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Medicare will cover pioneering cancer treatment nationwide Previously, regional administrators decided whether to cover the therapy.
Seema Verma, administrator of the Centers for Medicare and Medicaid Services, announced Wednesday that Medicare will cover CAR T-cell therapy, a groundbreaking treatment for blood cancers, nationwide. (Jabin Botsford)Seema Verma, administrator of the Centers for Medicare and Medicaid Services, announced Wednesday that Medicare will cover CAR T-cell therapy, a groundbreaking treatment for blood cancers, nationwide. (Jabin Botsford) By Laurie McGinley August 7, 2019 at 9:38 PM EDT Medicare will cover an innovative but high-priced cancer treatment nationwide, a step Trump administration officials said Wednesday would ensure patients have “consistent and predictable access” to a potentially lifesaving therapy.
In announcing the decision, Seema Verma, administrator of the Centers for Medicare and Medicaid Services, said that until now, Medicare’s regional administrators had decided whether to cover the treatment, which led to confusion.
Verma said the agency, which had scheduled this coverage decision originally for late May, has been struggling to figure out how to cover and pay for the treatment, called CAR T-cell therapy. The treatment costs $375,000 or $475,000, depending on whether it is used for advanced lymphoma or pediatric leukemia. Hospital stays can add hundreds of thousands of dollars to the cost of care. I RECALL TOTAL COSTS CAN APPROACH $1 MILLION Drugmakers note CAR T-cell therapy is designed to be given just once and to be a potential cure for patients who have run out of other options. But not all patients benefit from it and because it is so new, it’s too soon to know whether it will deliver long-term cures.
Such expensive treatments are “begging the question of how the system is going to pay for this over the long term,” Verma said, especially given Medicare’s considerable financial strains. “This is something we are extremely concerned about,” she added.
The coverage decision announced Wednesday is different in some important aspects from an agency proposal from February. That proposal called for “coverage with evidence development” — which would have required hospitals to collect and report data on patient outcomes over a long period. Hospitals said the data collection would be overly burdensome.
CMS’ final decision dropped that requirement. Instead, the agency said it will rely on patient information collected by the Food and Drug Administration and the National Cancer Institute. The FDA is requiring the two manufacturers of the therapy — Novartis and Gilead Sciences — to follow patients for years and report on outcomes. Such data will be put in a registry supported by NCI.
The Medicare decision also said it will cover the therapy when it is administered at health care facilities enrolled in an FDA-mandated safety program requiring special training on handling side effects. That means the treatment could be given on an outpatient basis, which would be much less costly.
The American Society of Hematology praised the decision, especially the elimination of the “coverage with evidence development” requirement. It said the proposed mandate would have prompted some hospitals not to provide CAR T-cell therapy.
The group also praised the decision for saying Medicare would cover all FDA-approved uses for CAR T-cell therapy and off-label uses recommended in CMS-approved compendiums, which are used to determine medically accepted uses of drugs and biologics.
The Medicare decision was the second step in a week taken by CMS to bolster CAR T-cell therapy. Last Friday, the agency said it would increase the reimbursement for CAR T-cell therapy, but didn’t go as far as many hospitals had wanted. Medical centers have complained they are losing money on Medicare patients because reimbursements have been too low.
The FDA approved two versions of CAR T-cell therapy, which stands for chimeric antigen receptor T-cell, in 2017. Kymriah is made by Novartis for certain types of lymphoma and childhood leukemia, and Yescarta is manufactured by Gilead Sciences for lymphoma. Other CAR T-cell therapy products are in development.
The complicated treatment involves extracting and genetically altering the patient’s T cells to attack a protein on the surface of cancer cells. The cells are then infused back into the patient
The response (IMHO very LIKELY MORE SHORTED in last 48 hrs as dry powder was absorbed:) as AXVL will be directly proportional to the strength of AVXL news on trial and other company progress.
I hope Missling knocks it out of the park!(Some post CC: Ambiguous; non transparent; extended trials[
Those recently affilated with nGBM( and DCVAX L clinicals closed)): CAN & WILL NWBO accept them under FDA's Project Facilitate (see below)since we know FDA Right to Try: RTT is a NO: NWBO can not with their limited resources do it for free)
How to Gain Access to Cancer Drugs Not Yet Approved by the FDA
'Project Facilitate' helps streamline the process for doctors' requests
In June 2019, the Food and Drug Administration (FDA) launched the federal government’s latest initiative to make it easier for Americans who have serious illnesses to gain access to experimental drugs.
Dubbed Project Facilitate, it’s a call center that oncologists can use to request help from the FDA in getting these drugs for their cancer patients from the companies developing them. Project Facilitate comes just one year after President Trump signed the “right to try” law, designed to give terminally ill patients the ability to use drugs before they’re approved by the FDA.
Despite these efforts on the part of the government, though, there’s still one big hurdle standing between patients and unapproved drugs: pharmaceutical companies can still decline requests for them. Even the FDA acknowledges that can be a challenge.
Project Facilitate is designed to make the process of requesting experimental drugs “as streamlined and efficient as possible,” says FDA press officer Amanda Turney, but the company developing the product “has the right to approve or disapprove the physician’s request.”
Doctor Says: First Try Clinical Trials
That’s just one reason many physicians who treat terminally ill patients recommend they try to get into clinical trials for investigational products before attempting to secure those drugs directly from the manufacturers.
“I favor clinical trials more than anything, because that’s how we learn whether drugs will truly be efficacious for patients,” says Dr. David Hong, director of investigational cancer therapeutics at the MD Anderson Cancer Center in Houston.
When patients try drugs outside of closely monitored trials, “there can be unseen consequences,” Hong says, such as side effects that physicians don’t know how to manage because of a lack of experience with the medications.
Patients with any disease can search for clinical trials online at ClinicalTrials.gov. Several cancer-focused nonprofits, such as BreastCancerTrials.org (in collaboration with the Susan G. Komen Foundation), offer free online tools that match patients to clinical trials based on the specifics of their diagnoses, as does the website EmergingMed.
Hong recommends cancer patients first request help identifying appropriate clinical trials from the oncologist managing their treatment plan. But it’s also important to get a second opinion, ideally from a large cancer center that runs several clinical trials. Why?
“There are thousands of new drugs in development,” Hong says, and there are so many clinical trials starting up all the time that your oncologist may not have the most up-to-date information.
Newly diagnosed cancer patients should also request “next-generation” gene sequencing of their tumors, suggests Dr. Leonard Lichtenfeld, interim chief medical and scientific officer of the American Cancer Society. These tests can identify hundreds of genetic irregularities, such as mutations, that may be targetable with experimental therapies — if not now, then perhaps sometime in the future.
“Say you have a genetic analysis done and there’s no trial available” of a drug targeted at your genetic results, Lichtenfeld says. “Six months later, you may find that a trial exists where there was none before.”
Working With Drug Companies
Another good reason to insist on next-generation gene sequencing is that pharmaceutical companies will often go out of their way to get targeted drugs to patients with rare cancers. That was the case when a small company called Loxo Oncology started developing what is now Vitrakvi (larotrectinib), a drug that targets tumors with a rare abnormality called NTRK gene fusion.
For several years, Loxo (now owned by Eli Lilly) worked with oncologists all over the world to enroll people with the abnormality in the clinical trials — even patients who lived nowhere near the main sites where the drug was being tested. The drug was approved by the FDA last November based on clinical trial data showing that 75% of patients in the trials had a partial or complete response to it.
Not all companies are so accommodating, however, which is why resources like Project Facilitate could be useful.
The program, which is in the pilot phase, includes a call center that will be staffed by oncology experts at the FDA. These experts will help physicians fill out the paperwork that companies require when requests for experimental drugs are made. The FDA staff member assigned to a request will act as a liaison between the physician and drug company.
Whenever a request is denied, the FDA liaison will follow up to find out why the drug maker said no. The program is an extension of Expanded Access, otherwise known as “compassionate use,” which lets patients with life-threatening diseases file requests online for investigational products.
Expanding Access to Drug Trials
It’s important for patients to understand that if they are ultimately able to get an unapproved drug outside of a clinical trial, their health insurer may refuse to pay for it. But insurers will cover the routine costs associated with participating in clinical trials of experimental products, and drug makers, generally, will supply medicines at no charge to patients.
In a perfect world, every patient who wants to try an experimental drug would be able to get into a clinical trial. That’s a long way off, Lichtenfeld says, but initiatives like Project Facilitate could help lessen the barriers.
“Only a small percentage of cancer patients even have the opportunity to participate in clinical trials,” he says. “We need to find patients wherever they are and figure out the best way to get them into the trials. The FDA making these resources available will be helpful.”
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The RA in Europe is way ahead on this. Trust me. They have been for years.
Thank you for your sharing the above as well as FDA's RWE both gathered from your physician network, given their professional experience and point of reference.
To my knowledge iwasadiver are joining both Longfellow95 and Maverick_1:
3 so far! plus maybe Meirluc
To wit: There is more media coverage just in the UK only re GBM than seen in the USA! despite the fact that the UK is just 1/5 in population than that of the UK!:
In total, the United Kingdom of Great Britain and Northern Ireland has a population of 63.18 million people in an area of 93,628 sq mi (242,495 sq km) vs the United States of America which has population of 309.35 million in an area of 3,805,927 sq mi (9,857,306 sq km).Jan 30, 2016
maverick_1 Monday, 08/05/19 11:27:15 PM Re: longfellow95 post# 238558 Post # 238596 of 238963 Quote: Longfellow re: This is exactly what Novocure are doing.Using a device with a broader application (such as in GBM) as a Humanitarian Device. Which is exactly what you are not supposed to do. These Humanitarian Device Exemptions are supposed to be for very rare populations, hence the lower evidence threshold and now the permission to profit is supposed to encourage the development of devices purely for that rare population.
But the FDA guidance page on this has gone missing in action.
Now you can make a profit and the limit has been doubled to 8000 a year..
A perfect fit for Novocure, even more so when they get Medicare reimbursement (75% of MPM sufferers)
maverick_1 Saturday, 12/27/14 01:53:23 PM Post # of 238594
WRAPUP on GBM device (approved for rGBM in 2011) and halted for efficacy,FDA approval 11/14 for newly diagnosed GBM) renamed Optune by Novocure:
Salient Points to Consider: 1.Only 3-4 mos better PFS 2.Monthly ongoing costs of $20K (SOME insurers cover it) 3. Greater than 18 hrs/day wearable application w/6 lb portable battery BACKpack with shaved scalp. Can one drive a car with it?, set off screening alarms@airport due to lithium ion batteries. 4.BUT HAS 150 centers in US SMSA areas for training, installation etc.
Privately held (by MDT;JNJ;PFE and two venture funds) but may be going for an IPO given the pulse of the IPO market (beginning with 1/12/15 JP Morgan HC Conf.... JP likely lead underwriter should it take place.)
For the above, there will be a market of demand, just a question of how large given the relatively small PFS improvements; ongoing costs (on lease!);level of personal obtrusiveness/convenience; receipt of CPT code for reimbursement considerations.
Key Facts from NovoCure http://www.novocure.com/ Founded in 2000 $250 million in development dollars Over 200 employees in 6 countries More than 150 global treatment centers 21 peer reviewed publications More than 1,690 patients treated
Fallen star investor Neil Woodford's funds and his protégé Mark Barnett's Invesco together hold about 23% of Burford's shares. Burford is the second-largest holding in the suspended Woodford Equity Income fund, and third-largest holding in both the Invesco High Income and Invesco Income funds
Dr. Khleif is the Director of the Georgia Cancer Center, the State cancer center of Georgia at Augusta University. He is a Georgia Research Alliance Distinguished Cancer Scientist and Clinician, and a professor of Medicine, Biochemistry, Cancer Biology and Graduate Studies. He also serves as the Director of the Immuno-Oncology and Immunetherapeutics Program. Dr. Khleif previously served as Chief of the Cancer Vaccine Section at the National Cancer Institute (NCI), and Professor of Medicine at Uniformed Services University of the Health Sciences. He also served as a Special Assistant to the FDA Commissioner from 2006-2009, where he led the FDA Critical Path for Oncology designed to restructure the oncology drug development process.
Dr. Khleif’s research group focuses on the development of novel immune therapies and cancer vaccines, and rational designs for combination immune therapy, based on understanding the molecular mechanisms of the interaction between cancer and the immune system (including cancer-induced immune suppression). His team designed and conducted some of the first cancer vaccine clinical trials, and they are currently developing novel combination approaches to enhance anti-tumor effects. During his tenure at NCI, Dr. Khleif was also detailed to serve for 4 years as the Director General and CEO of the King Hussein Cancer Center in Amman, expanding and strengthening the clinical operations and building that institution into a National Comprehensive Cancer Center of Excellence. Dr. Khleif serves on the board of the Society of Immune Therapy of Cancer (SITC), and he is a member of the National Cancer Policy Forum of the National Academy of Medicine where he leads the Immunotherapy Task Force. Dr. Khleif is the chair or member of many national committees on immuno-oncology and cancer research. He is the recipient of many awards including the National Cancer Institute (NCI) Director Gold Star Award, and the Public Health Service (PHS) Commendation Medal.
3) Perhaps big market players knew who did the SALE of 3.1 mln shares in ONE Trade on June 30Th and the rest of Park West's former original 3.5 mln were ALL SOLD.
Perhaps that large seller accounted for the rise in short interest.
4)BIG market players do monitor social media venues
5)Perhaps some AVXL holders are on Margin got Margin Calls: Put up more money or liquidate to satisfy margin account deficiencies..
SpursFan08 re:Yes. He cannot day whether or not SAP is done. He reiterated this is a 12 year long trial and he understands the frustration but they have released all they can release through PRs. He also said don’t believe anything on the message boards lol.
He said there a small company but they want to do everything right and correct and they don’t want to do something stupid and be in the rough with the FDA and other regulating agencies.
BOTH the above posts totally confirms what I’ve been stating that David Innes, who had NO professional experience as an Investor Relations professional, along with as a Oppenheimer "broker" that recommended NWBO at much higher prices.......
NOW has to REALIGN and CONFORM to ONE CONSISTENT COMMUNICATION MESSAGE versus the convoluting MESS on social media mechanisms which only provokes MORE needless NOISE & POLLUTION on top of the NEEDED Corporate silence! ........
ALL especially at this most critical end phase of an EPIC STRUGGLE.
Seen it ALL in FIVE years here:
along with the REAL SOURCE of this to date EPIC NWBO Debacle
Where Neil Woodford facing Karma! Live and Die by the Sword.
Longfellow re: This is exactly what Novocure are doing.Using a device with a broader application (such as in GBM) as a Humanitarian Device. Which is exactly what you are not supposed to do. These Humanitarian Device Exemptions are supposed to be for very rare populations, hence the lower evidence threshold and now the permission to profit is supposed to encourage the development of devices purely for that rare population.
But the FDA guidance page on this has gone missing in action.
Now you can make a profit and the limit has been doubled to 8000 a year..
A perfect fit for Novocure, even more so when they get Medicare reimbursement (75% of MPM sufferers)
maverick_1 Saturday, 12/27/14 01:53:23 PM Re: None 0 Post # of 238594
WRAPUP on GBM device (approved for rGBM in 2011) and halted for efficacy,FDA approval 11/14 for newly diagnosed GBM) renamed Optune by Novocure:
Salient Points to Consider: 1.Only 3-4 mos better PFS 2.Monthly ongoing costs of $20K (SOME insurers cover it) 3. Greater than 18 hrs/day wearable application w/6 lb portable battery BACKpack with shaved scalp. Can one drive a car with it?, set off screening alarms@airport due to lithium ion batteries. 4.BUT HAS 150 centers in US SMSA areas for training, installation etc.
Privately held (by MDT;JNJ;PFE and two venture funds) but may be going for an IPO given the pulse of the IPO market (beginning with 1/12/15 JP Morgan HC Conf.... JP likely lead underwriter should it take place.)
For the above, there will be a market of demand, just a question of how large given the relatively small PFS improvements; ongoing costs (on lease!);level of personal obtrusiveness/convenience; receipt of CPT code for reimbursement considerations.
Key Facts from NovoCure http://www.novocure.com/ Founded in 2000 $250 million in development dollars Over 200 employees in 6 countries More than 150 global treatment centers 21 peer reviewed publications More than 1,690 patients treated
Xena re: Park West still holds AVXL - they are required to file if they are no longer 5% owners.
Did you know their AVXL holdings are in an offshore tax haven:
CUSIP No. 032797300 (1) Names of Reporting Persons
Park West Investors Master Fund, Limited
(2) Check the Appropriate Box if a Member of a Group (a) [ ] (b) [ ]
(3) SEC Use Only (4) Citizenship or Place of Organization Cayman Islands Number of Shares Beneficially Owned by Each Reporting Person With (5) Sole Voting Power: 0 (6) Shared Voting Power: 3,154,122* (7) Sole Dispositive Power: 0 (8) Shared Dispositive Power: 3,154,122*
Xena re:XENALIVES re :Your post stinks to high heaven too!
If one goes to Stockcharts.com and under Sharp Charts:
Input as one indicator this:
Ulcer Index:
The following comes from Peter G. Martin himself:
“Ulcer Index measures the depth and duration of percentage drawdowns in price from earlier highs. The greater a drawdown in value, and the longer it takes to recover to earlier highs, the higher the UI. Technically, it is the square root of the mean of the squared percentage drawdowns in value. The squaring effect penalizes large drawdowns proportionately more than small drawdowns.”
It is hard to say it much better than that so we will leave it as is.
One of notable and previously cited books read and practiced:
Clean Gut Book by Alejandro Junger
In Clean Gut, Alejandro Junger, M.D, New York Times bestselling author of Clean and creator of the world-famous Clean Program, delivers a complete toolkit for reversing disease and sustaining life-long health. All of today’s most-diagnosed ailments can be traced back to an injured and irritated gut. ... Google Books Originally published: March 19, 2013
He discovered how penicillin kills bacteria. He helped solve the riddle of how our immune system can tell friend from foe.
Harvard biochemistry professor Jack Strominger has published over 1,000 scientific papers
Jack Strominger admits he's a scientific thrill-seeker, and always has been, ever since a quirk of fate landed him — an MD, not a PhD — in his own fully funded NIH lab at the crazy-young age of 26.
In the late 60s, inspired by the first human heart transplant, Strominger jumped to working on the immune system, hoping to understand why the body rejects transplants.
Jack Strominger admits he's a scientific thrill-seeker, and always has been, ever since a quirk of fate landed him — an MD, not a PhD — in his own fully funded NIH lab at the crazy-young age of 26.
"He's successful at getting grants because he's a great scientist. Because he's curious. Because he asks the right questions." Dr. Judy Lieberman, Harvard Medical School
He hopes to write a big paper called "Tolerance and Immunity" that lays out important mechanisms at work. He wants to keep solving immune-system mysteries
The EXTREMES of Capitalism has taken center Stage: From all forms of Health Care Costs that has marginalized everyone except the wealthy as the single biggest cause of personal bankruptcies to the stranglehold of $1.6 TRILLION emanating from the ongoing Student Debt Crisis where 70% of ALL US universities payroll costs is adjunct professors who average $40,000 per year while college annual costs are rising so much faster than average salaries: College students are scrounging, struggling with providing for daily FOOD expenditures! Generic pill costs could easily be 4-5 times different in the same metropolis!
Contrast that with higher taxes for FREE EDUCATION and Socialized Health Care!
Dr. Bala re:Having published 35 journal papers,[Invaluable INSIGHT along with VuBru's below ] I can say that the revision process can be very time consuming. Often the reviewers propose further work and when you resubmit after revision, they need to be satisfied that the paper was revised taking into consideration a majority, if not all, of their points. Otherwise, it will be sent back to the authors for another revision. When you are dealing with the DCVax-L trial and so many authors, this whole process could take years. Do you think that will make the investors happy?
Thank you Dr. Bala for sharing and being direct.
Vu Bru is one of many NWBO/AMRN shareholders like Marzan; ILoveTech ETC ETC that I in my Program OUTREACH to give a NEEDED TRANSFUSION of NEW BLOOD (CAPITAL to support NWBO in open market) AND INSIGHTS are the benefits! versus the witnessed financial devastation of Do It Yourself to save$ without the relevant professional experience.
Regarding Interim Analyses - I am a researcher and have been involved in a few FDA trials, and thought I would hopefully shed some light on the IA issues that have been raised based on my experience.......
Agreed, but I have doubts the FDA fully understood the science of pseudoprogression at the time of the partial halt. They are not science content experts, just bureaucrats - all they would see is an apparent worsening of patients in the treatmemnt group that needed to be explained. I can easily see them asking for a detailed response from the company to address this issue............
Boatload of Thanks to VuBru's professional and eruditely elaborate response vs all others to date (and for Doc_Logic for prompting the below).............
Longfellow95 to HapLiberian:You need to realize that the big name journals, their peer reviewers, so-called KOL's, the medical media, and of course the regulator, are all tied up with Big Pharma.
It's what maintains the status quo.
And makes everything doubly difficult for NWBO.
I fully suspect it was a 'big name' journal who faffed about asking for revision after revision for 6 months, which eventually forced NWBO to go with JTM. A wise decision.
Imo, JTM with its open access policy is more reliable, and ultimately open to greater scrutiny.
My opinion.
BEST COMPREHENSIVE EXPLANATION versus previous waffling criticisms underscoring the previous delays!: COMMENDABLE & THOROUGHLY THOUGHT OUT reply for his Legal brothers to oversee!
Significant Regulatory Milestone Towards Phase 2 in Recurrent GBM
COVINGTON, Ky., July 30, 2019 /PRNewswire/ -- Bexion Pharmaceuticals, Inc. an oncology-focused company today announced the completion of a Type B End-of-Phase 1 meeting with the U.S. Food and Drug Administration (FDA) to discuss the End of Phase 1 and the design of a Phase 2 clinical trial for BXQ-350, the Company's lead candidate, for the treatment of recurrent Glioblastoma Multiforme (rGBM).
In the Type B meeting, the FDA provided guidance on various design aspects of Bexion's proposed Phase 2 clinical trial. The trial is expected to enroll 55-60 patients.
"An End of Phase 1 meeting with the FDA is a significant regulatory milestone for any new clinical development program," said Dr. Richard Schwen, Regulatory Consultant for Bexion. "Such meetings are granted by the FDA only when significant data on the drug are available. Bexion's recent meeting signals the FDA's interest in the BXQ-350 program, and an opportunity for Bexion to obtain input to refine the remaining studies required for an FDA approval."
The Phase 2 trial is designed with learnings from the Phase 1 program, which dosed 93 patients, with 21 having rGBM. The Phase 1 program reached the highest planned dose and demonstrated a strong safety profile for BXQ-350.
Based upon the FDA's feedback, Bexion will submit a Phase 2 clinical trial protocol and statistical analysis plan along with the requested Chemistry, Manufacturing and Controls (CMC) information to the Investigational New Drug (IND) application.
"The FDA has provided us very valuable advice on the design of the Phase 2 trial, and we are grateful for their comprehensive feedback," said Dr. Ray Takigiku, CEO of Bexion Pharmaceuticals. "Recurrent GBM remains an urgent unmet medical need, and we believe the initiation of a Phase 2 clinical trial of BXQ-350 for this devastating disease is a major milestone for both patients and Bexion. We are working diligently to incorporate the FDA's comments into the IND, with the expectation of initiating the Phase 2 trial in the first quarter of 2020."
About Bexion Pharmaceuticals Bexion Pharmaceuticals is a clinical-stage biopharmaceutical company developing BXQ-350, a first-in-class agent composed of the multifunctional, lysosomal activator protein Saposin C and phosphatidylserine. BXQ-350 has demonstrated pre-clinical antitumor effects in vitro and in vivo, particularly in brain and other solid tumors, including those that may lead to brain metastases. Bexion has completed a multi-site first-in-human Phase 1 clinical trial of BXQ-350 for solid tumors and gliomas. A Phase 1 Pediatric Trial opened in April, 2019.
For more information, visit www.bexionpharma.com. Media Contact: Margaret van Gilse - 859.757.1652 - mvangilse@bexionpharma.com.
Health Canada approves VITRAKVI® (larotrectinib), the first tumour agnostic cancer treatment for advanced solid tumours harbouring an NTRK gene fusion MISSISSAUGA, ON, July 30, 2019 – Bayer announced today that there is now a treatment in Canada for Tyrosine Receptor Kinase (TRK) fusion protein-driven childhood and adult cancers. Neurotrophic Tyrosine Receptor Kinase (NTRK) gene fusions can result in the production of TRK fusion proteins that can lead to uncontrolled cell growth and cancer.
Health Canada issued a Notice of Compliance with Conditions (NOC/c) for VITRAKVI® (larotrectinib). VITRAKVI® is approved for the treatment of adult and pediatric patients with solid tumours that have an NTRK gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have no satisfactory treatment options. Treatment with VITRAKVI® should be initiated following confirmation of an NTRK gene fusion in a tumour specimen using a validated test.
This is the first time Health Canada has approved a tumour agnostic treatment, such that patients with advanced solid tumours harbouring an NTRK gene fusion may be eligible for treatment with VITRAKVI®, across multiple tumour types and ages. VITRAKVI® is a first-in class oral and highly selective TRK inhibitor that may shrink the tumour, or may slow or stop it from growing.
In the clinical trials that were the basis for this approval, TRK fusion cancer patients treated with larotrectinib experienced clinical benefit across numerous tumour types, including lung, thyroid, melanoma, GIST (gastrointestinal stromal tumour), colon, soft tissue sarcoma, salivary gland, and infantile fibrosarcoma. The overall response rate (ORR) was 75% (95% CI, 64%, 85%) with 22% of patients experiencing a complete response (CR) to treatment. The ORR observed was 90% in pediatrics and 69% in adults, and responses were rapid and durable. In the larger safety analysis set consisting of patients with and without TRK fusion cancer, the most commonly reported treatment emergent adverse events (TEAEs) (≥ 20%), in order of decreasing frequency, were fatigue, nausea, dizziness, vomiting, anemia, liver enzymes increased, cough, constipation, and diarrhea.
“The strong clinical data to date that supports VITRAKVI to target TRK fusion cancer is very encouraging. This is the first Health Canada approved tumour agnostic treatment,” says Dr. Sébastien Perreault, Clinical Assistant Professor, Department of Neurosciences, Faculty of Medicine, University of Montreal and Clinical Researcher, CHU Sainte-Justine. “As a neuro-oncologist treating patients with TRK fusion cancer, this medication represents a new approach to treatment.”
“The approval of a tumour-agnostic treatment is a major milestone in cancer treatment for pediatric and adult patients in Canada,” says Jackie Manthorne, President and Chief Executive Officer, Canadian Cancer Survivor Network. “Canadians with TRK fusion cancer across a variety of cancer types now have a meaningful treatment option. This underlines the importance of getting genomic testing, if appropriate.”
VITRAKVI® received approval under Health Canada’s NOC/c policy. This policy facilitates earlier access to promising new medicines that treat, prevent or diagnose serious, life-threatening and/or severely debilitating diseases for which there is no alternative medicine available in Canada, or where the new medicine offers a significant improvement through its risk/benefit profile over existing medicines.
Canadian Contribution
Twenty years ago, Dr. Poul Sorensen (then at BC's Children's Hospital, and now a Distinguished Scientist at the British Columbia Cancer Research Centre, Vancouver) and his team published a seminal paper (Knezevich et al, 1998) that reported on the discovery that led to the development of a new class of tyrosine kinase inhibitors, called TRK inhibitors.
“By using a molecular-based approach to studying a cancerous tumour, we were able to identify the NTRK gene fusion as the driver of what is now known as TRK fusion cancer,” says Dr. Sorensen. “This validated the idea of studying rare tumours and represented a new approach to cancer drugs and their regulation with one drug intended for a wide variety of cancers.”
Dr. Sorensen’s discovery uncovered the molecular and cellular pathways characterizing NTRK genes (and their encoded receptors, TrkA, TrkB, and TrkC) as oncogenic drivers that when altered through a gene fusion event can result in what oncologists and pathologists now refer to as TRK fusion cancer.
“The approval of VITRAKVI is reflective of Bayer’s commitment to being a leader in precision medicine and providing Canadians with innovative treatment solutions,” says Dr. Shurjeel Choudhri, Senior Vice President and Head, Medical & Scientific Affairs for Bayer Inc.
What is TRK Fusion Cancer?
TRK fusion cancer is rare and occurs when an NTRK gene fuses with another unrelated gene, producing a TRK fusion protein that becomes constitutively active or overexpressed, triggering a signaling cascade. These TRK fusion proteins act as oncogenic drivers promoting cell growth and survival, leading to TRK fusion cancer, regardless where it originates in the body. TRK fusion cancer is not limited to certain types of tissues and can occur in any part of the body. TRK fusion cancer occurs in various adult and pediatric solid tumours with varying frequency, including lung, thyroid, gastrointestinal cancers (colon, cholangiocarcinoma, pancreatic and appendiceal), sarcoma, CNS cancers (glioma and glioblastoma), salivary gland cancers (mammary analogue secretory carcinoma) and pediatric cancers (infantile fibrosarcoma and soft tissue sarcoma). TRK fusion proteins are rare in common cancers and common in rare cancers.
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to benefit people by supporting efforts to overcome the major challenges presented by a growing and aging global population. At the same time, the Group aims to increase its earning power and create value through innovation and growth. Bayer is committed to the principles of sustainable development, and the Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2018, the Group employed around 117,000 people and had sales of 39.6 billion euros. Capital expenditures amounted to 2.6 billion euros, R&D expenses to 5.2 billion euros. For more information, go to www.bayer.ca.
Scotty3371 re:Flipper explained it well. It's an extrapolation of an extrapolation of an extrapolation, it's not science. Its laughable the fda let's them market 5 yr is 13%. A complete 8 billion dollar farse!!!!
Been strident in my posts lately re NVCR's Optune after seeing too many high profile posters on this matter that have been misplaced IMHO in their posted views.
UNLESS you can OVERTURN The FDA Regulatory Labyrinth of Rules for Medical Devices vs Drugs ALL posts on this matter is Pissing in the Wind!
ADVFN re: So, if you are one of those on this board who are inclined to gloat over Woodford's woes, just remember that the ones who are really suffering are the more than a quarter of a million investors who put their trust in him.
Given your above response AND your previous query to me:
ADVFN_doclee Monday, 05/13/19 02:27:17 AM Re: maverick_1 post# 227068 Post # 227098 of 237896 Maverick, No matter what others may think about you "telling your own story", I, and I'm sure, many others will find it useful to know something of your background when it comes to evaluating the relevance of what you may say on this board.
Best wishes.
[ maverick_1 Tuesday, 05/21/19 12:01:38 AM Re: ADVFN_doclee post# 227098 Post # 228235 of 237896 Quote: Maverick, No matter what others may think about you "telling your own story", I, and I'm sure, many others will find it useful to know something of your background when it comes to evaluating the relevance of what you may say on this board. Second reply since only ADVN_doclee saw the FIRST reply!
Please EXPLAIN for ALL here your INCORRECT interpretation of:
So, if you are one of those on this board who are inclined to gloat over Woodford's woes[
Since you've only posted on NWBO March 2018 it does not appear you have been aware of my CONTRARY views on WHY Neil Woodford is the ACTUAL PIVOTAL REASON I've previously posted of NWBO EPIC DEBACLE.
Nobody even had that view and many more across the entire post 2015 NWBO EPIC stock price debacle since ALL here were on Cloud NINE! due to the bully pulpits here.
THE above is to SET THE RECORD STRAIGHT to preclude and CLOSE this ONE chapter of MANY in NWBO's EPIC debacle.
Around the corner Aug 5-6 Immuno Oncology Summit (not likely NWBO)
Cambridge Healthtech Institute’s 4th Annual Personalized Cancer Vaccines Advancing Personalized and Combination Immunotherapy AUGUST 8-9, 2019
Recent developments in personalized cancer vaccines are enabling the next generation of rational cancer immunotherapy. Cancer vaccines may now be combined with other immunotherapies, dendritic cells used for personalized immunotherapy, and neoantigens on the surface of cancer cells targeted, matching vaccine components to each patient. mRNA- and DNA-based vaccines present an additional opportunity for cancer therapeutics. Cambridge Healthtech Institute’s Fourth Annual Personalized Cancer Vaccines conference will cover applications in several types of cancer and progress in the understanding of the immune system that has significantly advanced the potential of cancer vaccines.
Thanks to our amazing #doingitforlaura supporters we’re in Cologne for day 1 of immunotherapy treatment. We’ll be back for 5 days every month for electrohyperthermia & oncolytic virus infusion & when chemo’s finished, dendritic cell vaccine. We wouldn’t be here without you.!
Photonic5 re: I’m pretty sure her time in London isn’t about patients. It’s most likely about speaking with lawmakers and regulators to gain support for the SAP and data release.
They have to educate pertinent stakeholders as to how game changing this has the potential to be so that approval can be swift following publishing data.
Thanks Photonic and TOTALLY agree for the NEEDED REDIRECTION:
ANY CEO and most especially CEO Linda Powers have LOTS of competing interests for their TIME which has to be dealing with TOP of the List Priorities versus sadly sometimes the minutiae that gains some currency on MB's.
My Mantra Do NOT put off till tomorrow what you can do TODAY: I literally had a checklist notepad at 17 yrs old that was my GPS.
All because I came from a POOR highly dysfunctional non harmonious family where I had NO childhood due to getting into select NYC commuter schools since 12 yrs of age when I started working one job and then two jobs at 15 and putting myself thru college and my first job was at the venerable Bernstein & Co working under https://en.wikipedia.org/wiki/Lewis_Sanders right across the street from now Trump Towers which was 3 story Bonwit Tellers: a high end womens boutique on 56th St/FIFTH Ave the PRIMEST of NYC real estate two blocks from The Plaza Hotel near the amazing The Pierre Hotel.https://en.wikipedia.org/wiki/The_Pierre
PATIENCE and being resolutely grounded in one's complete due diligence and constantly ADDING to one's skillsets will win the day despite temporary setbacks.
An important addendum re David Innes everyone has overlooked:
It was truly in David Innes interest to accept the Investors Relations job simply because David got ALL of OPCO clients into NWBO at almost 10 times what it is currently!
IF one does not or can not understand the above then you should have whatever of your shirt left to seek out a professional money manager where there is a mutuality of interests!
Lots of big NWBO shareholders have confided that to me: (they should have used a professional money manager rather than doing it themself!....even one poster that has more than 7 figure shares owned.
To wit: IT SHOWS temperamentally in many ways for those pre 2016 owners (those with avg cost exceeding $3)
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