New Heart Drug From Novartis Gets FDA Approval — Now The Major Pricing Battle Begins

It used to be that the major hurdle in getting a new drug approved was successfully completing phase 3 trials – the long-term studies needed to prove to regulators that your drug was both effective and safe. These trials can take years to run and cost hundreds of millions of dollars. Yet, despite doing successful studies for many years in preparation for phase 3, these late stage studies are not guaranteed to be successful. Many a phase 3 program has gone down in flames despite promising earlier trials. Thus, the successful completion of phase 3 and ultimately, approval of the drug by the FDA, is the cause of jubilation at the sponsoring company and especially by the hundreds of people involved in completing the successful program.

This is likely what happened yesterday at Novartis when it received FDA approval for its new drug to treat heart failure, Entresto (previously known by its code name LCZ696). This drug is the first of a new class called angiotensin receptor neprilysin inhibitors. In its development of Entresto, Novartis carried out an extensive clinical plan, the cornerstone of which being PARADIGM, an 8,442 patient trial of people with heart disease in which half of the patients received the Novartis drug and the other half received enalapril, a generic drug that is the current standard of care. The results of the trial proved remarkable as Entresto showed a 20% reduction in the risk of death from cardiovascular causes or hospitalization for worsening heart failure.

Novartis’ clinical plan was a risky one in that PARADIGM was designed to compare its drug against standard of care. Oftentimes, pharmaceutical companies are criticized for running clinical trials designed to make their drug look as good as possible by picking either a placebo or a weak drug as the comparator. That was not the case here. Furthermore, this was an expensive roll of the dice for Novartis as this trial likely cost hundreds of millions of dollars to run with no guarantee of success. But, by setting such a high bar for their drug with PARADIGM, they carried out the type of study that patients and physicians need by utilizing the drug in a real world setting and comparing it to the best treatment. The reward can be found in the response of cardiologists such as Clyde Yancy, the chief of cardiology at Chicago’s Northwestern Memorial Hospital who told the Wall Street Journal that Entresto is “one of the few times that we have identified a medication that is better than the standard. It’s clearly superior to what we have.” This is how clinical trials should be run and MUST be run in order to prove the value of a new drug to the world.

Normally, that would be the end of the story. But another hurdle now looms in the form of payers. Novartis has set a price of $12.50/day for Entresto (about $4,500/year). This is a far cry from the costs of breakthrough cancer or hepatitis C medications, which can often exceed $100,000/year. Novartis estimates that over 2 million Americans should be eligible for this drug and one quickly comes to the realization that this is going to be a multibillion dollar revenue generator for Novartis. Payers, however, are also painfully aware of this and might be reluctant to add another new drug to their cost basis. So now a new battle faces Novartis: convincing payers to allow access to this drug for patients in their healthcare systems. Despite the fact that Entresto is a drug that will allow heart failure patients to live longer and avoid hospitalizations, Novartis is now in discussions to justify the price of the drug with respect to the overall benefits to patients and the saving of downstream healthcare costs. In other words, it will be awhile before many heart failure patients will get access to this important medicine.

Getting FDA approval for a new important drug is always a wonderful event. Just as important in this new world of healthcare is getting “approval” of the payers – an approval that is proving challenging.

---

source : Forbes.com

Read More

Cancer patients treated in world-first clinical trial of Canadian viral therapy

Canadian researchers have launched the world’s first clinical trial of a novel investigational therapy that uses a combination of two viruses to attack and kill cancer cells, and stimulate an anti-cancer immune response. Previous research by this team and others worldwide suggests that this approach could be very powerful, and could have fewer side effects than conventional chemotherapy and radiation, although it will take years to rigorously test through this trial and others.

The therapy was jointly discovered and is being developed by Dr. David Stojdl (Children’s Hospital of Eastern Ontario, University of Ottawa), Dr. Brian Lichty (McMaster University) and Dr. John Bell (The Ottawa Hospital,University of Ottawa), and their respective research teams and colleagues. The clinical trial, which is funded by the Ontario Institute for Cancer Research and coordinated by the NCIC Clinical Trials Group, is expected to enroll up to 79 patients at four hospitals across Canada. Up to 24 patients will receive one of the viruses and the rest will receive both, two weeks apart.

Christina Monker, 75, a former nurse from Rockland, Ontario, is one of the first patients treated in the trial. She was diagnosed with cancer in 2012 and, despite six weeks of radiation therapy and two rounds of chemotherapy, the cancer spread to both her lungs. After completing another 30 rounds of chemotherapy, she enrolled in the trial at The Ottawa Hospital and was treated on June 2, 2015.

“The nausea of chemotherapy was worse than I ever could have imagined, but with the viral therapy I just felt like I had the flu for a couple of days, and the symptoms were easily managed,” said Ms. Monker. “It is too soon to know if I may have benefited from this therapy, but I’m very glad to contribute to this important research that could improve care for others.”

The idea of using viruses to treat cancer has been around for more than a century, with sporadic reports of cancer patients experiencing remarkable recoveries after viral infections. However, it is only in recent years that viral therapy has begun to be developed and tested in a rigorous way. Drs. Bell, Lichty and Stojdl began investigating viral therapies for cancer nearly 15 years ago when they worked together at The Ottawa Hospital.

“We found that when normal cells become cancerous, it’s like they are making a deal with the devil,” explained Dr. Bell, a senior scientist at The Ottawa Hospital and professor at theUniversity of Ottawa. “They acquire genetic mutations that allow them to grow very quickly, but these same mutations also make them more susceptible to viruses.”

The two viruses being tested in this clinical trial are called MG1MA3 and AdMA3. MG1MA3 is derived from a virus called Maraba, which was first isolated from Brazilian sandflies, while AdMA3 is derived from a common cold virus called Adenovirus. Both of these viruses have been engineered to stimulate an immune response against cancer cells that express a protein called MAGE-A3, but the Maraba virus also achieves an extra layer of anti-cancer activity by replicating inside many kinds of cancer cells and killing them directly. These viruses are manufactured in specialized facilities at The Ottawa Hospital and McMaster University.

“The idea behind this trial is to use the Adenovirus to prime the patient’s immune system to recognize their cancer, and then use the Maraba virus to directly kill their cancer and further stimulate their immune system to prevent the cancer coming back,” said Dr. Brian Lichty, associate professor at McMaster University. “We’re enthusiastic about the potential of this unique therapy.”

“We’re very excited about this first clinical trial,” said Dr. Stojdl, senior scientist at the Children’s Hospital of Eastern Ontario and associate professor at the University of Ottawa. “We’re continuing to push very hard to develop a suite of biological therapies with the goal of launching similar trials tailored to other types of tumours, including brain cancer and several devastating childhood cancers.”

Viral therapies are one component of a growing field of cancer research that seeks to use biological materials (including cells, genes, antibodies and viruses) to attack cancer cells and stimulate an anti-cancer immune response. This field of research has been called biotherapy or immunotherapy. Dr. Bell and his colleagues recently launched the$60M BioCanRx network to advance this area of research.

The Maraba virus is an important part of a broad biotherapeutics clinical trial development program in Canada that is combining viruses and vaccines with standard and emerging therapies to treat different types of tumours. Drs. Lichty, Bell and Stojdl and their institutions, in cooperation with the Fight Against Cancer Innovation Trust, have formed Turnstone Biologics in order to engage the private sector and to help fund further clinical trials.

“Immunotherapy is a very exciting field of cancer research, with antibody-based therapies showing the most promise in clinical trials so far,” said Dr. Derek Jonker, the overall lead for the clinical trial, a medical oncologist at The Ottawa Hospital and a professor at the University of Ottawa. “Viral therapies have also shown promise in laboratory studies, but it is too soon to know what impact they may have on patients. This clinical trial will help us find out and we’re very grateful to the patients who have participated.”

“Ontario is pleased to support innovative research through the Ontario Institute for Cancer Research,” said Reza Moridi, Ontario Minister of Research and Innovation. “Our investments have enabled our researchers to be at the forefront of this new therapy. Immunotherapy has the potential to vastly improve the way cancer is treated, and is another example of how research investment brings tangible benefits to Ontarians and people around the world.”

“The NCIC Clinical Trials Group is very pleased to conduct this trial, which offers a potential new therapeutic approach for cancer patients that has been developed by Canadian researchers,” said Dr. Janet Dancey, director, NCIC Clinical Trials Group and professor at Queen’s University in Kingston.

“Our Government is committed to investing in research that will accelerate efforts to find a cure for cancer, a disease that kills thousands of Canadians each year. The clinical trial announced today represents an innovative approach to treating cancer. We are proud to have contributed to the development of this therapy and wish the researchers and clinicians every success as they carry out this important study,” said the Honourable Rona Ambrose, Canada’sMinister of Health.

In addition to The Ottawa Hospital, the clinical trial is also taking place at the Juravinski Cancer Centre of Hamilton Health Sciences (under the leadership of Dr. Sebastien Hotte), Princess Margaret Cancer Centre of the University Health Network in Toronto (under the leadership of Dr. Albiruni R A Razak) and the Vancouver Centre of the BC Cancer Agency (under the leadership of Dr. Daniel Renouf). The trial was approved by Health Canada, the Ontario Cancer Research Ethics Board and the BC Cancer Agency Research Ethics Board. Further details about the trial are available at clinicaltrials.gov. Patients wishing to participate in the trial should speak with their own oncologist and ask for a referral to one of the participating hospitals. Further details for patients at The Ottawa Hospital are available online.

While this trial is primarily funded by the Government of Ontario through the Ontario Institute for Cancer Research, many other funding organizations have also supported the research of Drs. Bell, Lichty and Stojdl, including The Ottawa Hospital Foundation, CHEO Foundation, Canadian Cancer Society, Terry Fox Research Institute, Canadian Institutes of Health Research, Ontario Ministry of Research and Innovation, Canada Foundation for Innovation, Ottawa Regional Cancer Foundation, Hair Donation Ottawa, Angels of Hope, BioCanRx, Pancreatic Cancer Canada, NAV Canada and several philanthropic donors.

About the Partners

The Ottawa Hospital is one of Canada’s largest learning and research hospitals with over 1,100 beds, approximately 12,000 staff and an annual budget of over $1.2 billion. Our focus on research and learning helps us develop new and innovative ways to treat patients and improve care. As a multi-campus hospital, affiliated with the University of Ottawa, we deliver specialized care to the Eastern Ontario region, but our techniques and research discoveries are adopted around the world. We engage the community at all levels to support our vision for better patient care.www.ottawahospital.on.ca

McMaster University, one of four Canadian universities listed among the Top 100 universities in the world, is renowned for its innovation in both learning and discovery. It has a student population of 30,000, and more than 170,000 alumni in 137 countries. Its Michael G. DeGroote School of Medicine has a global reputation for educational advancement and its development of evidence-based medicine. McMaster and its academic hospital partner Hamilton Health Sciences are internationally known for their research intensity.

The CHEO Research Institute coordinates the research activities of the Children’s Hospital ofEastern Ontario (CHEO) and is affiliated with the University of Ottawa. Its three programs of research include molecular biomedicine, health information technology, and evidence to practice research. Key themes include cancer, diabetes, obesity, mental health, emergency medicine, musculoskeletal health, electronic health information and privacy, and genetics of rare disease. The CHEO Research Institute makes discoveries today for healthier kids tomorrow. For more information, visit www.cheori.org

The Ontario Institute for Cancer Research (OICR) is an innovative cancer research and development institute dedicated to prevention, early detection, diagnosis and treatment of cancer. The Institute is an independent, not-for-profit corporation, supported by the Government of Ontario. OICR’s research supports more than 1,700 investigators, clinician scientists, research staff and trainees located at its headquarters and in research institutes and academia across the Province of Ontario. OICR has key research efforts underway in small molecules, biologics, stem cells, imaging, genomics, informatics and bio-computing. For more information, please visit the website at www.oicr.on.ca.

The NCIC CTG is the only Canadian cooperative cancer trials group conducting the entire range of cancer trials from early phase studies to large international randomized controlled trials across all cancer types. Its primary mission is to assess the effectiveness of interventions to prevent the development of cancer or improve the care of those patients who do develop cancer. NCIC CTG trials have led to improved outcomes for cancer patients. It is a national research program of the Canadian Cancer Society. The NCIC CTG’s Central Operations and Statistics Office is located at Queen’s University in Kingston, Ontario, Canada

Read More

China to reform drug approval system

China has decided to reform its appraisal and approval system for drugs and medical instruments with the aim of improving drug safety and quality and encouraging innovation.

According to a guideline issued by the China State Council on Aug. 18, China aims to set up a more scientific and efficient system to ensure the safety and quality of medicines and medical instruments that come into the market.The relevant authorities will make efforts to strike a balance between the number of registration applications received and those that are approved by the end of 2016. They also will ensure that, by 2018, every application will be approved or rejected within a certain time limit.The State Council requested that the approval standards for medicines should be improved by adjusting the classification of drug registration. The appraisal and approval process of innovative drugs will be accelerated, including drugs to treat AIDS, cancer, serious infectious and rare diseases.

Institutions and staff involved in the R&D of drugs will be allowed to apply for the registration of new drugs. Once these drugs are transferred to enterprises for manufacturing, no further appraisal will be required.Approvals concerning the clinical trials of drugs and medical instruments will be further improved. Additionally, procedures on drug approvals will be simplified, so that applications for drugs and relevant packing and materials will be approved at the same time.In order to enhance the transparency of approvals, information concerning drug supply and demand, as well as registration applications, will be released in a timely manner. The public will be able to view the list of approvals and make enquiries, if required, and applicants also will be informed of the approval process and results.

Read More

Universal flu vaccine steps closer

Flu vaccines in the US are made to target a small number of viral strains based on public health experts’ predictions. Scientists hope that, one day, a universal flu vaccine will be developed that will be effective against all strains, and the findings of a new study may take researchers one step closer to this goal.

The study, published in Cell, found that chemical modifications to a specific region in antibodies could be used to improve current flu vaccines.

“We believe these results may represent a preliminary step toward a universal flu vaccine, one that is effective against a broad range of the flu viruses,” states senior author Jeffrey Ravetch, head of the Leonard Wagner Laboratory of Molecular Genetics and Immunology at the Rockefeller University, New York.

Vaccines in the US are typically formulated to target H1 and H3 influenza A viruses along with influenza B strains predicted by experts to be prevalent during the coming flu season. If the experts’ predictions are inaccurate, however, then the vaccines are rendered ineffective.

Just this January, the Centers for Disease Control and Prevention (CDC) reported that this season’s flu vaccine was only 23% effective across all age groups, due to a large proportion of this season’s viruses being “drift variants.”

“While the conventional flu vaccine protects only against specific strains, usually three of them, our experiments show that by including modified antibodies within the vaccine it may be possible to elicit broad protection against many strains simultaneously,” explains Ravetch.

Influenza can be a dangerous illness – particularly in adults over the age of 65 and people with other health conditions. The American Lung Association state that 90% of deaths from influenza occur in adults older than 65.It is a difficult virus for scientists to target with vaccines due to the wide variety of strains that exist, with new strains emerging regularly. Because of how elusive the virus can be, a universal vaccine represents a dream goal for scientists and has been the focus of many research projects.

The new study revolves around a new strategy involving the Fc region of antibodies – the region that connects with immune cells. The researchers, led by Taia Wang and Jad Maamary, already knew that modifying the Fc region affected how the antibodies interacted with immune cells, and so the team set out to investigate how changes to the region might improve an immune response.

Efficacy of improved vaccine: ‘no small accomplishment’

Healthy volunteers were vaccinated with a seasonal flu vaccine containing an inactivated strain of the H1N1 virus – also known as swine flu. Using blood samples, the researchers tracked how the participants’ immune systems responded, looking for chemical modifications to antibodies against a surface protein. Around a week later, the researchers noticed an increase in the number of sialylated antibodies present. Sialic acid is a molecule crucial to signaling, meaning that the presence of these antibodies indicated a positive response to the vaccine.

Experiments on cell cultures and mice revealed that sialylated Fc regions bind to a receptor protein called CD23 on specific immune cells called B cells. In turn, CD23 activates another receptor known as FcyRIIB that discourages B cells from producing low-affinity antibodies.

In short, sialylated Fc regions lead to the activation of B cells producing the highest affinity antibodies. The researchers found that this higher affinity resulted in broad protection against influenza viruses from the H1 subtype (to which H1N1 belongs).

Using this discovery, the researchers modified the H1N1 vaccine by adding sialylated antibodies against the virus’ protein. The new and improved vaccine was then tested on mice.

“When we immunized mice with just the H1 protein from one strain or with the sialylated complexes containing the same viral protein, we found both offered equal protection against the same strain of flu,” explains Maamary. “However, when we exposed them to strains expressing different versions of the H1 protein, only the sialylated immunizations offered protection.”

Wang believes the new mechanism they have uncovered could potentially be utilized to reduce the rates of morbidity and mortality attributable to seasonal influenza virus infections.

“We are now looking into applying this strategy toward improving existing vaccines; ideally, this would result in a vaccine that provides lifelong immunity against flu infections,” she states.

Earlier this year, Medical News Today reported on a study suggesting that a newly discovered class of antibodies could lead to a universal flu vaccine “within 5 years.”

Read More

Cystic fibrosis: yeast study may address root cause

Yast is helping to tackle the root cause of cystic fibrosis – a disease that results from faulty ion channels. A new study shows how a small molecule can take the place of a missing protein in yeast cells with faulty ion channels, enabling them to work again.

The study, by researchers from the University of Illinois at Urbana-Champaign, is published in the Journal of the American Chemical Society.

All organisms – from microbes to humans – rely on protein pumps and channels that transport ions across the cell membrane. Faulty ion channels in cells underlie many currently incurable human diseases.

Cystic fibrosis is caused by a genetic mutation that affects some of the proteins that make up ion channels, causing them to malfunction. The result is a thick build up of mucus in the lungs and other organs, making breathing difficult.

While treatments for cystic fibrosis exist, they do not fix the underlying cause. Treatments include inhalants, enzyme supplements and clearing the airways, which can usually only be done with help.

Yeast is a useful organism for researching human health and disease because yeast cells are very similar to human cells. On two occasions, yeast has featured in studies that have won Nobel prizes (one in 2001, the other in 2009) for their work on human cells.

The new study describes how a small molecule can take the place of a missing protein to restore the type of ion channel function that is missing in people with cystic fibrosis and similar diseases. The molecule – amphotericin B – was originally extracted from bacteria and is used to treat fungal infections.

THE SMALL MOLECULE CAUSED DEFICIENT YEAST TO GROW NEARLY AS WELL AS NORMAL YEAST

The team tested the small molecule at low doses in a strain of yeast that cannot grow because it has faulty ion channels.

Fast facts about cystic fibrosis

• People with cystic fibrosis have inherited two copies of the defective CF gene
• About 70,000 people worldwide are living with cystic fibrosis
• Most cases are diagnosed by the age of 2.

When they added the molecule, the researchers found the yeast grew nearly as well as a normal strain that they used as a control. They note:

“Here we report vigorous and sustainable restoration of yeast cell growth by replacing missing protein ion transporters with imperfect small molecule mimics.”

The authors say more research is needed to confirm whether the small molecule will work in human diseases like cystic fibrosis. In the meantime, however, they conclude that their study provides a “framework for pursuing such a therapeutic strategy.”

People with cystic fibrosis are at greater risk of lung infection because the thick, sticky mucus that builds up in the lungs allows germs to thrive and multiply.

Earlier this year, Medical News Today reported how researchers are reaching a better understanding of infections in cystic fibrosis. A small study of children with the disease reveals that the microorganisms that infect people with cystic fibrosis can survive on little to no oxygen.

Read More

Age-related immune system decline slowed by antioxidants

The thymus is an organ that produces T lymphocytes – white blood cells that are crucial to the immune system.

New research has demonstrated how the aging process damages the immune system, while showing how antioxidants in the diet could slow the build-up of this damage.

Findings from the study, published in Cell Reports, also lend support to the “free-radical theory” of aging, whereby reactive oxygen species such as hydrogen peroxide that are produced by normal metabolism cause damage to cells. This damage contributes to both aging and age-related diseases.

The study was conducted by researchers from The Scripps Research Institute (TSRI) who focused their attention on an organ called the thymus that is responsible for the production of T lymphocytes – also referred to as T cells.

T cells are white blood cells that control the body’s immune response. These cells are continuously lost, and it is the job of the thymus – located between the lungs – to replenish them, enabling the body to respond to new infections. However, the thymus is unable to continuously produce high levels of T cells.

“The thymus begins to atrophy rapidly in very early adulthood, simultaneously losing its function,” explains study author Dr. Howard Petrie. “This new study shows for the first time a mechanism for the long-suspected connection between normal immune function and antioxidants.”

Antioxidants are substances that could prevent or delay damage to cells. Examples include beta-carotene, vitamin C and vitamin E. They can often be found in fruits and vegetables and are also available in the form of supplements.

The researchers set out to explore the mechanisms behind the connection by developing a computational approach they could use to assess gene activity in two types of thymus cell in mice – stromal cells and lymphoid cells.

In the stromal cells, they observed that a deficiency in an antioxidant enzyme called catalase led to the production of reactive oxygen species through metabolism, which in turn sped up the rate at which damage occurred.

COMMON DIETARY ANTIOXIDANTS FOUND TO PRESERVE SIZE OF THE THYMUS

The researchers then tested the role of this antioxidant by increasing catalase levels in genetically altered animal models. By doing this, they were able to maintain the size of the thymus for a longer period.

In addition, the researchers were also able to preserve the size of the thymus in animals by giving them two common dietary antioxidants – including vitamin C.

The question of why the thymus decreases in size more rapidly than other body tissues remains unanswered, however. Dr. Petrie says that while other research has demonstrated the thymus is responsive to sex hormones, their new study shows that its aging process is the same as in other tissues.

“However, the process is accelerated in the thymus by a deficiency in the essential protective effects of catalase, which is found at higher levels in almost all other body tissues,” he continues.

The researchers also point out that while increasing catalase levels in stromal cells preserved the size of the thymus for a longer period, it did not prevent it from atrophying – as yet, there is no way to completely halt metabolic damage accumulated over time.

Read More

Commonly Used Drugs May Delay Brain Injury Recovery

New study shows that Anticholinergics, a category of drugs recommended to treat common problems which range from bladder issues, depression to insomnia, can in up to 50 percent of old age patients delay their recovery from brain injury.

Anticholinergics have in previous studies been identified to have side effects, such as short-term cognitive impairment, confusion and dizziness. But the new research lead by the University of East Anglia (UEA) in the UK is one of the first to examine their effects on people who have suffered from a brain injury.

The team of scientists, which includes researchers from Aston University, other UK research centers and the British National Health Service, presented their results in the journal Brain Injury. The study collected data from 52 subjects who had experienced brain or spinal cord injury and were undergoing treatment at a neuro-rehabilitation unit. Patients going through neuro-rehabilitation are usually given anticholinergics to reduce pain, bladder control and other problems. Researchers identified that the average length of stay among the subjects they examined was longer for those with greater levels of anticholinergic burden (ACB) in their system. The analysis of data revealed a direct connection between modifications in ACB and duration of hospital stay. Subjects whose ACB scores on discharge were greater than on admission remained longer in hospital compared to those whose ACB scores on discharge were reduced. However, the researchers point out their research was not developed to show cause and effect; it can only demonstrate that there is a link.

Anticholinergic medicines can conflict with rehabilitation

Senior author of the study Chris Fox, states that:

“This initial study shows the requirement for bigger studies to fully support the outcomes and calls for additional research into what long-term results these common drugs are having on the recovery of these patients.”

He describes that while it is usually essential to cure common problems of brain and spinal cord injuries with anticholinergic medicines, drugs side effects like cognitive impairment can get in the way of sufferers rehabilitation, causing in a longer hospital stay. Co-author Dr. Ian Maidment, says their results add to the proof that, where possible, anticholinergics must be prevented in a extensive range of populations.“Regular drugs review by a nurse, physician or pharmacist may be a way of guaranteeing that drugs with anticholinergic effects are used properly,” he suggests.

The research is significant not just for clinical reasons but also due to the fact one of the measures of hospital efficiency is length of stay, and there are financial rewards for discharging sufferers as soon as is safe.

Prof. Fox concludes:

“Determining things which might negatively affect the length of a patient’s stay can have significant financial along with quality of life implications.”

For that reason, he adds, the study’s results may help enhance the performance of health care by decreasing the time patients spend in rehabilitation.

Read More

Roche Continues to be at the Top in Oncology Sales

Swiss drug maker Roche continues to be at the top of the heap in oncology sales for several years and it’s no wonder with its best selling drugs such as Avastin, Herceptin, Rituxan and Triumvirate. Roche generated revenue of over $25 billion via its cancer drugs in the previous year, which is more than twice the revenue generated by its nearest competitor Novartis.

But things are transforming. While Roche will not lose its top place in the near future, not with its well performing new products such as Perjeta and Gazyva, and a PD-L1 medicine on its way, however it is important to note that Roche’s cancer portfolio increased by just 2% in 2014, in comparison to double digit increases at US based drug makers such as Celgene, Johnson & Johnson and Bristol-Myers Squibb, which took the 3rd, 4th and 5th place in oncology sales respectively. Novartis with a larger baseline to begin with, saw its cancer medicines increase by 8% for the year reaching an estimated $10.2 billion.

And with the No. 2 Novartis taking on GlaxoSmithKline’s oncology portfolio, novel immuno-oncology drugs hitting the scene and other hot new oncology products on the prowl, competitor pharmaceutical companies are gaining.

Novartis definitely will add considerably to its total for 2015, with GSK’s medicines now the fold. Those medicines brought in some $1.8 billion in 2014, which would have increased Novartis past $12 billion previous year. If it can maintain the increasing sales of Tasigna and Afinitor as they have been, each delivered raises of a minimum of 20% last year – that’s one more big chunk.

Looking further ahead, the fight for second place could heat up. Estimations for 2020 sales put Clegene’s Revlimid (It is used in people with myelodysplastic syndrome triggered by an abnormal chromosome) at $10.1 billion all on its own, and the firm’s Abraxane is predicted to provide in over $2 billion. It also has Pomalyst, a novel multiple myeloma medicine, to play a crucial role with the $2.4 billion in its 2020 prediction. Clegene’s CEO Bob Hugin states that his organization can reach $20 billion in sales by then, though some of that predicted increase is pegged on an autoimmune disease candidate.

And then there’s immuno-oncology, which appears to be ready to take cancer therapy by surprise. How it will all play out is not clear at this point. US drug maker Bristol-Myers’ Opdivo data from ASCO put its long term  $6 billion prediction in question, for instance, but those medicines could change the cancer rankings significantly.

Read More

FDA Awards $19 Million for 18 New Rare Disease Research Grants

The U.S. Food and Drug Administration (FDA) has awarded 18 new research grants totaling more than $19 million to enhance the development of orphan drugs for rare disease patients.

The grants are intended for clinical studies evaluating the safety and effectiveness of products that could either result in, or substantially contribute to, the FDA approval of products.

Since its creation in 1983, the Orphan Products Grants Program has provided more than $350 million to fund more than 570 new clinical studies and supported the marketing approval of more than 50 products.

Since many rare conditions are also pediatric conditions, it is no surprise that 10 of the 18 awards fund studies that enroll pediatric patients as young as newborns. Some of the rare conditions under investigation in the grants include sickle cell anemia, amyloidosis, autosomal dominant polycystic kidney disease, prader willi syndrome, as well as numerous rare cancers,

THE GRANT RECIPIENTS FOR FISCAL YEAR 2015 ARE:

• Albert Einstein College of Medicine (Bronx, New York), Deepa Manwani (principle investigator)
  • Phase 2 Study of Gamunex (Intravenous Gammaglobulin) for the Treatment of Sickle Cell Acute Pain
  • About $1.6 million over 4 years
• Baylor College of Medicine (Houston, Texas), Andrew Sikora
  • Phase 2 Study of ADXS11-001 Vaccine for the Treatment of HPV-Related Oropharyngeal Cancer
  • About $1.2 million over 3 years
• Beckman Research Institute of the City of Hope (Duarte, California), Behnam Badie
  • Phase 1 Study of Cellular Immunotherapy Using Optimized IL13Ra2 Specific CAR T Cells for the Treatment of Malignant Glioma
  • $600,000 over 3 years
• Columbia University (New York, New York), Suzanne Lentzsch
  • Phase 1A/B Study of 11-1F4 mAb for the Treatment of AL Amyloidosis
  • $600,000 over 3 years
• Edimer Pharmaceuticals Inc. (Cambridge, Massachusetts), Neil Kirby
  • Phase 2 Study of EDI200 for the Treatment of X-Linked Hypohidrotic Ectodermal Dysplasia
  • $1.6 million over 4 years
• Emory University (Atlanta, Georgia), Claudia Morris
  • Phase 2 Study of L-Arginine Therapy for the Treatment of Pediatric Sickle Cell Disease Pain
  • $1.6 million over 4 years
• Indiana University-Purdue University at Indianapolis (Indianapolis, Indiana), Kent Robertson
  • Phase 2 Study of Imatinib for the Treatment of Airway Tumors in Children with Neurofibromatosis Type 1
  • $1.6 million over 4 years
• Indiana University-Purdue University at Indianapolis (Indianapolis, Indiana), Sharon Moe
  • Phase 1 Study of Low Dose Pioglitazone for the Treatment of Autosomal Dominant Polycystic Kidney Disease
  • About $600,000 over 3 years
• New York University School of Medicine (New York, New York), Horacio Kaufmann
  • Phase 2 Study of Carbidopa for the Treatment of Familial Dysautonomia
  • About $1.1 million over 3 years
• Northshore University Healthsystem (Evanston, Illinois), Eli Ehrenpreis
  • Phase 1 Study of Naltrexone for the Treatment of Mesenteric Panniculitis
  • About $220,000 over 3 years
• Rhythm Metabolic Inc. (Boston, Massachusetts), Keith Gottesdiener
  • Phase 2 Study of the Melanocortin 4 Receptor Agonist RM-493 for the Treatment of Prader Willi Syndrome
  • About $1 million over 3 years
• Scioderm Inc. (Durham, North Carolina), Robert Ryan
  • Phase 2 Study of SD-101 for the Treatment of Epidermolysis Bullosa
  • $400,000 for 1 year
• Sloan-Kettering Institute Cancer Research (New York, New York), Mrinal Gounder
  • Phase 3 Study of Sorafenib for the Treatment of Desmoid Tumors or Aggressive Fibromatosis
  • About $900,000 over 4 years
• Transderm Inc. (Santa Cruz, California), Roger Kaspar
  • Phase 1 Study of Sirolimus for the Treatment of Pachyonychia Congenita
  • About $400,000 over 2 years
• University of California San Diego (La Jolla, California), Santosh Kesari
  • Phase 2 Study of Vascular-Targeted Prodrug (G-202) for the Treatment of Recurrent Glioblastoma
  • About $1.6 million over 4 years
• University of Kansas Medical Center (Kansas City, Kansas), Mazen Dimachkie
  • Phase 2 Study of Arimoclomol for the Treatment of Sporadic Inclusion Body Myositis
  • About $1.6 million over 4 years
• University of Michigan (Ann Arbor, Michigan), Meghan Arnold
  • Phase 3 Study of Standard vs Reduced IV Fat for the Prevention of Parenteral Nutrition-Associated Cholestasis (PNAC)
  • About $1.6 million over 4 years
• Innovative Biotherapies Inc. (Ann Arbor, Michigan), H. David Humes (medical device awardee)
  • Phase 2 Study of Selective Cytopheretic Device for the Treatment of Pediatric Patients with Acute Kidney Injury
  • About $1.6 million over 4 years

---

Source

FDA awards 18 grants to stimulate product development for rare diseases [news release]. Washington, DC: US Food and Drug Administration; September 21, 2015.http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm463539.htm

Read More

Sensitive Blood Test May Help Rule Out Heart Attack

WEDNESDAY Oct. 7, 2015, 2015 — A new, highly sensitive blood test may help doctors quickly rule out heart attack for almost two-thirds of people who seek emergency room treatment for chest pain, a new study suggests.

Researchers said their findings could potentially reduce unnecessary hospital admissions and substantially lower health-care costs. “Until now, there were no quick ways to rule out a heart attack within the emergency department,” said the study’s lead author, Dr. Anoop Shah, from the University of Edinburgh in Scotland.

“Over the last two decades, the number of hospital admissions due to chest pain has tripled. The overwhelming majority of these patients do not have a heart attack,” Shah said. Assessing a possible heart attack requires lengthy stays in the ER or hospitalization for repeat testing, the study authors pointed out.

The new test is more sensitive than the standard version, Shah’s team said. It can detect far lower blood levels of troponin, a protein released when heart muscle is damaged. The more damage that occurs, the higher blood levels of troponin will be. A slight increase in troponin suggests some damage has occurred, while very high levels indicate a person has had a heart attack, the researchers explained.

Using this new test, doctors could potentially double the number of low-risk patients able to be safely discharged from the emergency room, the researchers reported in the Oct. 8 issue ofThe Lancet.”Use of this approach is likely to have major benefits for both patients and health-care providers,” Shah said in a journal news release.

For the study, the researchers measured troponin levels in more than 6,000 patients admitted to the hospital with chest pain, and assessed their risk for heart attack and death from heart attack within 30 days.

The investigators found that 61 percent of the patients with a troponin level below 5 ng/L (nanograms per liter of blood) were at very low risk of heart attack and could have been discharged early, regardless of age, gender, and risk factors for heart disease. One year out, these patients had a three times lower risk of heart attack and cardiac death than those with higher troponin levels, the researchers said.

The authors of an accompanying editorial in the journal said patient follow-up will be needed to validate use of this test in routine practice.”Trials are needed to assess the safety and effectiveness of clinical pathways that involve no further testing for such patients,” wrote Martin Than from Christchurch Hospital, New Zealand, and colleagues.

---

More information

The American Heart Association describes the symptoms of heart attack.

Read More