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Sunday, October 7, 2018

Teen Youngest Person in History of IDWeek to Present

Thirteen-year old Devyn Stek, from Pottstown, Pennsylvania, made history here at IDWeek 2018 as the youngest person ever to present at the conference.

Her work, which was originally her seventh-grade science project at St. Teresa of Calcutta Education Center in Schwenksville, Pennsylvania, is anenvironmental surveillance study of Toxocara (roundworm) contamination levels in selected parks in Montgomery County. The abstract will be published in the Journal of Infectious Disease after the conference.

The idea for the project came when Stek and her family got their three cats — all strays — dewormed.

“The veterinarian first introduced me to roundworm and all types of parasitic worms. That’s when I got into microbiology,” she explained. “I did some research and found out that you can isolate Toxocara from soil. I just kept going further with it.”

“I didn’t know what to expect because the testing had never been done in Pennsylvania,” she added.

About 13.9% of people in the United States have antibodies to Toxocara. “This suggests that tens of millions of Americans may have been exposed to the Toxocara parasite,” according to the Centers for Disease Control and Prevention website. And often, people don’t know they have been exposed.

Stek collected six soil samples at each of six different parks in the county. She looked at the parasitic worms that grow in the intestines of dogs and cats and studied the prevalence of eggs under a microscope. Overall, 35 of the 36 samples tested positive for Toxocara eggs, although the level of contamination varied widely by park and sample. The most contaminated spots were around dog parks and places pets found food or relieved themselves.

Stek has shared her results with some of the parks and hopes that the findings will result in better messaging about the value of frequent hand-washing, she told Medscape Medical News.

She also hopes to educate people about toxocariasis — which can cause eye, lung, heart, and neurologic damage in humans — and the necessity of getting pets dewormed.

Science Teacher “Thrilled”

Devyn is the kind of student science teachers hope for every year, said Dave Dusza, Stek’s science teacher at St. Teresa.

“I am so very proud of what Devyn has done,” he told Medscape Medical News. “When Devyn came to me about her topic, I was thrilled that she was going to do something on this scale. After the school science fair, she advanced to the Montgomery County science fair. She won a prize there and was invited to speak at Merck on her findings.”

Stek’s father, Jon, is a medical writer for Merck vaccines, and his father was a Navy doctor who did work with parasitic worms. Both men have presented at IDWeek in the past, which is how Stek set her sights on this conference.

She found out she was accepted when her mom got the invitation confirmation in June and told her, “You’re going to San Francisco.” Much dancing and shouting followed, her dad said.

No other presenter at IDWeek has ever come close in age, said Andrew Pavia, MD, IDWeek cochair for the Infectious Diseases Society of America (IDSA).

Youngest by a Longshot

“We’ve definitely had projects submitted with college students on the author list, but I’m not sure we’ve had someone who hadn’t started medical school present here before,” Pavia told Medscape Medical News.

He said that when the work was first reviewed by experts looking at the public health abstracts, they were impressed because of the content and public interest and gave it a high score.

“Then they noticed there were no physicians on it,” he said. A member of the review group realized Stek’s grade level and called her home to verify that she was indeed the person who had done the research.

Her father confirmed that Devyn had conceived and designed the project, conducted the research, and did all the analysis working with a statistician. He said his contribution was providing the microscope at home so she didn’t have to do outside lab work and providing poster templates.

When the chairs of the meeting received the abstract, said Pavia, and “we were very excited by the newsworthiness of the subject and, more than anything, about how this brought a young person into the field, doing really good science.”

IDSA and the conference’s partner societies have actively been trying to reach out to younger researchers, he added.

“To be honest, we thought we just had to reach medical students and, perhaps, people finishing up their college careers. We’ve got to rethink that now,” he told Medscape Medical News. “This raises a whole new opportunity to reach even younger students and we need to figure out what to do with that.”

There is broad concern that some of the best young technical minds get swept into areas other than medicine, he explained.

“We’ve got to get people excited about the contributions they can make and the opportunities and the fun of the science, and change the perception that it’s all about starting a company and making your first billion,” he said.

Devyn Stek meets NASA astronaut Kate Rubins, PhD, shortly after Rubins delivered the keynote address at IDWeek 2018.

Stek is already excited about the future of her work and her future in general. She said she plans to keep testing the 36 samples to see if the prevalence of Toxocara is affected by the seasons. This will be next year’s science project and, she hopes, the subject of her presentation at IDWeek 2019. Stek plans to submit the completed paper on her current work to a journal for publication.

When she’s not studying, she splits her time between Girl Scouts, swimming, soccer, dance, church activities, and being president of her school’s student council.

As for career plans, “anything in science would be great,” said Stek. But first she has to choose her high school; she has four options.

She also said she knows what she won’t be doing: studying Guinea worm disease. In her sampling of IDWeek sessions, she saw just enough to know that not all work with worms suits her.

“They’re really gross,” she said.

Devyn and Jon Stek, Dusza, and Pavia have disclosed no relevant financial relationships.

IDWeek 2018: Poster 438. Presented October 4, 2018.

https://www.medscape.com/viewarticle/903078#vp_1

Drug trade war: Chinese fentanyl is fueling the US opioid crisis

The U.S.-China trade relationship has dominated headlines in recent months, but it’s the illicit drug trade that some lawmakers and experts believe should be moved to the top of the list of issues between the two countries.

Last year, more than 49,000 Americans died from opioid-related overdoses. The majority of those deaths, 60 percent, involved fentanyl, a synthetic opioid up to 50 times more potent than heroin. Between 2014 and 2016, the number of fentanyl-related deaths skyrocketed by almost 600 percent.

China leads the world in the production and supply of fentanyl and the chemical precursors used to manufacture the drug. Roughly 68 percent of all global fentanyl movements originate in China, according to the Defense Intelligence Agency.

Data provided by the Defense Intelligence Agency. Chart produced by Sen. Dianne Feinstein’s office for a Senate Drug Caucus hearing, Tues. Oct. 2, 2018.

The United States is currently pouring billions of dollars into combating the opioid epidemic. Law enforcement agents have been given emergency authorities to try to control the flow of fentanyl and target opioid “kingpins.” Border and customs officials have seized record amounts of fentanyl and other synthetic opioids this year but the drug crisis continues to get worse.

At a Tuesday hearing of the Senate Caucus on International Narcotics Control, lawmakers, drug control officials and experts argued for addressing the source of the problem, namely, China’s outsized role in fueling the U.S. opioid epidemic.

TRACKING THE FLOW OF FENTANYL

“China is the number one problem when it comes to illegal fentanyl coming into the United States,” said Sen. Chuck Grassley, R-Iowa.

China is one of the world’s largest producers of the precursor chemicals used to manufacture fentanyl and other synthetic opioids. It also leads the world in making the chemicals used to process heroin, cocaine and methamphetamines. That production is largely unregulated by Chinese authorities, making it easy for local actors to divert supplies to overseas buyers.

U.S.-based traffickers and consumers can go online and easily order fentanyl off the dark web at up to 90 percent purity and have it shipped overseas through the U.S. Postal Service or other international mail carriers. Congress passed legislation last month — the Synthetics Trafficking and Overdose Prevention Act, or STOP Act — to prevent the exploiting of the Postal Service by drug traffickers. That included provisions for sophisticated equipment to detect opioids.

“A single individual with a computer, P.O. Box, and a pill press can order fentanyl directly from China to his or her home,” Grassley said, describing the rise of a mom and pop shop model of drug trafficking. “For the cost of a few thousand dollars of raw product, the sole proprietor can turn that raw fentanyl into thousands of pills worth literally millions of dollars on the street.”

The Drug Enforcement Agency has estimated a kilogram of fentanyl priced at roughly $5,000 can yield $1.5 million in profits when pressed into counterfeit pills, cut into heroin or sold on the street.

The profit potential has attracted Mexican drug cartels who represent “the most significant criminal threat to the United States,” according to DEA Global Enforcement deputy chief of operations, Paul Knierim.

“Now Chinese and Mexican nationals are increasingly operating in concert, resulting in an alignment responsible for the proliferation of heroin, fentanyl and related synthetics coming across the South West border,” Knierim asserted.

IMPROVING INTERNATIONAL COOPERATION

In recent years, the United States has reached out to China, Mexico and other partners to improve cooperation and stem the flow of drugs into the United States. At the United Nations last week, President Donald Trump acknowledged the international dimensions of the U.S. opioid epidemic and secured a commitment by 130 nations to crack down on the global drug trade.

In 2017, as part of President Trump’s first high-level meeting with Chinese President Xi Jinping, the two agreed to a framework to improve bilateral counternarcotics cooperation. Since that time, U.S. and Chinese law enforcement have worked together to take down chemical laboratories and criminal networks. In 2019, the DEA plans to open a new office in Guangzhou, China the origin of a substantial amount of fentanyl consumed in the United States.

The United States has also worked with China to help issue and implement regulatory controls on 175 psychoactive substances, including 25 fentanyl analogs and two fentanyl chemical precursors.

The State and Justice Departments are now pushing their Chinese counterparts to schedule fentanyl and fentanyl analogs as an entire class of drug with no accepted medical use. Scheduling fentanyl as a class would help law enforcement in both countries target illicit laboratories and drug trafficking networks involved in the trade.

“Identifying a technical path forward on China class scheduling is our number one drug control ask of the Chinese government,” said Kirsten Madison, Assistant Secretary of State for International Narcotics and Law Enforcement Affairs.

The United States took a similar step in February when the Justice Department invoked emergency authorities and scheduled the entire category of fentanyl and fentanyl-related drugs, making it illegal to manufacture, sell or possess. That order was unprecedented and will remain in effect until 2020.

PRESSURING CHINA TO CUT SUPPLY

Despite progress in some areas, the continued rise in overdose deaths and the steady flow of illicit drugs into the United States is proof that the current efforts are not working, said Sen. Dianne Feinstein, D-Calif. “I really believe we need to hold China responsible for some of this. I really believe that we need to see that China change course,” she argued.

Citing DIA figures, she noted that between 2016 and 2015, there were 1,072 shipments of fentanyl seized by U.S. officials before they entered the country. In China, there were only four occasions where fentanyl was interdicted before it was shipped out.

China has publicly downplayed its role in the deadly opioid epidemic, instead blaming American demand. In August, the Chinese government responded to a tweet from President Trump who said, “It is outrageous that Poisonous Synthetic Heroin Fentanyl comes pouring into the U.S. Postal System from China.”

Yu Haibin of the Chinese Ministry of Public Security’s Narcotics Control Bureau said Trump’s comments were “completely unacceptable.” He argued, “The United States has no proof that most fentanyl in the country comes from China,” he said. “It’s highly irresponsible to draw such a conclusion based on some individual cases.”

In a recent interview with ABC’s “20/20,” Yu Haibin again denied China’s role as chief exporter. “I want to stress that as far as I know, it’s impossible for fentanyl that’s produced legally here to be exported to the U.S.,” he claimed. “So it must be done by criminals using underground processing plants and the internet.”

On Tuesday, Derek Scissors, an expert in U.S.-China relations at the American Enterprise Institute, emphasized that the Chinese government “undoubtedly has the capacity to curb large-scale export of illegal drugs to the U.S. It has intentionally or unintentionally decided these actions are not worth their costs.”

He told the committee there is “no reason for the Chinese not to be good cooperative partners” in stopping the export of fentanyl. “But they will only make these changes if it’s a priority for them.”

Improving China’s level of cooperation will be another test of America’s willingness to pressure President Xi Jinping, Scissors continued. “For us to get them to take action that they have not been willing to take for the safety of their own citizens, it is going to require a coercive threat,” he said.

Specifically, the Trump administration could sanction specific Chinese provinces or cities that are not cooperating with international drug control efforts. The State Department could decertify those regions, cutting off foreign aid and assistance.

Scissors also suggested charging Chinese government officials for criminal negligence. The government would not extradite Chinese nationals to face the U.S. justice system, but it would send a message, he noted.

It is not clear how much pressure the United States will be willing to put on China to address the drug issue. Between the trade war and President Trump’s latest threat to impose tariffs on essentially all Chinese imports, the bilateral relationship is fraught.

“I think we have the capacity to affect that judgment on the part of the Chinese,” Sen. Sheldon Whitehouse, D-R.I., said, noting it will be a challenge. “I think obviously in the trade war we’re at right now, it’s a little hard to highlight that because there are so many points of conflict that have emerged.”

Lawmakers are currently pushing forward a number of additional drug control bills including the bipartisan Stop the Importation and Trafficking of Synthetic Analogues Act, or SITSA.

The Justice Department and Department of Homeland Security support the legislation which would give law enforcement agencies the authority to quickly classify and schedule dangerous opioids and fentanyl analogs. Critics of the bill have argued it will expand the war on drugs by criminalizing a new class of drugs, synthetic opioids. Sponsors of the bill have noted the legislation only targets the manufacturers and traffickers of illicit opioids. Those in simple possession of the drugs are exempted.

https://kutv.com/news/nation-world/chinese-fentanyl-is-fueling-the-us-opioid-crisis-drug-trade-tensions-escalate

Major Advance Reported in Understanding Antibiotic Resistance

Scientists at the University of Montana (UM) and the University of Washington have discovered how pathogenic bacteria resist antibiotic treatment and recently published their findings in an article titled (“Entropically-driven aggregation of bacteria by host polymers promotes antibiotic tolerance in Pseudomonas aeruginosa”) in the Proceedings of the Natural Academy of Sciences.

“Bacteria causing chronic infections are generally observed living in cell aggregates suspended in polymer-rich host secretions, and bacterial phenotypes induced by aggregated growth may be key factors in chronic infection pathogenesis. Bacterial aggregation is commonly thought of as a consequence of biofilm formation; however, the mechanisms producing aggregation in vivo remain unclear. Here we show that polymers that are abundant at chronic infection sites cause bacteria to aggregate by the depletion aggregation mechanism, which does not require biofilm formation functions. Depletion aggregation is mediated by entropic forces between uncharged or like-charged polymers and particles (e.g., bacteria),” write the investigators.

“Our experiments also indicate that depletion aggregation of bacteria induces marked antibiotic tolerance that was dependent on the SOS response, a stress response activated by genotoxic stress. These findings raise the possibility that targeting conditions that promote depletion aggregation or mechanisms of depletion-mediated tolerance could lead to new therapeutic approaches to combat chronic bacterial infections.”

“Antibiotic resistance is a major problem,” says Patrick Secor, Ph.D., assistant professor in UM’s division of biological sciences and lead researcher on the paper. “However, it is often the case that if you take bacteria that survive antibiotic treatment from someone’s infected lungs and treat those same bacteria with antibiotics in the lab, the bacteria die. We wanted to understand why.”

We found that bacteria living in high concentrations of polymers get a little stressed out,” adds Lia Michaels, a researcher at UM and co-author of the paper. “Basically, the polymer-rich environment activates stress responses in the bacteria, causing them to tolerate higher levels of antibiotics.”

“I like to compare it to the stress our bodies undergo when we exercise,” notes Dr. Secor. “Exercising today allows you to run a little further or lift a little more weight later on. This is analogous to the stress responses turned on in bacteria living in airway mucus—exposure to stress today allows the bacteria to survive the stress of antibiotic exposure later on.”

The researchers found that stress responses induced by mucus polymers pressing on the bacteria were a result of mild DNA damage in the bacterial cells.

“One thing that this DNA damage did was slow bacterial growth,” says Laura Jennings, Ph.D., UM research assistant professor and co-author of the paper. “Because most antibiotics work best on rapidly dividing cells, these slow-growing bacteria were more difficult to kill with antibiotics.”

The researchers speculate that the mechanisms by which polymers turn on bacterial stress responses could be targeted therapeutically to treat long-term bacterial infections.

“Our hope is that we could come up with new ways to treat bacterial infections or increase the efficacy of antibiotic treatment,” according to Dr. Secor.

https://www.genengnews.com/gen-news-highlights/major-advance-reported-in-understanding-antibiotic-resistance/81256292

FDA approves HPV vaccine for people up to 45

The Food and Drug Administration expanded its approval of the HPV vaccine to include men and women between 27 and 45, an effort to protect more people from several types of cancer caused by the human papillomavirus.

The vaccine, called Gardasil 9, previously was approved for people ages 9 through 26. The vaccine is typically given in two doses several months apart for those who are 9 through 14, and in three doses for individuals 15 through 26. For those older than 26, the recommended regimen will be three doses.

Most sexually active individuals in the United States will become infected with HPV in their lifetimes. In most cases, the virus is cleared by the body’s immune system, but when that doesn’t occur, HPV infections can lead to cervical, anal, vaginal, penile and throat cancers.

The approval “represents an important opportunity to help prevent HPV-related diseases and cancers in a broader age range,” said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research.

Experts say the vaccine, which protects against nine HPV strains, is most effective when administered before the initiation of sexual activity. But data also indicate that the vaccine can benefit the older group. That’s because even though many adults have been exposed to some types of HPV, most have not been exposed to all nine types covered by the vaccine.

Merck, which manufacturers the vaccine, requested the expanded age range this year. In June, the FDA granted the application priority review.

The original version of the vaccine, called simply Gardasil, was approved by the FDA in 2006 and covered four strains of HPV; it is no longer available in the United States. Gardasil 9 was approved in 2014. The two versions are manufactured similarly and cover four of the same HPV types.

The agency said it based its expanded age approval on data on the original Gardasil vaccine involving 3,200 women ages 27 through 45. The data, and long-term follow-up, showed that Gardasil was effective in preventing persistent infection, genital warts, various precancerous lesions and cancers related to HPV types covered by the vaccine. The FDA said the effectiveness for men was inferred based on the data for women, a small trial for men ages 27 through 45 and the experience of younger males.

The Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices, which is made up of medical and public health experts who make recommendations on the use of vaccines, is expected to review the expanded age range at its meeting later this month and to vote on it next year. If the CDC committee recommends that the older group receive the vaccine, insurance companies are much more likely to cover the cost.

In August, CDC data for 2017 showed that HPV vaccination rates are rising, although not as fast as medical experts would like. Nearly half of adolescents ages 13 to 17 had received all the recommended doses for HPV vaccination, while two-thirds had received the first dose. For both groups, that was a five-percentage-point increase from the previous year.

But HPV-related cancers also are increasing. More than 43,000 people developed HPV-associated cancer in 2015, compared with about 30,000 in 1999, the CDC said.

https://www.washingtonpost.com/health/2018/10/05/fda-approves-hpv-vaccine-people-up/?utm_term=.5a1975642864

CVS Offering Point-Of-Sale Rebates In Part D Next Year With SilverScript Plan

Premiums will average about $80 for enhanced plan, well above average $32.50 premium for basic stand-alone Medicare Part D coverage in 2019.

https://pink.pharmaintelligence.informa.com/PS124005/CVS-Offering-PointOfSale-Rebates-In-Part-D-Next-Year-With-SilverScript-Plan

Promising rare conditions treatments could upend the market hierarchy

With less than 14% of Phase I to III drug candidates winning FDA approval, unforeseen roadblocks can derail even the most promising clinical research programs. But with a steadfast attitude and transparent messaging, biopharma companies can recover from unexpected obstacles — and set themselves up to thrive.

That’s a feat demonstrated by three mid- to late-stage candidates currently working their way through clinical trials. The first comes from Solid Biosciences, a company with a single-minded focus on Duchenne muscular dystrophy. Solid’s gene therapy candidate, SGT-001, hit a rough patch earlier this year after a serious adverse event prompted the FDA to place a clinical hold on the trial. But the company developed a contingency plan to ensure patient safety, a move that paid off when the FDA lifted the clinical hold only three months later.

Meanwhile, Celgene’s approval application for its multiple sclerosis candidate, ozanimod, was met with a surprise refuse-to-file letter in February, prompting the company to reassess its application materials.

Finally, Alexion stumbled into a Wall Street squabble over the efficacy of its paroxysmal nocturnal hemoglobinuria drug ravulizumab, with some investors questioning whether the drug marks an improvement over the company’s existing blockbuster, Soliris. Alexion has remained steadfast in its assertion ravulizumab will benefit patients because of its superior dosing schedule — a message that’s resonating with physicians and patients.

SGT-001
Solid Biosciences, Phase I/II
Indication: Duchenne muscular dystrophy

Catalyst: SGT-001 is an investigational gene therapy for Duchenne muscular dystrophy (DMD). But the FDA placed a hold on the IGNITE DMD Phase I/II trial after one patient experienced a serious adverse event in the form of reduced platelet count. Solid promptly promised to monitor patients and treat them for low platelet count should the same problem crop up again.

Those steps were enough for the FDA, which lifted IGNITE DMD’s clinical hold after just three months. Despite the adverse event, enthusiasm remains high for this candidate, notes inThought Research medical adviser Dr. Leon Henderson-MacLennan.

Solid’s chief scientific officer Dr. Carl Morris says the company hopes to have interim data available in the second half of 2019. “Right now, there is no good treatment for the majority of patients with Duchenne muscular dystrophy. It is 100% fatal,” he notes.

Competitive landscape: Solid isn’t the only company advancing a gene therapy for DMD. Both Sarepta Therapeutics and Pfizer are moving ahead with their own candidates, which are similar to SGT-001. Despite the competition, Morris welcomes the arrival of other new treatment options for this incurable disease. “We are excited by the progress,” he says.

Henderson-MacLennan believes it’s too soon to predict which candidate will make it to market first. In terms of the efficacy and safety profiles, he notes, “The devil is going to be in the details of the clinical trials that are ongoing.”

Messaging strategy: Because SGT-001 is a gene therapy, it could be argued that patients who are treated successfully will be “cured.” However, Morris doesn’t necessarily see it that way. “It’s a treatment,” he explains. “We hope it will slow or stop the continued progression of the disease. But ‘cure’ means you come back to a normal phenotype, and we don’t think that’s possible.”

According to Henderson-MacLennan, it’s smart for Solid to take a conservative approach — which is rare in the splashy world of gene therapies.

That isn’t to say Solid won’t eventually earn those bragging rights.

“Current non-steroidal therapy for DMD only addresses a small subset of patients with a certain mutation. SGT-001 will be the first therapy in the space that will work regardless of mutation status,” says Heartbeat SVP, medical director Madhuri Fletcher. “Ideal messaging will focus on treating all, regardless of mutation status or disease stage.”

Marketing strategy: Although it’s early days for SGT-001, Solid and its competitors are likely to follow a relatively straightforward marketing plan for rare diseases with high unmet need. “It will be important to identify families early. Solid will need to partner with the strong support communities in the DMD space and integrate with the multidisciplinary approach to DMD management,” explains Fletcher. “There will also be an opportunity to demonstrate commitment to the DMD community by coming in at a more reasonable price than the controversially expensive in-market competitor.”

Advocacy groups want transparency, Morris adds. “They want to help educate patients and the community about what gene therapy can provide and what it can’t.”

Ozanimod
Celgene, Phase III
Indication: Multiple sclerosis

Catalyst: Ozanimod, which has been tested in two Phase III trials for relapsing multiple sclerosis, was assumed to be a sure-fire blockbuster — that is, until the FDA issued a surprise refuse-to-file letter in February, sending the company’s shares tumbling 9%.

But the regulatory delay won’t crush ozanimod’s blockbuster potential, according to inThought Research president Ben Weintraub. “It was a business setback, but I didn’t see anything concerning about the way the drug works,” he explains.

To that end, Celgene clarified it won’t have to conduct any additional in-human studies and plans to refile early next year. This puts ozanimod back on the map for a tentative launch in 2020.

Competitive landscape: Celgene is seeking approval for relapsing MS based on Phase III data that demonstrated ozanimod’s superiority to Biogen’s Avonex (interferon beta-1a). Although Celgene is back on track to win approval, there’s already lots of competition in the MS market, including Avonex and Roche’s Ocrevus (ocrelizumab).

Celgene is “on the clock to get it out there before Gilenya [fingolimod] goes generic,” Weintraub says.

Considering the market competition, ozanimod’s real potential may lie in other indications. Weintraub notes the drug is being tested in ulcerative colitis and Crohn’s disease, where few oral treatment options exist.

Messaging strategy: Celgene shouldn’t worry about the FDA’s refuse-to-file when it comes to messaging the drug, according to Strategic Marketing Solutions principal Richard Meyer. Instead, patients and physicians will be focused on the drug’s side-effect profile. MS patients are extremely active on social media, he notes, so Celgene will have to produce data that convinces them and physicians ozanimod dramatically improves quality of life.

“That could be difficult,” he adds. “It’s kind of hard to be the new kid on the block.”

Marketing strategy: Meyer believes to gain an edge, Celgene should price its drug at or below the wholesale cost of its competitors. It also makes sense for the brand team to “plunge into social media.”

But TV is still crucial, even in today’s digital world. “The number one way people find out about new products is still a TV ad,” Meyer continues. “I would go on TV as heavy as I could, then measure the awareness among MS patients. Once you get awareness to a certain level, you start talking about advantages over the competition.”

Ravulizumab
Alexion, Phase III
Indication: Paroxysmal nocturnal hemoglobinuria

Catalyst: Ravulizumab, also called ALXN1210, is Alexion’s next-gen treatment for paroxysmal nocturnal hemoglobinuria (PNH). The drug is slated to replace Alexion’s existing blockbuster, Soliris. In two Phase III studies, treatment with weight-based ravulizumab proved non-inferior to Soliris across 11 primary and secondary endpoints. But the drug’s key selling point is its dosing schedule, with Soliris requiring dosing every two weeks and ravulizumab every two months. Normally the FDA takes an entire year to approve applications, but Alexion used one of its priority review vouchers, which is likely to speed up the review process by at least four months.

Competitive landscape: Ravulizumab isn’t facing any steep competition, Henderson-MacLennan notes. Heartbeat VP, strategy Simon Beins adds, “Ravulizumab, like Alexion’s 11-year-old Soliris, have a lot in common: They share a mechanism of action; their efficacy results, so far, look similar; and they have no competition from any other drugmaker.”

Messaging strategy: According to Beins, ravulizumab’s messaging priority will be clear: convenience. “PNH usually strikes younger adults at prime working and child-raising ages, so a less disruptive administration schedule will be compelling,” he explains. Because Soliris has a black box warning, ravulizumab is likely to have one, too. “The chance of meningococcal infections is high. So, Alexion will have to demonstrate there are no surprises hidden in its safety profile,” Beins continues.

Marketing strategy: Considering the high price tag for Soliris — around half a million dollars per year — Alexion’s marketing strategy for ravulizumab could hinge on the price execs decide to set. “Given ravulizumab will likely cannibalize much of Soliris’ business, we’ll see exactly how Alexion balances its reputation [it has faced criticism and legal action over Soliris’ price tag] with its bottom line,” Beins says.

In the wake of Soliris’ patient support program, the team marketing ravulizumab will benefit from the learnings and infrastructure Alexion has built interacting with PNH patients. “This high-touch approach also extends to HCPs,” Beins adds, noting PNH is so rare, many have never seen a case of it.

https://www.mmm-online.com/home/channel/pipeline/promising-rare-conditions-treatments-could-upend-the-market-hierarchy/

Fitbit rolling out Charge 3 with focus on sleep tracking

Fitbit on Thursday said it will begin rolling out its latest wearable fitness tracking device, the Charge 3, on Sunday and will go live with a beta version of its Fitbit Labs Sleep Score program in November.
Participants accepted to the program will be able to track breathing disruptions that could signal health problems. Fitbit said that in addition to the sleep score program, it is conducting clinical research aimed at validating its software for use in detecting health conditions such as sleep apnea and atrial fibrillation.
The San Francisco-based company said pre-orders for the Charge 3, which will cost $149.95, are exceeding those for its earlier-generation Versa device in the first six weeks.

Data from the Fitbit devices with a relative SpO2 sensor will inform its Fitbit Labs Sleep Score beta program, which will be available for a limited time to qualifying Fitbit users in the United States, Australia and United Kingdom.

“Data from this sensor will feed into the upcoming launch of our Fitbit Labs Sleep Score beta, which will allow users to better understand metrics like their sleep quality and breathing disturbances. In the future, with FDA approval, this data could be used to screen and detect more serious health conditions, such as sleep apnea,” Fitbit CEO James Park said in a statement.

With digital health applications from such names as Apple, Fitbit and Johnson & Johnson making inroads into the medical device space, FDA is moving quickly to develop a software precertification program to address products at the intersection of both worlds.

Fitbit is one of nine companies in FDA’s digital health software precertification pilot program for Software as a Medical Device.

The program could replace the need for a premarket submission for certain medical software products and allow for a streamlined review of marketing submissions for others. Manufacturers eligible for the new regulatory framework have shown “a robust culture of quality and organizational excellence,” according to FDA.

“Because of software’s faster iterative design, development, and validation, traditional implementation of the premarket requirements may impede or delay patient access to advances in software technology that would improve public health,” FDA has said.

The new Charge 3 device features updated health and fitness sensors and algorithms, a large touchscreen display and up to seven days of battery life. It is also waterproof.

Fitbit said 54% of pre-orders on Fitbit.com have come from customers who previously owned two or more of its devices before ordering Charge 3.

https://www.healthcaredive.com/news/fitbit-rolling-out-charge-3-with-focus-on-sleep-tracking/539001/

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Sunday, October 7, 2018