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Influenza Antiviral Medications: Summary for Clinicians
Previously oseltamivir and zanamivir only approved , now RAPIVAB approved Dec 19
CDC web pages being updated to reflect new flu drug
Recent publicity about number of FDA approvals, role of Breakthough therapy designation, and use of FDASIA suggest the agency has felt some public and political pressure to reform congested review processes. This is probably positive for Sarepta because if it can get over the safety hurdle where FDA requested new patient data, there is little reason not to move forward quickly with eteplirsen. As to drisa v eteplirsen hasn't Prosensa talked about an adv committee--and there can't be adv committee on drisa w/o considering etep.
We have activated a multi-agency response plan following the confirmation of highly pathogenic H5 avian influenza in a backyard flock in Benton County. WSDA will work closely with the USDA’s Animal Plant Health Inspection Service (USDA-APHIS) on this response.
There is no immediate public health concern and avian influenza does not affect poultry meat or egg products, which remain safe to eat.
The owners contacted WSDA after losing 50 birds in the past week. You can read the full news release here:
It should be approved under AA see Draft guidance for DMD pp 32 and chapter on biomarkers
So we are looking at
1. Verification of dystrophin masurement
2. Safety both for existing participants and new enrollees
3. Clinical benefit v natural history for untreated DMD course
Then FDA should apply the following:
... Primary Outcome Measures The approval of a drug for the treatment of DMD based on the use of a biomarker as a single primary surrogate efficacy measure can be considered under accelerated approval. As described in the biomarkers chapter that follows, at the present time, there is a great deal of research...
Sarepta comments on Draft DMD drug development guidance:
1. Safety is important to get benefits
2. Natural history is understood, agressive, predictable despite variation.
Safety plays a critical role in informing benefit-risk for patients and caregivers, especially when concerning chronic, life-long therapies for patients starting in childhood. The guidance does not currently include a section on safety considerations. A key consideration of risk includes the severity and reversibility of adverse events and the ability to manage risks in the post-market setting. The ability to continue taking a treatment and continue to benefit from it hinges on the treatment’s safety profile. In general, products with a favorable safety profile may result in a more favorable benefit-risk assessment by reducing the uncertainty related to risks of the drug and improving the trade-off decision. We encourage FDA to clarify how safety considerations, combined with the DMD community’s clear benefit-risk tolerance, will factor into the benefit- risk framework for DMD treatments.
2. NATURAL HISTORY
The draft guidance provides a very thorough account of the natural history and course of progression of DMD. While sources of variability and heterogeneity are described in detail, and it is important to consider causes of such variability when designing and interpreting clinical studies, we caution FDA not to lose sight of the fact that despite some level of variability, DMD follows a common, predictable, and aggressive disease course.
Earlier this week, Pfizer Inc. (PFE) announced that it has acquired a controlling interest in Redvax Gmbh, a spin-off from Swiss biopharma company Redbiotec AG. With this acquisition, Pfizer’s vaccine pipeline will gain a preclinical human cytomegalovirus (CMV) vaccine candidate.
While financial details of the agreement were not disclosed, Pfizer said that together with the vaccine candidate it has also bagged intellectual property and a technology platform related to a second, undisclosed vaccine program.
With the addition of the CMV vaccine program, Pfizer will be one of the leaders in CMV research and development. CMV, a herpes virus, is estimated to infect about 50% - 90% of adults. According to the U.S. Centers for Disease Control and Prevention (CDC), in the U.S. alone, every year about 5,000 children develop lasting health problems caused by CMV.
Information for Health Care Professionals
Additional Resources on Influenza Antiviral Drugs
More information about oseltamivir, zanamivir, peramivir, amantadine, and rimantadine.
What antiviral drugs are recommended this flu season?
There are three FDA-approved influenza antiviral drugs recommended by CDC this season to treat influenza. The brand names for these are Tamiflu® (generic name oseltamivir), Relenza® (generic name zanamivir), and Rapivab® (generic name peramivir). Tamiflu® is available as a pill or liquid and Relenza® is a powder that is inhaled. (Relenza® is not for people with breathing problems like asthma or COPD, for example.) Rapivab® is administered intravenously by a health care provider.
How long should antiviral drugs be taken?
To treat the flu, Tamiflu® and Relenza® are usually prescribed for 5 days, although people hospitalized with the flu may need the medicine for longer than 5 days. Rapivab® is administered intravenously for 15 to 30 minutes.
So, if you have asthma and are at high risk, no relenza, choice is Tamiflu or Rapivab according to. CDC
AP Report CDC Pushes antivirals
The CDC sent an advisory to physicians last month, warning them this could be a potentially bad flu season and encouraging prompt treatment with antivirals. CDC officials said doctors should not wait for test results confirming the flu if they are dealing with an elderly patient, someone who is very sick from the flu, or someone with pre-existing conditions like asthma, diabetes and heart disease.
The CDC sent a second alert to doctors Friday that repeated earlier recommendations and noted a new antiviral was approved by the U.S. Food and Drug Administration last month. It's called Rapivab, and is an infusion that can be given to sick patients who aren't able to take Tamiflu pills or another, inhalable antiviral medicine called Relenza.
For outpatients with acute uncomplicated influenza, oral oseltamivir, inhaled zanamivir, or intravenous peramivir may be used for treatment.
The recommended treatment course for uncomplicated influenza is two doses per day of oral oseltamivir or inhaled zanamivir for 5 days, or one dose of intravenous peramivir for 1 day.
As interpreted by one source of analysis SRPT patients "showed improvement"
People who interpret lesser declines as negative have no knowledge of NH or understanding of
what progressive disease means.
In addition to the 6MWT data, several other outcomes have been measured in this study, including those looking at respiratory muscle function. Interestingly, the boys taking eteplirsen showed improvement in respiratory muscle function. From baseline through Week 168 in the Intent-to-Treat population (n=12), as measured by maximum inspiratory and expiratory pressure (MIP and MEP), showed a 11.1% mean increase in MIP and a 14.7% mean increase in MEP.
Biocryst gains on Europe orphan drug deisgnation
good news for DMD boys and their families; Just getting NH and safety data will help build NDA filing, likely approval for all applicable exons to treat, stabilize, extend lives and QOL.
Real question is, is Prosensa or now BMRN saying anything, esp anything similar to FDA. While FDA communications about a company's drug under approval are confidential, communications by company A about company B's competing drug should not be, so a FOIA request to FDA about any Prosensa / BMRN statements about eteplirsen may be in order. Also, if the Prosensa acquisition occurs, look for BMRN to bury the old P rosensa presentations, because of inclusion of statements like this
The most common adverse events in drisapersen-treated patients were injection-site reactions and renal events (mostly subclinical proteinuria - eight patients in the continuous group and eleven in the intermittent group)
He actually dropped a little nitro at the end, in the Q&A, said they were still working with FDA to make them comfortable on all the data, there was still a possibility for accelerated approval, and approval in 2015. If I heard that correctly on breakout discussion. Lots of talk about safety, history, and different chemistry. I couldn't make out the name of another company Imersan? which he said was an analogue on safety issues making FDA decisions tough.
Adam Feuerstein, who was there, tweeted it was ISIS mipomersen, which got approved, but with heavy labels for warnings for toxicity in US, but was denied approval in Europe-so you can get a thumbs up, or thumbs down, or approval with warnings (which affect doctor and patient choice)
Europe gives thumbs down to Sanofi's cholesterol drug mipomersen
March 22, 2013 | By Emily Mullin
Although recently approved by the FDA, Europe's Committee for Medicinal Products for Human Use has decided not to recommend approval for Isis and Genzyme's cholesterol-lowering drug mipomersen.
EMA committee shoots down Sanofi's cholesterol drug mipomersen
December 14, 2012 | By Ryan McBride
A European Medicines Agency panel handed down a negative opinion late this week on the antisense drug from Isis Pharmaceuticals for treating a genetic disease that causes blood cholesterol to jump, citing safety concerns such as signs of liver toxicity and cardiovascular risks in patients in clinical trials.