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FDA Continues tough Support of Innovation in the Development of Gene Therapy Products

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FDA Continues tough Support of Innovation in the Development of Gene Therapy Products

Jan 28, 2020: This is a pivotal time in the field of the gene therapy as the FDA continues its efforts in order to support innovators developing new medical products for Americans and others around the world. Up till now, the FDA has approved four gene therapy products, which insert new genetic material into the patient’s cells.

The agency think likely for many more approvals in the coming years, as evidenced by the more than 900 investigational new drug (IND) applications for ongoing clinical studies in this area, this will provide patients and providers with increased therapeutic choices.

In that spirit, today, the FDA is announcing the release of the number of important policies: six final guidances on the gene therapy manufacturing and clinical development of products and a draft guidance, Interpreting Sameness of the Gene Therapy Products Under the Orphan Drug Regulations

.One of the most important steps the FDA can take to support safe innovation in this field is to create policies that can provide product developers with the meaningful guidance to answer critical questions as they research and design their gene therapy products.

Overall, these policy documents are representative of the efforts to help advance product development in the field of gene therapy.

The FDA is an agency inside the U.S. Department of Health and Human Services which protects the public health by assuring the safety, efficiency, and security of the human and veterinary drugs, vaccines and other biological products for human use, and medical devices.

The agency also is responsible for the safety and protection of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products. https://www.fda.gov/news-events/press-announcements/fda-continues-strong-support-innovation-development-gene-therapy-products

Clozaril, Fazaclo ODT, Versacloz (clozapine): Drug Safety Communication – FDA reinforce Warning That Untreated Constipation Can Lead to Serious Bowel Problems

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Clozaril, Fazaclo ODT, Versacloz (clozapine): Drug Safety Communication – FDA reinforce Warning That Untreated Constipation Can Lead to Serious Bowel Problems

Jan 28,2020: FDA is underpinning an existing warning that constipation caused by the schizophrenia medicine clozapine (Clozaril, Fazaclo ODT, Versacloz, generics) can, unusually, progress to serious bowel complications. This can lead to hospitalization or even death if the constipation is not diagnosed and treated quickly.

Background: Clozapine affects how the intestines (bowels) function in the widely held patients. It produces effects ranging from constipation (trouble having a bowel movement), which is a frequent incident, to serious but unusual bowel problems, including complete blockage of the bowel.

Recommendation: Patients should contact to their health care professional if:

  • Their bowel movements are not as much than normal.
  • Not have a bowel movement for at least three times a week.
  • Have hard or dry stools.
  • Have difficulty passing gas.
  • Patients should contact your health care professional immediately if you have symptoms which can be associated with the serious bowel problems such as: nausea, vomiting, bloating or belly swelling, or belly pain.

Health care professionals should:

  • Evaluate the bowel function before starting the patient on clozapine.
  • Avoid co-prescribing clozapine with other anticholinergic medicines (cause gastrointestinal hypomotility).
  • Advise patients frequently of the significant risk of the constipation and life-threatening bowel issues and the need in order to stay hydrated to prevent constipation.
  • Question patients concerning the frequency and quality of their bowel movements throughout treatment.

Healthcare professionals and patients are encouraged to account adverse events or side effects related to the use of these products to the FDA’s MedWatch Safety Information and Adverse Event Reporting Program. https://fda.einnews.com/pr_news/508279977/clozaril-fazaclo-odt-versacloz-clozapine-drug-safety-communication-fda-strengthens-warning-that-untreated-constipation-can-lead-to-serious

Phase II DESTINY-Gastric01 trial of Enhertu (trastuzumab deruxtecan) versus chemotherapy met primary endpoint

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Phase II DESTINY-Gastric01 trial of Enhertu (trastuzumab deruxtecan) versus chemotherapy met primary endpoint

Jan 27, 2020: High-level results from the positive registrational Phase II trial DESTINY-Gastric01 showed AstraZeneca’s and Daiichi Sankyo Company, Limited (Daiichi Sankyo)’s Enhertu (trastuzumab deruxtecan), achieved a statistically considerable and clinically meaningful development in objective response rate (ORR) and overall survival (OS) in patients with HER2-positive unresectable or metastatic gastric or gastroesophageal junction cancer that had developed following two or more treatment regimens including trastuzumab and chemotherapy.

The trial met its primary endpoint of an improvement in ORR, as measured by an independent review committee, in patients treated with Enhertu compared to the investigator’s choice of the chemotherapy (irinotecan or paclitaxel monotherapy). 

Enhertu showed a statistically important and clinically meaningful improvement in OS, a key secondary endpoint.

Adverse effect: In general, safety and tolerability profile of Enhertu in DESTINY-Gastric01 was reliable with that seen in the published Phase I trial in which the most common adverse actions (≥30 percent, any grade) were haematologic and gastrointestinal that includes neutrophil count decrease, anaemia, nausea and decreased appetite.

Also, there were cases of treatment-related interstitial lung disease (ILD) and pneumonitis, the popular of which were Grade 1 and 2 with two Grade 3 and one Grade 4. No ILD-related deaths (Grade 5) occurred in the gastric patients in the Phase I trial or in the DESTINY-Gastric01 trial.Data from DESTINY-Gastric01 will be presented at a upcoming medical meeting.

In addition to the planned discussion with the Japan Ministry of Health, Labour and Welfare (MHLW), both companies plan to discuss their data with other health authorities. 

Recently Enhertu  received Accelerated Approval in the US for HER2-positive unresectable or metastatic breast cancer following two or more prior anti-HER2 based treatment regimens and the additional global regulatory submissions in HER2-positive metastatic breast cancer are underway.

Enhertu is being jointly developed and commercialised worldwide with AstraZeneca except in the Japan where Daiichi Sankyo maintains exclusive rights.

DESTINY-Gastric01: DESTINY-Gastric01 is the registrational Phase II, open-label, multi-centre trial assessing the safety and effectiveness of Enhertu in 189 patients from Japan and South Korea with HER2-expressing advanced gastric cancer or gastroesophageal junction adenocarcinoma (defined as IHC3+ or IHC2+/ISH+) who have progressed on two or more prior treatment regimens that  includes fluoropyrimidine (5-FU) and platinum chemotherapy and trastuzumab.

https://www.astrazeneca.com/media-centre/press-releases/2020/phase-ii-destiny-gastric01-trial-of-enhertu-versus-chemotherapy-met-primary-endpoint-27012020.html

AstraZeneca’s Brilinta met primary endpoint in Phase III THALES trial in stroke

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AstraZeneca’s Brilinta met primary endpoint in Phase III THALES trial in stroke

Jan 27, 2020: High-level results from the Phase III THALES trial showed AstraZeneca’s Brilinta (ticagrelor) 90 mg used daily two times a day and taken with aspirin for 30 days, reached a statistically considerable and clinically meaningful reduction in the risk of the primary composite endpoint of the stroke and death, compared to aspirin alone.

THALES was conducted in more than 11,000 patients who had a minor acute ischaemic stroke or high-risk transient ischaemic attack (TIA) in the 24 hours prior to treatment initiation.

The initial safety findings in the THALES trial were reliable with the known profile of Brilinta, with an increased bleeding rate in the treatment arm.

The full THALES trial results will be presented at the forthcoming medical meeting.

Brilinta is approved in over 110 countries for the treatment of acute coronary syndrome (ACS) and in over 70 countries for the secondary prevention of cardiovascular (CV) events among high-risk patients who have experienced a heart attack.

Stroke: Stroke is the second main cause of death worldwide, with 6.2 million stroke-related deaths in 2017, from which 2.7 million were due to ischaemic stroke.

Patients who experience an acute ischaemic stroke or TIA are at high risk of the developing subsequent ischaemic events, with predominantly high risk within 30 days after the initial event and highest risk period being the first 24 hours after the initial event.

THALES:THALES is an AstraZeneca-sponsored, randomised, placebo-controlled, double-blinded, international, multicentre, event-driven trial involving more than 11,000 patients in order to test the hypothesis whether Brilinta and aspirin is superior to aspirin alone in preventing  composite of the  stroke and death in patients with minor acute ischaemic stroke or high-risk TIA. https://www.astrazeneca.com/media-centre/press-releases/2020/brilinta-met-primary-endpoint-in-phase-iii-thales-trial-in-stroke-27012020.html

FDA accepts for priority review Sanofi’s Dupixent® (dupilumab) for children aged 6 to 11 years with moderate-to-severe atopic dermatitis

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FDA accepts for priority review Sanofi’s Dupixent® (dupilumab) for children aged 6 to 11 years with moderate-to-severe atopic dermatitis

Jan 28, 2020: The U.S.FDA has accepted Priority Review the supplemental Biologics License Application (sBLA) for Sanofi’s Dupixent® (dupilumab) as an add-on maintenance treatment for children aged 6 to 11 years with moderate-to-severe atopic dermatitis whose disease is not effectively controlled with topical prescription therapies or when those therapies are not advisable, target action date for the FDA decision is May 26, 2020.

Regardless of standard-of-care therapy, many young children suffering with moderate-to-severe atopic dermatitis continue to experience uncontrolled, persistent symptoms.

These children live with intense, persistent itching, skin lesions along with skin dryness, cracking, redness or darkness, crusting and oozing.

The sBLA is supported by the data that includes pivotal Phase 3 results on the efficiency and safety of Dupixent shared with topical corticosteroids (TCS) in children with severe atopic dermatitis.

Results of the trial shows, children treated with Dupixent and TCS experienced considerably improved measures of overall disease severity, skin clearing, itching and health-related quality of life, compared to TCS alone. 

Adverse events more commonly observed with Dupixent included conjunctivitis, nasopharyngitis and injection site reactions, which is consistent with the previously documented safety profile of Dupixent in older populations. 

In 2016, the FDA granted Breakthrough Therapy designation to review Dupixent for the treatment of severe atopic dermatitis in the childrens of 6 months to 11 years of age not well controlled on topical prescription medications.

MOA: Dupixent is a fully human monoclonal antibody that inhibits signaling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) proteins.

Data from the Dupixent clinical trials have shown that IL-4 and IL-13 are key drivers of the type 2 inflammation that plays a key role in atopic dermatitis, asthma and chronic rhinosinusitis with nasal polyposis (CRSwNP).

https://fda.einnews.com/pr_news/508230379/sanofi-fda-accepts-for-priority-review-dupixent-dupilumab-for-children-aged-6-to-11-years-with-moderate-to-severe-atopic-dermatitis

AstraZeneca divests rights to Inderal, Tenormin, Tenoretic, Zestril and Zestoretic to Atnahs Pharma (Atnahs) to established hypertension medicines

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AstraZeneca divests rights to Inderal, Tenormin, Tenoretic, Zestril and Zestoretic to Atnahs Pharma (Atnahs) to established hypertension medicines

Jan 27, 2020: AstraZeneca has decided to sell the global commercial rights to Inderal (propranolol), Tenormin (atenolol), Tenoretic (atenolol, chlorthalidone fixed-dose combination), Zestril (lisinopril) and Zestoretic (lisinopril, hydrochlorothiazide fixed-dose combination) to Atnahs Pharma (Atnahs).

The agreement excludes the rights in the US and India, which were previously deprived, and in Japan, which will be reserved by AstraZeneca.

These medicines, used primarily to treat hypertension, have lost their patent protection worldwide. AstraZeneca will continue to manufacture and supply to Atnahs during a transition period.

Financial considerations: Atnahs will make an candid payment of $350m to AstraZeneca. AstraZeneca may also be received future sales-contingent payments of up to $40m between 2020 and 2022.

In 2018, Inderal, Tenormin, Tenoretic, Zestril and Zestoretic produced annual sales of $132m in the markets covered by this agreement. 

The divestment is expected to be completed in the first quarter of 2020, subject to customary closing conditions and regulatory clearances.                                                                     

https://www.astrazeneca.com/media-centre/press-releases/2020/astrazeneca-divests-rights-to-established-hypertension-medicines-27012020.html

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

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

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

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

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

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

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

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/

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