I want more stock here........................................haaa
after years of research i agree with them..............The first drug that has ever worked to slow the progression of DMD..facts of science and natural history.
CDER April 25, 2016 8:00 a.m. to 5:30 p.m.
College Park Marriott Hotel and Conference Center
3501 University Blvd. East
Hyattsville, MD 20783
The committee will discuss new drug application (NDA) 206488, eteplirsen injection for intravenous infusion, sponsored by Sarepta Therapeutics, Inc., for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.
FDA intends to make background material available to the public no later than 2 business days before the meeting. If FDA is unable to post the background material on its Web site prior to the meeting, the background material will be made publicly available at the location of the advisory committee meeting, and the background material will be posted on FDA’s Web site after the meeting. Background material is available at: 2016 Meeting Materials, Peripheral and Central Nervous System Drugs Advisory Committee
Public Participation Information
Interested persons may present data, information, or views, orally or in writing, on issues pending before the committee.
Written submissions may be made to the contact person on or before April 11, 2016.
Oral presentations from the public will be scheduled between approximately12:40 p.m. and 2:40 p.m. Those individuals interested in making formal oral presentations should notify the contact person and submit a brief statement of the general nature of the evidence or arguments they wish to present, the names and addresses of proposed participants, and an indication of the approximate time requested to make their presentation on or before April 1, 2016.
Time allotted for each presentation may be limited. If the number of registrants requesting to speak is greater than can be reasonably accommodated during the scheduled open public hearing session, FDA may conduct a lottery to determine the speakers for the scheduled open public hearing s
Friday, April 15, 2016
Race for the first drug approval by FDA for treating Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) is an inherited disorder that involves muscle weakness, which quickly gets worse. Duchenne muscular dystrophy is caused by a defective gene for dystrophin (a protein in the muscles). However, it often occurs in people without a known family history of the condition. Because of the way the disease is inherited, it usually affects boys. Duchenne muscular dystrophy occurs in about 1 out of every 3,600 male infants. DMD is a rare disease and is an orphan disease indication for the drug development.
In June 2015, FDA issued its draft guidance for industry “Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment” which delineated the details about FDA’s thinking about the drug development for treating Duchenne Muscular Dystrophy.
FDA recognized that Duchenne Muscular Dystrophy is life-threatening and severely debilitating disease with unmet medical need (no FDA-approved drug up to this date). The statutory standards for effectiveness apply to drugs for dystrophinopathies just as they do for all other drugs. FDA has long stressed, however, that it is appropriate to exercise flexibility in applying the statutory standards to drugs for serious diseases with important unmet needs, while preserving appropriate guarantees for safety and effectiveness.
Sarepta is grossly undervalued so much ...platforms worth billions..The drug Eteplirsen is so under estimated
My opinion ,The 36 experts already had a real adcom ,,,It is no better true experts out there for dmd disease that knows more than the 36.
Could be anything now...........................................................................................getting real
Primary Outcome Measures:
Change in 6-Minute Walk Test (6MWT) distance from baseline [ Time Frame: Baseline, Weeks 12, 24, 36, 48 ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
The percentage of dystrophin-positive fibers [ Time Frame: Baseline, Weeks 24/48/72/96 ] [ Designated as safety issue: No ]
Maximum inspiratory/expiratory pressure percent predicted (MIP/MEP % predicted) [ Time Frame: Baseline, Weeks 12, 24, 36, 48 ] [ Designated as safety issue: No ]
Other Outcome Measures:
Evaluate the long-term effects of eteplirsen up to 96 weeks [ Time Frame: Week 1-96 ] [ Designated as safety issue: No ]
Estimated Enrollment: 160
Study Start Date: September 2014
they also think if bad news comes they can make money also. Can they really cover that quick with 18million short ? after hours covering? huh the way it looks it could rise 28 to 32.00 a share more overnight or in 5 hours or less
I am wondering if they are going to show some a week before adcom or during adcom or just when new briefing documents arrive ?or after adcom/
It would be correct to include new data on more boys and a notice to review ahead of adcom ..fyi
Knowing the science here drives me , many can't understand and that is the only reason it has taken so long with avi/sarepta and of coarse some bad ceo-s did not help. Just because some see the real future of new direct drugs does not make them a broken record ,it makes them way ahead of others and over excited.
Growth from here short term...could be any price in 3 years ,the sky is the limit
I guess they don't in any way want to step on the FDA in any sensitive manor.
it was put off in the past and the lack of not knowing the science is the only problem ''The 36 recent dmd experts tried and hopefully got through to the not knowing to help clarify to spaces in the brain of those empty heads.