The following slide deck was published by Bristol-Myers Squibb Company in conjunction with this event.
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Monday, September 30, 2019
Dementia Outcomes Improved With Supportive Care
Dementia patients’ quality of life improved over 12 months with
team-based telephone and Internet support, a survey of dementia
caregivers in the Care Ecosystem randomized trial showed.
Care delivered by a trained, unlicensed care team navigator and managed by an advanced-practice nurse, a social worker, and a pharmacist improved multiple facets of care, reported Katherine Possin, PhD, of the University of California San Francisco, and coauthors in JAMA Internal Medicine.
These included measures of patient quality of life, emergency department visits, caregiver depression, and caregiver burden.
“This study demonstrates that effective phone-based dementia care that addresses the needs of the person with dementia and the caregiver can be provided from a hub across large geographic areas, irrespective of the patients’ healthcare system affiliations,” Possin told MedPage Today.
“The Care Ecosystem addresses gaps in our healthcare system around dementia,” she continued. “While the Care Ecosystem won’t cure dementia, it changes the experience of dementia for both the patients and the caregivers, so that they may live as well as possible.”
A model like the Care Ecosystem can expand the reach of the dementia specialist workforce, Possin added. “We work at an academic medical center, where we have a lot of dementia specialists, and we are able to provide high-quality dementia care,” she noted. “But many people living with dementia in this country have limited or no access to dementia specialists because of where they live or because it is hard for them to travel to appointments.”
Earlier data from the Care Ecosystem research group showed that caregiver and dementia health were intertwined: dementia patients were nearly twice as likely to use an emergency department if their caregiver had depression, for example.
“Dementia is a public health challenge, a family disease, and a major strain on our healthcare system,” observed Jennifer Tjia, MD, MSCE, of the University of Massachusetts in Worcester, in an accompanying editorial. “Possin and colleagues have provided evidence that achieving the goals of the triple aim of improving care, health outcomes, and reducing cost can be met by providing dementia training to clinically supervised, unlicensed care team navigators in a scalable way.”
The single-blind, pragmatic Care Ecosystem trial was conducted in California, Nebraska, and Iowa in urban (San Francisco and Omaha) and rural settings. It randomized 780 dyads of dementia patients and caregivers — 512 to the Care Ecosystem intervention and 268 to usual care — from March 2015 to February 2017, tracking each dyad for 1 year.
All eligible patients had a dementia diagnosis from a treating
provider and were enrolled in or eligible for Medicare or Medicaid.
About half (49.8%) of patients had mild dementia; 28.7% had dementia,
and 21.5% had severe dementia. Participants were informed of their
randomization result and continued to receive services from other
healthcare professionals throughout the study.
In the Care Ecosystem group, patients and caregivers received telephone-based, collaborative care from a team of dementia specialists. The team navigator was an unlicensed, trained guide who served as the primary point of contact for patients and caregivers under nurse supervision. Navigators called dyads approximately monthly; the number of telephone calls averaged 15.3 per dyad over 12 months. The nurse, social worker, and pharmacist were available for situations beyond the navigator’s scope, including safety concerns, behavioral symptoms, or complex legal and financial circumstances.
In the usual care group, participants were offered contact information for the Family Caregiver Alliance, the Alzheimer’s Association, and Area Agencies on Aging. They also were sent quarterly newsletters with general dementia-related articles and word games.
All outcomes were based on caregivers’ answers to telephone survey questions. The primary outcome measure was the Quality of Life in Alzheimer’s Disease (QOL-AD) score, which rated 13 aspects of patient quality of life — such as physical health, energy level, mood, memory, ability to do things for fun, and family relationships — on a four-point scale of poor, fair, good, or excellent.
For the primary outcome, caregivers reported that the Care Ecosystem improved patient quality of life (β 0.53, 95% CI 0.25-1.30; P=0.04). In secondary outcomes, the Care Ecosystem program was shown to do the following:
The study had several limitations, the researchers noted. Dyads were
aware of their randomization, and the sample selected likely had fewer
unmet needs than patients and caregivers in other populations. Economic
outcomes were reported only for 12 months and were based on survey
results.
“For many families, supportive dementia care such as the Care Ecosystem can change the experience of living with dementia,” Possin said. Supportive care can reduce stress and burdens on caregivers, she noted: “They are not alone, and can actually plan ahead for challenges that are coming, rather than feeling they are in a constant state of crisis.”
Care delivered by a trained, unlicensed care team navigator and managed by an advanced-practice nurse, a social worker, and a pharmacist improved multiple facets of care, reported Katherine Possin, PhD, of the University of California San Francisco, and coauthors in JAMA Internal Medicine.
These included measures of patient quality of life, emergency department visits, caregiver depression, and caregiver burden.
“This study demonstrates that effective phone-based dementia care that addresses the needs of the person with dementia and the caregiver can be provided from a hub across large geographic areas, irrespective of the patients’ healthcare system affiliations,” Possin told MedPage Today.
“The Care Ecosystem addresses gaps in our healthcare system around dementia,” she continued. “While the Care Ecosystem won’t cure dementia, it changes the experience of dementia for both the patients and the caregivers, so that they may live as well as possible.”
A model like the Care Ecosystem can expand the reach of the dementia specialist workforce, Possin added. “We work at an academic medical center, where we have a lot of dementia specialists, and we are able to provide high-quality dementia care,” she noted. “But many people living with dementia in this country have limited or no access to dementia specialists because of where they live or because it is hard for them to travel to appointments.”
Earlier data from the Care Ecosystem research group showed that caregiver and dementia health were intertwined: dementia patients were nearly twice as likely to use an emergency department if their caregiver had depression, for example.
“Dementia is a public health challenge, a family disease, and a major strain on our healthcare system,” observed Jennifer Tjia, MD, MSCE, of the University of Massachusetts in Worcester, in an accompanying editorial. “Possin and colleagues have provided evidence that achieving the goals of the triple aim of improving care, health outcomes, and reducing cost can be met by providing dementia training to clinically supervised, unlicensed care team navigators in a scalable way.”
The single-blind, pragmatic Care Ecosystem trial was conducted in California, Nebraska, and Iowa in urban (San Francisco and Omaha) and rural settings. It randomized 780 dyads of dementia patients and caregivers — 512 to the Care Ecosystem intervention and 268 to usual care — from March 2015 to February 2017, tracking each dyad for 1 year.
In the Care Ecosystem group, patients and caregivers received telephone-based, collaborative care from a team of dementia specialists. The team navigator was an unlicensed, trained guide who served as the primary point of contact for patients and caregivers under nurse supervision. Navigators called dyads approximately monthly; the number of telephone calls averaged 15.3 per dyad over 12 months. The nurse, social worker, and pharmacist were available for situations beyond the navigator’s scope, including safety concerns, behavioral symptoms, or complex legal and financial circumstances.
In the usual care group, participants were offered contact information for the Family Caregiver Alliance, the Alzheimer’s Association, and Area Agencies on Aging. They also were sent quarterly newsletters with general dementia-related articles and word games.
All outcomes were based on caregivers’ answers to telephone survey questions. The primary outcome measure was the Quality of Life in Alzheimer’s Disease (QOL-AD) score, which rated 13 aspects of patient quality of life — such as physical health, energy level, mood, memory, ability to do things for fun, and family relationships — on a four-point scale of poor, fair, good, or excellent.
For the primary outcome, caregivers reported that the Care Ecosystem improved patient quality of life (β 0.53, 95% CI 0.25-1.30; P=0.04). In secondary outcomes, the Care Ecosystem program was shown to do the following:
- Reduce emergency department visits (β −0.14, 95% CI −0.29 to −0.01; P=0.04)
- Decrease caregiver depression (β −1.14, 95% CI −2.15 to −0.13; P=0.03)
- Lessen caregiver burden (β −1.90, 95% CI −3.89 to −0.08; P=0.046)
“For many families, supportive dementia care such as the Care Ecosystem can change the experience of living with dementia,” Possin said. Supportive care can reduce stress and burdens on caregivers, she noted: “They are not alone, and can actually plan ahead for challenges that are coming, rather than feeling they are in a constant state of crisis.”
Last Updated September 30, 2019
This project was funded by the Department of Health and Human
Services, Centers for Medicare & Medicaid Services, Global Brain
Health Institute, National Institute on Aging, and National Institute on
Neurological Disorders and Stroke.
The researchers reported relationships with the Centers for Medicare & Medicaid Services, National Institute on Aging, National Institute of Neurological Disorders and Stroke, Global Brain Health Institute, National Center for Advancing Translational Sciences, National Palliative Care Research Center, the John A. Hartford Foundation, the West Foundation, the Patient Centered Outcomes Research Institute, the American Academy of Hospice and Palliative Medicine, and Cornell University, as well as receiving UCSF grants.
The editorialist reported grants from the National Institutes of Health and the Cambia Health Foundation, personal fees from the Donaghue Foundation, and personal fees and nonfinancial support from CVS Health outside the study.
The researchers reported relationships with the Centers for Medicare & Medicaid Services, National Institute on Aging, National Institute of Neurological Disorders and Stroke, Global Brain Health Institute, National Center for Advancing Translational Sciences, National Palliative Care Research Center, the John A. Hartford Foundation, the West Foundation, the Patient Centered Outcomes Research Institute, the American Academy of Hospice and Palliative Medicine, and Cornell University, as well as receiving UCSF grants.
The editorialist reported grants from the National Institutes of Health and the Cambia Health Foundation, personal fees from the Donaghue Foundation, and personal fees and nonfinancial support from CVS Health outside the study.
Primary Source
JAMA Internal Medicine
Secondary Source
JAMA Internal Medicine
Source Reference: Tjia
J “A Telephone-Based Dementia Care Management Intervention — Finding
the Time to Listen” JAMA Intern Med 2019;
DOI:10.1001/jamainternmed.2019.4111.
https://www.medpagetoday.com/neurology/dementia/82480Cancer immunotherapy boom showing no sign of slowdown
- Nearly twice as many cancer immunotherapies are now in development as were in testing two years ago, according to a new report from the Cancer Research Institute that highlights how the field has rapidly become a top target of drugmaker investment.
- Just under 3,900 active drugs are now under preclinical or clinical study, up 91% from 2017, researchers from CRI found. A large portion of that growth came in cell therapy, with 797 new agents added to the industry’s pipeline over the past two years.
- While roughly two-thirds of the drug candidates tracked by the institute are in preclinical stages, the number of immunotherapies in clinical testing grew significantly, too.
The industry’s pipeline continues to expand rapidly, with more than 5,000 active immuno-oncology studies listed in the federal database clinicaltrials.gov.
Researchers at CRI counted 60% more organizations, including academic and research groups, were actively conducting testing of cancer immunotherapies in 2019 than in 2017, according to data published Friday in Nature Drug Discovery.
“This tremendous investment and commitment from different sectors have laid the foundation for 31 approvals by the FDA for IO drugs in the past 2 years,” the report’s authors wrote.
For companies like Merck & Co., Bristol-Myers Squibb and Roche, cancer immunotherapies are now a crucial part of revenue growth. But others, like Novartis, Celgene, Amgen and Eli Lilly, have expanded their pipelines in hopes of gaining ground on the field’s initial leaders.
Much of that research, however, focuses on similar biological pathways. There are more than 190 CD19-targeted immunotherapies in some form of testing, for example, as well as roughly 200 PD-1 and PD-L1 inhibitors.
Such overlap has raised concerns about duplicative efforts, as companies chase already proven drug classes.
Encouragingly, however, CRI found that 205 more targets were being studied in 2019 than in 2017, bringing the total ot 468 active targets. Some of the more common targets without an approved treatment include NY-ESO-1 and STAT3.
CRI’s numbers for clinical immunotherapy candidates are higher than the nearly 450 counted this year by IQVIA, a research group, although differences in definitions could explain the gap.
In its May 2019 report, IQVIA noted growth in the overall number of cancer drugs in development and found more than 212,000 Americans were treated with PD-1 or PD-L1 drugs last year, up nearly 100-fold from 2014.
https://www.biopharmadive.com/news/cancer-immunotherapy-research-growth-drug-study-oncology/563881/
What’s in your CBD? Buyer beware
CBD (or cannabidiol) products, containing a compound found in marijuana, have skyrocketed in popularity and seem to be for sale everywhere, from grocery stores, to coffee shops and gas stations.
CBD itself does not get you high, but advocates say it can help with anything from muscle aches to anxiety. It’s forecast to become a $22 billion industry by 2024.
But without wide federal oversight, there is no way of really knowing what’s inside CBD.
So, we decided to find out.
CBS News partnered with Mile High Labs, in Colorado, to test nine CBD oil samples purchased from around the country.
It took two days for senior lab associate Joshua Cogell to test our nine samples, checking for CBD and for THC (the ingredient in marijuana that gives a high), and also for dangerous impurities, like pesticides and heavy metals.
“We test for four different heavy metals: mercury, arsenic, cadmium and lead,” said Cogell.
“They all sound bad,” said correspondent Barry Petersen.
“They are very, yes, things you do not want in your body.”
The CBD craze took off when President Trump signed the Farm Bill last December. The bill allows farmers to legally grow hemp, the source of CBD. It is a member of the marijuana plant family, but it produces only trace amounts of THC. Under the bill, the government allows less than 0.3% THC in CBD products.
Here’s what Mile High found in our samples:
“That’s really concerning to me,” said Steve Mueller, who founded Mile High Labs in Loveland, Colorado, and is its CEO. He testified at FDA hearings arguing for federal regulation to ensure accuracy in labeling.
“Right now, there’s no one enforcing any of those things,” Mueller said. “It’s sort of up to the companies to do it themselves.”
“So, that really is the Wild West — whatever you say is what ends up on the label, is what people think they’re buying?” asked Petersen.
“Yeah, you can see that from the results here that it is the Wild West,” Mueller said. “And what you get on the shelf is, you don’t really know.”
To find out if what we don’t know can hurt us, Petersen went to the emergency room at U.C. Health University of Colorado Hospital. There he met Dr. Andrew Monte, a toxicologist and emergency medicine physician who has treated people who’ve ingested too much CBD.
“Patients actually can become more somnolent than would be expected,” Dr. Monte said. “So, they could become very sleepy. Patients can also get nausea, vomiting and diarrhea. Those are the most common side effects from oral CBD.”
And here is another “buyer beware”: don’t turn to CBD and turn away from medications your doctor prescribed.
“It seems to me that people have no problem with paying $50 for a very small vial of cannabidiol, yet they don’t wanna pay their $5 copay for a medicine that’s actually been tested in a randomized controlled clinical trial,” Dr. Monte said.
Now, it’s worth mentioning Dr. Monte tells CBS News that cannabinoids do interact with prescription drugs. But because we lack reliable controlled trials, we don’t have enough detail to understand all the interactions.
https://www.cbsnews.com/news/cbd-cbs-news-tests-cannabidiol-products-for-cbd-thc-and-impurities-dosages/
CBD itself does not get you high, but advocates say it can help with anything from muscle aches to anxiety. It’s forecast to become a $22 billion industry by 2024.
But without wide federal oversight, there is no way of really knowing what’s inside CBD.
So, we decided to find out.
CBS News partnered with Mile High Labs, in Colorado, to test nine CBD oil samples purchased from around the country.
It took two days for senior lab associate Joshua Cogell to test our nine samples, checking for CBD and for THC (the ingredient in marijuana that gives a high), and also for dangerous impurities, like pesticides and heavy metals.
“We test for four different heavy metals: mercury, arsenic, cadmium and lead,” said Cogell.
“They all sound bad,” said correspondent Barry Petersen.
“They are very, yes, things you do not want in your body.”
The CBD craze took off when President Trump signed the Farm Bill last December. The bill allows farmers to legally grow hemp, the source of CBD. It is a member of the marijuana plant family, but it produces only trace amounts of THC. Under the bill, the government allows less than 0.3% THC in CBD products.
Here’s what Mile High found in our samples:
- None had pesticides or heavy metals above federal standards.
- The THC levels were all within federal guidelines.
- Four samples were pretty much right on.
- Two samples cheated you, giving only 60 to 80% of the advertised dosage.
- Then there were the over-performers: A thousand-milligram sample was really 1,100 milligrams — 10% higher.
- And one was way over – 210% of what the label said.
“That’s really concerning to me,” said Steve Mueller, who founded Mile High Labs in Loveland, Colorado, and is its CEO. He testified at FDA hearings arguing for federal regulation to ensure accuracy in labeling.
“Right now, there’s no one enforcing any of those things,” Mueller said. “It’s sort of up to the companies to do it themselves.”
“So, that really is the Wild West — whatever you say is what ends up on the label, is what people think they’re buying?” asked Petersen.
“Yeah, you can see that from the results here that it is the Wild West,” Mueller said. “And what you get on the shelf is, you don’t really know.”
To find out if what we don’t know can hurt us, Petersen went to the emergency room at U.C. Health University of Colorado Hospital. There he met Dr. Andrew Monte, a toxicologist and emergency medicine physician who has treated people who’ve ingested too much CBD.
“Patients actually can become more somnolent than would be expected,” Dr. Monte said. “So, they could become very sleepy. Patients can also get nausea, vomiting and diarrhea. Those are the most common side effects from oral CBD.”
And here is another “buyer beware”: don’t turn to CBD and turn away from medications your doctor prescribed.
“It seems to me that people have no problem with paying $50 for a very small vial of cannabidiol, yet they don’t wanna pay their $5 copay for a medicine that’s actually been tested in a randomized controlled clinical trial,” Dr. Monte said.
Now, it’s worth mentioning Dr. Monte tells CBS News that cannabinoids do interact with prescription drugs. But because we lack reliable controlled trials, we don’t have enough detail to understand all the interactions.
https://www.cbsnews.com/news/cbd-cbs-news-tests-cannabidiol-products-for-cbd-thc-and-impurities-dosages/
FDA issues warning letters to websites selling illegal opioids
The U.S. Food and Drug Administration and the Drug Enforcement
Administration (DEA) jointly issued warning letters to four online
networks for illegally marketing unapproved and misbranded versions of
opioid medicines, the agencies said.
The networks which were issued the warning letters on Monday are Divyata, Euphoria Healthcare Pvt Ltd, JCM Dropship and Meds4U, which operate a total of 10 websites.
The move comes at a time when health regulators in the country are
combating the rising use of opioids, which were responsible for 47,600
overdose deaths in the United States in 2017.
Misbranded drugs are those that fail to bear adequate directions for their intended use, the FDA said.
The networks also violated the Controlled Substances Act by not registering the online pharmacies with the DEA despite advertising for the sale of opioids, the agencies said.
In case the online networks fail to take corrective action within 15 working days, the websites’ domain name registrars may be informed, the agencies said.
In June last year, the FDA issued warning letters to nine other online networks operating a total of 53 websites, to stop illegally marketing unapproved versions of opioid medications.
https://www.reuters.com/article/us-usa-opioids/fda-issues-warning-letters-to-websites-selling-illegal-opioids-idUSKBN1WF1UE
The networks which were issued the warning letters on Monday are Divyata, Euphoria Healthcare Pvt Ltd, JCM Dropship and Meds4U, which operate a total of 10 websites.
Misbranded drugs are those that fail to bear adequate directions for their intended use, the FDA said.
The networks also violated the Controlled Substances Act by not registering the online pharmacies with the DEA despite advertising for the sale of opioids, the agencies said.
In case the online networks fail to take corrective action within 15 working days, the websites’ domain name registrars may be informed, the agencies said.
In June last year, the FDA issued warning letters to nine other online networks operating a total of 53 websites, to stop illegally marketing unapproved versions of opioid medications.
https://www.reuters.com/article/us-usa-opioids/fda-issues-warning-letters-to-websites-selling-illegal-opioids-idUSKBN1WF1UE
AnaptysBio -13.5% after GALLOP trial data
AnaptysBio (NASDAQ:ANAB) has slipped 13.5% postmarket after posting some positive topline data from its interim analysis of the GALLOP Phase 2 clinical trial.
That analysts was conducted with the first two
patients completing a 16-week ANB019 monotherapy study for treating
moderate-to-severe generalized pustular psoriasis.
That’s a chronic, life-threatening, rare inflammatory disease that has no approved therapies, the company says.
Enrollment is ongoing and the company expects additional clinical data and a regulatory strategy update in 2020.
https://seekingalpha.com/news/3502749-anaptysbio-minus-13_5-percent-gallop-trial-dataBiotech Investors: Mark Your Calendar For These October PDUFA Dates
September turned out to be a fruitful month for FDA approvals. The regulatory agency gave its nod to Ardelyx Inc ARDX 2.89%‘s Ibsrela, a new molecular entity indicated to treat irritable bowel syndrome with constipation in adults.
With Ibsrela, NME approvals for the year totaled 27, lower than the 41 approvals given by the same time last year.
Some of the therapies that crossed the FDA hurdle included Johnson & Johnson JNJ 0.66%‘s Janssen unit’s Erleada for the expanded indication of metastatic castration-sensitive prostate cancer; Novo Nordisk A/S NVO 0.17%‘s oral semaglutide for Type 2 diabetes; and Merck & Co., Inc. MRK 1.54%‘s HIV drugs Pifeltro (doravirine) and Delstrigo (doravirine/lamivudine/tenofovir disoproxil fumarate).
The following are the key PDUFA dates for the upcoming month.
Type of Application: sNDA
Candidate: Descovy (emtricitabine 200 mg and tenofovir alafenamide 25 mg tablets)
Indication: Pre-exposure prophylaxis, or PrEP, for HIV-1 infection
Date: Oct. 4 (Estimated, assuming 6-month review period due to the priority voucher submitted with the application)
Descovy was initially approved in April 2016 to be used in combination with other antiretroviral agents for the treatment of HIV-1 infection in adults and pediatric patients ages 12 and older.
Gilead is now eyeing approval for Descovy as a preventive treatment for reducing the risk of sexually acquired HIV-1 infection among individuals who are HIV-negative and at risk for HIV.
An FDA panel that met in August voted 16-2 to recommend approval of Descovy as PrEP to reduce the risk of HIV acquisition in men having sex with men and transgender women. The panel voted 8-10 against approving it for cisgender women.
Type of Application: NDA
Candidate: Scenesse
Indication: prevention of phototoxicity and anaphylactoid reactions in adult patients with erythropoietic protoporphyria, or EPP
Date: Oct. 6
Scenesse is a controlled release injectable implant containing afamelanotide, which is being developed as a first-line treatment for patients with EPP, a rare genetic metabolic disorder that causes phototoxicity and anaphylactoid reactions when patients expose their skin to light.
In June, the FDA communicated its decision to extend the review period by three months to provide it with more time for the review of the full NDA submission.
Type of Application: NDA
Candidate: PF708
Indication: Osteoporosis
Date: Oct. 7
F708 is a therapeutic equivalent of Eli Lilly And Co LLY 0.53%‘s Forteo, which was approved in 2002 to treat osteoporosis in men and menopausal women who are at high risk of having a fracture.
Forteo’s global sales stood at $1.6 billion in 2018.
Type of Application: sBLA
Candidate: MabThera/Rituxan in combination with glucocorticoids
Indication: treatment of granulomatosis with polyangiitis and microscopic polyangiitis in children two years of age and older
Date: Oct. 11. This is estimated, assuming the filing of the application two months prior to the sBLA acceptance date of June 12.
Granulomatosis with polyangiitis and microscopic polyangiitis are rare, potentially life-threatening diseases affecting small- and medium-sized blood vessels.
Type of Application: sNDA
Candidate: Xarelto
Indication: prevention of venous thromboembolism in medically ill patients
Date: Oct. 13 (estimated)
Xarelto, an anticoagulant, was initially approved for prophylactic treatment of deep vein thrombosis that may lead to pulmonary embolism in people undergoing knee or hip replacement surgery. Since then, J&J had secured several label expansions for the drug.
Type of Application: sNDA
Candidate: Zilretta
Indication: repeat administration for osteoarthritis of the knee
Date: Oct. 14
Zilretta was initially approved in Oct. 2017 as an extended-release, intra-articular injection for osteoarthritis knee pain. In the recent second quarter, Zilretta sales rose 60% quarter-over-quarter to $17 million.
Type of Application: BLA
Candidate: Brolucizumab
Indication: wet age-related macular degeneration, or AMD
Date: Oct. 15 (Estimated, based on the submission filing date of April 15)
Type of Application: sBLA
Candidate: Nplate
Indication: treatment of adult patients with immune thrombocytopenia for 12 months or less
Date: Oct. 18 (Estimated, based on the submission filing date of Dec. 19)
Nplate was approved in 2008 as a long-term treatment of adults with chronic immune thrombocytopenia, or low platelet count. Later, Nplate was approved for pediatric patients with the same indication.
Type of Application: NDA
Candidate: Xipere
Indication: treatment of macular edema associated with uveitis
Date: Oct. 19
Xipere, chemically triamcinolone acetonide ophthalmic suspension, is formulated as suprachoroidal Injection for the treatment of macular edema associated with uveitis.
Type of Application: NDA
Candidate: Cosyntropin
Indication: Diagnostic drug for detecting adrenocortical insufficiency
Date: Oct. 19
Long-acting cosyntropin is an alcohol-free formulation of a synthetic analogue of adrenocorticotropic hormone. It’s secreted by the pituitary gland and is responsible for the stimulation of the adrenal cortex. Contingent on FDA approval, the company expects to launch the product in the U.S. by early 2020.
Type of Application: sBLA
Candidate: Ultomiris
Indication: Atypical hemolytic uremic syndrome, or aHUS
Date: Oct. 19
aHUS, also known as complement-mediated thrombotic microangiopathy, is a severe and chronic ultra-rare disease that can cause progressive damage to vital organs, predominantly the kidneys, leading to kidney failure and premature death.
Ultomiris was previously approved in December 2018 for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria.
Type of Application: NDA
Candidate: FMX101
Indication: treatment of inflammatory lesions of non-nodular moderate-to-severe acne vulgaris in patients 9 years and older
Date: Oct. 20
Type of Application: NDA
Candidate: ET-202
Indication: ready-to-use injectable formulation of phenylephrine
Date: Oct. 21
Type of Application: sNDA
Candidate: Baxdella
Indication: community-acquired bacterial pneumonia, or CABP
Date: Oct. 24
BAXDELA, in both capsule and IV formulations, was approved by the FDA in 2017 for the treatment of adult patients with acute bacterial skin and skin structure infections caused by designated susceptible bacteria.
Type of Application: sNDA
Candidate: Zejula
Indication: Ovarian cancer
Date: Oct. 24
Zejula, or niraparib, came into Glaxo’s stable following its acquisition of Tesaro.
Glaxo is now seeking approval of Zejula for the treatment of advanced ovarian, fallopian tube or primary peritoneal cancer patients who have been treated with three or more prior chemotherapy regimens and whose cancer is associated with either BRCA mutation or homologous recombination deficiency.
Previously, Zejula was approved as a maintenance treatment of women with recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy.
Type of Application: NDA
Candidate: Higher dose naloxone spray
Indication: opioid overdose
Date: Oct. 31
Adamis recently said it was conducting additional pharmacokinetic studies comparing its long-acting naloxone spray, provisionally given the name Zimhi, to a relevant comparator. The company said it will forward data from the study to the FDA later this month.
The Bone, Reproductive and Urologic Drug Advisory Committee is scheduled to meet on Tuesday, Oct. 29 to discuss AMAG Pharmaceuticals, Inc. AMAG 4.55%‘s sNDA for Makena, which is being evaluated for reducing the risk of recurrent preterm birth or improving neonatal mortality and morbidity.
The same committee will discuss Wednesday, Oct. 30 Agile Therapeutics Inc AGRX 5.8%‘s NDA for Twirla — a low-dose combo hormonal contraceptive patch — to be used for birth control.
https://www.benzinga.com/general/biotech/19/09/14491876/attention-biotech-investors-mark-your-calendar-for-these-october-pdufa-dates
With Ibsrela, NME approvals for the year totaled 27, lower than the 41 approvals given by the same time last year.
Some of the therapies that crossed the FDA hurdle included Johnson & Johnson JNJ 0.66%‘s Janssen unit’s Erleada for the expanded indication of metastatic castration-sensitive prostate cancer; Novo Nordisk A/S NVO 0.17%‘s oral semaglutide for Type 2 diabetes; and Merck & Co., Inc. MRK 1.54%‘s HIV drugs Pifeltro (doravirine) and Delstrigo (doravirine/lamivudine/tenofovir disoproxil fumarate).
The following are the key PDUFA dates for the upcoming month.
Gilead Seeks Label Expansion For HIV Combo Drug
Company: Gilead Sciences, Inc. GILD 0.76%Type of Application: sNDA
Candidate: Descovy (emtricitabine 200 mg and tenofovir alafenamide 25 mg tablets)
Indication: Pre-exposure prophylaxis, or PrEP, for HIV-1 infection
Date: Oct. 4 (Estimated, assuming 6-month review period due to the priority voucher submitted with the application)
Descovy was initially approved in April 2016 to be used in combination with other antiretroviral agents for the treatment of HIV-1 infection in adults and pediatric patients ages 12 and older.
Gilead is now eyeing approval for Descovy as a preventive treatment for reducing the risk of sexually acquired HIV-1 infection among individuals who are HIV-negative and at risk for HIV.
An FDA panel that met in August voted 16-2 to recommend approval of Descovy as PrEP to reduce the risk of HIV acquisition in men having sex with men and transgender women. The panel voted 8-10 against approving it for cisgender women.
Will Clinuvel’s Phototoxicity Drug See The Light?
Company: Clinuvel Pharmaceuticals Ltd CLVLY 1.26%Type of Application: NDA
Candidate: Scenesse
Indication: prevention of phototoxicity and anaphylactoid reactions in adult patients with erythropoietic protoporphyria, or EPP
Date: Oct. 6
Scenesse is a controlled release injectable implant containing afamelanotide, which is being developed as a first-line treatment for patients with EPP, a rare genetic metabolic disorder that causes phototoxicity and anaphylactoid reactions when patients expose their skin to light.
In June, the FDA communicated its decision to extend the review period by three months to provide it with more time for the review of the full NDA submission.
Pfenex Seeks Nod For Therapeutic Equivalent of Lilly’s Osteoporosis Drug
Company: Pfenex Inc PFNX 0.94%Type of Application: NDA
Candidate: PF708
Indication: Osteoporosis
Date: Oct. 7
F708 is a therapeutic equivalent of Eli Lilly And Co LLY 0.53%‘s Forteo, which was approved in 2002 to treat osteoporosis in men and menopausal women who are at high risk of having a fracture.
Forteo’s global sales stood at $1.6 billion in 2018.
Roche’s Blood Vessel Inflammation Drug Awaits FDA Nod
Company: Roche Holdings AG Basel ADR RHHBY 0.03%Type of Application: sBLA
Candidate: MabThera/Rituxan in combination with glucocorticoids
Indication: treatment of granulomatosis with polyangiitis and microscopic polyangiitis in children two years of age and older
Date: Oct. 11. This is estimated, assuming the filing of the application two months prior to the sBLA acceptance date of June 12.
Granulomatosis with polyangiitis and microscopic polyangiitis are rare, potentially life-threatening diseases affecting small- and medium-sized blood vessels.
J&J’s Seeks Another Label Expansion For Anticoagulant Drug Xarelto
Company: J&J’s Janssen unitType of Application: sNDA
Candidate: Xarelto
Indication: prevention of venous thromboembolism in medically ill patients
Date: Oct. 13 (estimated)
Xarelto, an anticoagulant, was initially approved for prophylactic treatment of deep vein thrombosis that may lead to pulmonary embolism in people undergoing knee or hip replacement surgery. Since then, J&J had secured several label expansions for the drug.
FDA To Rule On Second Indication For Flexion’s Osteoarthritis Drug
Company: Flexion Therapeutics Inc FLXN 1.47%Type of Application: sNDA
Candidate: Zilretta
Indication: repeat administration for osteoarthritis of the knee
Date: Oct. 14
Zilretta was initially approved in Oct. 2017 as an extended-release, intra-articular injection for osteoarthritis knee pain. In the recent second quarter, Zilretta sales rose 60% quarter-over-quarter to $17 million.
Will FDA See Merit In Novartis’ Wet AMD Drug?
Company: Novartis AG NVS 0.55%Type of Application: BLA
Candidate: Brolucizumab
Indication: wet age-related macular degeneration, or AMD
Date: Oct. 15 (Estimated, based on the submission filing date of April 15)
Second Label Expansion In The Cards For Amgen’s Nplate?
Company: Amgen, Inc. AMGN 0.73%Type of Application: sBLA
Candidate: Nplate
Indication: treatment of adult patients with immune thrombocytopenia for 12 months or less
Date: Oct. 18 (Estimated, based on the submission filing date of Dec. 19)
Nplate was approved in 2008 as a long-term treatment of adults with chronic immune thrombocytopenia, or low platelet count. Later, Nplate was approved for pediatric patients with the same indication.
Go or No-go For Clearside’s Macular Edema Drug?
Company: Clearside Biomedical Inc CLSD 4.28%Type of Application: NDA
Candidate: Xipere
Indication: treatment of macular edema associated with uveitis
Date: Oct. 19
Xipere, chemically triamcinolone acetonide ophthalmic suspension, is formulated as suprachoroidal Injection for the treatment of macular edema associated with uveitis.
Assertio Seeks Assent For Adrenocortical Insufficiency Diagnostic Drug
Company: Assertio Therapeutics Inc ASRT 2.29%Type of Application: NDA
Candidate: Cosyntropin
Indication: Diagnostic drug for detecting adrenocortical insufficiency
Date: Oct. 19
Long-acting cosyntropin is an alcohol-free formulation of a synthetic analogue of adrenocorticotropic hormone. It’s secreted by the pituitary gland and is responsible for the stimulation of the adrenal cortex. Contingent on FDA approval, the company expects to launch the product in the U.S. by early 2020.
Can Alexion’s Ultra-Rare Disease Drug Snag Second Approval?
Company: Alexion Pharmaceuticals, Inc. ALXN 0.17%Type of Application: sBLA
Candidate: Ultomiris
Indication: Atypical hemolytic uremic syndrome, or aHUS
Date: Oct. 19
aHUS, also known as complement-mediated thrombotic microangiopathy, is a severe and chronic ultra-rare disease that can cause progressive damage to vital organs, predominantly the kidneys, leading to kidney failure and premature death.
Ultomiris was previously approved in December 2018 for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria.
Can Foamix Acne Treatment Clear FDA Hurdle?
Company: Foamix Pharmaceuticals Ltd FOMX 0.98%Type of Application: NDA
Candidate: FMX101
Indication: treatment of inflammatory lesions of non-nodular moderate-to-severe acne vulgaris in patients 9 years and older
Date: Oct. 20
Eton Braces For Decision On Ready-to-use Phenylephrine
Company: Eton Pharmaceuticals Inc ETON 1.94%Type of Application: NDA
Candidate: ET-202
Indication: ready-to-use injectable formulation of phenylephrine
Date: Oct. 21
Melinta On Track For Another Approval For Antibacterial Drug?
Company: Melinta Therapeutics Inc NASDAQ: MLNT) and Ligand Pharmaceuticals Inc. LGND 0.6%Type of Application: sNDA
Candidate: Baxdella
Indication: community-acquired bacterial pneumonia, or CABP
Date: Oct. 24
BAXDELA, in both capsule and IV formulations, was approved by the FDA in 2017 for the treatment of adult patients with acute bacterial skin and skin structure infections caused by designated susceptible bacteria.
GlaxoSmithKline’s Ovarian Cancer Drug to Add Another Indication?
Company: GlaxoSmithKline plc GSK 0.8%Type of Application: sNDA
Candidate: Zejula
Indication: Ovarian cancer
Date: Oct. 24
Zejula, or niraparib, came into Glaxo’s stable following its acquisition of Tesaro.
Glaxo is now seeking approval of Zejula for the treatment of advanced ovarian, fallopian tube or primary peritoneal cancer patients who have been treated with three or more prior chemotherapy regimens and whose cancer is associated with either BRCA mutation or homologous recombination deficiency.
Previously, Zejula was approved as a maintenance treatment of women with recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy.
Adamis’ Opioid Overdose Drug Awaits Clearance
Company: Adamis Pharmaceuticals Corp ADMP 5.94%Type of Application: NDA
Candidate: Higher dose naloxone spray
Indication: opioid overdose
Date: Oct. 31
Adamis recently said it was conducting additional pharmacokinetic studies comparing its long-acting naloxone spray, provisionally given the name Zimhi, to a relevant comparator. The company said it will forward data from the study to the FDA later this month.
Adcom Schedule
FDA’s Antimicrobial Drugs Advisory Committee will meet Wednesday, Oct. 16 to discuss SHIONOGI & CO L/ADR SGIOY 1.95%‘s NDA for cefiderocol lyophilized powder for intravenous administration for the treatment of complicated urinary tract infection, including pyelonephritis due to gram-negative bacteria in patients with limited or no alternative treatment options.The Bone, Reproductive and Urologic Drug Advisory Committee is scheduled to meet on Tuesday, Oct. 29 to discuss AMAG Pharmaceuticals, Inc. AMAG 4.55%‘s sNDA for Makena, which is being evaluated for reducing the risk of recurrent preterm birth or improving neonatal mortality and morbidity.
The same committee will discuss Wednesday, Oct. 30 Agile Therapeutics Inc AGRX 5.8%‘s NDA for Twirla — a low-dose combo hormonal contraceptive patch — to be used for birth control.
https://www.benzinga.com/general/biotech/19/09/14491876/attention-biotech-investors-mark-your-calendar-for-these-october-pdufa-dates
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