30 days hath September, April June and November, all the rest, ie Dec and Jan have 31. 60 days is
up the day after Presidents Day, Feb. 18. So BCRX is 8 days after 60 day acceptance or rejection.
Happens BCRX reporting is, however, on same day as ACIP meeting, devoting first 2 hours to influenza updates at CDC Atlanta.
PLoS One. 2014; 9(1): e83400.
Published online 2014 January 8. doi: 10.1371/journal.pone.0083400
6 Minute Walk Test in Duchenne MD Patients with Different Mutations: 12 Month Changes
Seems as if the race to approval of Eteplirsen might be in the final Furlong-getting on board the bandwagon for this safe, effective drug as an example of "how the patient voice can best be utilized to speed access"
Webinar: How You Can Help PPMD’s Advocacy Agenda at Online Webinar - Eastern Time Zone
February 11, 2014 from 1pm to 2pm
PPMD’s advocacy strategy is multi-agency (FDA, NIH, CDC) and multi-layered. Yes, we focus on legislation like the MD-CARE Act which has transformed the landscape in Duchenne. But we also work to shape regulatory policies that reflect the needs of our community, meeting with the FDA and EMA about how the patient voice can best be utilized to speed access to potential therapies in Duchenne.Join Pat Furlong, Ryan Fischer and Dave Zook (FaegreBD) on February 11 at 1pm eastern to learn about PPMD’s…
Co called Biocryst (BCRX) had an IV antiviral, for flu cases, but a lousy phase III trial design because they were trying to enroll critical cases in hospital, which is difficult, so finally terminated trial, and said they would spend no more money on trials of their government sponsored drug development. Guess what - FDA said OK, we can use Japan clinical trials (which showed efficacy) and BARDA said they would fund the cost of filing
the NDA - so by playing hardball, got funding, filed an NDA Dec. 20, and now are on glide path to
FDA acceptance of the approach and data around Feb. 20. So, you do what you have to do with FDA,
like telling them as SRPT did last summer, we don't have enough extra drug to let people use it
under compassionate use - you either let us proceed with NDA or that's it for the patients. Now its up to the
FDA again - deny access to a drug with good safety profile and strong evidence of efficacy, backed up by recently released pulmonary function data, and be keelhauled by congress and patients advocates. Or use new data, and new people in key positions, to make a more rational judgement based on "new information" even if they have to craft some kind of open label trial/AA/ confirmatory reporting by users. Like Parkinson's debilitating disease require more than thumb twiddling if a possible therapy to halt downward spiral is available.
A FAIR HAND FOR THE FAST TRACK
Possibly because of fewer NDA submissions in 2013, the FDA approved only 27 new medicines last year compared to 39 in 2012. But one new issue drew even more attention than drug approvals during the year: expedited development. Of 120 applicants for breakthrough status, the agency awarded it to only 36 candidates. A separate, accelerated-approval program also disappointed some companies, as well as patients and their families.
A case in point was Sarepta’s drug eteplirsen for Duchenne muscular dystrophy (DMD). In the opinion of its supporters, the drug caused significant improvement in patients’ walking ability along with boosting the levels of dystrophin, a putative biomarker for DMD symptoms. When the FDA asked the company for more information from its dataset on eteplirsen to support its request for accelerated approval, the head of the main DMD patient group sent a long, petulant email to Woodcock in response.
No one expects Woodcock to comment specifically on the eteplirsen case, but she has this to say: “Sometimes people do pitch their case to me, and they’re just so convinced they have a breakthrough. They may come in with incredible clinical data, but we say, ‘Well, just repeat it, open label; take another 30 people and show you get the same results, and then we’ll talk about accelerated review. It depends on what the results are and how convincing the data are.”
Woodcock says she identifies with the patient-community advocates and could even see herself joining them if she faced a disease such as ALS or AIDS. “HIV was the instigation for accelerated approval; we said we were going to approve drugs based on a surrogate marker if it was reasonably likely to predict clinical benefit. And we’ve done a large number of accelerated approvals since then, many of them for orphan or rare diseases but also for cancer and so forth. But the data, even if it’s Phase 2, must be convincing.”
Data quality in general needs industry’s attention, Woodcock says. Academic data is especially problematic, with some estimates that up to 50 percent of it cannot be reproduced. She believes pharma companies are much more careful about validating the research data they generate, considering the money at stake in selecting development candidates.
Yet, she says, strange as it may seem, companies still routinely fall short with clinical research data, often because of poor practices by individual investigators. But poor trial design, rather than poorly run trials, may compromise trial results as well, she believes. A solution? More data transparency, ideally shared through a “trusted third party” or custodian as proposed by GSK and others, would allow companies to learn from past errors and successes, according to Woodcock Less
Don't need an offering to get cash - here's the sequence FDA acceptance with accelerated approval/advisory committee meeting/FDA approval in summer/stockpile order = each 10,000 courses is $20+ million,. say 30000 course = $60 million
And here's why all this will happen= sayeth Dr. Frieden of CDC in Time today
Bird Flu is no longer just China's problem
If H7N9 were to become a pandemic, there is a vaccine that researchers have developed, but Frieden says it’s not very effective and may require more than one dose, which could complicate distribution if there were an urgent need to immunize the population. But other measures for containing outbreaks that have proven effective in the past, including reducing the contact between people and infected animals, implementing social distancing strategies to prevent rapid spread of the virus, and dispensing medications to treat the severely ill, could help to contain an outbreak. “Flu reminds us that we are all connected by the air we breathe,” says Frieden. “There is no way for us to know if a pandemic will happen tomorrow or 10 years from now or never. But what we can do is be even better prepared than we have in the past if it happens.”
Following is the transcript of remarks made by the Secretary for Food and Health, Dr Ko Wing-man, after attending a public function today (January 29):
Reporter: (On measures the Government will take after confirmation of the fourth human case of avian influenza A (H7N9).)
Secretary for Food and Health: This is the fourth case of Hong Kong citizens contracting H7N9 virus after visiting the Mainland, mainly Shenzhen because of the geographical proximity. There is no way that we can stop the human traffic between the Mainland and Hong Kong. There are bound to be many HK citizens paying a visit to the Mainland, so the most important thing is to take precautions. If you are just a visitor to the Mainland, please avoid getting into contact with live poultry or wild birds and avoid visiting or getting close to wet markets with live poultry. If you are living or staying in the affected area for a longer time, and there is no way that you can avoid going to a wet market, then one should adopt more stringent personal protection, such as carrying a mask, and very importantly, washing hands before touching eyes, nose or mouth.
(The real problem is if there are any wet markets near pig farms - if H7N9 infected birds should mix, mingle, be fed to, eat from or otherwise potentially reassort with H3N2 or H1N1 infected pigs, we could be in deep
And Jack, good luck with the BOTA, I'll stick with Biocryst through the FDA determinations on or about Feb. 20. to accept or accelerate peramivir NDA approval.
The huge price difference in 4G services between the Chinese mainland and Hong Kong has drawn criticism of and suspicion about Chinese telecom operators.
"I do not think it is fair for the same company to charge differently for similar services," said 27-year-old Zhang Hui in Shanghai. She said she wanted the speed that 4G can provide but balked at paying the high cost.
"But what can you do? We do not have a choice," she said.
Gao Shu, a spokeswoman for China Mobile's Beijing branch, refused to comment on the monthly fee, saying that it is a subject for users.
Gao added that China Mobile does have an extra package of 180 yuan for 5GB of mobile data. There will be 1GB free of charge, if subscribers make the purchase before March 31.
Telecom analysts on the mainland attributed the high initial cost to the lack of real competition. In Hong Kong, they said, the stiff competition among the many carriers and service providers is instrumental in keeping prices low.
Jane Zhang, principal research analyst in carriers and mobile services with Gartner Inc, a US information technology research and advisory firm, said it is comparatively easier for carriers to promote 4G services in Hong Kong, given the benign infrastructure and popularity of mobile Internet. However, the mainland requires a huge initial investment to cultivate the market.
"For the time being, the 4G network by China Mobile can only cover major parts of Beijing. It takes time before the services and network become mature. The company has to set a high price to cover the initial cost."
Ashley Sheng, a telecom analyst with Shanghai-based securities company Shenyin & Wanguo Securities Co, said the price gap comes from the fierce competition among various telecom operators in Hong Kong.
"China Mobile is in a monopoly position on the mainland, so it can set a very high price for the new technology. While in Hong Kong, where 4G has been quite popular and China Mobile has so many local competitors, the company has to follow the local scenario."
April 18 Forbes discussed Taiwan's warily watching H7N9 on mainlaind
Currently, there are daily briefings being held at the CDC’s Central Epidemic Command Center in Taipei, where updates on China infection numbers and the latest figures are presented to the media.
Taiwan’s CDC had earlier formulated a National Influenza Pandemic Preparedness Plan, which details when and how measures are rolled out in the face of a pending and actual pandemic occurrence. This blueprint is driving the process at the moment. Such measures cover border control, agricultural measures including testing birds and livestock, tightening access to farms and netting off open-air duck farms, for example. Already a ban on the killing of live poultry in traditional markets is set to be in effect in mid-June, although the ban would be implemented immediately if a human case of the flu emerges on the island before then. Other measures include checking and stockpiling antivirals such as Tamiflu, Relenza and Rapiacta, and putting a plan in place to produce a vaccine.
Cubist says FDA will make a decision on its skin infection treatment tedizolid by June 20
December 30, 2013 10:24 AM
LEXINGTON, Mass. (AP) -- Federal regulators have accepted an experimental antibiotic from Cubist Pharmaceuticals for expedited review, the company said Monday.
That means that a decision on Tedizolid could come from the Food and Drug Administration by June 20.
The antibiotic designed to treat serious skin infections. Cubist acquired tedizolid when it bought Trius Therapeutics in September. The deal was valued around $818 million.
Shares of Cubist Pharmaceuticals Inc. rose 12 cents to $67.48 in morning trading.
louie 27 days ago 0 1
I had a post surgical MRSA infection four years ago.
I took your antibiotic "Cubicin" on a daily basis for two weeks through an IV.
It saved my life....Thank You.
Did Wells Fargo even mention peramivir?
their Jan 23 slides have lengthy discussions of antivirals,but limited availability of peramivir
2013-2014 Influenza Season: Updates and Recommendations for Clinicians
Which leads to reasonable expectation that FDA review will be accelerated, not standard, when announced in Feb. - another data point will be ACIP Feb meeting
This influenza season, CDC has received a number of reports of severe respiratory illness among young and middle-aged adults, many of whom were infected with influenza A (H1N1) pdm09 (pH1N1) virus. Multiple pH1N1-associated hospitalizations, including many requiring intensive care unit (ICU) admission, and some fatalities have been reported. For the 2013-14 season, if pH1N1 virus continues to circulate widely, illness that disproportionately affects young and middle-aged adults may occur. Annual influenza vaccination is recommended for all persons aged 6 months and older, and is the best way to prevent influenza. However, available evidence consistently indicates that antiviral treatment, when initiated as early as possible in patients with confirmed or suspected influenza, can reduce severe outcomes of influenza. During this COCA conference call, critical care physicians will comment on their recent experiences caring for patients with severe influenza, and a CDC subject matter expert will summarize the 2013-14 season to date and review CDC recommendations for health care providers including the use of antiviral medications for the treatment of influenza.
(Will anyone ask them if an IV antiviral would be useful?)
What are Filoviruses?
Filoviruses belong to a virus family called Filoviridae and can cause severe hemorrhagic fever in humans and nonhuman primates. So far, only two members of this virus family have been identified: Marburg virus and Ebola virus. Four species of Ebola virus have been identified: Ivory Coast, Sudan, Zaire, and Reston. Ebola-Reston is the only known filovirus that does not cause severe disease in humans; however, it can be fatal in monkeys.
Publish filovirus infection animal model POC data for BCX4430 (1Q:14)
Acceptance of peramivir NDA file by FDA (1Q:14)
BCX4161 posters at AAAAI (1Q:14)
Before the JPM14 presentation, there was not a definite date range for the filovirus publication, FDA acceptance, or posters. Now these are all projected for the first quarter of 2014, could drive BCRX to
Not sure that FDA would be subject to a lawsuit, but possible congressional hearings. The problem is that some in FDA reason by analogy with regard to drugs targeting same disease when the
PK and MOA are different (witness all the holding back on HepC from one company's problems) If they don't have a good understanding, they lump everybody under one concept, when they are in fact not comparable as to SAEs, pharmacology, etc. But the patients who are the ones harmed by unjustified denial can keep the pressure on the FDA. Patients and efficacy for them are what its really all about, not shareholders.
If we can see effect on exposure and correlating effect on enzyme we know we'll have a question of when, not if we have a product or something like that - results by June 2014