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FDA review raises safety concern about FARXIGA (dapagliflozin, BMS, Astra Zeneca) for Type 2 Diabetes

Dapagliflozin is a new molecular entity in the antidiabetic class known as SGLT2 inhibitors. Through selective and reversible inhibition of SGLT2, dapagliflozin causes renal elimination of glucose. The magnitude of HbA1c reduction observed in the dapagliflozin clinical program is
relatively consistent across trials and similar to that of other recently approved antidiabetic drugs, including the SGLT2 inhibitor, canagliflozin. In addition to improvement in glycemic control, dapagliflozin produces modest reductions in body weight and systolic blood pressure. Consistent with the results of previous meta-analyses, the updated meta-analysis of major cardiovascular events in the pool of 21 Phase 2b and Phase 3 clinical trials included in this NDA resubmission
continues to meet the December 2008 Guidance, ruling out the unacceptable increase in CV risk of greater than 80% above comparator groups. Furthermore, divergence in the Kaplan-Meier curves, in favor of dapagliflozin, were observed for the primary CV composite endpoint after approximately eight months, suggesting potential benefit with prolonged use. However, there appears to be a numeric imbalance in early MACE events not favoring dapagliflozin. A similar
observation of an early imbalance in cardiovascular events was seen with canagliflozin, also not favoring canagliflozin. For dapagliflozin, discordant with the results of the updated meta­ analysis, a pool of two large, well-designed clinical trials enriched with individuals at high risks
for CV events—and in whom statistically significant reductions in HbA1c, systolic blood pressure, and body weight were observed—achieved a point estimate and upper bound 95% confidence interval for the hazard ratio of the composite MACE endpoint which exceeded 1 and
1.8, respectively. When considering efficacy of dapagliflozin, potential benefits should be balanced against credible safety concerns identified during clinical development. There continues to be a numeric
imbalance in cases of bladder cancer, not favoring dapagliflozin. An imbalance in bladder cancer events was not seen with canagliflozin. Additionally, a potential case of drug-induced liver
injury was observed during the first review cycle for which an association with dapagliflozin remains plausible. The Applicant has provided more than three years of follow-up data for this case to support a possible reclassification of the diagnosis. Overall, the occurrence of marked
liver laboratory test abnormalities remains relatively balanced between dapagliflozin and the comparator treatment arms.

 

 

 

 

Krishan Maggon 's insight:

FDA review has raised important safety concern about drug induced liver toxicity, increased cardiovascular risk and bladder cancer. Expert panel meeting on 12 December to discuss safety and efficacy issues.

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Pharma Biotech Industry Review (Krishan Maggon)
Review and updates of latest developments in the pharma biotech industry dealing with new drugs, market research and global health. It will mainly cover FDA and EMA approvals of new drugs, regulatory filings ( NDA, BLA, MAA ), sales and marketing data of to selling drugs, blockbuster drugs, best selling drugs and R&D budget and R&D productivity/efficiency. Sales and marketing forecast and cost of newly approved drugs will be discussed. http://knol.google.com/k/krishan-maggon-knols#" rel="nofollow">http://knol.google.com/k/krishan-maggon-knols#
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FDA New Drug Approvals 2014

FDA New Drug Approvals 2014 | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it

FDA approves Farxiga (dapaglifozin, Astra Zeneca, BMS) to treat type 2 diabetes. 

 

 

Krishan Maggon 's insight:

For a free updated ongoing review of new drugs (NCE/NME) approved by the FDA in 2014 with a list of new NDA/BLA filings, PDUFA dates. 

 

FDA first approved drug Farxiga (dapaglifozin, Astra Zeneca, BMS) to treat type 2 diabetes is reviewed with links, references,

clinical data and regulatory history and its competition with J&J first approved SGTP2 inhibitor Invokana. 

 

Approvals of generics, combination products or new formulations is not covered.

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FDA approves Zydelig (idelalisib, Gilead) for three types of blood cancers

FDA approves Zydelig (idelalisib, Gilead) for three types of blood cancers | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
The U.S. Food and Drug Administration today approved Zydelig (idelalisib) to treat patients with three types of blood cancers.

 

Zydelig is being granted traditional approval to treat patients whose chronic lymphocytic leukemia (CLL) has returned (relapsed. The FDA is also granting Zydelig accelerated approval to treat patients with relapsed follicular B-cell non-Hodgkin lymphoma (FL) and relapsed small lymphocytic lymphoma (SLL), another type of non-Hodgkin lymphoma. 

 

Zydelig is to be used in patients for whom Rituxan alone would be considered appropriate therapy due to other existing medical conditions (co-morbidities). Zydelig is the fifth new drug with breakthrough therapy designation to be approved by the FDA and the third drug with this designation approved to treat CLL.

 

The FDA approved Gazyva (obinutuzumab) in November 2013, Imbruvica (ibrutinib) in February 2014 and a new use for Arzerra (ofatumumab) in April 2014 to treat CLL. Both Gazyva and Arzerra also received breakthrough therapy designation for this indication. Like the other two drugs, Zydelig was also granted orphan product designation because it is intended to treat a rare disease.

Zydelig’s safety and effectiveness to treat relapsed CLL were established in a clinical trial of 220 participants who were randomly assigned to receive Zydelig and Rituxan or placebo and Rituxan. The trial was stopped for efficacy following the first pre-specified interim analysis point, which showed participants treated with Zydelig and Rituxan had the possibility of living at least 10.7 months without their disease progressing (progression-free survival) compared to about 5.5 months for participants treated with placebo and Rituxan. Results from a second interim analysis continued to show a statistically significant improvement for Zydelig and Rituxan over placebo and Rituxan.

Zydelig’s safety and effectiveness to treat relapsed FL and relapsed SLL were established in a clinical trial with 123 participants with slow-growing (indolent) non-Hodgkin lymphomas. All participants were treated with Zydelig and were evaluated for complete or partial disappearance of their cancer after treatment (objective response rate, or ORR). Results showed 54 percent of participants with relapsed FL and 58 percent of participants with SLL experienced ORR.

The FDA is approving Zydelig to treat FL and SLL under the agency’s accelerated approval program, which allows approval of a drug to treat a serious or life-threatening disease based on clinical data showing the drug has an effect on a surrogate endpoint reasonably likely to predict clinical benefit to patients. This program provides earlier patient access to promising new drugs while the company conducts confirmatory clinical trials.

Zydelig carries a Boxed Warning alerting patients and health care professionals of fatal and serious toxicities including liver toxicity, diarrhea and colon inflammation (colitis), lung inflammation (pneumonitis) and intestinal perforation that can occur in Zydelig-treated patients. Zydelig is also being approved with a Risk Evaluation and Mitigation Strategy (REMS) comprised of a communication plan to ensure healthcare providers who are likely to prescribe Zydelig are fully informed about these risks.

Common side effects include diarrhea, fever (pyrexia), fatigue, nausea, cough, pneumonia, abdominal pain, chills and rash. Common laboratory abnormalities include decreased levels of white blood cells (neutropenia), high levels of triglycerides in the blood (hypertriglyceridemia), high blood sugar (hyperglycemia) and elevated levels of liver enzymes.

Zydelig is marketed by Foster City, California-based Gilead Sciences. Rituxan and Gazyva are marketed by Genentech, a member of the Roche Group, based in South San Francisco, California. Imbruvica is co-marketed by Sunnyvale, California-based Pharmacyclics and Raritan, New Jersey-based Janssen Biotech, Inc. Arzerra is marketed by Research Triangle Park, North Carolina-based GlaxoSmithKline.

 

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Gilead first entry int the lucrative cancer market.  

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Free HCV, HIV and TB R&D 2014 Pipeline Report

Free HCV, HIV and TB R&D 2014 Pipeline Report | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it

Introduction and Executive Summary

HIV

The Antiretroviral Pipeline

Preventive Technologies: Antiretroviral and Vaccine Development

Research Toward a Cure and Immune-Based and Gene Therapies

Fit for Purpose: Treatment Optimization

The Pediatric Antiretroviral Pipeline

HCV

Hepatitis C Pipeline: BONANZA! The Gold Rush Is Under Way

2013/2014 HCV Drug Pipeline Update

Global Update: Hepatitis C Treatment Activism

TB

The Tuberculosis Diagnostics Pipeline

Tuberculosis Drug Development Hobbles Forward

Playing Catch-Up: Pediatric Tuberculosis Treatment Pipeline

The Tuberculosis Vaccines Pipeline

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Krishan Maggon 's curator insight, Today, 4:20 AM

Great initiative to provide open access to drug pipeline report and updates in AIDS, Hepatitis C and Tuberculosis including immunotherapies and vaccines.

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Free HIV, HCV and TB 2014 Pipeline Report

Free HIV, HCV and TB 2014 Pipeline Report | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it

Introduction and Executive Summary

HIV

The Antiretroviral Pipeline

Preventive Technologies: Antiretroviral and Vaccine Development

Research Toward a Cure and Immune-Based and Gene Therapies

Fit for Purpose: Treatment Optimization

The Pediatric Antiretroviral Pipeline

HCV

Hepatitis C Pipeline: BONANZA! The Gold Rush Is Under Way

2013/2014 HCV Drug Pipeline Update

Global Update: Hepatitis C Treatment Activism

TB

The Tuberculosis Diagnostics Pipeline

Tuberculosis Drug Development Hobbles Forward

Playing Catch-Up: Pediatric Tuberculosis Treatment Pipeline

The Tuberculosis Vaccines Pipeline

Krishan Maggon 's insight:

Great initiative to provide open access to drug pipeline report and updates in AIDS, Hepatitis C and Tuberculosis including immunotherapies and vaccines.

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Istodax (romidepsin, Celgene) lymphoma drug can drive HIV out of hiding: study

Istodax (romidepsin, Celgene) lymphoma drug can drive HIV out of hiding: study | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
LONDON (Reuters) - An anti-cancer drug made by the U.S. biotech firm Celgene can re-activate hidden HIV in patients so that it can be detected, bringing researchers closer to being able to treat it, Danish
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Dr Dev Kambhampati | FDA- Paving the Way for Personalized Medicine

Dr Dev Kambhampati | FDA- Paving the Way for Personalized Medicine

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The $300000 Drug Kalydeco (Ivacaftor, Vertex) for Cystic Fibrosis - New York Times

The $300000 Drug Kalydeco (Ivacaftor, Vertex) for Cystic Fibrosis - New York Times | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
The $300000 Drug
New York Times
“I still pinch myself every day,” says Emily Schaller, 32, who has been taking the drug since she participated in its Phase III trials five years ago. “I can take deep breaths.
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Since the CF patients have to take the drug for the rest of their lives, Ivacaftor will cost millions per patient. 

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Diseases, symptoms, genes, and proteins linked together in giant network - Medical Xpress

Diseases, symptoms, genes, and proteins linked together in giant network - Medical Xpress | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
Medical Xpress
Diseases, symptoms, genes, and proteins linked together in giant network
Medical Xpress
The data was extracted from millions of PubMed bibliographic records with at least one disease or symptom term in the metadata field.
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Novel Therapies for Tuberculosis: Tuberculosis Control and its Discontents

Novel Therapies for Tuberculosis: Tuberculosis Control and its Discontents | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it

The barriers to access TB services are caused mainly by poverty and its complex socioeconomic determinants, but this key variable is hard to represent into the mathematical models [8]. Could TB be controlled through technology despite a world of increasing poverty?

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Citation: Portero JL, Rubio M (2006) Novel Therapies for Tuberculosis: Tuberculosis Control and its Discontents. PLoS Med 3(10): e461. doi:10.1371/journal.pmed.0030461

 

 

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AbbVie buys Shire for $54.8 Billion as Drug Deals Surge

AbbVie Inc. (ABBV) agreed to buy Shire Plc (SHP) for about 32 billion pounds ($54.8 billion), becoming the latest U.S. health-care company to shift its tax residence abroad in a record surge in industry deals.
Krishan Maggon 's insight:

AbbVie has paid a very high price for Shire, almost double of the market cap of before the bid was made public. Shire has a mixture of amphetamines to control ADHD in children and relatively poor R&D pipeline. 

 

Tax Inversion M&A are becoming costly for US companies. 

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Johnson & Johnson Reports 2014 Second-Quarter Results

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The top selling human medicinal brands  half year sales $ billions

 

Remicade  3.4

Olysio          1.2

Stelara        0.98

 

 

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Rare Diseases: A Report on Orphan Drugs in the Pipeline

Rare diseases, when taken together, are not that rare at all.

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Novartis licenses Google “smart lens” technology to develop smart contact lens

Novartis licenses Google “smart lens” technology to develop smart contact lens | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
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Pharm Exec's 2014 Dealmakers Outlook

After fading to dreary summer stock for the past few years, dealmaking is today back to center stage, but with the major roles reversed— small biotech, yesterday’s understudy, now gets top billing, while big Pharma has to work harder for its...
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FDA Approves Eagle Pharmaceuticals’ Ryanodex® (dantrolene) for the Treatment of Malignant Hyperthermia

WOODCLIFF LAKE, N.J.--(BUSINESS WIRE)--

Eagle Pharmaceuticals, Inc. (“Eagle” or “the Company”) (Nasdaq:EGRX) today announced that the U. S. Food and Drug Administration (FDA) has approved Ryanodex® (dantrolene sodium) for injectable suspension indicated for the treatment of malignant hyperthermia (MH), along with the appropriate supportive measures. MH is an inherited and potentially fatal disorder triggered by certain anesthesia agents in genetically susceptible individuals. FDA had designated Ryanodex as an Orphan Drug in August 2013. Eagle has been informed by the FDA that it will learn over the next four to six weeks if it has been granted the seven year Orphan Drug market exclusivity.

 

Eagle is the exclusive licensee of four U.S. patents for Ryanodex. Approval of Ryanodex represents two major milestones: in addition to adding to the Company’s portfolio of approved compounds, Ryanodex represents the first product to be solely marketed by the Company.

 

Ryanodex is the first significant enhancement to MH treatment options in more than three decades, reformulated to improve performance in managing MH. The product has the potential to become a new standard of care for the treatment of malignant hyperthermia, because it enables anesthesiologists to deliver a therapeutic dose of the only antidote for MH (dantrolene sodium) in a much more expedient manner than currently possible with existing formulations of IV dantrolene sodium, potentially saving lives and reducing MH-related morbidity. Ryanodex can be prepared and administered in less than one minute by a single healthcare practitioner.

Utilizing innovative nanosuspension technology, Ryanodex now provides a therapeutic loading dose of dantrolene sodium in a single vial. 250 mg of Ryanodex is mixed with only 5 mL of sterile water and administered to the patient in less than one minute. Other dantrolene sodium formulations require multiple 20mg vials reconstituted in large volumes of sterile water, a process that can take 15 to 20 minutes to mix reconstitute and administer.

“When a patient experiences malignant hyperthermia during surgery, it is a life-threatening emergency requiring immediate treatment including the administration of the ‘antidote’ drug dantrolene sodium,” said Henry Rosenberg, MD, CPE, a founder and President of the Malignant Hyperthermia Association of the United States (MHAUS). “The ability for healthcare professionals in hospitals and surgery centers to more quickly prepare and administer this new formulation of the antidote dantrolene sodium is expected to bring the crisis under control more rapidly and prevent severe complications from MH.”

Ryanodex was granted priority review status by the FDA in March 2014, a regulatory review process that expedites the review of drugs that treat life threatening and serious conditions and provide a significant improvement in safety or effectiveness over the existing therapies. Ryanodex will be available to order through national and regional drug wholesalers in August with product shipping shortly after. For more information about Ryanodex, please visit RYANODEX.COM.

About Ryanodex

Ryanodex (dantrolene sodium) is a novel formulation of the antidote for management of malignant hyperthermia (MH), a potentially fatal disorder described in more detail below. Ryanodex is available in single-use vials containing 250mg of dantrolene sodium in lyophilized powder form. It is formulated for rapid reconstitution and administration in less than one minute to patients in malignant hyperthermia crisis. Ryanodex should be administered by continuous rapid intravenous push beginning with a loading dose of 2.5 mg/kg, and continuing until symptoms subside. Ryanodex is the first significant enhancement to the management of malignant hyperthermia in more than three decades, and has the potential to become the new standard of care in malignant hyperthermia management.

About Malignant Hyperthermia

Malignant Hyperthermia is a condition that can be triggered when genetically susceptible individuals come in contact with certain inhaled (volatile) anesthetics or the muscle relaxant succinylcholine. These patients can experience tachycardia, elevated blood pressure, raised carbon dioxide levels, and very high body temperature levels. If not treated immediately, the hypermetabolic episode can be fatal.

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A Dearth in Innovation for Key Drugs

A Dearth in Innovation for Key Drugs | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it

There is clearly something wrong with pharmaceutical innovation.

Antibiotic-resistant infections sicken more than two million Americans every year and kill at least 23,000. The World Health Organization has warned that a “post-antibiotic era” may be upon us, when “common infections and minor injuries can kill.” Even the world’s tycoons consider the proliferation of antibiotic-resistant bacteria one of the crucial global risks of our times, according to a survey by the World Economic Forum.

Yet the enthusiasm of the pharmaceutical industry for developing drugs to combat such a potential disaster might be best characterized as a big collective “meh.”

 

Continue reading the main storyRelated Coverage

 Antibiotic-Resistant Infections Lead to 23,000 Deaths a Year, C.D.C. FindsSEPT. 16, 2013 Breaking the Seal on Drug ResearchJUNE 29, 2013 Federal Research Center Will Help Develop MedicinesJAN. 22, 2011Understanding the Role of the F.D.A. in Drug RegulationSEPT. 29, 2008

 

No major new type of antibiotic has been developed since the late 1980s, according to the W.H.O. From 2011 to 2013, the Food and Drug Administration approved o nly three new molecular entities to combat bacterial diseases — the lowest rate since the 1940s. “No sane company will develop the next antibiotic,” said Michael S. Kinch, who led a team at the Yale Center for Molecular Discovery tracking the evolution of pharmaceutical innovation over the last two centuries.

 

Continue reading the main story More Bugs, Fewer Drugs

The pace of development of new antibiotic molecules has slowed sharply since its peak in the 1980s, even as drug-resistant bacteria and other factors have made old antibiotics obsolete.

Average new antibiotic molecules per year

DEVELOPED

MADE OBSOLETE

+

3

molecules per year

+

2

+

1

0

1

2

3

4

’40s

’50s

’60s

’70s

’80s

’90s

’00s

’10s

(through

2013)

Source: Michael S. Kinch, Denton Hoyer, et. al., Yale Center for Molecular Discovery. This is a link to the paper.

 

 

And this is hardly the drug industry’s only problem. Antibiotics, Professor Kinch told me, “are the canary in the coal mine.”

This is particularly striking at a time when the pharmaceutical industry is unusually optimistic about the future of medical innovation. Dr. Mikael Dolsten, who oversees worldwide research and development at Pfizer, points out that if progress in the 15 years until 2010 or so looked sluggish, it was just because it takes time to figure out how to turn breakthroughs like the map of the human genome into new drugs. The pipeline today, which includes tailored treatments for cancer, newfangled vaccines and therapies for tough diseases like hepatitis C, is robust.

So far this decade, the F.D.A. has approved drugs at a pace second only to the 1990s. In 2012, the FDA approved 37 new drugs, the most in 15 years.

But the economics of the drug development, argues Professor Kinch, who in July was appointed associate vice chancellor of the University of Washington in St. Louis, are not conducive to creating the highest levels of public health.

More and more antibiotics are going out of circulation every year — either because of bacteria have become resistant to them or because they have been replaced by better or less toxic drugs. The pharmaceutical arsenal against bacterial infections shrank to only 96 different molecules by the end of last year, 17 fewer than at the turn of the century.

Nevertheless, many of the big drug companies that produced the antibiotic breakthroughs of the past have decided to drop this line of research. And few new entrants are jumping in.

“It has become very difficult to find new drug classes to fight infections,” Dr. Dolsten of Pfizer acknowledged. “There haven’t been enough incentives for the industry to take on 10 or 15 years of research.”

Antibiotics face a daunting proposition. They are not only becoming more difficult to develop, but they are also not obviously profitable. Unlike, say, cancer drugs, which can be spectacularly expensive and may need to be taken for life, antibiotics do not command top dollar from hospitals. What’s more, they tend to be prescribed for only short periods of time.

Continue reading the main story

Importantly, any new breakthrough antibiotic is likely to be jealously guarded by doctors and health officials for as long as possible, and used only as a drug of last resort to prevent bacteria from developing resistance. By the time it became a mass-market drug, companies fear, it could be already off patent and subject to competition from generics that would drive its price down.

Antibiotics are not the only drugs getting the cold shoulder, however. Research on treatments to combat H.I.V./AIDS is also drying up, according to the research at Yale, mostly because the cost and time required for development are increasing. Research into new cardiovascular therapies has mostly stuck to less risky “me too” drugs.

Neuropsychiatric diseases, including Alzheimer’s and depression, are the leading cause of disability across most of the industrial world. And they are going to get worse. Yet researchers have underscored a dearth of investment into these diseases.

Instead, pharmaceutical and biotechnology firms are betting on personalized therapies — mostly targeting specific varieties of cancers — and drugs for so-called orphan diseases, which affect very small populations. “More people are studying orphan diseases than have orphan diseases,” Professor Kinch said jokingly. Of the new drugs that the F.D.A. approved in 2013, about 70 percent were specialty drugs — which are used by less than 1 percent of the population, according to the drug benefits manager Express Scripts.

The problem, of course, lies in the industry’s incentives. The cost of developing a new drug has skyrocketed over the last three decades. A research paper by scientists from Eli Lilly suggested that in 2010, it cost $1.8 billion to bring a big new drug from conception to rollout, through the costly gantlet of clinical trials needed to prove that it is both safe and more effective than existing therapies.

Developing orphan drugs is cheaper. They receive expedited approval from the F.D.A. Clinical trials are inherently less expensive because the drugs are aimed at a small population. And insurance companies are willing to pay $100,000 a year for a drug that few patients will use.

“Companies are flocking to rare diseases,” said John LaMattina, a former head of research at Pfizer who now writes a blog about pharmaceutical research. “They might only make $500 million in sales a year, but their costs are much lower.”

Similar considerations have pushed pharmaceutical companies into newfangled biological drugs at the expense of old-fashioned compounds. Standard brand-name drugs lose 80 percent of the market within a year of patent expiration. Biologicals face much less generic competition, protected both by regulation and the fact that it is tough to determine the equivalency of different biological agents.

The wave of protests over the $84,000 cost per course of Gilead’s blockbuster new drug to treat hepatitis C, Sovaldi, highlights the kind of strain that can be caused when mass market therapies are priced like niche specialty drugs.

Continue reading the main story Continue reading the main storyContinue reading the main story

“I’ve seen nothing as potentially harmful as the exorbitant pricing displayed by Gilead,” wrote Dr. Steve Miller, the chief medical officer of Express Scripts. Regardless of whether that is worth it for the individual patient or society at large — which it probably is — the price could bankrupt Medicaid budgets around the country.

Can drug makers’ incentives be fixed? Some argue that the patent system governing drug innovation is not up to the task, and suggest handing over most drug research and development to the National Institutes of Health, which already spend tens of billions on basic research.

Tweaking the existing system might be a more feasible proposition, however. Research on new antibiotics could be encouraged by allowing shorter clinical trials for the promising molecules or guaranteeing minimum returns for groundbreaking drugs.

Patricia Danzon of the Wharton School of the University of Pennsylvania suggests recalibrating the regulatory burden to favor research in drugs with a broader potential footprint. “The decks have been stacked in favor of orphan drugs,” she said.

At the same time, new mechanisms are needed to constrain prices.

The National Health Service in Britain may have a bad reputation in the United States, but Americans could benefit from something like the country’s National Institute for Health and Care Excellence, which determines what therapies will be covered, based on their efficacy and their price.

“There’s a myth in the United States that market forces are working to control prices,” Professor Danzon said. It’s clear that they aren’t. But the market isn’t delivering the innovation we need, either.

 

 


Via Technical Dr. Inc.
Krishan Maggon 's insight:

The author raises the question of lack of newer antibiotics to combat drug resistant strains.

 

The problems of the Industry are well known

 

R&D and marketing cost of a new broad spectrum antibiotics  $ 2 billion.

 

Multiplication of infections, indications, clinical trials, market segments

 

Stringent and additional regulatory burden and requirements for approval of new antibiotics

 

Clinicians preference not to use new antibiotics for general use and opposition of healthcare providers to new antibiotics restricting market success.

 

Industry inability to charge higher prices for new antibiotics

 

 

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A Massive New Scientific Database Will Map the Spread of Infectious Disease - Motherboard

A Massive New Scientific Database Will Map the Spread of Infectious Disease - Motherboard | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
A Massive New Scientific Database Will Map the Spread of Infectious Disease Motherboard EID2 accesses and pulls metadata on pathogens' DNA and RNA sequences from NCBI's (National Center for Biotechnology Information at the US National Library of...
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A great tool to map out the local or global pandemics 

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Allergan cuts jobs and R&D to fight Valeant bid

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Allergan is better off than being a part of Valeant with its troubled past history.

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Fat burner weight loss banned drug DNP (2,4-dinitrophenol) causes deaths and severe clinical toxicity in the UK -- Kamour et al. -- Emergency Medicine Journal

Abstract

Objective 2,4-Dinitrophenol (DNP) increases energy consumption by uncoupling oxidative phosphorylation. Although not licensed as a medicine, it is sometimes used by ‘body sculptors’ and for weight loss as a ‘fat burning’ agent. This research was performed to characterise patterns of presentation, clinical features and outcomes of patients reported to the National Poisons Information Service (NPIS) in the UK after exposure to DNP.

Methods NPIS telephone enquiry records and user sessions for TOXBASE, the NPIS online information database, related to DNP, were reviewed from 1 January 2007 to 31 December 2013.

Results Of the 30 separate systemic exposures to DNP reported by telephone to NPIS during the study period (27 males, 3 females, with a median age of 23.5 years), there were 3 during 2007–2011 (inclusive), 5 during 2012 and 22 during 2013. TOXBASE user sessions also increased sharply from 6 in 2011 to 35 in 2012 and 331 in 2013. The modes of exposure reported in telephone enquiries were chronic (n=2), acute (n=12) and subacute (n=16). Commonly reported clinical features were fever (47%), tachycardia (43%), sweating (37%), nausea or vomiting (27%), skin discolouration or rash (23%), breathing difficulties (23%), abdominal pain (23%), agitation (13%) and headache (13%). There were five (17%, 95% CI 6.9% to 34%) fatalities, four involving acute overdose.

Conclusions The study indicates a substantial recent increase in clinical presentations with toxicity caused by exposure to DNP in the UK with an associated high mortality. Further steps are needed to warn potential users of the severe and sometimes fatal toxicity that may occur after exposure to this compound.

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Emerg Med J doi:10.1136/emermed-2013-203335

Original articleIncreasing frequency of severe clinical toxicity after use of 2,4-dinitrophenol in the UK: a report from the National Poisons Information ServiceAshraf Kamour1, Nathan George1, David Gwynnette1, Gillian Cooper2, David Lupton3,Michael Eddleston3, John Paul Thompson2, John Allister Vale4,Harry Krishna Ruben Thanacoody1, Simon Hill1, Simon Hugh Lynton Thomas1

+Author Affiliations

1National Poisons Information Service, Newcastle Unit, Wolfson Unit, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK2National Poisons Information Service, Cardiff Unit, University Hospital Llandough, Penarth, Vale of Glamorgan, UK3National Poisons Information Service, Edinburgh Unit, Royal Infirmary of Edinburgh, Edinburgh, UK4National Poisons Information Service, Birmingham Unit, City Hospital, Birmingham, UKCorrespondence toProf. Simon H L Thomas, Wolfson Unit of Clinical Pharmacology, Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne NE2 4HH, UK; simon.thomas@ncl.ac.ukReceived 11 November 2013Revised 17 March 2014Accepted 1 April 2014Published Online First 23 June 2014
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One injection stops diabetes in its tracks - Salk Institute - News Release

One injection stops diabetes in its tracks - Salk Institute - News Release | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
One injection stops diabetes in its tracks
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New Approaches to Filling the Gap in Tuberculosis Drug Discovery

New Approaches to Filling the Gap in Tuberculosis Drug Discovery | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
PLOS Medicine is an open-access, peer-reviewed medical journal that publishes outstanding human studies that substantially enhance the understanding of human health and disease.
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Casenghi M, Cole ST, Nathan CF (2007) New Approaches to Filling the Gap in Tuberculosis Drug Discovery. PLoS Med 4(11): e293. doi:10.1371/journal.pmed.0040293

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FDA approves Ruconest (human recombinant C1-esterase inhibitor, Pharming, Salix) for hereditary angioedema (HAE).

The U.S. Food and Drug Administration yesterday approved Ruconest, the first recombinant C1-Esterase Inhibitor product for the treatment of acute attacks in adult and adolescent patients with hereditary angioedema (HAE).
Krishan Maggon 's insight:

The U.S. Food and Drug Administration yesterday approved Ruconest, the first recombinant C1-Esterase Inhibitor product for the treatment of acute attacks in adult and adolescent patients with hereditary angioedema (HAE).

Hereditary angioedema, which is caused by having insufficient amounts of a plasma protein called C1-esterase inhibitor, affects approximately 6,000 to 10,000 people in the United States. People with HAE can develop rapid swelling of the hands, feet, limbs, face, intestinal tract, or airway. These acute attacks of swelling can occur spontaneously, or can be triggered by stress, surgery or infection. Swelling of the airway is potentially fatal without immediate treatment.

 

The safety and efficacy of Ruconest was evaluated in a multicenter controlled clinical trial. Forty-four adult and adolescent patients with acute attacks were treated with Ruconest. The most common adverse reactions reported in patients treated with Ruconest were headache, nausea and diarrhea.

Ruconest received orphan-drug designation for acute attacks by the FDA because it is intended for treatment of a rare disease or condition.

Ruconest is manufactured by Pharming Group NV, Leiden, the Netherlands, and will be distributed in the United States by Santarus Inc., a wholly owned subsidiary of Salix Pharmaceuticals Inc., Raleigh, North Carolina.

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Pfizer NONACOG ALFA (BeneFIX®) Once-Weekly Prophylaxis For Hemophilia B Effective in Phase III trial

Pfizer Inc. (NYSE:PFE) today announced the positive results of a Phase 3 study comparing a prophylaxis regimen of BeneFIX® Coagulation Factor IX (Recombinant) 100 IU/kg once-weekly to on-demand treatment in people with moderately severe to severe hemophilia B. The top-line results of the study showed that the primary study endpoint was met and hemophilia B patients taking once-weekly BeneFIX (100 IU/Kg) showed a statistically significant reduction in the annualized bleeding rate (ABR) (P < 0.0001) relative to on-demand treatment with BeneFIX.

In the study, the median ABR value, a commonly used measure of efficacy for prophylaxis regimens in hemophilia, was 2.0 for the prophylaxis regimen, compared to 33.6 for the on-demand regimen, representing a 94% decrease in bleeding rates. The mean ABR value was 3.6 for the prophylaxis period, compared to 32.9 for the on-demand treatment, which represents a reduction of 89% (P < 0.0001).


About BeneFIX

BeneFIX is a recombinant coagulation factor IX product indicated for the control, prevention and perioperative management of bleeding episodes in adult and pediatric patients with hemophilia B. BeneFIX received FDA approval in the U.S. on February 11, 1997, and was approved in the European Union later that year. It has been studied in clinical trials in both previously treated and untreated patients, and established in both on-demand and preventive care, additionally shown to help control bleeds in major and minor surgeries. BeneFIX is not approved for prophylaxis use in the United States.

BeneFIX Indications and Usage

BeneFIX is an injectable medicine that is used to help control and prevent bleeding in people with hemophilia B. Hemophilia B is also called congenital factor IX deficiency or Christmas disease.

BeneFIX is NOT used to treat hemophilia A.


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Therapeutic targeting of microRNAs: current status and future challenges

Therapeutic targeting of microRNAs: current status and future challenges | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it

MicroRNAs (miRNAs) are evolutionarily conserved small non-coding RNAs that have crucial roles in regulating gene expression. Increasing evidence supports a role for miRNAs in many human diseases, including cancer and autoimmune disorders. The function of miRNAs can be efficiently and specifically inhibited by chemically modified antisense oligonucleotides, supporting their potential as targets for the development of novel therapies for several diseases. In this Review we summarize our current knowledge of the design and performance of chemically modified miRNA-targeting antisense oligonucleotides, discuss various in vivo delivery strategies and analyse ongoing challenges to ensure the specificity and efficacy of therapeutic oligonucleotides in vivo. Finally, we review current progress on the clinical development of miRNA-targeting therapeutics.

  
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NATURE REVIEWS DRUG DISCOVERY | REVIEW

 Therapeutic targeting of microRNAs: current status and future challengesZhonghan Li& Tariq M. RanaAffiliationsCorresponding authorNature Reviews Drug Discovery (2014) doi:10.1038/nrd4359Published online 11 July 2014  
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Newer agents for psoriasis in adults. BMJ

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Newer agents for psoriasis in adultsZ K Jabbar-Lopez, academic clinical fellow, K C P Wu, Wellcome Trust clinical research fellow, N J Reynolds, professor of dermatology and honorary consultant dermatologist

Author affiliations

Correspondence to: N J Reynolds nick.reynolds@ncl.ac.uk

BMJ 2014; 349 

doi: http://dx.doi.org/10.1136/bmj.g4026 ;

(Published 09 July 2014)

Cite this as: BMJ 2014;349:g4026

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Brexpiprazole (Otsuka, Lundbeck) NDA filed in US for Schizophrenia & Depression

Otsuka Pharmaceutical Co., Ltd.|2014 July 14|News Release|Otsuka and Lundbeck Submit New Drug Application in The Us for Brexpiprazole for The Treatment of Schizophrenia and as Adjunctive Therapy for The Treatment of Major Depressive Disorder |Central Nervous System Area|Pharmaceuticals
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