Nathan Brown on 05/20/2015 ... On May 14, 2015, The House Energy and
Commerce's Health Subcommittee approved an ... that are meant to expedite the
development and release of new medical treatments, cures, and devices. ... This
revised draft of the bill, as approved by the subcommittee, includes a proposal
could see something anytime or by months end i would think.
Sarepta Therapeutics Completes NDA Submission to FDA for Eteplirsen for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 SkippingBusiness Wire (Mon, Jun 29)
Keep in mind that it really only takes 24 weeks to confirm that the drug is working...
Condition Intervention Phase
Duchenne Muscular Dystrophy
Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A 48-Week, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy
Possible class approval for Sarepta.You must remember that all the exons use the same drug ,it is only sequenced differently per exon..... One must think now that Sarepta's share price is now worth 3 times of the highest upgrade...fyi
For years, almost all attention surrounding Sarepta Therapeutics has been on its drug for the most common gene mutation that causes Duchenne muscular dystrophy, called eteplirsen. This week, the company quietly disclosed plans to start late-stage trials of two further drugs for other genetic causes of DMD.
Together, the three experimental drugs would treat about 31 percent of the total worldwide patients with the rare disease. That’s more than double the number that would be treated by eteplirsen, which is now being considered for approval in the U.S.
“We’re really filling out our pipeline,” said Ian Estepan, a spokesman for Cambridge-based Sarepta (Nasdaq: SRPT).
The company’s goal in the long run has always been to develop drugs to treat as many different mutations as possible that cause the disease, which degenerates muscles to the extent that patients are forced to use wheelchairs by the age of 12. Eteplirsen is aimed at boys with a mutation in a part of the DNA called exon 51. That’s the most common one, responsible for about 15 percent of patients with the disease, and is also the target population of a competing drug developed by California-based BioMarin Pharmaceuticals (Nasdaq: BMRN). Both drugs will likely be reviewed by an FDA advisory committee later this year.
Sarepta Therapeutics Announces First Patient Dosed in European Phase I/II Study of SRP-4053 in Duchenne Muscular Dystrophy PatientsBusiness Wire (Wed, Jan 14)
class approval ,,to costly for all exons one by one..after all we are talking about the same drug ''just a different sequence .Say you have 12 master locks and all the locks are alike but have different keys ''should only need a trial for that one lock and that one key only.
Further study details as provided by National Institutes of Health Clinical Center (CC):
Primary Outcome Measures:
•To evaluate the safety and tolerability of AVI-7100 in healthy adults, following single- or multiple-dose, intravenous administration at escalating dose-levels [ Time Frame: 2 years ]
Secondary Outcome Measures:
•To evaluate the pharmacokinetics of intravenously administered AVI-7100 in healthy adults, following single- or multiple-dose, intravenous administration at escalating dose-levels [ Time Frame: 2 years ]
Estimated Enrollment: 150
Study Start Date: November 2012
Estimated Study Completion Date: December 2016
Estimated Primary Completion Date: December 2016 (Final data collection date for primary outcome measure)
Drug: AVI-7100 versus placebo
dose-escalating AVI-7100 versus placebo
Despite currently available antivirals, influenza causes significant morbidity and mortality (estimated 36,000 deaths each year in the United States alone). More agents are needed in the armamentarium of anti-influenza medications including novel mechanisms of action and parenteral agents.
This study will evaluate the safety of a novel RNA based therapeutic AVI-7100. Beginning with a low single-dose, subjects are randomized to receive either AVI-7100 or placebo and evaluated on Study Days 0, 1, 2, 4, 10, 28. The safety and tolerability is evaluated using symptoms, clinical laboratory tests, ECG, and pharmacokinetics. Utilizing a series of stopping rules and a medical monitor, the dose will be escalated as safety and tolerability are established. Once the maximum tolerated dose (MTD) is established in the first part of this study (either limited by adverse effects or up to maximum anticipated dose), the safety and tolerability of multi-dose administration will begin (replicating anticipated clinical use). Subjects again are randomized to either AVI-7100 or placebo daily for 5 days, and evaluated on Study Days 0, 1
The stock is going to do this until more news...can't do nothing but wait''''short term holders that take profits and shorts sell along with it but long term is outstanding.. healthy trading
in the 100-s range next year...,,who cares about the short term games really.. if youre long...
and this older one.
this site is also behind.
fda site clinicaltrialsdotgov
Active, not recruiting Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy Condition: Duchenne Muscular Dystrophy (DMD)
Intervention: Drug: AVI-4658 (Eteplirsen)