Does FDA ever send out any approval after 8pm, or 6pm? Just curious. How often do they miss the PDUFA date by several days? Heard that FDA likes to approve on Fridays. Could it be this coming Friday? I am kind of surprised that FDA missed the due date.
Its not ridiculous at all. Insurance already pays tens of thousands a month to keep CF kids alive and it gets worse and worse. Kalydeco will halt the progress and allow reduction in other meds and keep them out of expensive hospitals. The math says its worth it.
Sentiment: Strong Buy
" to the combination of lumacaftor and ivacaftor. We submitted the NDA and MAA in the U.S. and EU respectively in November 2014 that children and adults with CF ages 12 and older who have 2 copies of the F508del mutation. In the U.S. we received priority review with the PDUFA date of July the 5th 2015.
In the EU we were granted accelerated assessment and we anticipate EU approval around Q4 of this year if discussions with regulators progress as expected. All in regulatory approval in the EU individual reimbursement discussions will begin on a country by country basis....
scalped this from their Conference Call in January.... the other question for me post this Phase 2B 12 week study when will they raise cash for 2016 events... I am long since $87.00
Sentiment: Strong Buy
Does the patent for Ivacafter protect against someone using deuterated Ivacafor? If not I believe that Concert could develop and sell deuterated Ivacaftor without any agreement or assistance from Vrtx.
The title of your message is correct, but it is something that has not happened. The FACTS are that the insurance companies ARE already paying for the drug. Apparently, you are not well informed. I suggest you read the available facts and then if you have any common sense cover your short albeit at a loss. But that is at least better than losing a lot lot more if you wait too long. Oh and BTW don't tell me you are not short or you are not trading options!
Stock price moving to new highs today anticipating CF drug approvals with PDUFA for expanded pediatric label for Kalydeco due tomorrow, and earlier than anticipated 809/770 approval soon to follow........
The zinc-finger nuclease (ZFN) technology used by Crane et al. is a very old technology. Vertex is focused on gene editing technologies that are specific to the target gene (CFTR) and efficient unlike ZFN. But both technologies have to overcome huge problems of delivering to human cells in the body.
. . . repair (HDR). We observed an exquisitely sensitive, homology-dependent preference for targeting one CFTR allele versus the other. The corrected cystic fibrosis iPSCs, when induced to differentiate in vitro, expressed the corrected CFTR gene; importantly, CFTR correction resulted in restored expression of the mature CFTR glycoprotein and restoration of CFTR chloride channel function in iPSC-derived epithelial cells.
Please cite this article in press as: Crane et al., Targeted Correction and Restored Function of the CFTR Gene in Cystic Fibrosis Induced
Pluripotent Stem Cells, Stem Cell Reports (2015), http://dx.doi.org/10.1016/j.stemcr.2015.02.005
You are correct verity, if I remember correctly, a few years ago CFF contracted with Genzym, now part of Sanofy, to try the many compounds it had in its portfolio (3 M?) to see if any of them would be effective for CF. Then when CF monitized its royalty rights for VRTX's CF franchise, it indicated the reason was to fund CF research efforts of other drug companies. So all of the listings in their "pipeline" is funding of other companies' effort for CF drugs.
I am not aware of any partnership or agreement with VRTX. So how do they get hold of Ivacafter so they can modify it? Do they buy it from VRTX? Also even if they are successful in improving Ivacafter, they still need to negotiate an arrangement with VRTX since Ivacafter is patented. Unless they have an agreement it seems to me VRTX may only give them pittance for using its patented product -- that is assuming it is successful in being better.
FYI: a few of my other current medical science company investments: (Do your own due diligence before investing!)
MACK (Merrimack Pharmaceuticals) An oncology company with a it's first drug to be approved at the end of March or early April 2015..Large insider ownership with many shares recently purchased on the open market like the rest of us. Has a partnership with Baxter to market it's most advanced drug outside the U.S. Has a deep pipeline of other drugs that will change the paradigm for treating solid tumors on the molecular level, analogous to individualizing treatment of the various mutations of CF
DXCM (Dexcom) The leader in CGM (continuous glucose monitoring) in the treatment of Type 1 DM. Partnerships with insulin pump makers JNJ (Animus division) and Insulet, and working to eliminate the need for finger stick bloodsugar testing; Has apps to I-phone and Android smart phones allowing parents of pediatric patients, and caregivers monitoring of blood sugars to avoid highs and lows, preventing dangerous complications and hospitalizations. Collaborating it's technology to develop artificial pancreas closed loop insulin delivery system not requiring finger stick blood sugar testing. Just became profitable in the past quarter.
QDEL (Quidel Corp) I'm a fan. (hence my moniker). A leader in the point of care and molecular rapid diagnostics market. Has developed a whole new group of rapid POC (point of care diagnostics) that is starting to gain traction in sales for detection of multiple infections, Huge insider ownership and superb management. It's pipeline has a number of innovative new diagnostic platforms to test for multiple pathogens both quantitatively as well as qualitatively to monitor response to treatment of diseases like HIV.