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Medtronic Launches Efficio™ Software to facilitate Clinicians Efficiently Manage Targeted Drug Delivery Therapy with SynchroMed™ II

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Jan. 23, 2020: Medtronic announced the launch of Efficio™, a cloud-based data management software for use with the SynchroMed™ II intrathecal drug delivery system, that will allow clinicians to more powerfully manage their targeted drug delivery pump practices in order to treat patients with chronic pain, cancer pain and severe spasticity.

Efficio management software allows clinicians in order to manage patients’ pumps with greater effectiveness and ease by consolidating, organizing and providing visual summaries of the patient data from the clinician programmer in a secure cloud environment accessible at anytime, anywhere.

Additional benefits include the ability to proactively schedule patient appointments, assist prescription drug order tracking for the pump refills, and printing reports on-demand. With Efficio, clinicians can handle patient data without the use of spreadsheets and simply export data into their electronic medical records systems as appropriate.

Efficio software supports HIPAA compliance and is designed in order to safeguard protected health information. Data is secluded using encryption and authentication. Access restrictions and login time limits are also in the place to help protect data.

SynchroMed™ II Intrathecal Drug Delivery System: The SynchroMed II intrathecal drug delivery system offers a safe, verified, and effective way to manage chronic pain, cancer pain and severe spasticity for the suitable patients.

SynchroMed II provides efficient pain relief at a fraction of the oral dose with less side effects and has been shown in order to reduce or eliminate the use of oral opioids. http://newsroom.medtronic.com/news-releases/news-release-details/medtronic-launches-efficiotm-software-help-clinicians

FDA approves Tazverik (tazemetostat) for the treatment of adults and pediatric patients with metastatic or locally advanced epithelioid sarcoma

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Jan 23, 2020: U.S. FDA granted accelerated approval to Tazverik (tazemetostat) for the treatment of adults and pediatric patients aged 16 years and older with metastatic (when cancer cells spread to other parts of the body) or locally advanced (when the cancer has grown outside the organ it started in, but has not yet spread to distant parts of the body) epithelioid sarcoma not eligible for the complete resection (surgically removing all of the tissue, structure, or organ).

Epithelioid sarcoma is a rare sub-type of the soft tissue sarcoma that often occurs in young adults. “Epithelioid sarcoma accounts for less than one percent of all soft tissue sarcomas,”

MOA: Tazverik blocks activity of the EZH2 methyltransferase, which could help keep the cancer cells from growing.

The majority cases of epithelioid sarcoma begin in the soft tissue under the skin of an extremity, although it can start in other areas of the body. Surgical removal is considered the key treatment when the cancer is localized to one area of the body.

Chemotherapy or radiation may also be given. nevertheless, there is a high likelihood for the local and regional spread of the disease even with treatment and around 50% of patients have metastatic disease at the time of diagnosis.

Tazverik’s approval was based on results of a clinical trial enrolling 62 patients with the metastatic or locally advanced epithelioid sarcoma. During this trial, patients received 800 milligrams (mg) of Tazverik two times a day until the disease progressed or the patient reached an intolerable level of toxicity.

Tumor response assessments were performed every eight weeks throughout the clinical trial. The overall response rate of the trial was 15%, with 1.6% of patients having a complete response and 13% having a partial response.

Of the nine patients that had a response, six (67%) patients have  a response lasting six months or longer.

Side effects: Patients treated with Tazverik are at increased risk of developing secondary malignancies including: T-cell lymphoblastic lymphoma (a type of blood cancer that affects the lymphatic system generally found in the lymph nodes), myelodysplastic syndrome (a disorder resulting from the poorly formed or dysfunctional blood cells) and the acute myeloid leukemia (a cancer of the blood and bone marrow).

Tazverik granted Accelerated Approval, which enables the FDA in order to approve drugs for the serious conditions to fill an unmet medical need based on a result that is reasonably likely to predict a clinical benefit to patients.

Tazverik also received Orphan Drug designation, which provides incentives to support and encourage the development of drugs for rare diseases.The FDA also granted approval of Tazverik to Epizyme Inc.

https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-option-specifically-patients-epithelioid-sarcoma-rare-soft-tissue

Celularity Announces FDA Clearance of IND for CYNK-001, an Allogeneic, Off-the-Shelf Cryopreserved NK Cell Therapy for glioblastoma multiforme (GBM)

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Jan 22, 2020: U.S. Food and Drug Administration (FDA) has cleared the Celularity’s Investigational New Drug (IND) Application for CYNK-001 in patients with glioblastoma multiforme (GBM).

The clinical investigation of the CYNK-001 in patients with GBM is expected to be the first clinical trial in U.S. in order to investigate intratumoral administration of an allogeneic NK cell therapy.

The Company plans to initiate the first-in-human clinical testing of CYNK-001 administered intravenously or intratumorally, to evaluate the safety, feasibility, and tolerability of multiple doses of CYNK-001 in subjects with relapsed GBM.

Dose: Single dose of the CYNK-001 was well-tolerated and showed enhanced in vivo anti-tumor activity against glioblastoma multiforme (GBM).

CYNK-001 investigated for the treatment for acute myeloid leukemia (AML), multiple myeloma (MM), and as a potential treatment option for various solid tumors.

About CYNK-001:CYNK-001 is the only cryopreserved allogeneic, off-the-shelf NK cell therapy being developed from the placental hematopoietic stem cells as a potential treatment option for various hematologic cancers and solid tumors.

NK cells are a exceptional class of immune cells, innately capable of targeting cancer cells and interacting with adaptive immunity.

NK cells derived from the placenta are essentially safe and versatile, allowing potential uses across a range of organs and tissues. https://www.businesswire.com/news/home/20200122005061/en/Celularity-Announces-FDA-Clearance-Landmark-IND-CYNK-001

FDA Approves Medtronic Micra™ AV, the World’s Smallest Pacemaker with atrioventricular (AV) synchrony for the treatment of patients with AV block

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Jan. 22, 202: U.S. FDA grants approval for Medtronic’s Micra™ AV, the world’s smallest pacemaker with atrioventricular (AV) synchrony for the treatment of patients with AV block. It is a condition in which the electrical signals between the chambers of the heart (the atria and the ventricle) are impaired.

Now Medtronic offers the first and only FDA-approved leadless pacemaker portfolio, expanding number of potential candidates for this groundbreaking technology in U.S.

In the past, patients with AV block have been treated with the traditional dual-chamber pacemakers which are implanted in the upper chest, under the skin beneath the collar bone, and connected to the heart by using thin wires known as “leads.”

Micra AV has several additional internal atrial sensing algorithms which detect cardiac movement that allows device to adjust pacing in the ventricle in order to coordinate with the atrium, providing “AV synchronous” pacing therapy to the patients with the AV block.

The Micra AV approval is based on data from the MARVEL 2 (Micra Atrial Tracking Using A Ventricular accELerometer) study that evaluates the safety and effectiveness of accelerometer-based atrial sensing algorithms.

The study evaluated the ability of the Micra’s internal sensor in order to monitor and detect atrial contractions and facilitate coordinated pacing between the atrium and ventricle, thereby providing AV synchrony.

About the Micra Transcatheter Pacing System (TPS)
Approved by the FDA in 2016, the Micra TPS is a leadless pacemaker option for the patients who only require pacing in the right ventricle.

Comparable in size to a large vitamin, Micra is less than one-tenth the size of the traditional pacemakers so far delivers advanced pacing technology to patients via a simply invasive approach.

During the implant procedure, the device is attached to the heart with small tines and delivers electrical impulses that pace the heart through an electrode at the end of the device.

http://newsroom.medtronic.com/news-releases/news-release-details/fda-approves-medtronic-micratm-av-worlds-smallest-pacemaker

Akcea and Ionis report positive topline Phase 2 results of AKCEA-APOCIII-L Rx for the treatment of hypertriglyceridemia

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Jan 22, 2020: Akcea Therapeutics and Ionis Pharmaceuticals announced positive topline results from the Phase 2 study of AKCEA-APOCIII-LRx in the treatment of patients with hypertriglyceridemia, patients who have established cardiovascular disease (CVD).

The objective of the Phase 2 study was to evaluate the safety and efficacy of altered doses and dosing frequencies of AKCEA-APOCIII-LRx.

The multicenter, randomized, double-blind, placebo-controlled, dose-ranging study that includes 114 patients with the clinical diagnosis of CVD or who are at high risk of CVD.

Participants were administered AKCEA-APOCIII-LRx or placebo via SC injection for at least six months with some patients being treated up to a year.

Weekly, bi-weekly, and monthly dosing was explored in the four cohorts with doses ranging from 10 mg to 50 mg of total monthly dose.

Observations from the AKCEA-APOCIII-LRx study included: Statistically considerable dose-dependent reductions in the fasting triglycerides compared to placebo at all dose levels.

Maximum once monthly dose of 50 mg, more than 90% of patients achieved serum triglycerides of ≤ 150 mg/dL, compared to below 5% of patients in the placebo group; mean triglyceride levels of patients at the baseline was 285 mg/dL, Major reductions in multiple additional risk factors, including apoC-III, very low-density lipoprotein (VLDL-C) and remnant cholesterol, compared to placebo.

Statistically considerable increases in the high-density lipoprotein cholesterol (HDL-C) compared to placebo at all dose levels. Around 85% of the patients completed treatment and the rate of treatment discontinuation was comparable between the active and the placebo groups.

About AKCEA-APOCIII-LRx
AKCEA-APOCIII-LRx (ligand conjugated antisense (LICA) drug) was discovered by the Ionis and co-developed by Akcea and Ionis.

It is an antisense drug developed using Ionis’ proprietary LIgand Conjugated Antisense (LICA) technology platform and is designed in order to inhibit production of apolipoprotein C-III (apoC-III), a protein produced in the liver that plays a essential role in the regulation of serum triglycerides. https://ir.akceatx.com/news-releases/news-release-details/akcea-and-ionis-report-positive-topline-phase-2-results-akcea

European Commission approves Darzalex® in combination with bortezomib, thalidomide and dexamethasone for multiple myeloma

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Jan 22, 2020: The European Commission (EC) approved marketing authorisation for Janssen Pharmaceutical’s Darzalex (daratumumab) in combination with bortezomib, thalidomide and dexamethasone (VTd) for the treatment of multiple myeloma.

The approval, granted is based on the results of part one of the company’s Phase III CASSIOPEIA study, published in The Lancet.

The Phase III preliminary is a two-section study; section one exhibited that the total reaction rate was essentially higher in the daratumumab with VTd gathering, at 29 percent, contrasted with VTd alone, at 20 percent.

Movement free endurance (PFS) was altogether improved in the daratumumab-VTd set contrasted with exclusively VTd, the expansion of daratumumab to VTd brought about a 18-month PFS pace of 93 percent contrasted with 85 percent for VTd alone.

Most common side effects observed were: neutropenia, lymphopenia, stomatitis and thrombocytopenia. In the daratumumab-VTd blend arm, implantation related responses happened in 35 percent of patients. https://www.europeanpharmaceuticalreview.com/news/111080/ec-approves-darzalex-in-combination-with-vtd-for-multiple-myeloma/

Jazz Pharmaceuticals reported that EU Marketing Authorisation for Sunosi® (solriamfetol) for Excessive Daytime Sleepiness in Adults with Narcolepsy or Obstructive Sleep Apnea

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Jan 22, 2020:Jazz Pharmaceuticals reported that the European Commission endorsed Sunosi® (solriamfetol) to improve alertness and lessen unnecessary daytime languor (EDS) in grown-ups with narcolepsy (with or without cataplexy) or obstructive rest apnea (OSA) whose EDS has not been acceptably treated by essential OSA treatment, for example, ceaseless positive aviation route pressure (CPAP).

Mechanism of action: Sunosi is the primary double acting dopamine and norepinephrine reuptake inhibitor endorsed to treat EDS in grown-ups living with narcolepsy or OSA and the main authorized treatment in the European Union for the treatment of EDS in grown-ups living with OSA.

Sunosi is approved with doses of 75 mg and 150 mg once-daily for people with narcolepsy and doses of 37.5 mg, 75 mg and 150 mg for the patients with OSA. 

At Week 12 of the Phase 3 clinical trial, 150 mg of solriamfetol for the narcolepsy patients and both 75 mg and 150 mg doses for OSA patients established improvements in restlessness compared to the placebo as assessed via the maintenance of wakefulness test from approximately one hour post-dose through around nine hours post-dose.

Solriamfetol has the potential to be an important treatment option for the patients living with extreme daytime sleepiness as a result of OSA or narcolepsy.

The Marketing Authorisation Application (MAA) for Sunosi is based on the facts from four randomised placebo-controlled studies included in the Treatment of Obstructive sleep apnea and Narcolepsy Excessive Sleepiness (TONES) clinical trial program.

Data from the studies in the TONES program established the superiority of solriamfetol relative to the placebo. The most commonly reported adverse reactions were headache (11.1%), nausea (6.6%) and decreased appetite (6.8%).

The most serious and frequently occurring adverse reactions were increased blood pressure and palpitations.

In the TONES 2 and TONES 3 12-week clinical studies in the narcolepsy and OSA patients, respectively, around 68-74% of people taking solriamfetol at the 75 mg dose and 78-90% of people taking solriamfetol at the 150 mg dose reported enhancement in their overall clinical condition, as assessed by the Patient Global Impression of Change (PGIc) scale. https://investor.jazzpharma.com/news-releases/news-release-details/jazz-pharmaceuticals-receives-eu-marketing-authorisation-sunosir

AstraZeneca’s ‘Desire Zero Carbon’ system to take out outflows by 2025 and be carbon negative over the whole worth chain by 2030

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Jan 22, 2020: AstraZeneca revealed a goal-oriented program for zero carbon outflows from its worldwide activities by 2025 and to guarantee its whole worth chain is carbon negative by 2030, presenting decarbonisation designs by over 10 years.

Propelled today at the World Economic Forum (WEF) Annual Meeting in Davos, Switzerland, AstraZeneca’s ‘Desire Zero Carbon’ technique quickens the Company’s current science-based targets, multiplying vitality profitability and utilizing sustainable power source for both force and warmth, just as changing to a 100% electric vehicle armada five years in front of timetable.

The Company will contribute up to $1bn to accomplish these objectives and to build up the cutting edge respiratory inhalers with close to zero Global Warming Potential (GWP) fuels.

‘Desire Zero Carbon’ embarks to make AstraZeneca’s worldwide tasks answerable for zero carbon discharges without depending on counterbalance plans to arrive at zero emanations on total.

Planning for 50-million tree reforestation(‘AZ Forest’): Likewise they plan, is ‘AZ Forest’, a 50-million tree reforestation activity that will be turned out throughout the following five years, Trees naturally remove CO2 and are essential to mitigate the effects of climate change.In association with nearby governments and One Tree Planted, a non-benefit association concentrated on worldwide reforestation, the primary trees will go into the ground in Australia in February 2020, with France, Indonesia and different nations to follow. This activity underpins WEF’s recently propelled ‘1T.org – The Champions for a Trillion Trees’ foundation. As a further advance to help shape approach and discover cross-industry answers for quickly decarbonise the worldwide economy, Pascal Soriot, Chief Executive Officer, will likewise take an interest in the debut meeting of the Sustainable Markets Council (SMC). The Council was built up by His Royal Highness, the Prince of Wales, with the help of WEF, as a warning group of open private-charitable pioneers to drive manageable arrangements in accordance with the United Nation’s Sustainable Development Goals. Next-generation inhalers: AstraZeneca commits to launch its next-generation inhaler for the  treatment of asthma and chronic obstructive pulmonary disease (COPD) with near-zero GWP propellants by 2025.Also,the propellant used in the next generation pressurised metered-dose inhalers (pMDI) to encompass a GWP that is 90-99% lower than propellants used in older pMDIs. https://www.astrazeneca.com/media-centre/press-releases/2020/astrazenecas-ambition-zero-carbon-strategy-to-eliminate-emissions-by-2025-and-be-carbon-negative-across-the-entire-value-chain-by-2030-22012020.html

Free “LabXchange” Science Education Accelerator Launched By the Amgen Foundation And Faculty of Arts and Sciences at Harvard

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Jan 22, 2020: The Amgen Foundation alongside the Faculty of Arts and Sciences at Harvard University (Harvard FAS) declared the worldwide dispatch of LabXchange™, a free online science training stage that furnishes clients with access to customized guidance, virtual lab encounters and systems administration openings over the worldwide academic network.

LabXchange is reason worked to drive more consideration in the logical procedure and sparkle coordinated effort to manufacture innovative, group based ways to deal with genuine issues.

“For numerous students, science can feel like a collection of the facts in order to memorize – which is different to what the scientific process is – it is a journey that requires bold thinking and deep imagination.

With the LabXchange, more students can come in concert and experience the joy of discovery.”

Including virtual lab tests created at LabXchange alongside other world-class resources made by approved accomplices, LabXchange breathes life into the logical procedure.

By mimicking key procedures in sub-atomic and cell science, such as utilizing CRISPR to address hereditary imperfections, understudies can investigate a wide scope of logical techniques and assemble their intuition in saddling science to take care of genuine issues.

LabXchange is designed in order to level the playing field for the students and to promote science literacy for all.

Key features of the LabXchange includes:
Free access to the library of world-class educational content including videos, interactive simulations and the assessments.

Ability to mix and match materials, empowering teachers in order to create flexible learning pathways for classes or individual students that harmonize existing science curriculums.

Global networking functionality, enabling teachers to collaborate further than a single classroom, school or district. https://www.amgen.com/media/news-releases/2020/01/free-labxchange-science-education-accelerator-launched-by-amgen-foundation-and-harvards-faculty-of-arts-and-sciences/

US FDA grants priority review of GlaxoSmithKline’s belantamab mafodotin for patients with relapsed or refractory multiple myeloma

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Jan 21, 2020: GlaxoSmithKline declared that the US FDA allowed a need audit for the organization’s Biologics License Application (BLA) looking for endorsement of belantamab mafodotin for the treatment of patients with backslid or obstinate various myeloma whose earlier treatment incorporated an immunomodulatory specialist, a proteasome inhibitor and an enemy of CD38 counter acting agent.

The BLA depends on information from the urgent DREAMM-2 (DRiving Excellence in Approaches to Multiple Myeloma) study, as of late distributed in The Lancet Oncology, which selected intensely pre-treated patients who had effectively advancing different myeloma that had declined in spite of current standard of care.

In 2017, belantamab mafodotin was allowed Breakthrough Therapy assignment by the FDA, which is expected to encourage the improvement of investigational medications that have demonstrated clinical guarantee for conditions where there is noteworthy neglected need.

The function of BCMA is to promote plasma cell survival by transduction of signals from two known ligands, BAFF (B-cell activating factor) and APRIL (a proliferation-inducing ligand).Belantamab mafodotin is an investigational immunoconjugate which contain a humanised anti-B cell maturation antigen (BCMA) monoclonal antibody conjugated to cytotoxic agent auristatin F via non-cleavable linker. https://www.gsk.com/en-gb/media/press-releases/us-food-and-drug-administration-fda-grants-priority-review-of-belantamab-mafodotin-for-patients-with-relapsed-or-refractory-multiple-myeloma/

The U.S.FDA approved Horizon Therapeutics Tepezza (teprotumumab-trbw) for the treatment of adults with thyroid eye disease

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Jan 21, 2020:  The U.S. FDA approved Horizon Therapeutics Tepezza (teprotumumab-trbw) for the treatment of adults with thyroid eye disease, a rare condition where the muscles and fatty tissues behind the eye turned inflamed, causing the eyes to be pushed forward and bulge outwards (proptosis).

This approval represents the first drug approved for the treatment of thyroid eye disease. Thyroid eye disease is linked with the outward bulging of the eye that can cause a variety of symptoms such as eye pain, double vision, light sensitivity or complexity closing the eye. More women than men affected with this disease.

Thyroid eye disease can be devastating. For example, the troubling ocular symptoms that leads to the progressive inability of people with thyroid eye disease in order to perform important daily activities, such as driving or working.

Tepezza was affirmed dependent on the aftereffects of two investigations (Study 1 and 2) comprising of an aggregate of 170 patients with dynamic thyroid eye ailment who were randomized to either get Tepezza or a fake treatment.

Of the patients who were controlled Tepezza, 71% in Study 1 and 83% in Study 2 showed a more noteworthy than 2 millimeter decrease in proptosis (eye projection) when contrasted with 20% and 10% of subjects who got fake treatment, individually.

Tepezza ought not be utilized if pregnant, and ladies of youngster bearing potential ought to have their pregnancy status confirmed before starting treatment and ought to be directed on pregnancy anticipation during treatment and for a half year following the last portion of Tepezza.

The FDA allowed this application Priority Review, notwithstanding Fast Track and Breakthrough Therapy Designation. Also, Tepezza got Orphan Drug assignment, which gives motivating forces to help and empower the improvement of medications for uncommon sicknesses or conditions.

Improvement of this item was additionally to a limited extent bolstered by the FDA Orphan Products Grants Program, which gives awards to clinical investigations on wellbeing and viability of items for use in uncommon maladies or conditions.                https://fda.einnews.com/pr_news/507754416/fda-approves-first-treatment-for-thyroid-eye-disease

Supernus Announces U.S. FDA Acceptance for Review of the New Drug Application for SPN-812 for the Treatment of ADHD

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Jan. 21, 2020: Supernus Pharmaceuticals reported that the U.S. FDA has discovered the New Drug Application (NDA) for SPN-812(serotonin norepinephrine modulating agent) for the treatment of kids and youths with consideration shortfall hyperactivity issue (ADHD) worthy for audit.

The FDA has appointed a Prescription Drug User Fee Act (PDUFA) target activity date of November 8, 2020. If FDA approve SPN-812, will be the first new therapy to treat ADHD.

The NDA for SPN-812 depends on information from a broad improvement program comprising of four Phase III clinical preliminaries that concentrated the pediatric patient populace from the age of 6 to 17 years, two Phase II clinical preliminaries, a few Phase I preliminaries, a long haul open name augmentation study, preclinical testing, and medication fabricating information.

Every one of the four crucial clinical preliminaries indicated a decrease in ADHD-RS-5 all out score as ahead of schedule as Week 1 and proceeding until the finish of the clinical investigation, just as progress in both hyperactivity/impulsivity and negligence subscales.

The impact was factually critical for the examinations for 100mg, 200mg, and 400mg portions. SPN-812 had a satisfactory security profile with low frequency of antagonistic occasions and low suspension rates.

What’s more, during the final quarter of 2019, the Company started a Phase III program to examine SPN-812 in the grown-up ADHD persistent populace. https://fda.einnews.com/pr_news/507773640/supernus-announces-fda-acceptance-for-review-of-new-drug-application-for-spn-812-for-the-treatment-of-adhd