FDA Approves Consensi (amlodipine and celecoxib) for Treatment of Hypertension and Osteoarthritis Pain

  

In continuation of my update on amlodipine and celecoxib

Kitov Pharma Ltd. an innovative bio pharmaceutical      co.,  announced  that the U.S. Food and Drug Administration (FDA) has approved Consensi (amlodipine and celecoxib) oral tablets for marketing.                               amlodipine                                                     celecoxib      

Consensi is a patent-protected combination of celecoxib, a non-steroidal anti-inflammatory drug (NSAID), and amlodipine besylate, an antihypertensive calcium channel blocker. Consensi was approved for once daily use in three dosage forms, corresponding to the current approved dosages of amlodipine (2.5, 5, and 10 mg) for hypertension and a 200 mg dose of celecoxib for the treatment of osteoarthritis pain.

“We are very pleased with Consensi’s approval and would like to thank the members of Kitov’s team, consultants and investigators, as well as the FDA’s Division of Cardiovascular and Renal Products, for all of their support and assistance,” said Dr. J. Paul Waymack, Chairman of Kitov's Board and Chief Medical Officer. “Consensi provides a safe and effective combination treatment option for the millions of Americans who suffer from osteoarthritis pain and hypertension.

“Now that Consensi has been approved for marketing, our clinical and regulatory teams will focus on leveraging their drug development expertise to advance NT219, an exciting investigational new drug candidate currently in development for various oncology indications.”

Isaac Israel, Kitov’s CEO, added: “This approval demonstrates the Kitov team’s ability and experience in expertly guiding Consensi through clinical trials and regulatory review, from Investigational New Drug (IND) submission to FDA approval in less than four years.

“Over 50 million Americans suffer from osteoarthritis. About 1 of 3 U.S. adults or about 75 million people have high blood pressure*, known as the “silent killer” due to the absence of noticeable symptoms. As a result, patients’ adherence to the hypertension treatment regimen is low. We believe that Consensi, as a single pill combination treatment for osteoarthritis and hypertension, presents a unique value proposition of potentially increasing treatment adherence.

“We recently expanded our commercialization network for Consensi by securing a second licensing agreement in Asia with a major Chinese pharmaceutical company. The FDA approval of Consensi puts us in a stronger position towards securing commercial partnerships for the U.S. and other key territories.”

The FDA-approved Consensi New Drug Application included the positive results from the Company’s Phase III clinical trial. These data demonstrated that the study met its primary endpoint of showing that the drug lowers daytime systolic blood pressure by at least 50% of the reduction in blood pressure achieved in patients treated with amlodipine besylate only, with statistical significance of p=0.001. Kitov also submitted the positive results from its randomized double-blind, placebo-controlled renal function Phase III/IV clinical trial of Consensi. Data from this study validated the primary efficacy endpoint achieved in the completed Phase III clinical trial. This study also demonstrated that treatment with Consensi led to a statistically significant reduction of serum creatinine, a marker of renal function, from its baseline value (p=0.0005), demonstrating improved renal function in patients treated with the combination. In contrast, neither amlodipine besylate nor placebo lowered creatinine to a statistically significant level.

Drug to treat alcohol addiction also helps with suppression of HIV

In continuation of my update  on naltrexone

A medication commonly prescribed to treat alcohol use disorder also appears to help maintain or improve suppression of HIV among individuals at risk for a lapse in HIV treatment, Yale researchers said.

In a new study, a research team led by Dr. Sandra Springer conducted a placebo-controlled, randomized trial involving individuals incarcerated in Connecticut who had both HIV and alcohol use disorders. Upon release, the 100 study participants were given either extended-release naltrexone—an FDA-approved drug that treats alcohol addiction—or a placebo. The researchers followed the individuals for six months from the time of release.

At the end of the study period, the research team found that the study participants taking extended-release naltrexone were more likely to have either maintained or improved suppression of HIV.

The study findings confirm the benefit of extended-release naltrexone for people who have alcohol use disorders and HIV disease, and demonstrate its effectiveness in helping patients to meet the goals of HIV treatment. The paper is published in the Journal of Acquired Immune Deficiency Syndrome.

More information: Sandra A. Springer et al. Extended-release naltrexone improves viral suppression levels in prisoners with HIV and alcohol use disorders who are transitioning to the community, JAIDS Journal of Acquired Immune Deficiency Syndromes (2018)

Ribavirin for treating Crimean Congo hemorrhagic fever—latest Cochrane review

In continuation of my update on Ribavirin

In a viral haemorrhagic disease where up to 40% of people developing it die, it is remarkable that doctors still do not agree whether the only recognised treatment, an antiviral drug called ribavirin, makes a difference. In a new Cochrane Review a team of authors at LSTM, along with colleagues in London, The Philippines and in Greece, evaluated the evidence to assess the effectiveness of treating Crimean Congo haemorrhagic fever (CCHF).

Crimean Congo haemorrhagic fever is spread by the bite of an infected tick, and is becoming more common, with outbreaks in Turkey, Eastern Europe and the Eastern Mediterranean. Doctors treat the infection in hospital with intravenous fluids, blood and good nursing care. The debates around ribavirinare common amongst clinicians treating the disease, with strong advocates on one side, and others who have policies not to use it, so the authors hoped the review would settle the debate.

The review authors found just one trial with 136 participants, and some observational comparative studies of 612 participants: overall the analysis did not provide a clear answer. When the authors examined studies that were often quoted as showing benefit, they were critically biased. Although fewer people died in groups receiving ribavirin, the apparent effect could be due to the drug, or equally because those getting the drug may have also been less sick, or received high quality nursing and medical care earlier in the disease.

Lead author, LSTM's Dr. Samuel Johnson, said: "Some doctors advocate giving ribavirin, and state that not to give it is even unethical. The problem is that the studies claiming to demonstrate benefit from the drug are designed in such a way we cannot separate the effect of the drug from other factors, and thus we do not know if ribavirin is effective at all."

The review clarifies the need for reliable research from a randomised control trial to establish whether ribavirin is effective. "The irony is that the strong beliefs and the widespread use of the drug may make it difficult to actually carry out the research needed" states Dr. Johnson. "What we need to know is whether it works, when it works, and how good it is."

But is there any harm in just giving it in case it works? Dr. Johnson points out, "Using unreliable research as evidence of benefit if it doesn't work could potentially waste resources and harm patients, we would also need to investigate other options. On the other hand, if ribavirin does work, then it needs to be rolled out to all patients who could benefit, which is currently not the case."

Whilst research into emerging infectious diseases and during outbreaks is difficult, the team hopes that the reviewprovides an opportunity to strengthen the call for greater steps to be taken to facilitate rigorous research providing reliable results in outbreaks of infectious diseases.

Ref : http://cochranelibrary-wiley.com/doi/10.1002/14651858.CD012713.pub2/abstract;jsessionid=9510BA3ECC4167F7B1CC8995CA6675B1.f03t03

Coffee helps teams work together, study suggests

In continuation of my update on coffee

Good teamwork begins with a cup of coffee for everyone, a new study suggests.

Researchers found that people gave more positive reviews for their group's performance on a task—and their own contribution—if they drank caffeinated coffee beforehand.

A second study showed that people talked more in a group setting under the influence of caffeinated coffee—but they also were more on-topic than those who drank decaf.

Coffee seems to work its magic in teams by making people more alert, said Amit Singh, co-author of the study and a doctoral student in marketing at The Ohio State University's Fisher College of Business.

"We found that increased alertness was what led to the positive results for team performance," Singh said.

"Not surprisingly, people who drank caffeinated coffee tended to be more alert."

Singh conducted the study with Vasu Unnava and H. Rao Unnava, both formerly at Ohio State and now with the Graduate School of Management at the University of California, Davis. The study appears online in the Journal of Psychopharmacology.

While many studies have looked at how caffeine affects individual performance, this is the first to examine the impact it has on teams, Singh said.

The first study involved 72 undergraduate students who said they were coffee drinkers. They were instructed not to drink coffee before the experiment.

Half of them first participated in what they were told was a coffee-tasting task. They were split into groups of five. After drinking a cup of coffee and rating its flavor, they were given 30 minutes of filler tasks to give the caffeine a chance to kick in. The other half of the participants did the coffee tasting at the end of the experiment.

Each group then read about and were asked to discuss a controversial topic—the Occupy movement, a liberal movement that highlighted social and economic inequality. After a 15-minute discussion, group members evaluated themselves and the other group members.

Results showed that those who drank the coffee before the discussion rated themselves and their fellow team members more positively than did those who drank coffee after the discussion, Singh said.

The second study was similar, except that 61 students all drank coffee at the beginning of the study. However, half drank decaf and the others drank caffeinated brew.

http://journals.sagepub.com/loi/jop

'Miracle treatment' long-term success for babies with diabetes

Over a decade, Emma Matthews has progressed from fearing for her son's life every night to being safe in the knowledge that his diabetes is well managed thanks to the long-term success of "miracle treatment" 

Until he was four, Jack frequently had life-threatening low blood sugar (hypos) at night as a result of the insulin injections he took several times a day to treat his neonatal diabetes. He was among the first to switch from insulin injections to sulphonylurea tablets 14 years ago, thanks to research led by the University of Exeter 'involving an international collaboration with groups in Norway, Italy, France and Poland. . Today, the team's new research in The Lancet Diabetes and Endocrinology reveals that the treatment is just as successful over the long-term and still providing excellent sugar control after 10 years.

Neonatal diabetes is diagnosed before the age of six months. Half of all cases are caused by a mutation in their KCNJ11 gene—which is involved in keeping insulin-producing cells in the pancreas working properly. This results in life threatening diabetes soon after birth. Led by the University of Exeter, the new decade-long analysis of 81 patients from 20 different countries was funded by the Wellcome Trust and Diabetes UK. It shows that these people can be treated successfully through sulphonylurea tablets, with excellent blood sugar control in the long term.

Professor Andrew Hattersley is the lead of the genetic diabetes research team at the University of Exeter Medical School. In 2006, they discovered that around half of people with neonatal diabetes can come off insulin injections and be treated more effectively with sulphonylurea tablets. In these people, the tablets provide the key to unlocking the closed door of the insulin-producing beta cells.Professor Hattersley said: "Switching from regular insulin injections was life-changing for these people who had been on insulin all their life; many described it as "a miracle treatment". Not only does this eradicate the need to inject with insulin several times a day, it also means much better blood sugar control. This is the first study to establish that this treatment is safe and works excellently for at least 10 years and all indications are that it will continue to work for decades more. This is great news for the thousands of patients who have made the switch from insulin."tablets.

Dr. Pamela Bowman, of the University of Exeter Medical School, who led the study, said: "It was incredibly exciting to help people make the switch from insulin to simple tablets—but the question was, would the benefits last? Half of people with type 2 diabetes treated with sulphonylureas no longer have good blood sugar control after five years. Our study has found that in neonatal diabetes, the tablets are safe and they work long term—with 93% of people in the study remaining on sulphonylureas alone after 10 years, with excellent blood sugar control."

Today, Jack's diabetes is so well managed that Emma has not had to worry about his condition since he changed treatment.

"It's like we've gone from living in a horror film to living in a rom com," said Emma, a nurse from Essex in the UK. "Before Jack switched treatment, every single night was a living nightmare. His blood sugar levels were all over the place, and I didn't think he was going to be alive when I went into his bedroom in the morning."

Now, Jack has recently celebrated his 18th birthday, with 150 guests. "We're so proud of our charming young man," said Emma. "He makes friends wherever he goes. Dealing with Jack's diabetes was particularly hard because of his severe learning difficulties. I honestly don't think he'd still be with us if it wasn't for the research at Exeter."

Dr. Elizabeth Robertson, Director of Research at Diabetes UK, said: "It's so important that people living with rare forms of diabetes, like neonatal diabetes, receive the right diagnosis and treatment. That's why we are delighted to have been able to help fund this vital work, demonstrating for the first time that sulphonylurea tablets are a safe and effective way for some people with neonatal diabetes to manage their condition for the long term. Moving forward, we hope research will uncover ways to prevent the developmental issues people with neonatal diabetes face.

"Nine out of ten people with this condition can switch from insulin therapy when they get the right diagnosis, so we would like all children diagnosed with diabetes under six months to be tested for neonatal diabetes, so the right treatment can help them get the best start in life."

Ref : http://www.exeter.ac.uk/news/research/title_662952_en.html

Cancer fighting effects of aspirin revealed in bowel tumor study

Researchers have shed light on how taking aspirin can help to stave off bowel cancer.

Experts found that the painkiller blocks a key process linked to tumour formation.

Regular use of aspirin is known to reduce a person's risk of developing colon cancer but the drug's tumour fighting properties have not been well understood.

Researchers at the University of Edinburgh focused on a structure found inside cells called the nucleolus.

Activation of the nucleolus is known to drive tumour formation and dysfunction has also been linked to Alzheimer's and Parkinson's.

The team at the University's Cancer Research UK Edinburgh Centre tested the effects of aspirin on cells grown in the lab and on tumour biopsies removed from colon cancer patients.

They found that aspirin blocks a key molecule called TIF-IA, which is essential for the nucleolus to function.

Not all colon cancer patients respond to aspirin but the researchers say their findings could help pinpoint those most likely to benefit.

Aspirin has side effects that include internal bleeding and it can cause certain types of stroke. Long term use is not recommended. The researchers say the study paves the way for the development of new, safer therapies that mimic aspirin's effects.

The research, published in Nucleic Acids Research, was funded by the Medical Research Council and the Biotechnology and Biological Sciences Research Council. Worldwide Cancer Research, Bowel and Cancer Research and The Rosetrees Trust also supported the work.

Dr. Lesley Stark, of the Cancer Research UK Edinburgh Centre at the University of Edinburgh, said: "We are really excited by these findings as they suggest a mechanism by which aspirin may act to prevent multiple diseases. A better understanding of howaspirin blocks TIF-IA and nucleolar activity provides great promise for the development of new treatments and targeted therapy."

Experimental drug restores some bladder function after spinal cord injury, study finds

An experimental drug that blocks abnormal neural communication after spinal cord injury could one day be the key to improving quality of life by improving bladder function, new research suggests.

Researchers at The Ohio State University tested the drug—which is currently available only for research—to gauge its potential to improve bladder function after spinal cord injury in mice and saw promising results.

The experimental drug (LM11A-31) appears to help by blocking the dual activity of pro-nerve growth factor (proNGF) and a receptor called p75. ProNGF is known to be secreted from the cell after nerve injury.

After a month-long treatment after spinal cord injury in mice, bladder volume decreased significantly to a level close to normal, said lead researcher Sung Ok Yoon, an associate professor of biological chemistry and pharmacology at Ohio State.

The study appears in The Journal of Clinical Investigation.

In humans, spinal cord injuries sever the communication between the bladder and the brain, leading to the loss of the normal ability to urinate at will. This leads to bladder over-filling, which causes high pressure and bladder enlargement due to thickening of the muscular bladder walls. This can cause urine to return to the kidney, which can lead to kidney infection and disease, Yoon said.

People with spinal cord injury typically rely on a catheter to pass urine.

Eventually, Yoon said, a new circuit of nervous-system communication is formed within the spinal cord, and it allows urine to be expelled unexpectedly, causing incontinence.

"There are no approved medications to treat bladder dysfunction brought on by spinal cord injuries, something that is a major concern and diminishes quality of life for these patients," Yoon said."This drug appears to help maintain near-normal bladder pressure and less unexpected expulsion of urine in mice."

Because the experimental drug does not restore normal communication between the bladder and the brain, it is certainly not a cure, Yoon said. Based on the mouse study, however, patients are likely to experience reduced bladder volume, which would lower the risk of bladder infection and reflux to the kidney, and less incontinence.

"The structure as well as the integrity of the neural communication in the bladder is expected to improve as well, contributing to the overall health of the bladder," she said.

Yoon and her collaborators also confirmed—through samples from two recent spinal-cord injury patients—the presence of proNGF in the urine within hours after injury. Urine from healthy people does not contain the growth factor. Yoon said that since the drug counteracts proNGF action, these findings could potentially be extended to further research into other types of bladder dysfunction besides spinal cord injury.

https://www.jci.org/articles/view/97837

Ref : https://www.jci.org/

Spironolactone may be an alternative to antibiotics in women's acne treatment

In continuation of my update on spironolactone

In a finding that suggests the potential for practice change that would reduce the use of antibiotics in dermatology, researchers in the Perelman School of Medicine at the University of Pennsylvania have found the diuretic drug spironolactone may be just as effective as antibiotics for the treatment of women's acne. The study, published this month in the Journal of Drugs and Dermatology, found patients who were originally prescribed spironolactone changed to a different drug within one year at almost the same rate as those who were prescribed antibiotics. The prescription change is a proxy for ineffectiveness, since switching is often the result of treatment failure due to lack of efficacy, side effects, cost, or other factors.

Acne is one of the most common diseases in the world. It affects 85 percent of people under the age of 18, but it also regularly impacts adults. More than 50 percent of women in the United States are treated for acne between the ages of 20 and 29, while more than 35 percent are treated between the ages of 30 and 39.

Oral antibiotics are the most common systemic treatment for acne, and when combined with the large patient population, the result is that dermatologists prescribe the highest level of antibiotics per provider among all medical specialties, according to the Centers for Disease Control—a fact that contributes to concerns about increased resistance to antibiotics across all fields of medicine.

"It's clear that a safe alternative to oral antibiotics could have a huge benefit, and our data show spironolactone may be that alternative," said the study's lead author John S. Barbieri, MD, MBA, Dermatology chief resident at Penn. David J. Margolis, MD, Ph.D., a professor of Dermatology, was the study's senior author.

Spironolactone, marketed under the name aldactone, is currently approved to treat high blood pressure, heart failure, and conditions that cause people to retain fluid. It blocks the effects of male hormones like androgen, meaning it's not an option to treat acne in men. However, those same anti-hormonal effects can help prevent acne outbreaks in women. As a result, some dermatologists use it to treat female acne patients.

Researchers compared data on 6,684 women and girls taking spironolactone to 31,614 who were prescribed antibiotics. Within a year, 14.4 percent of spironolactone patients and 13.4 percent of antibiotic patients had switched to alternative treatments, suggesting each treatment was working at almost the same rate, despite the fact that tetracycline-class antibiotics are prescribed five times as frequently.

"These numbers suggest dermatologists should consider spironolactone first instead of antibiotics when it comes to women with acne," Barbieri said.

In addition to the benefits for antibiotic stewardship, Barbieri pointed to several studies showing long-term oral antibiotic use may be associated with antibiotic resistance, lupus, inflammatory bowel disease, and even colon and breast cancer.

"This indicates spironolactone may have a better safety profile than oral antibiotics, which is another factor that makes it such an appealing option," Barbieri said. He also noted spironolactone is less expensive, which may be relevant to patients with high deductibles or who are uninsured.

Spironolactone is not approved for the treatment of acne by the U.S. Food and Drug Administration despite expert opinion supporting its use, and Barbieri says the findings of this study should be confirmed by a randomized controlled trial that directly compares the two treatment options.

FDA Advisory Committee Recommends the Approval of Baricitinib 2mg, but not 4mg, for the Treatment of Moderately-to-Severely Active Rheumatoid Arthritis

Eli Lilly and Company (NYSE: LLY) and Incyte Corporation (NASDAQ: INCY) announced  that the U.S. Food and Drug Administration's (FDA) Arthritis Advisory Committee recommended approval of the 2-mg dose of baricitinib, a once-daily oral medication for the treatment of moderately-to-severely active rheumatoid arthritis (RA) for adult patients who have had an inadequate response or intolerance to methotrexate. While the Advisory Committee unanimously supported the efficacy of the 4-mg dose of baricitinib, it did not recommend approval of the 4-mg dose of baricitinib for the proposed indication based on the adequacy of the safety and benefit-risk profiles.

"We are confident that baricitinib, if approved, can help people in the U.S. manage the challenges of living with RA," said Christi Shaw, president of Lilly Bio-Medicines. "While we are disappointed with the Advisory Committee's assessment of the data for the 4-mg dose, we are confident in the positive benefit-risk profile of both the 2-mg and the 4-mg doses. We look forward to continuing our work with the FDA on our New Drug Application (NDA) and are hopeful that baricitinib will receive approval in the coming months."

Baricitinib 2-mg and 4-mg doses are approved in more than 40 countries, including the member states of the European Union and Japan.

For both doses, the Advisory Committee voted to support the assessment that baricitinib's data provide substantial evidence of efficacy. For the 2-mg dose, the Advisory Committee voted in favor of the assessment that baricitinib's safety data adequately support its approval. For the 4-mg dose, the Advisory Committee voted against the assessment that baricitinib's safety data was adequate to support its approval based on the proposed indication.

The Advisory Committee's recommendation was based on baricitinib's global development program, which included four completed Phase 3 studies. In total, 3,492 patients, who represented a range of treatment experiences, received baricitinib in the global RA development program. The Phase 3 studies evaluated baricitinib's treatment impact related to RA signs and symptoms, physical function, joint damage progression and other patient-reported outcomes. The Phase 3 program also evaluated recognized risks for RA patients, including serious infection, malignancy, major adverse cardiovascular events (MACE), venous thromboembolism (VTE), and gastrointestinal perforations, along with key laboratory changes. The safety profile of baricitinib is based on 7,860 patient-years of exposure.

"Despite advances in the management of RA over the last 20 years, which include early treatment, optimized use of traditional therapies for rheumatic disease and the advent of newer medications such as biologics, many patients are still struggling to meet treatment targets, and live with debilitating pain, fatigue and other symptoms of RA," said Peter Taylor, MA, PhD, professor, University of Oxford, an expert who attended the Advisory Committee Meeting. "Baricitinib could be a promising option for RA patients in the U.S. who are not achieving adequate disease control with currently available treatments."

The FDA is not required to follow the Advisory Committee's recommendation, but will consider it during its review of the NDA for baricitinib.

FDA Advisory Committee Recommends the Approval of Baricitinib 2mg, but not 4mg, for the Treatment of Moderately-to-Severely Active Rheumatoid Arthritis

Alkermes Announces FDA Acceptance for Review of New Drug Application for ALKS 5461 for the Adjunctive Treatment of Major Depressive Disorder

In continuation of my update on ALKS-5461  .................

We know that, Buprenorphine/samidorphan (developmental code name ALKS-5461) is a combination drug formulation of buprenorphine and samidorphan acting as a μ-opioid receptor antagonist which is under development by Alkermes as an adjunct to antidepressant therapy in treatment-resistant depression (TRD).

Buprenorphine 

 Samidorphan

Alkermes plc (Nasdaq: ALKS)announced that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for ALKS 5461, a novel, once-daily, oral investigational medicine for the adjunctive treatment of major depressive disorder (MDD) in patients with an inadequate response to standard antidepressant therapies. FDA's target action date for the ALKS 5461 NDA is Jan. 31, 2019.

FDA's acceptance of the ALKS 5461 NDA and rescission of the Refusal to File letter issued March 30, 2018 follows productive interactions with the Agency in which Alkermes clarified certain aspects of the NDA submission. No additional data or analyses were submitted by Alkermes to FDA.

"FDA's filing of the ALKS 5461 application is a positive step forward for patients suffering from major depressive disorder, a serious disease where inadequate response to existing antidepressants remains a well-known and significant treatment limitation, and where there have been no new pharmacological treatment approaches in 30 years," stated Craig Hopkinson, M.D., Chief Medical Officer and Senior Vice President of Medicines Development and Medical Affairs at Alkermes. "We will continue to engage with the FDA throughout the review process, as we work to bring this important medicine to patients."

The NDA filing for ALKS 5461 is based on results from a clinical efficacy and safety package with data from more than 30 clinical trials and more than 1,500 patients with MDD. Throughout the clinical development program, ALKS 5461 demonstrated a consistent profile of antidepressant activity, safety and tolerability in the adjunctive treatment of MDD.

Achaogen Announces FDA Advisory Committee Voted Unanimously in Favor of Plazomicin for Treatment of Adults with Complicated Urinary Tract Infections

Achaogen, Inc. (NASDAQ:AKAO), a late-stage biopharmaceutical company developing innovative antibacterials addressing multi-drug resistant (MDR) gram-negative infections, announced that the U.S. Food and Drug Administration's (FDA) Antimicrobial Drugs Advisory Committee voted on the two points for Advisory Committee consideration as follows:

1. Has the applicant provided substantial evidence of the safety and effectiveness of plazomicin for the treatment of complicated urinary tract infections?

Result: (15-0-0) There were 15 yes votes and zero no votes. No members of the panel abstained.

2. Has the applicant provided substantial evidence of the safety and effectiveness of plazomicin for the treatment of bloodstream infections in patients with limited or no treatment options?

Result: (4-11-0) There were four yes votes and 11 no votes. No members of the panel abstained.

There were 16 panel members at the meeting, one of whom departed prior to the vote and was therefore not present for the voting.

"We are encouraged by the Committee's unanimous vote in favor of plazomicin for complicated urinary tract infections (cUTI). The discussion underscored the real-world challenges that healthcare providers face every day given limited or inadequate treatment options for certain pathogens," said Blake Wise, Achaogen's Chief Executive Officer. "Regarding bloodstream infections, the Limited-Population Antibacterial Drug pathway, or LPAD, is a novel approach that enables the FDA to consider the benefits and risks for the sickest patients who have few or no available treatment options, and to approve antibiotics like plazomicin that we believe, have the potential to address these limited patient populations."

The FDA is not bound by the Committee's votes but takes its input into consideration when reviewing marketing applications. Plazomicin has a Prescription Drug User Fee Act (PDUFA) date of June 25, 2018. If the FDA approves plazomicin by this target action date, Achaogen expects to launch plazomicin in the U.S. soon thereafter.

http://investors.achaogen.com/releasedetail.cfm?ReleaseID=1066053

FDA Advisory Committee Votes in Favor of Waylivra (volanesorsen) for Treatment of Familial Chylomicronemia Syndrome

Akcea Therapeutics, Inc. (NASDAQ:AKCA), an affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. (NASDAQ:IONS)   announced that the U.S. Food and Drug Administration’s (FDA) Division of Metabolism and Endocrinology Products Advisory Committee voted 12-8 to support approval of Waylivra (volanesorsen) for the treatment of people with familial chylomicronemia syndrome (FCS). The committee’s non-binding recommendation will be considered by the FDA in its review of Akcea’s New Drug Application for Waylivra. The PDUFA date for completion of the review of Waylivra is August 30, 2018.

3’—A*—G*—mC*—T*—T*—dmC—dT—dT—dG—dT—dmC—dmC—dA—dG—dmC—T*—T*—T*—A*—T*—5’




* = 2’-O-(2-methoxyethyl)

m = 5-methyl

d = 2’-deoxy

“We thank the committee members for their time and their comments today. The Committee’s majority vote in favor of approval is an important positive step to bring Waylivra to people with FCS who have no adequate treatment options,” said Paula Soteropoulos, chief executive officer of Akcea Therapeutics. “We look forward to working with the FDA to complete the final stages of regulatory review for Waylivra. We are committed to the FCS community and will continue to focus on bringing Waylivra, potentially the first and only treatment, to people living with this serious and potentially fatal disease.”

“Given the severity of FCS and the burden it places on patients, the need for a therapy is critical. The progress in development of a potential first-ever treatment is very encouraging. The planned efforts in monitoring and education are designed to achieve the highest levels of patient adherence, compliance and safety,” said Dr. Seth Baum, president, American Society for Preventive Cardiology. “The medical community is eager to have a medicine to treat our patients with FCS.”

The Advisory Committee reviewed data from two Phase 3 clinical trials, APPROACH and COMPASS, as well as the ongoing APPROACH Open Label study for Waylivra. Results from the phase 3 APPROACH trial, the largest study ever conducted in patients with FCS, show that patients with FCS treated with Waylivra achieved a statistically significant mean reduction in triglycerides of 77% from baseline and decreased risk of pancreatitis. The most common adverse events in the APPROACH study were injection site reactions and platelet declines. The Committee's input will be considered by the FDA in its review of the New Drug Application for Waylivra. The FDA is not bound by the Committee's guidance, but takes its advice into consideration when reviewing investigational medicines. Waylivra is also under regulatory review in the European Union and Canada.

“People with FCS have severely elevated triglycerides, which lead to multiple severe daily and chronic symptoms, such as abdominal pain and increased risk for pancreatitis, which can be fatal. Waylivra is the first drug to demonstrate substantial triglyceride lowering in clinical trials in people with FCS,” said Brett P. Monia, chief operating officer at Ionis. “Waylivra illustrates how our antisense technology can create targeted drugs for people living with severe diseases who currently have no available therapeutic options.”

Ref : http://ir.akceatx.com/news-releases/news-release-details/fda-advisory-committee-votes-favor-waylivra-treatment-familial

AcelRx Announces FDA Acceptance of NDA for DSUVIA

In continuation of my update on  sufentanil

AcelRx Pharmaceuticals, Inc. (Nasdaq: ACRX), (AcelRx or the Company), a specialty pharmaceutical company focused on innovative therapies for use in medically supervised settings, today announced the acceptance of its New Drug Application (NDA) resubmission for DSUVIA™ by the U.S. Food and Drug Administration (FDA). The FDA considers the DSUVIA NDA resubmission a complete class 2 response to their October 2017 action letter. Therefore, the FDA has assigned a PDUFA (Prescription Drug User Fee Act) goal date of November 3, 2018.

The acceptance of the DSUVIA NDA resubmission is yet another important milestone achieved by the Company this year," stated Vince Angotti, Chief Executive Officer of AcelRx. "We are one step closer to potentially delivering a new, non-invasive treatment option for the management of moderate-to-severe acute pain for adult patients in medically supervised settings. We believe DSUVIA, if approved, also has the opportunity to help U.S. hospitals manage through the intravenous opioid shortage1 they are currently experiencing in their facilities," continued Angotti.

About DSUVIA™ (sufentanil sublingual tablet), 30 mcg

DSUVIA™ (sufentanil sublingual tablet, 30 microgram), known as DZUVEO™ outside the United States, has a proposed indication for the management of moderate-to-severe acute pain in medically supervised settings, in adult patients and was designed to eliminate dosing errors associated with IV administration via its non-invasive single-dose applicator (SDA) administered by health care professionals. Sufentanil is an opioid analgesic currently marketed for intravenous (IV) and epidural anesthesia and analgesia. The sufentanil pharmacokinetic profile when delivered sublingually avoids the high peak plasma levels and short duration of action observed with IV administration. In Europe, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion, recommending approval of DZUVEO. A decision by the European Commission on whether to grant the marketing authorization is expected in the third quarter of 2018.

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AcelRx Announces FDA Acceptance of NDA for DSUVIA

FDA to Review Zynquista (sotagliflozin) as Potential Treatment for Type 1 Diabetes

The U.S. Food and Drug Administration (FDA) has accepted Sanofi's regulatory filing for Zynquista (sotagliflozin). The investigational oral treatment would be used in addition to insulin therapy to improve blood sugar control in adults with type 1 diabetes.

"If approved, Zynquista would be the first oral antidiabetic drug approved in the U.S. for use by adults with type 1 diabetes, in combination with insulin." says Jorge Insuasty, Senior-Vice President, Global Head of Development, Sanofi. "We look forward to working with the FDA through the review process with a view towards bringing this investigational medicine to adults with type 1 diabetes in the U.S."

Developed in partnership with Lexicon Pharmaceuticals, Inc., Zynquista is an investigational oral dual inhibitor of SGLT-1 and SGLT-2, proteins that influence how the intestines and kidneys absorb and eliminate sugar (glucose) resulting in improved glucose control and additional clinical benefits.

"After decades of little change and innovation, the treatment of type 1 diabetes has begun to shift significantly and, if approved, our dual SGLT-1 and SGLT-2 inhibitor, Zynquista, would be the first approved oral therapy used in combination with insulin to improve glycemic control and patient outcomes for adults in the United States who are living with type 1 diabetes," said Pablo Lapuerta, M.D., executive vice president and chief medical officer, Lexicon. "The acceptance of the NDA filing moves us closer to providing a meaningful option for people with type 1 diabetes and we look forward to continuing to work closely with the FDA during the review process."

The FDA New Drug Application for sotagliflozin is based on data from the inTandem clinical trial program which includes three Phase 3 clinical trials assessing the safety and efficacy of Zynquista in approximately 3,000 adults with inadequately controlled type 1 diabetes.1–3 The safety and efficacy data have not yet been evaluated by any regulatory authority.

The target FDA action date under the Prescription Drug User Fee Act (PDUFA) is anticipated to be March 22, 2019. Sanofi also submitted a regulatory application to the European Medicines Agency earlier this year.

TherapeuticsMD Announces FDA Approval of Imvexxy (estradiol vaginal inserts) for the Treatment of Dyspareunia Due to Menopause

In continuation of my update on estradiol

TherapeuticsMD, Inc., an innovative women's healthcare company,  announced that the United States Food and Drug Administration (FDA) has approved Imvexxy (estradiol vaginal inserts) for the treatment of moderate-to-severe dyspareunia (vaginal pain associated with sexual activity), a symptom of vulvar and vaginal atrophy (VVA), due to menopause. Imvexxy is the only product in its therapeutic class to offer a 4 mcg and 10 mcg dose, the 4 mcg representing the lowest approved dose of vaginal estradiol available.

"Imvexxy is a bio-identical vaginal estrogen product that offers a fraction of the estrogen contained in the average doses of many existing products currently on the market," said Brian Bernick, MD, Chief Clinical Officer of TherapeuticsMD. "Imvexxy is the only product specifically designed to be applicator-free. It dissolves completely without mess or additional clean-up, and can be used anytime of day. It allows women the freedom to immediately return to their normal daily activities. Studies showed that, in patients who used Imvexxy, systemic absorption of estradiol remained within postmenopausal range."

"We are excited to bring Imvexxy to market as TherapeuticsMD's first FDA-approved drug as we strive to be the premier Women's Health Company," said Robert Finizio, Chief Executive Officer of TherapeuticsMD. "Imvexxy reflects our long-standing corporate mission and commitment to health solutions that women want, based on the concepts of medical need, efficacy, safety, simplicity, and affordability. Imvexxy will be offered at a price in parity with other products that have been on the market for 10 to 30 years. By ensuring patients can access Imvexxy at an affordable price, TherapeuticsMD is doing the right thing for women."