Monthly Archives: June 2021

Many more people who have breast cancer access complementary therapies than oncologists believe, according to new study results presented at the 2021 Annual Meeting of the American Society of Clinical Oncology (ASCO).

Cancer is a complex disease that affects every component of a patients life. While conventional medicine is effective for curing disease, it can fall short in helping patients heal, said Wayne Jonas, MD, a study coauthor and the executive director of integrative health programs at the Samueli Foundation, according to The ASCO Post, the societys publication.

Complementary medicine, also known as integrative medicine, refers to a range of practices that are used in conjunction with standard medical ones, according to the National Institutes of Health. These include yoga, tai chi, qi gong, meditation, mindfulness and acupuncture. (When these are employed instead of conventional medicine, by contrast, they are referred to as alternative medicine and are associated with poorer outcomes.)

While many of these practices have been shown to improve quality of life and health during and after cancer treatment, people with cancer need expert guidance to integrate them safely and effectively into their regimens. When oncologists fail to discuss complementary medicine with their patients, their patients often must resort to tracking down information and resources on their own.

"Many oncologists are generally supportive of integrating complementary and lifestyle therapies with conventional medical treatment, but the education and guidance given to patients varies widely," Terri Crudup, the studys first author and the senior principal of primary intelligence at the data science company IQVIA, told The ASCO Post.

The new study involved national surveys that were administered to 115 clinical oncologists who treat breast cancer and 164 people with breast cancer who had been diagnosed less than two years before. The surveys were conducted by IQVIA in late 2020, according to the Post.

The majority of patients (73%) surveyed reported accessing one or more types of complementary medicine in the wake of their diagnosis. By contrast, the majority of oncologists surveyed were under the impression that only a minority of patients (43%) did. The oncologists also revealed that they discussed complementary medicine with only roughly 50% of their patients.

Both populations saw the potential value of complementary medicine. The majority of oncologists (66%) and the majority of patients (65%) believed that accessing complementary medicine improved quality of life. In addition, the majority of patients (60%) thought that such treatments improved their health outcomes.

However, oncologists and patients occasionally disagreed on the effectiveness of specific practices. Perhaps as a result, patients reported that their oncologists provided fewer recommendations for spiritual services and meditation and mindfulness than they did for, say, nutrition consultations, support groups, psychological support and exercise.

The study also found that patients preferred learning about complementary medicine from their oncologists rather than from oncology nurses or patient navigators.

"Oncologists and the institutions at which they practice should look for methods to educate and expose patients to a variety of safe and effective complementary and lifestyle therapies to find the ones that will help their patients most, Crudup said, according to the Post.

For more on cancer patients and nontraditional medicine, read People With Cancer Are Using Complementary MedicineWithout Telling Their Doctors.

See the original post here:

Oncologists Underestimate the Number of Breast Cancer Patients Who Use Complementary Medicine - Cancer Health Treatment News

SAN RAFAEL, Calif., June 28, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the company resubmitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A.In May 2021, the EMA granted the Company's request for accelerated assessment. Accelerated assessment potentially reduces the time frame for the EMA Committee for Medicinal Products for Human Use (CHMP) and Committee for Advanced Therapies (CAT) to review a MAA for an Advanced Therapy Medicinal Product (ATMP).A CHMP opinion is anticipated in the first half of 2022.

The submission includes safety and efficacy data from the 134 subjects enrolled in the Phase 3 GENEr8-1 study, all of whom have been followed for at least one year after treatment with valoctocogene roxaparvovec, as well as four and three years of follow-up from the 6e13 vg/kg and 4e13 vg/kg dose cohorts, respectively, in the ongoing Phase 1/2 dose escalation study.

"This is an important step to deliver the potential first gene therapy for people with hemophilia A. The data package for this submission includes the largest Phase 3 study in any gene therapy for hemophilia A supported by up to four years of observation in a Phase 1/2 study," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. "We look forward to the EMA reviewing the robust data set. We believe that valoctocogene roxaparvovec represents a scientific innovation that may become an important treatment choice for those people who have unmet medical needs."

"This submission is important for the bleeding disorders community given the need for continued scientific and medical innovation to provide a treatment option to address unmet needs for many people with hemophilia A," said Brian O Mahony FACSLM, Chief Executive, Irish Haemophilia Society. "We believe that therapeutic choices are critical for our community to provide access to alternative treatment approaches with the potential to address individual patient needs."

Applications are eligible for accelerated assessment if the CHMP and CAT decide the product is of major interest for public health, particularly from the point of view of therapeutic innovation. Evaluating a MAA under the EMA centralized procedure can take up to 210 days, not counting clock stops when applicants are requested to provide additional information. On request, the CHMP and CAT can reduce the time frame to 150 days if the applicant provides sufficient justification for an accelerated assessment, although an application initially designated for accelerated assessment can revert to the standard procedure during the review for a variety of reasons. The decision to grant accelerated assessment has no impact on the eventual CHMP and CAT opinion on whether a marketing authorization should be granted.

Regulatory Status

In the United States, BioMarin intends to submit two-year follow-up safety and efficacy data on all study participants from the Phase 3 GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec, as previously requested by the Food and Drug Administration (FDA). BioMarin is targeting a Biologics License Application (BLA) resubmission in the second quarter of 2022, assuming favorable study results, followed by an expected six-month review by the FDA.

The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec in March 2021. RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are expected to address an unmet medical need in patients with serious conditions. The RMAT designation is complementary to Breakthrough Therapy Designation, which the Company received in 2017.

In addition to the RMAT Designation and Breakthrough Therapy Designation, BioMarin's valoctocogene roxaparvovec also has received orphan drug designation from the FDA and EMA for the treatment of severe hemophilia A.The Orphan Drug Designation program is intended to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions.

Robust Clinical Program

BioMarin has multiple clinical studies underway in its comprehensive gene therapy program for the treatment of hemophilia A. In addition to the global Phase 3 study GENEr8-1 and the ongoing Phase 1/2 dose escalation study, the Company is actively enrolling participants in a Phase 3b, single arm, open-label study to evaluate the efficacy and safety of valoctocogene roxaparvovec at a dose of 6e13 vg/kg with prophylactic corticosteroids in people with hemophilia A. The Company is also running a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with hemophilia A with pre-existing AAV5 antibodies, as well as another Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with hemophilia A with active or prior FVIII inhibitors.

About Hemophilia A

People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A (FVIII levels <1%) often experience painful, spontaneous bleeds into their muscles or joints.Individuals with the most severe form of hemophilia A make up approximately45 percent of the hemophilia A population.People with hemophilia A with moderate (FVIII 1-5%) or mild (FVIII 5-40%) disease show a much-reduced propensity to bleed.The standard of care for adults with severe hemophilia A is a prophylactic regimen of replacement Factor VIII infusions administered intravenously up to two to three times per week or 100 to 150 infusions per year.Despite these regimens, many people continue to experience breakthrough bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life.

Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have Hemophilia A.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare and ultra-rare genetic diseases. The Company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visitwww.biomarin.com. Information on BioMarin's website is not incorporated by reference into this press release.

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about (i) the development of BioMarin's valoctocogene roxaparvovec program generally, (ii) the anticipated timing of a CHMP opinion in the first half of 2022, (iii) BioMarin's intention to submit to the U.S. Food and Drug Administration (FDA) two-year follow-up safety and efficacy data on all study participants from the GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec, (iv) BioMarin targeting resubmission of a Biologics License Application in the second quarter of 2022 assuming favorable study results, followed by an expected six-month review procedure by the FDA, and (v) the timing of the regulatory activities in the U.S and Europe, including validation and timing of potential approvals and the expected review procedures. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned preclinical studies and clinical trials of valoctocogene roxaparvovec; additional data from the continuation of the Phase 1/2 trial and the Phase 3 trial, any potential adverse events observed in the continuing monitoring of the participants in the clinical trials; the content and timing of decisions by the FDA, the European Commission and other regulatory authorities; the content and timing of decisions by local and central ethics committees regarding the clinical trials; our ability to successfully manufacture valoctocogene roxaparvovec for the clinical trials and commercially, if approved; and those other risks detailed from time to time under the caption "Risk Factors" and elsewhere in BioMarin's Securities and Exchange Commission (SEC) filings, including BioMarin's Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, and future filings and reports by BioMarin. BioMarin undertakes no duty or obligation to update any forward-looking statements contained in this press release as a result of new information, future events or changes in its expectations.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc.

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Debra Charlesworth

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

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A review of data from more than 315 clinical trials of weight loss supplements has returned results suggests very little strong evidence exists to support use of dietary supplements and alternative therapies as weight loss interventions in adults.

A systematic conducted by investigators at the University of North Carolina (UNC), results of the study suggest risk of bias and sufficiency varied widely across the studies examined and less than 17% of trials were considered to be of low risk of bias and sufficient to support efficacy.

"Our findings are important for clinicians, researchers, and industry alike as they suggest the need for rigorous evaluation of products for weight loss," said lead investigator John Batsis, MD, associate professor in the Division of Geriatric Medicine at the UNC School of Medicine and in the Department of Nutrition at the UNC Gillings School of Global Public Health, in a statement. "Only then can we produce data that allows clinicians to provide input and advice with a higher degree of certainty to our patients."

As the obesity epidemic continues to plague the US and other developed nations, the popularity of weight loss supplements and different dietary approaches has skyrocketed. With an interest in exploring claims of efficacy made by many manufacturers in the dietary supplement industry, Batsis and a team of colleagues sought to evaluate the current available evidence base related to use of these supplements and dietary approaches.

To do so, Batsis and colleagues designed the study as a systematic review of work assessing dietary supplements and alternative therapies for weight loss from within the Medline, Cochrane Library, Web of Science, CINAHL, and Embase databases. For inclusion in the review, studies needed to be designed as randomized clinical trials, published in English, contain patients with overweight or obesity, and examine a patient population 18 years of age or older.

A total of 20,504 records were identified in the investigators' initial query. After exclusion of duplicates and those not meeting inclusion criteria, 1743 full-text manuscripts were identified for assessment and 315 were ultimately included in the final review. These 315 examined 14 different types of dietary interventions, including calcium and vitamin D supplements, chitosan, chocolate/cocoa, green tea, guar gum, mind-body, and pyruvate.

Upon assessment, investigators found the risk of bias varied widely across the studies included in the review Overall, 52 (16.5%) studies were classified as having a low risk of bias. Of these, 16 (31%) demonstrated significant pre/post weight changes compared to placebo therapy. Investigators pointed out the weight loss observed in these studies was 0.3-4.93 kg. Trials demonstrating significant weight loss included 1 trial on chitosan, 1 on chromium, 5 on ephedra or caffeine, 2 on garcinia, 2 on green tea, and 1 on conjugated linoleum acid.

In a perspective statement from The Obesity Society, authors underlined the need for clinicians and providers to be aware of the lack of evidence surrounding many of these supplements, despite unregulated claims related to efficacy.

Our recommendation to clinicians is to consider the lack of evidence for non-FDA-approved dietary supplements and therapies and guide their patients toward tested weight management approaches. Public and private entities should provide adequate resources for obesity management. Finally, we call on regulatory authorities to critically examine the dietary supplement industry, including their role in promoting misleading claims and marketing products that have potential harm to patients, wrote investigators.

This study, A Systematic Review of Dietary Supplements and Alternative Therapies for Weight Loss, was published in Obesity.

Read more from the original source:

UNC-Led Review Details Lack of High-Quality Evidence to Support Supplements, Alternative Therapies for Weight Loss - Endocrinology Network