the GMP guidance document

This presentation, which has been pre-approved by RAPS as eligible for up to 12 credits towards a participant’s RAC recertification upon full completion, will review the GMP guidance document and discuss how it may be integrated with the recommendations of the guidance documents on CMC requirements. In one source, it will present the regulations and guidelines that apply to early phase products. In some cases these may not be regulations, but needs that, if met, will increase the efficiency of activities as a product proceeds through the development process. Steven will present these items in the order of product development from the point of R & D activities to the completion of Phase 2 clinical trials.

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This course is very useful for any pharmaceutical personnel who must deal with products both in early and later phases of development, as it will make them aware of the regulatory requirements that will affect operations dealing with these products. The modifications to the GMPs for early phase products have altered the GMPs in such a way as to reduce requirements to allow more efficient work. At the same time, some of the things that may appear to have changed, have not, and personnel in the pharmaceutical sector should be aware of this. This is the learning that Steven will emphasize at this seminar.

It will cover the following areas:

  • Very Early Stages
  • GLP requirements
  • Early Pre-IND Studies
  • Meetings and Preparing for the IND
  • GMPs for Phase 1 IND products
  • Requirements for Phase 2 INDs
  • Preparing for IND Meetings

FDA Guideline on Process Validation

FDA Guideline on Process Validation.

Process Validation (PV), as defined by the FDA, has undergone a change from the past 25 years or so. The meaning of PV is what the FDA assigns to it. So, any understanding of PV has to be tied to FDA’s guidelines which offer a definition of the term, as well as updates from time to time.

The previous and the latest guidelines…

The latest FDA guideline on PV came in 2011, and was consequential to the earlier one of 1987. In view of the changes in the industry, the definition of the scope of PV also changed. Earlier, the FDA described PV as the process of “…establishing documented evidence which provides a high degree of assurance that a specific process will consistently produce a product meeting its pre-determined specifications and quality characteristics”.

The guideline of 2011

Considering the insufficiency of this definition, the FDA issued another guideline, an updated one, in January 2011 to make the definition more wide-ranging. Expectedly, it is more comprehensive and emphasizes process understanding and control as a means of focusing on product quality. PV now means “the collection and evaluation of data, from the process design stage through commercial production which establishes scientific evidence that a process is capable of consistently delivering quality product”.

How is the 2011 Guideline different?

As defined more comprehensively in the January 2011 Guideline, the following changes may be discerned from the 1987 Guideline:

• There is added emphasis on process design. Risk is now incorporated into process design

• It now covers activities ranging over the entire process lifecycle, which is an ongoing program that is carried out in three defined stages

• Greater emphasis is laid on the role of objective measures and statistical tools

• Knowledge, detection and control of variability have got a greater thrust from this Guideline.

References:

http://www.fda.gov/downloads/Drugs/Guidances/UCM070336.pdf

http://www.ispeboston.org/files/handouts_-_morrison.pdf

http://www.pharmout.net/downloads/white_paper_fda_process_validation_guidance_final.pdf