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
History of Changes
Looks like HHS is getting ready to order antivirals April 8 FBO
April 8 Notice of Intent to Sole Source Contract 1 year plus 4 years of options for tamiflu
Added: Apr 08, 2015 3:26 pm
The Department of Homeland Security (DHS), Office of Procurement Operations (OPO), intends to issue a sole source, firm-fixed price contract under the authority of FAR 6.302-1, "Only one responsible source and no other supplies or services will satisfy agency requirements." The Government intends to award to Genentech USA, Inc. at 1 DNA Way, South San Francisco, CA 94080 for the antiviral medicine Oseltamivir (trade name Tamiflu).
DHS, Office of Health Affairs (OHA), requires this antiviral medicine for the DHS Antiviral Stockpile for pandemic and avian influenza preparedness. The stockpile is needed in order to have a readily available supply of medicine to protect DHS personnel in the event of a pandemic to ensure the continuity of its essential services and overall critical mission. The medicine must be approved by the Food and Drug Administration and demonstrated by the Centers for Disease Control to be effective in treating Influenza A, H1N1 Influenza and H5N1 Avian Influenza. Market research has shown that no other known contractors presently exist who could get the necessary CDC/FDA approvals to produce the product in sufficient quantities and deliver it in time to meet the current requirement.
Expedited Access Guidance excerpt from April 13 Guidance for
Devices treating serious conditions with unmet need.
In some cases, patients may be willing to assume greater risk for earlier access to a medical device. This may be particularly true for patients with conditions for which no treatments exist or
those who face serious or life-threatening conditions.
FDA must ensure that if access to medical devices is expedited, FDA is reasonably assured the device is safe and effective. Reducing premarket data requirements while increasing postmarket requirementsfor devices subject to a PMA, when appropriate,can assist FDA in making medical devices available to patients sooner than if following the traditional premarket review pathway…
The Data Development Plan should outline all data the sponsor
intends to collect in support of device approval including what data will be collected premarket and postmarket.
FDA’s EAP program contains features from CDRH’s Innovation Pathway, piloted in 2011 to facilitate the development and expedite the review of breakthrough technologies.
In addition, the EAP programis based in part on FDA’s
experience with the Center for Drug Evaluation and
Research (CDER) and Center for Biologics Evaluation
and Research (CBER) programs that are intended to facilitate and expedite development and review of new drugs to address unmet
medical needs in the treatment of serious or life-threatening conditions(“FDA drug expedited programs”).
(Comment-presumably part of any expedited/AA Sarepta NDA is a pre- and post market data plan for eteplirsen.)
In Egypt, as of 6 April 2015, the Ministry of Health and Population has reported 134 human cases of influenza A(H5N1), including 38 deaths. Since 2006, Egypt has reported 336 human cases, according to the World Health Organization (WHO)/Food and Agriculture Organization of the United Nations (FAO).
The number of laboratory-confirmed human cases of avian influenza A(H5N1) virus infection in Egypt with onsets of illness from January to March 2015 is the highest reported for a three-month period since the start of the epidemic in 2006. New cases have been reported every month since the surge that started in November 2014 (Figure). No similar surge in cases has been reported in any other country.
1. Parent pressure and quality of life reports demonstrate effectiveness of eteplirsen, without significant safety issues.AA approval has been created for such unmet medical need of serious progressive disease.
2. 6Mwt data on which Prosensa/Biomarin rely are at least as variable and dependent on treatment exogenous factors as dystrophin measurement: height, weight, other steroid treatment, age, maturity, etc.
3. No use having multiple advisory committees: FDA wants to move forward and so one DMD therapy advisory committee, sometime second half of 2015, after both NDAs on file, likely factors into FDA decision. And if both drugs are approved, patient choice and competitive pressure on price (no single treatment premium cost to patients and insurance companies) would make a lot of sense.
Of course, all of this assumes the people making the decision have a lot of sense. But at least the FDA CDER knows it is under intense scrutiny by patients, families, congress.
Avian Flu Diary has published an on-line report picked up from several news sources in Viet Nam of 2 people infected with the recently emerged A/H5N6 avian virus in Nghe An Province. Dr. Michael Coston, [editor, Avian Flu Diary] feels the report is accurate, as just over 2 weeks ago, the virus was reported in the adjacent province to the north (see comment below).
An outbreak of avian influenza A / H5N6 outbreak in Quy Hop district, Nghe An, caused an infection in 2 people. The Local Veterinary Authority has issued 11 000 doses of vaccine to control the outbreak. The source Pioneer said outbreaks of avian influenza occurred in the neighborhood of Dinh Xuan Nghia, Quy Hop district, Nghe An. It is a location with concentrated population.
Rapivab for flu and Flu(v). ie variants and worse, HAE drug and followon; Filovirus /Ebola/Mers possibly via animal rule pathway. Looks fine to me. Now just wait for earnings and stockpile sitting patiently,buy on dips not on margin.
Its an issue of process under 2106. But the exon skipping proceeds by different processes - the materials (PMO) are different. All BMRN can do is litigate and delay, but if they do that, the patients and mothers will chain themselves to Biomarin's doors. You can't patent uranium fission and then claim rights over plutonium fission by claiming the idea of fission.
Step 1: Is the claim directed to one of the four patent-eligible subject matter categories: process, machine, manufacture, or composition of matter? The subject matter of the claim must be directed to one of the four subject matter categories. If it is not, the claim is not eligible for patent protection and should be rejected under 35 U.S.C. 101, for at least this reason. A summary of the four categories of invention, as they have been defined by the courts, are:
i. Process – an act, or a series of acts or steps. See Gottschalk v. Benson, 409 U.S. 63, 70, 175 USPQ 673, 676 (1972) ("A process is a mode of treatment of certain materials to produce a given result. It is an act, or a series of acts, performed upon the subject-matter to be transformed and reduced to a different state or thing." (emphasis added) (quoting Cochrane v. Deener, 94 U.S. 780, 788, 24 L. Ed. 139, 1877 Dec. Comm'r Pat. 242 (1876))); NTP, Inc. v. Research in Motion, Ltd., 418 F.3d 1282, 1316, 75 USPQ2d 1763, 1791 (Fed. Cir. 2005) ("[A] process is a series of acts." (quoting Minton v. Natl. #$%$’n. of Securities Dealers, 336 F.3d 1373, 336 F.3d 1373, 1378, 67 USPQ2d 1614, 1681 (Fed. Cir. 2003))). See also 35 U.S.C. 100(b); Bilski v. Kappos, 561 U.S. ___, 130 S. Ct. 3218, 95 USPQ2d 1001 (2010).