Information for Healthcare Professionals (Drugs)
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Information for Healthcare Professionals (Drugs)
FDA Drug Info Rounds Video
Know Your Source: Protecting Patients from Unsafe Drugs
Global Alliance of Drug Information Specialists (GADIS)
Drug Safety Information
Transcript: Accelerated Approval Program video
Captain Mary Kremzner: Now Cat, exactly how does the FDA determine when an expedited approval is warranted?
Commander Catherine Chew: Generally drugs are approved under Accelerated Approval if they have the potential to impact factors, things like survival, day‑to‑day function or the likelihood of stopping a disease from progressing and becoming an even more serious condition.
Captain Mary Kremzner: Now Cat earlier you mentioned filling an unmet medical need. How would you define that?
Commander Catherine Chew: An unmet medical need simply means providing a therapy when none exists or providing a new therapy that may be superior to or less toxic than an existing one.
Captain Mary Kremzner: So without the standard clinical outcomes how does FDA determine if a treatment is potentially effective?
Commander Catherine Chew: Great question Mary. Accelerated Approvals use what we call a surrogate end point in clinical trials. These are markers or a physical sign of sorts used as an indirect measurement to predict a clinically meaningful outcome like survival or symptom improvement.
Captain Mary Kremzner: So exactly how does the surrogate end point save time in the drug approval process?
Commander Catherine Chew: Well for example, instead of having to wait to learn if a drug prolonged survival in cancer patients the FDA has approved drugs based on evidence that they shrink tumors. As a surrogate end point, tumor shrinkage is reasonably linked to real clinical benefit.
Captain Mary Kremzner: Okay, so let me make sure I have this. Let’s say a drug goes through Accelerated Approval and gets to market, how do we know if it really provides that clinical benefit?
Commander Catherine Chew: Any approvals given based on surrogate end points are on the condition that the sponsor will conduct post-marketing clinical trials to verify the anticipated benefit. If these trials, called Phase 4 Confirmatory Trials, shows the drug does in fact provide a clinical benefit then and only then does the FDA grant traditional approval for that drug.
Somebody needs a good DNA vaccine for pandemic influenze-US Gov just awarded GSK $200 million plus
Contract No. HHSO100201500010C was awarded on May 22, 2015 to GlaxoSmithKline LLC in the amount of $79,998,267 for the base period and either a $120,000,000 or $112,000,344 option period to support the development of cell-based influenza virus vaccines against influenza subtypes with pandemic potential.
Contracting Office Address:
Office of the Assistant Secretary for Preparedness & Response (ASPR)
Department of Health and Human Services
330 Independence Ave. SW
You don't need much of a sales force when all the neurology centers across the country are participating in DMD trials and all the Duchenne charities make patient and therapy information available on their blogs. If FDA approves it, the boys will get it.
J&A Statutory Authority:
FAR 6.302-1 - Only one responsible source (except brand name)
Contract Award Date:
September 30, 2015
Contract Award Number:
Added: May 22, 2015 4:11 pm
Notification of a Justification and Approval in accordance FAR 6.302-1 to procure the antiviral drug, brincidofovir, from Chimerix, Inc. for the treatment of smallpox.
The documentary was successfully funded, just made it over the finish line, so coming out
later this year, probably Sep-Oct (about the same time as the AdComm???)
About the DMD mothers and their campaign to get FDA approval for a drug for their boys. If you made
money, as I did, contribute to finish this documentary, 4 days left.
May 8 PT 36
CREDIT SUISSE SECURITIES RESEARCH & ANALYTICS
(SRPT)SMALL & MID CAP RESEARCH
Q1 Earnings: FDA Meeting for Eteplirsen Around the Corner
SRPT expects to have a meeting with the FDA in Q2 to review the available
eteplirsen data and hopefully receive a green-light to file the NDA shortly thereafter.
SRPT recently completed the fourth biopsy in eleven out of
twelve patients.We are encouraged by the
progress withthe ongoing studies, including the on-track
enrollment of the confirmatory study.
Fourth round of biopsies
completed:SRPT has collected
11/12 biopsies and intends to run the necessary analyses
using new protocols developed in
conjunction with the FDA, including Western blot,
RT-PCR, and immunohistochemistry assays
As indicated, major news had to be publicly released. That 4:03 Press Release was the Regulation FD path for news, now the Sarepta CEO can tell us a lot more at PPMD conference about patient quality of life, follow on exon trials 53, 44 , methods of dystrophin measurement confirmation by multiple methods.
cannot release material information (which would cause person. tobuy or sell stock) without releasing generally under SEC regn FD. So if there is new news, has to be released before or after PPMD panels. but can review public data like status of various clinical trials apart from results.
Amendment 3 Platforms for rapidly optimizing monoclonal antibodies for influenza or other infectious diseases will be considered
FDA still hasn't published what they said they were going to show
A summary of the scientific workshop's highlights will be made available for review at the Division of Dockets Management and at [regulationsdotgov] You may submit a request to obtain a hard copy by sending a request to the Division of Freedom of Information (ELEM-1029), Office of Management Programs, Food and Drug Administration, 12420 Parklawn Dr., Element Bldg., Rockville, MD 20857.
Probably because everything is so close to being pending as two competing NDAs at this point, don't want to upset any applecarts. If they ever publish highlights with a decent interval for responding, say May 10 or so (after SRPT update?), comments due May 20.
Prosensa Announces 3rd Quarter 2014 Financial Results and Recent Corporate Developments
Rolling NDA submission drisapersen underway
EMA filing for conditional approval anticipated to follow NDA submission
Re-dosing has commenced in United States, Belgium & Sweden
Cash & cash equivalents at quarter-end €62M
LEIDEN, The Netherlands, Nov. 17, 2014 (GLOBE NEWSWIRE) -- Prosensa Holding N.V. (Nasdaq:RNA), the biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today reported financial results for the third quarter ending September 30, 2014 and provided an update on the regulatory plans and re-dosing for drisapersen, our lead exon-skipping drug candidate, and next steps for our follow-on exon skipping compounds for the treatment of Duchenne muscular dystrophy (DMD).
Hans Schikan, CEO of Prosensa said, "We are incredibly pleased with the substantial progress we have accomplished during this period. We have delivered on our promise of initiating the re-dosing process for patients who were previously in drisapersen clinical trials with a total of 15 boys now back on treatment, and our rolling New Drug Application (NDA) submission with the US Food & Drug Administration (FDA) is currently well underway. We continue to work as diligently and quickly as possible to fulfill our promise of bringing much needed therapies to boys with DMD."
The influenza A(H5N1) virus, commonly known as bird flu, is fatal in about 60 percent of human infections. Sporadic cases continue to be reported, usually after contact with sick or dead poultry from certain Asian and African countries. No human cases have been reported from Europe.
missed on rev, so shorts pile on, confirms guidance, snoopy movie, .earns 54 trades at half Starbucks price. with PE of 8-10. not selling anything here
says they have lots of data, says drisapersen does exon skipping, says this may have a therapeutic benefit BBBUUUTTT does not say drug produces dystrophin. probably because they are relying on 6MWT and because they never had good baseline dystrophin slides. Any advisory committee will ask, well does it produce dystrophin ?
Lots of flu variants have come out of China where pigs and waterfowl are in close proximity. Now we have HPAI all over several states - the biggest risk is disposing of any culled chickens as feed for pigs, because
that is a devils brew for potential new variants of H5N2 and H3N2 reassortments.
Sarepta is starting their own new trial, 20 4-6 year olds and 20 controls not amenable to Exon 51 skipping now, for observation, Must have muscle tissue in arm ( biopsies show dystrophin, remember) Dont think they would do this unless they had some clue about where FDA was on eteplirsen
First received: April 10, 2015
Last updated: April 14, 2015
Last verified: March 2015
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