Verity. Thanks for your thoughtful and insightful reply. I realize how very frustrating delays like this are to you and the CF community affected by these decisions. Obviously, the FDA has to be cautious with young children, but the safety of Ivacaftor in the pediatric population is well established and giving the drug a conditional approval requiring clinical phase 4 data to document efficacy over a longer time period in all 23 residual function mutations would give the proof the FDA is demanding and save lives at the same time. Maybe the White House advisers with an interest in 'personalized medicine' could be asked to have the FDA submit this idea to an FDA advisory committee to allow input from experts and the CF community on the idea of an expedited conditional approval for this sub-population of CF patients likely to benefit?
Verity- Are CF advocacy groups not expressing their concern to congressional representatives with FDA oversight over this FDA decision regarding the rejection of the sNDA to treat residual function mutations with Ivacaftor?
The sad part is that these residual function CF patients' health will deteriorate further waiting even though the efficacy and safety in the use of this drug has been proven. The FDA's bureaucracy is now demonstrating that 'fast track' and 'breakthrough' designation status offers little in terms of expediting approval. Ivacaftor was the first drug to get this designation by the FDA. Seems to me the FDA is clearly not making this designation a path for expedited approval, as was intended by the law that by creating this designation, was mandating streamlining the approval process for a drug that makes such a difference in the lives of patients.
With today's announced rejection by the FDA of the sNDA submitted by VRTX to treat CF patients ages 2-5 with residual function mutations with Ivacaftor, it is clear why it drugs like Ivacaftor cost so much. Vertex bears the financial burden of additional clinical trials to prove what is already apparent with regard to Ivacaftor's efficacy and safety in this CF subpopulation, which is then factored into the drugs pricing, and the affected CF patients are forced to wait even longer to receive this life saving treatment. Interesting that Elijah Cummings and Hilary do not seem to make comments about the impact that these kind of FDA decisions have on drug prices
Estimates also get revised up as well, depending on news flow.Let's see what happen to these estimates on Feb 7. They will likely go up when 1500 more CF patients with residual function mutations in the US are approved for treatment with Kalydeco monotherapy. Represents 400+ million more in revenue. Factor in approval of 2500 508dd CF patients ages 6-11 likely to be approved for treatment in the US with Orkambi later this year, and you can double that additional revenue number over the next two years. This is on top of the continued adoption of use of Orkambi by the great majority of 5088dd patients approved for use in patients ages 12 and older in this country and the EU and Canada. Election rhetoric about the cost of pharmaceuticals will die down after November, and if Republicans maintain majority control of the House, it will not likely translate into any legislation to control drug pricing which would crush risk taking destroying advances research and innovation in medicine.
Last night's CC made it clear that VRTX will not risk giving Orkambi sales estimates for 2016 yet, and that created uncertainty. So as a consequence the street is punishing the stock price by pricing in any potential 'disappointment' in case sales in the future were to disappoint. Great opportunity to buy since on Feb 7 FDA will likely expand Kaleydeco monotherapy to residual function CF patients and Orkambi will likely get approval for treating children ages 2-6 later this year. None of this is factored into 2016 guidance. Today's selling is just another sign of biotech bubble deflating and all moves are exaggerated. Great for traders, and buying opportunity for long term investors. My disappointment is lack of discussion of the non- CF pipeline and it's potential on top of the steady growth in revenue expected from CF throughout the year.
Thank you for sharing your kids experience. Clearly Orkambi does provide some benefit while waiting for the optimial next generation of treatments. While waiting for 661, Pharion's drug, and 2nd gen correctors, and perhaps someday CRISPR treatments, at least in the meantime, Orkambi can slow progression of the damage to your kids lungs, and the rest of the 508dd CF patient population who see some benefit. Insurance companies cannot stop paying for drugs that show benefit in patients. My best wishes to you and your family as you await further breakthroughs in CF treatment from Vertex.
How are your kids doing on Orkamkbi after being on it for a few months? It reportedly decreases the rate of re-hospitalizations (but does not eliminate the need for any future hospitalizations) and is the first drug to hopefully slows the progression of loss of lung function in 508dd CF patients. Do you see any benefit for your kids?
Pre-release of Q42015 revenue beat and guidance for 2016 at high end of expectations portends a positive analyst response to this afternoon's presentation at JPM. Positive Market response so far this morning will likely have follow through with analyst pushing up price targets based on what they hear this afternoon.
Not a word from TSC's article tonight by Adam Feuerstein, about all the drugs Vertex is entering in clinical trials in CF and multiple other diseases representing breakthrough drug potential in the treatment of multiple cancers, spinal cord injury, pain, influenza, sickle cell anemia, as well as well as improving treatment for the entire CF population. Only a headline and discussion about the deferral of guidance regarding Orkambi sales until later this year when more data is known about Orkambi prescription approval and renewals. Either AF has a bias against Vertex favoring shorts, and doesn't want to give the complete picture of Vertex 2016 business plans beyond Orkambi and Kalydeco projected sales or he's just too lazy to give his readers the complete picture of Vertex diverse and robust pipeline that is actively being developed with the proceeds from it's CF franchise. He also neglects to mention the ongoing reimbursement negotiations in the EU that will enhance Orkambi revenues throughout 2016 and the pending FDA approval of use of Kalydeco in residual function mutations in the US and in approved mutations in patients ages 6-11 which will double the treatable population with Kaldeco monotherapy by year end to over 8000 patients. The company's revenues and earnings will be exploding upward over the next few years on CF alone. Factor in the potential of the non-CF pipeline and the stock is hugely undervalued. Hopefully analysts tomorrow will spell out the complete picture.
Happy New Year to all patient longs! Leiden's presentation next Monday morning at JP Morgan will likely lay out the broad business plan and R&D pipeline milestones planned for 2016 and beyond. Analysts will finally be hearing about the non-CF pipeline in more detail and hopefully will start adding potential value for non CF assets in development for treatment of cancer, pain, spinal cord injury, Alzheimers, Hungtington's disease, influenza, to their future price targets for VRTX in addition to the value from the expanding CF drug portfolio in development (including Pharion's drug and 2nd gen CFTR correctors to treat the 'het min' CF population). Growing revenue and becoming profitable this year from growing Kalydeco and Orkambi sales in the US and abroad and NDA submission for VX 661 this year will make Vertex stand out as a biotech investment this year and going forward. Lots to look forward to in clinical trial results in the first half of the year!
Yahoo will not allow posting cut and paste from the tumbler web site. You can find the blog under "dreaming of breathing" when you type orkambi blog in the google search bar.
Actually side effect and drop out rate (about 4%) are about the same as in TRAFFIC and TRANSPORT Phase 3 trials on which FDA approval was based for Orkambi. Patients are aware that adverse reactions usually go away over a period of weeks and patients are significantly improved thereafter in rates of serious infections weight gain, quality of life, and re-hospitalization. As a results, the great majority of CF patients and their caregivers are prepared for tolerating the initial adverse respiratory effects noted in the first weeks of Orkambi treatment as the patients mobilize the chronic infected secretions in their lungs, and finally benefit from the drugs effects in reversing the underlying cause of the illness by improving CFTR function. The blog below is from a young adult CF patient that exemplifies the Orkambi experience in the majority of CF patients.
In short it keeps reinvesting in R&D to further improve it's technology to maintain it's lead in this market. The investment community sees the potential for CGM replacing finger sticks, expanding market in the type 1 market which is only 20-30% penetrated and with the google collaboration the potential to expand market to the vastly larger type 2 DM market.
Nice to get this recommendation going into the JP Morgan healthcare investor conf in three weeks
Everyone is entitled to opinions. His theme is that the DXCM's CGM platform will be made obsolete by competitor's devices yet to be developed and FDA approved. As you are aware, the majority of Type 1 diabetics that are using CGM are using and prefer
Dexcom CGM over MDT Abbott etc despite the larger resources these companies have. The reasons for DXCM leading in market share and rate of growth are numerous, with most important factors being accuracy and blue tooth connectivity ease of use and customer service. The reason the company is not yet profitable is simply Dexcom being focused on further advancing it's CGM platform, so that the competition continues to have to compete against a new higher standard offered by Dexcom allowing it to continue it's dominance in this market. IMHO it's evolving CGM is anything but becoming obsolete. Fourth qtr revenues will show it's impressive growth in the Type 1 DM market. Have patience and you will be rewarded.