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Posts Tagged ‘drug approvals

Science Policy Around the Web – July 3, 2018

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By: Jennifer Patterson- West, PhD

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Women’s Health

Pregnant Women: Avoid Soft Cheeses, But Do Get These Shots

During pregnancy, expecting mothers are advised to avoid a slew of activities, foods and medications to protect their unborn child including alcohol, unpasteurized cheese, lunch meat, ibuprofen and even hot baths.  What may come as a surprise is that the American College of Obstetricians and Gynecologist (ACOG) recently released an immunization guide that advise expecting mothers on which vaccine to receive during pregnancy and which to avoid.  It also highlights vaccines that should only be given to high risk patients or immediately following birth.

The influenza vaccine is given to protect the mother who is more likely to get seriously sick if she contracts the flu. The Tdap vaccine is recommend at 27-36 weeks of gestation during each pregnancy to boost the maternal immune system in order to protect the newborn.  In addition to this recommendation, the CDC also recommends anyone who plans to come in contact with the baby receive the Tdap vaccine at least 2 weeks prior if they are not up-to-date with their vaccines.

In 2015, 20,762 cases of pertussis, the infectious agent responsible for whooping cough, were reported to the CDC by the State Health Department. Although this is a 37% decrease compared to 2014, these numbers can be further improved by improved coverage of those in contact with unvaccinated infants.

The goal of these guidelines is to further reduce the number of cases of whooping cough in babies younger then 3 months old, a time when the disease is most fatal.  For babies that contract whooping cough, half of them will end up in the hospital and some will die. Although these recommendations are not new, many expecting parents may be unaware of what vaccinations should be received during pregnancy. The CDC estimates that only half of pregnant women in the United States receive the Tdap vaccine.

(Selena Simmons-Duffin, NPR)

Drug Approvals

FDA approves Country’s first medicine made from marijuana

On June 25, the FDA announced the approval of the first drug with an active ingredient derived from marijuana.  Epidiolex is an oral solution approved for the treatment of seizures associated with  Lennox-Gastaut syndrome and Dravet syndrome.

Both are rare and severe forms of epilepsy.  Lennox-Gastaut syndrome typically presents between the ages of 3 and 5 as frequent seizures. The majority of children with the syndrome exhibit learning and intellectual disabilities and delayed motor skills.

Dravet syndrome is a rare genetic disorder that presents as frequent fever-related seizures during the first year of life.  Children with this disorder commonly have underdeveloped language and motor skills. With age, other seizure types and symptoms typically arise that are potentially life-threatening. No drug had previously been approved specifically for the treatment of Dravet syndrome, which is why FDA granted Priority Review to the application and orphan drug designation.

The approval of Epidiolex has the potential to increase the quality of life for many patients with these rare syndromes The active ingredient derived from marijuana is cannabidiol (CBD), which was shown to be effective at reducing the frequency of seizures compared to a placebo in clinical trials.  Epidiolex does not contain THC, the psychoactive component of marijuana, that causes a euphoric high.

CBD is currently classified as a Schedule I substance in accordance with the Controlled Substance Act (CSA).  Schedule I substances include drugs or chemicals with no accepted medical use and a high potential for abuse.  Although more than thirty states have passed legislation that permits the use of medical marijuana or CBD, cannabis is still categorized as a Schedule I substance under the CSA.

The approval of Epidiolex provides a path forward for the approval of other marijuana-derived medications, or treatment of additional indications, that do not conflict with federal law.  The FDA Commissioner, Dr. Scott Gottlieb, stated that “We’ll continue to support rigorous scientific research on the potential medical uses of marijuana-derived products and work with product developers who are interested in bringing patients safe and effective, high quality products.”

(Andrew Joseph, STAT News)

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July 3, 2018 at 2:19 pm

Science Policy Around the Web – February 13, 2018

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By: Saurav Seshadri, PhD

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Experimental drugs

Trump Endorses “Right to Try” for Terminally Ill Patients

Proponents of the ‘right to try’ received some encouragement from President Trump’s recent State of the Union address, in which he announced his support for such legislation at the federal level.  Right to Try laws are designed to allow terminally ill patients to obtain unapproved but possibly lifesaving drugs directly from pharmaceutical companies, without involving the FDA.  While such laws already exist in 38 states, they are currently superseded by the Food, Drug, and Cosmetic Act; a bill that would eliminate this legal conflict was passed by the Senate last August, but has yet to be approved by the House of Representatives.

In general, Right to Try laws permit terminal patients, with their informed consent, to access investigational treatments if recommended by a physician.  However, they do not mandate that the manufacturer provide the drug or that insurance cover it, and in some cases, they absolve drugmakers and physicians from liability for adverse outcomes.  In addition, the FDA already offers a path to treatment for terminal patients under its ‘expanded access’ program, in which patients are treated as clinical trial participants and their doctor’s office becomes a satellite site, with appropriate regulatory oversight.  Opponents of Right to Try legislation, including FDA Commissioner Scott Gottlieb, argue that bypassing such oversight would critically undermine the clinical trial process (for example, a patient death from a drug obtained under a Right to Try law would not factor into the FDA’s consideration of that drug for approval).  They also suggest that these laws provide false hope for desperate patients – experimental drugs need only clear the safety phase of FDA trials, meaning no data exists on their efficacy – and open patients up to risks of physical harm and medical fraud.

Despite these concerns, Right to Try laws have gained momentum on the strength of anecdotal success stories, and politicians’ unwillingness to appear heartless towards patients suffering from terminal diseases.  Yet in reality, without securing financial support for patients, these laws are likely to result in some patients going bankrupt. Without requiring that treatments be demonstrated to be beneficial and at least safe, these laws are likely to result in patients pursuing ineffective treatments, while reducing their quality of life by enduring side effects, risking complications, and forgoing hospice care.  The future of Right to Try legislation may be influenced by new Health and Homeland Security Secretary (and former Eli Lilly executive) Alex Azar, who seems likely to support Trump’s agenda, though he didn’t mention the right to try in his response to the State of the Union address.  Ideally, the final bill will prioritize the existing drug review process, ensuring safety for the majority of patients while still providing hope for the sickest.

(Ike Swetlitz, STAT news)

Chemical safety

The truth about glyphosate may be getting lost in the weeds

The World Health Organization (WHO) kicked off a massive controversy in 2015 with its report labeling glyphosate, a component of an herbicide marketed by Monsanto, as ‘probably carcinogenic to humans’.  The report has faced stiff opposition from Republican Representatives on the US House Science, Space, and Technology Committee, largely fueled by a pair of Reuters reports suggesting that key data was suppressed by the WHO to support its conclusion.  Now Dr. Christopher Wild, Director of the group that conducted the research (the IARC, International Agency for Research on Cancer) has sent a detailed response to the Committee to rebut these criticisms and defend its original finding.

The response, which was presented at a recent Committee hearing by Democratic Representative Suzanne Bonamici, specifically addresses two issues raised by Reuters.  First, that a senior scientist failed to disclose data that would have exonerated glyphosate: the data was unpublished and therefore didn’t meet IARC’s criteria for consideration.  Second, that the published version of the report had several changes from an earlier draft, all of which involved deleting or revising statements that cast doubt on glyphosate’s link to cancer.  Dr. Wild claims that most of these changes were related to a single review article, whose conclusions were reconsidered when it was found to have been ghostwritten by a Monsanto scientist, and that its drafts are works in progress and therefore confidential.  Still, the response doesn’t explain the IARC’s discrepancy with other regulatory agencies: the European Food Safety Authority (EFSA) and US Environmental Protection Agency (EPA) have both found glyphosate to be safe, and claim their review processes are more transparent than the IARC’s.

The IARC’s stance on glyphosate puts it in a delicate position with the US government, from which it receives ‘valuable support’, especially as the topic becomes more partisan.  Republican lawmakers have already threatened to pull funding to the IARC, ostensibly over its refusal to provide a witness for the hearing (Dr. Wild invited them to visit his facility in France instead).  On the other side, the EPA’s assessment has been called into question by the discovery that an EPA official may have colluded with Monsanto to ‘kill’ investigation into glyphosate, leading Democratic Representative Ted Lieu to request a probe into the issue.  In the midst of a heated debate on climate change, the glyphosate story may initially seem to be another case of Republicans denying science to fight regulations and side with big business; however, the reality may be more complicated.  A recent protest in Paris by farmers, opposed to a proposed ban on glyphosate, highlights how those most affected by such policies must balance their economic stability against potential health risks.  Ultimately, though lawmakers may earn political points by siding with these individuals, if the price is discrediting accurate science and eroding public trust in regulatory agencies, no one wins.

(Corbin Hiar, E&E News)

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February 13, 2018 at 6:01 pm

Science Policy Around the Web – February 6, 2018

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By: Liuya Tang, PhD

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Cancer treatment

Breast cancer treatments can raise risk of heart disease, American Heart Association warns

Common cancer treatments include surgery, chemotherapy and radiation therapy. Chemotherapy and radiation therapy are always applied before or after surgical removal of a tumor, or applied to cancer patients when surgery cannot be performed. Not only will they attack tumor cells, chemotherapy or radiation therapy will also damage normal cells at the same time, which increases risks for other diseases. A recent report in the journal Circulation said that breast cancer treatments can raise risk of heart disease. It has been noticed that “breast cancer survivors who are 65 and older and were treated for their cancer are more likely to die of cardiovascular problems than breast cancer.” The possible cardiovascular consequences of breast cancer treatments may not be new to oncologists, but new cancer treatments have complex side effects which may not fully understood as they work differently from conventional cancer treatments. For example, the newly-developed cancer treatment, immunotherapy, stimulates the patient’s immune system to attack tumors, but sometimes the surging immune response can overshoot its target and attack healthy tissues and organs.

It is not a good idea to stop cancer treatment due to side effects, as saving ones life from a dangerous cancer is critical. But for this double-edged sword, how to make one edge blunt while keeping the other edge sharp? This requires surgeons and oncologists to work together to make a personalized treatment plan. As suggested by Dr. Deanna Attai, a breast surgeon at the University of California at Los Angeles, the patients with less-aggressive tumor may skip chemotherapy based on the test results on the cancer’s risk of recurrence. In addition, adopting different ways to deliver chemo drugs and developing more-targeted radiation can reduce the risks of cardiac damage for breast cancer patients.

It is not solely a doctor’s responsibility to monitor the side effects of cancer treatments, patients also need to be aware of what types of treatments and what the possible side effects are. Wrong treatments of side effects can aggravate symptoms, which may lead to severe problems. The new emerging immunotherapy presents a big challenge to the health care system as the side effects are not thoroughly understood. Doctors’ organizations and nonprofit groups are joining information campaigns to narrow the knowledge gap on immunotherapy, which will help patients better understand procedures of cancer treatment and manage any side effect if it occurs.

(Laurie McGinley, The Washington Post)

 

Drug development

Racing to replace opioids, biopharma is betting on pain drugs with a checkered past

The opioid epidemic has become a significant problem in the US, as 116 people died every day from opioid-related drug overdoses in 2016. To resolve this issue, biopharma continues to develop pain drugs. The class of drugs are called NGF inhibitors, which were halted by FDA in 2010 due to their severe side effects. NGF is short for nerve growth factor, which is a neuropeptide. When an injury occurs, the production of NGF is increased, which helps the brain perceive the pain. Theoretically antibodies that specifically bind NGF before it reaches cell receptors could be a good choice to inhibit NGF function, therefore treating people with chronic pain. But it was found that NGF antibodies are not suitable for a subset of patients with osteoarthritis, for whom treatment lead to dramatic joint deterioration. To obtain FDA’s approval of entering further clinical trials, drug companies showed that NGF drugs will probably be safe for patients not at risk of joint deterioration and shouldn’t be taken with nonsteroidal anti-inflammatory drugs such as Advil. So the clinical study was resumed in 2015. Will it become a replacement drug of opioids? Will the benefits outweigh its risks? The results will be put on table this year after drug companies finish their Phase 3 studies.

 

The severity of the opioid epidemic and the high need of non-addictive painkillers have kept drug companies optimistic about developing NGF drugs despite the side effects. However, there are opposite voices. The watchdog group Public Citizen criticized that the side effects are obvious and “further pursuit of testing in humans was an unreasonable course of action”. Criticisms also come from the business side. Leerink analyst Geoffrey Porges has warned Regeneron’s NGF drug would carry “all of the liabilities” of the past and scolded their continuing to pour money into the project. The failure has already been seen in the development of fulranumab, which is one type of NGF antibody. Even if NGF antibodies were approved by FDA, doctors would have concerns for prescribing a medication with potentially dangerous outcomes for patients with certain conditions.

(Damian Garde, STAT News)

 

 

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February 6, 2018 at 10:53 pm

Science Policy Around the Web – August 28, 2015

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By: Sylvina Raver, Ph.D.

Photo source: pixabay.com

Drug Policy

FDA approves drug for female libido amid controversy and lingering questions

On August 18, the FDA approved the drug flibanserin to treat female sexual dysfunction. Flibanserin, which will be sold under the brand name Addyi, has been billed as “female Viagra” and as a remedy for Female Sexual Interest/Arousal Disorder. With its recent approval, flibanserin becomes the first drug approved to specifically address female sexual problems, compared to the 26 pharmaceuticals approved for this purpose in men. Indeed, highlighting this stark gender inequality in treatment options was instrumental in the drug’s FDA approval. Sprout Pharmaceuticals, Addyi’s manufacturer, joined with non-profit and health care organizations in 2013 to form Even The Score, an advocacy organization that waged a hugely successful lobbing campaign to seek FDA approval of flibanserin by framing its approval as one “women’s sexual health equality.” Critics say that an accusation that the FDA is biased against women, championed by Even The Score, had greater influence on the drug’s approval than did data on the compound’s efficacy and safety. Addyi was found to have an effectiveness rate of between 8 and 13 percent, and led to side effects ranging from dizziness to sudden drops in blood pressure that were exacerbated by alcohol or hormonal contraception. Flibanserin will carry a warning that it can not be taken with alcohol, despite the odd fact that the alcohol safety study submitted to the FDA by Sprout Pharmaceuticals listed 23 of the 25 participants as men. This has led to lingering questions about how women – the intended users of the drug – would react to a flibanserin alcohol interaction, particularly because, unlike Viagra, Addyi is taken daily rather than just before a sexual encounter. The mild effectiveness of flibanserin, combined with concerns about its safety, resulted in two prior rejections of FDA approval prior to the third successful attempt. Addyi comes to market in October 2015. (Cari Romm, The Atlantic; Editorial, Nature)

Health Policy

A universal flu vaccine may soon be a reality

Every fall, millions of people are vaccinated against the flu with vaccines that are developed by scientists who predict which influenza strains are most likely to be problematic that particular year. New shots are required every year because there are thousands of influenza strains that constantly mutate, and one shot cannot protect against them all. Sometimes, the predictions are correct; sometimes, like in 2014, they miss the mark and tens of thousands of US citizens die from influenza. Furthermore, because flu vaccines are currently based on portions of the influenza virus that evolve throughout the flu season, protection is not guaranteed. Two independent groups of scientists have recently reported considerable progress towards a universal flu vaccine by using a novel approach. Both labs targeted hemagglutin, a protein found on the surface of the H1N1 influenza virus, that is composed of a head region that mutates and varies between different flu strains, and a stem region that is constant between different strains and does not mutate.

The two reports – published in Nature Medicine and Science on Wednesday, August 26 – employed different molecular engineering techniques to stabilize the stem portion of hemagglutin when it is isolated from the head region, thus producing a stable structure for the vaccine that is common between different strains. This new method resulted in almost 100% immunity in mice against the lethal H5N1 flu strain, which is distantly related to the H1N1 strain, and partial protection in ferrets and non-human primates. More research is needed to determine whether immunity extends to other strains of the influenza virus, as well as to determine the degree of protection in humans using a vaccine derived with these new approaches. (Hanae Armitage, Science; The Economist)

Public Health and Drug Control Policy

Illicit Version of Painkiller Fentanyl Makes Heroin Deadlier

An extremely potent opioid analgesic called fentanyl, often administered prior to surgical procedures or prescribed for severe cancer-related pain, is increasingly being added to heroin and causing deadly consequences for heroin users across the United States and Canada. Fentanyl is 30-50 times more potent than heroin, and nearly 80-100 times more potent than morphine, and results in a drug combination that is much stronger than what heroin users expect to be administering. The flood of fentanyl-laced heroin is exacerbating the ongoing epidemic of heroin-related overdose deaths, as authorities in some states report a more than 600% increase in fentanyl-related deaths from 2013 to 2014, and see no sign of a slowdown. Drug dealers are increasingly adding fentanyl to heroin in order to restore the potency of the drug that’s been previously diluted by those higher in the distribution chain. Law enforcement officials and policymakers are scrambling to keep pace with the problem. In the past two years, Mexican drug cartels have increased production of a synthetic form of the compound, acetyl fentanyl, that is not yet included in many screens for toxic drugs in the US, and is currently classified as a banned substance in only a few states. Last year the US DEA added acetyl fentanyl to its list of federally banned substances, and in March 2015 the agency issued a warning that fentanyl poses a “threat to health and public safety.” Drug enforcement and public health authorities are attempting to boost public awareness of the dangers of fentanyl-laced heroin, and are alerting local communities when a compound drug batch is detected. (Fred Bever, NPR; Nadia Whitehead, NPR)

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August 28, 2015 at 9:00 am