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New WHO guidance seeks to put an end to human rights violations in mental health care

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New WHO guidance seeks to put an end to human rights violations in mental health care

June 10, 2021: “Globally, the majority of mental health care continues to be provided in psychiatric hospitals, and human rights abuses and coercive practices remain all too common.

But providing community-based mental health care that is both respectful of human rights and focused on recovery is proving successful and cost-effective, according to new guidance released today by the World Health Organization.

Mental health care recommended in the new guidance should be located in the community and should not only encompass mental health care but also support for day-to-day living, such as facilitating access to accommodation and links with education and employment services.  

WHO’s new “Guidance on community mental health services: promoting person-centred and rights-based approaches” further affirms that mental health care must be grounded in a human rights-based approach, as recommended by the WHO Comprehensive Mental Health Action Plan 2020-2030 endorsed by the World Health Assembly in May 2021.

Much faster transition to redesigned mental health services required

“This comprehensive new guidance provides a strong argument for a much faster transition from mental health services that use coercion and focus almost exclusively on the use of medication to manage symptoms of mental health conditions, to a more holistic approach that takes into account the specific circumstances and wishes of the individual and offers a variety of approaches for treatment and support,” said Dr Michelle Funk of the Department of Mental Health and Substance Use, who led the development of the guidance.

Since the adoption of the Convention on the Rights of Persons with Disabilities (CRPD) in 2006, an increasing number of countries have sought to reform their laws, policies and services related to mental health care.

However, to date, few countries have established the frameworks necessary to meet the far-reaching changes required by international human rights standards.

Reports from around the world highlight that severe human rights abuses and coercive practices are still far too common in countries of all income levels.

Examples include forced admission and forced treatment; manual, physical and chemical restraint; unsanitary living conditions; and physical and verbal abuse.

The majority of government mental health budgets still goes to psychiatric hospitals

According to WHO’s latest estimates, governments spend less than 2% of their health budgets on mental health. Furthermore, the majority of reported expenditure on mental health is allocated to psychiatric hospitals, except in high-income countries where the figure is around 43%.

The new guidance, which is intended primarily for people with responsibility for organizing and managing mental health care, presents details of what is required in areas such as mental health law, policy and strategy, service delivery, financing, workforce development and civil society participation in order for mental health services to be compliant with the CRPD.

It includes examples from countries including Brazil, India, Kenya, Myanmar, New Zealand, Norway and the United Kingdom of community-based mental health services that have demonstrated good practices in respect of non-coercive practices, community inclusion, and respect of people’s legal capacity (i.e. the right to make decisions about their treatment and life).

Services include crisis support, mental health services provided within general hospitals, outreach services, supported living approaches and support provided by peer groups.

Information about financing and results of evaluations of the services presented are included.

Cost comparisons provided indicate that the community-based services showcased produce good outcomes, are preferred by service users and can be provided at comparable cost to mainstream mental health services.

“Transformation of mental health service provision must, however, be accompanied by significant changes in the social sector,” said Gerard Quinn, UN Special Rapporteur on the Rights of Persons with Disabilities.

“Until that happens, the discrimination that prevents people with mental health conditions from leading full and productive lives will continue.”

https://www.who.int/news/item/10-06-2021-new-who-guidance-seeks-to-put-an-end-to-human-rights-violations-in-mental-health-care

Pfizer and BioNTech Provide COVID-19 Vaccine to U.S. Government for Donation to Poorest Nations

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Pfizer and BioNTech Provide COVID-19 Vaccine to U.S. Government for Donation to Poorest Nations

June 10, 2021: “Pfizer and BioNTech announced plans to provide the U.S. government at a not-for-profit price 500 million doses of the companies’ COVID-19 vaccine, 200 million doses in 2021 and 300 million doses in the first half of 2022, to further support the multilateral efforts to address the surge of infection in many parts of the world and to help end the pandemic.

The government will, in turn, donate the Pfizer-BioNTech vaccine doses to low- and lower middle-income countries and organizations that support them.

As part of the plan, the United States will allocate the vaccine doses to 92 low- and lower middle-income countries and economies as defined by Gavi’s COVAX Advance Market Commitment (AMC) and the 55 member states of the African Union.

The U.S. government and the companies will work with COVAX to ensure these vaccines are delivered to the specified countries around the world in a way that is most efficient and equitable.

These doses are part of Pfizer and BioNTech’s previously announced pledge to provide two billion doses of the COVID-19 vaccine to low- and middle-income countries over the next 18 months.

“Our partnership with the U.S. government will help bring hundreds of millions of doses of our vaccine to the poorest countries around the world as quickly as possible.

COVID-19 has impacted everyone, everywhere, and to win the battle against this pandemic, we must ensure expedited access to vaccines for all.

I want to thank President Biden for his leadership in protecting the least advantaged of our global neighbors,” said Albert Bourla, Chairman and Chief Executive Officer, Pfizer.

“Fair and equitable distribution has been our North Star since Day One and we are proud to do our part to help vaccinate the world, a massive but an achievable undertaking.”

“As a vaccine developer, we felt the duty to develop a well-tolerated and highly effective vaccine and make it available to as many people worldwide as possible.

Today’s agreement underlines that the joint efforts of the private and the public sector are providing solutions to help end this pandemic,” said Ugur Sahin, M.D., CEO and Co-Founder of BioNTech.

“We are also committed to realizing sustainable solutions by supporting the establishment of manufacturing networks on various continents.

Our first step has been the establishment of our Regional Headquarters for south east Asia in Singapore which will also include mRNA manufacturing capacities for regional and global supply.

It is our goal to leverage our proprietary mRNA technology to help improve the health of people around the world.”

Deliveries of the 200 million doses will begin in August 2021 and continue through the remainder of the year.

The 300 million doses for 2022 will be delivered between January and end of June 2022. The U.S. government also has the option for additional doses in 2022.

The plan is to produce the doses being purchased by the U.S. government in Pfizer’s U.S. facilities.

Those U.S.-based sites involved in the production of the COVID-19 vaccine include Kalamazoo, MI, Andover, MA, Chesterfield, MO, Groton, CT, and McPherson, KS.

To date, Pfizer and BioNTech have shipped 700 million doses to more than 100 countries or territories around the world.

The companies have direct supply agreements in place with 122 countries and discussions are ongoing with many more on the supply of the companies’ COVID-19 vaccine.

Based on current projections, Pfizer and BioNTech expect to manufacture up to 3 billion doses of the COVID-19 vaccine in 2021.

The production capacity has consistently grown due to continued enhancements to the vaccine’s supply chain, which include expanding existing facilities, adding more suppliers, and bringing on additional Pfizer/BioNTech sites and contract manufacturers around the world to produce the vaccine.

Pfizer and BioNTech have an existing agreement in place to supply vaccine doses to the COVAX Facility, a mechanism established by Gavi, the Vaccine Alliance, the Coalition for Epidemic Preparedness Innovations (CEPI) and World Health Organization (WHO) that aims to provide governments with early access to a large portfolio of COVID-19 candidate vaccines using a range of technology platforms, produced by multiple manufacturers across the world.

Pfizer-BioNTech doses allocated through COVAX have reached countries in every region of the world, including Rwanda, South Korea, Colombia, Peru, Cabo Verde, Tunisia, Angola, West Bank and the Gaza Strip, Moldova, El Salvador, Mongolia, the Maldives, Bosnia and Herzegovina, Georgia, the Ukraine, Bolivia, Kosovo, Bhutan, Bangladesh, Laos, Pakistan and the Philippines.

Deliveries to 47 countries and territories around the world are planned through June 2021 as part of the COVAX second round allocation of Pfizer-BioNTech COVID-19 Vaccine doses.

Through both Pfizer and The Pfizer Foundation*, a number of other innovative assistance efforts have been supported, including:

  • Working with International Rescue Committee and the Jordanian Ministry of Health to provide essential primary health services and vaccinations to refugees in Jordan;
  • Collaborating with the UPS Foundation, which is donating freezers to countries that need assistance with building out their ultra-cold chain capacity; and
  • Partnering with Zipline through funding and technical expertise, to design and test a delivery solution that can safely and effectively distribute all COVID-19 vaccines in difficult to reach areas of the countries where it operates.

The U.S. government, the companies and COVAX will finalize the plan and further operational details in the coming weeks.

The Pfizer-BioNTech COVID-19 Vaccine, which is based on BioNTech proprietary mRNA technology, was developed by both BioNTech and Pfizer.

BioNTech is the Marketing Authorization Holder in the European Union, and the holder of emergency use authorizations or equivalent in the United States (jointly with Pfizer), Canada and other countries in advance of a planned application for full marketing authorizations in these countries.

The Pfizer-BioNTech COVID-19 Vaccine has not been approved or licensed by the U.S. Food and Drug Administration (FDA), but has been authorized for emergency use by FDA under an Emergency Use Authorization (EUA) to prevent Coronavirus Disease 2019 (COVID-19) caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) for use in individuals 12 years of age and older.

The emergency use of this product is only authorized for the duration of the declaration that circumstances exist justifying the authorization of emergency use of the medical product under Section 564 (b) (1) of the FD&C Act unless the declaration is terminated or authorization revoked sooner.

Please see Emergency Use Authorization (EUA) Fact Sheet for Healthcare Providers Administering Vaccine (Vaccination Providers) and Full EUA Prescribing Information available at www.cvdvaccine-us.com.

AUTHORIZED USE IN THE U.S.:

The Pfizer-BioNTech COVID19 Vaccine is authorized for use under an Emergency Use Authorization (EUA) for active immunization to prevent coronavirus disease 2019 (COVID-19) caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) in individuals 12 years of age and older.

IMPORTANT SAFETY INFORMATION FROM U.S. FDA EMERGENCY USE AUTHORIZATION PRESCRIBING INFORMATION:

  • Do not administer Pfizer‑BioNTech COVID-19 Vaccine to individuals with known history of a severe allergic reaction (e.g., anaphylaxis) to any component of the Pfizer‑BioNTech COVID-19 Vaccine
  • Appropriate medical treatment used to manage immediate allergic reactions must be immediately available in the event an acute anaphylactic reaction occurs following administration of Pfizer‑BioNTech COVID-19 Vaccine
    Monitor Pfizer-BioNTech COVID-19 Vaccine recipients for the occurrence of immediate adverse reactions according to the Centers for Disease Control and Prevention guidelines (https://www.cdc.gov/vaccines/covid-19/clinical-considerations/managing-anaphylaxis.html)
  • Syncope (fainting) may occur in association with administration of injectable vaccines, in particular in adolescents. Procedures should be in place to avoid injury from fainting
  • Immunocompromised persons, including individuals receiving immunosuppressant therapy, may have a diminished immune response to the Pfizer‑BioNTech COVID-19 Vaccine
  • The Pfizer‑BioNTech COVID-19 Vaccine may not protect all vaccine recipients
  • In clinical studies, adverse reactions in participants 16 years of age and older included pain at the injection site (84.1%), fatigue (62.9%), headache (55.1%), muscle pain (38.3%), chills (31.9%), joint pain (23.6%), fever (14.2%), injection site swelling (10.5%), injection site redness (9.5%), nausea (1.1%), malaise (0.5%), and lymphadenopathy (0.3%)
  • In a clinical study, adverse reactions in adolescents 12 through 15 years of age included pain at the injection site (90.5%), fatigue (77.5%), headache (75.5%), chills (49.2%), muscle pain (42.2%), fever (24.3%), joint pain (20.2%), injection site swelling (9.2%), injection site redness (8.6%), lymphadenopathy (0.8%), and nausea (0.4%)
  • Severe allergic reactions, including anaphylaxis, and other hypersensitivity reactions, diarrhea, vomiting, and pain in extremity (arm) have been reported following administration of the Pfizer-BioNTech COVID-19 Vaccine outside of clinical trials
    Additional adverse reactions, some of which may be serious, may become apparent with more widespread use of the Pfizer-BioNTech COVID-19 Vaccine
  • Available data on Pfizer‑BioNTech COVID-19 Vaccine administered to pregnant women are insufficient to inform vaccine-associated risks in pregnancy
  • Data are not available to assess the effects of Pfizer‑BioNTech COVID-19 Vaccine on the breastfed infant or on milk production/excretion
  • There are no data available on the interchangeability of the Pfizer‑BioNTech COVID‑19 Vaccine with other COVID-19 vaccines to complete the vaccination series. Individuals who have received one dose of Pfizer‑BioNTech COVID-19 Vaccine should receive a second dose of Pfizer‑BioNTech COVID-19 Vaccine to complete the vaccination series
  • Vaccination providers must report Adverse Events in accordance with the Fact Sheet to VAERS online at https://vaers.hhs.gov/reportevent.html. For further assistance with reporting to VAERS call 1-800-822-7967.

    The reports should include the words “Pfizer-BioNTech COVID-19 Vaccine EUA” in the description section of the report
  • Vaccination providers should review the Fact Sheet for Information to Provide to Vaccine Recipients/Caregivers and Mandatory Requirements for Pfizer-BioNTech COVID-19 Vaccine Administration Under Emergency Use Authorization.”

    https://www.pfizer.com/news/press-release/press-release-detail/pfizer-and-biontech-provide-500-million-doses-covid-19

FDA Provides Guidance on Measuring Patient-Reported Outcomes in Cancer Clinical Trials

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FDA Provides Guidance on Measuring Patient-Reported Outcomes in Cancer Clinical Trials

June 09, 2021: “The following quote is attributed to Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research“Patients would like to better understand symptoms they may experience and how a cancer therapy can affect their quality of life.

One way to accomplish this is to ask patients in clinical trials about the severity of their symptoms and ability to function using rigorously developed patient-reported outcomes.

However, achieving sufficient consistency and quality of these data in cancer drug applications submitted to the FDA has been a challenge.

The FDA’s Oncology Center of Excellence has undertaken a sustained effort to identify methods to rigorously collect patient-reported outcomes in cancer clinical trials.

We’ve been engaging with patients and outcomes research experts through a series of public workshops and publications External Link Disclaimer on which outcomes to measure, how frequently to assess them and the tools available to do so.

The draft guidance we’re releasing today is intended to improve the quality and consistency of data in order to inform patients with cancer about the symptoms and impacts they may experience during treatment with a cancer therapy.”

Additional Information

  • Today, the U.S. Food and Drug Administration issued a draft guidance document, “Core Patient-Reported Outcomes in Cancer Clinical Trials,” which, when finalized, will provide recommendations to sponsors on which patient-reported outcomes (PRO) concepts to measure in cancer clinical trials.
  • The draft guidance addresses how to incorporate assessment of PRO in clinical trials of drugs and biological products that are intended to support product labeling describing anti-tumor (preventing or inhibiting the formation or growth of tumors) activity in patients with cancer.
  • The draft guidance also addresses the frequency of these PRO assessments within cancer trials, highlighting that PRO assessments should be of sufficient frequency to take into account the administration schedule of the products being studied.

    It also discusses labeling considerations for PROs submitted with oncology product applications.”

    https://www.fda.gov/news-events/press-announcements/fda-brief-fda-provides-guidance-measuring-patient-reported-outcomes-cancer-clinical-trials

Roche obtains CE mark for the SARS-CoV-2 Antigen Self Test Nasal allowing for rapid self testing of COVID-19 at home

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June 08, 2021: “Roche announced it has received CE mark for its SARS-CoV-2 Antigen Self Test Nasal for at-home testing.

The test will be available in countries accepting the CE mark through pharmacies and other locations, in packs of five tests.

An early version of the test has already been available as a home-test in a number of European markets under local special approval pathways since February 2021.

With the CE Mark, the SARS-CoV-2 Antigen Self Test Nasal for rapid self testing of COVID-19 test has received official approval following the traditional registration pathway and can now also be used in markets that have not established regulatory exemption pathways.

By following simple instructions, individuals can perform the test at home using a nasal swab without special training or the supervision of a healthcare worker.

The test provides results in as little as 15 minutes and can help people to conveniently check if they are likely to be infectious from the comfort of their home.

In the case of children under 18 years of age, the test must be performed by an adult or under close adult supervision.

As societies begin to reopen and in line with local health regulations, the convenient test allows individuals planning to attend an event or gathering to use the test as a tool to confirm that they are not likely to be carriers of a substantial amount of the virus thus helping them make informed decisions and reduce the risk of transmission to others.

Complementary to the SARS-CoV-2 Antigen Self Test Nasal, Roche is offering NAVIFY® Pass.

This digital solution allows individuals and healthcare professionals to remotely store, display, and share their COVID-19 test results and vaccine status through a personalised QR code.

Having easy digital access to test results and vaccination status could potentially be used by both individuals and companies to facilitate access to locations with COVID-19 entry protocols, such as restaurants or entertainment venues, as well as to validate safety to travel.

“As the world prepares to reopen, high-quality, home-based testing will play an important role in the battle against the pandemic,” said Thomas Schinecker, CEO, Roche Diagnostics.

“Regular self-testing at home can reduce pressure on healthcare systems.

It can quickly identify people with the highest potential to be infectious so they can take immediate action to seek medical advice, manage their infection and protect others.”

This test is part of a partnership with SD Biosensor Inc., with whom Roche has also launched a SARS-CoV-2 Rapid Antibody Test in July 2020 and two SARS-CoV-2 Rapid Antigen Tests for professional use in September 2020 and February 2021.

These tests will continue to play an important role in fighting this pandemic and remain available for healthcare professional testing.

In addition to diagnostic testing, preventive measures remain key to protecting yourself and others against SARS-CoV-2.

It is recommended to continue wearing masks, socially distance and practice good hygiene, especially if you have symptoms or known contact with others who have tested positive for the virus.

Roche continues to expand its comprehensive COVID-19 portfolio to support healthcare systems in diagnosing SARS-CoV-2 infection.

About the SARS-CoV-2 Antigen Self Test Nasal for at-home patient self-testing
The SARS-CoV-2 Rapid Antigen Self Test Nasal is a rapid chromatographic immunoassay (lateral flow assay) for the detection of the nucleocapsid protein of SARS-CoV-2 in human nasal samples.

Each test contains a unique QR code to enable individuals to share their test results and vaccine status using NAVIFY Pass, Roche’s digital solution.

The clinical performance of the test was measured by head to head comparison with Roche’s highly sensitive reverse transcriptase polymerase chain reaction (RT-PCR) test using nasopharyngeal swab samples as a comparator, the gold standard sampling and detection method for SARS-CoV-2 detection.

Combined study results showed that the relative sensitivity of the SARS-CoV-2 Antigen Self Test Nasal was 91.1%.** The overall relative specificity was 99.6 %, which represents the ability of the test to correctly identify patients without the virus.

In one comparative independent self testing study where patients followed written and illustrated instructions to sample, test and read-out the results themselves, the majority of study participants considered the procedures easy to perform.

https://www.roche.com/media/releases/med-cor-2021-06-08.htm

FDA approved ADUHELM™ as the first and only Alzheimer’s disease treatment

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FDA approved ADUHELM™ as the first and only Alzheimer’s disease treatment

June 07, 2021: Biogen and Eisai announced that the U.S. FDA has granted accelerated approval for ADUHELM™ (aducanumab-avwa) as the first and only Alzheimer’s disease treatment to address a defining pathology of the disease by reducing amyloid beta plaques in the brain.

The accelerated approval has been granted based on data from clinical trials demonstrating the effect of ADUHELM on reducing amyloid beta plaques, a biomarker that is reasonably likely to predict clinical benefit, in this case a reduction in clinical decline.

Continued approval for ADUHELM’s indication as a treatment for Alzheimer’s disease may be contingent upon verification of clinical benefit in confirmatory trial(s).

“This historic moment is the culmination of more than a decade of groundbreaking research in the complex field of Alzheimer’s disease.

We believe this first-in-class medicine will transform the treatment of people living with Alzheimer’s disease and spark continuous innovation in the years to come,” said Michel Vounatsos, Chief Executive Officer at Biogen.

“We are grateful for the contributions of thousands of patients and caregivers who participated in our clinical trials, as well as for the dedication of our scientists and researchers.

Together with the healthcare community, we are ready to bring this new medicine to patients and begin to address this growing global health crisis.”

“Eisai has been working on the creation of new treatments for Alzheimer’s disease since the early 80s through our relentless pursuit to understand the root causes of this disease, and we have spent more than a quarter of a century with people living with Alzheimer’s disease to understand their needs,” said Haruo Naito, Chief Executive Officer at Eisai.

“We are very pleased to be able to open a new chapter in the history of Alzheimer’s disease treatment with the approval of ADUHELM.

This approval has the potential to bring hope to the future of global health, society and, most importantly, the patients and their families, and represents a great step toward the advancement of holistic ecosystem solutions for this devastating disease.”

The efficacy of ADUHELM was evaluated in two Phase 3 clinical trials—EMERGE (Study 1) and ENGAGE (Study 2)—in patients with early stages of Alzheimer’s disease (mild cognitive impairment and mild dementia) with confirmed presence of amyloid pathology.

The effects of ADUHELM were also assessed in the double-blind, randomized, placebo-controlled, dose-ranging Phase 1b study, PRIME (Study 3).

In these studies, ADUHELM consistently showed a dose- and time-dependent effect on the lowering of amyloid beta plaques (by 59 percent [p<0.0001] in ENGAGE, 71 percent [p<0.0001] in EMERGE, and 61 percent [p<0.0001] in PRIME).  

The ADUHELM safety profile is well characterized in over 3,000 patients who received at least one dose of ADUHELM.

The most frequently reported adverse event was radiographic detection of events termed Amyloid Related Imaging Abnormalities, or “ARIA.”

ARIA (-E and/or -H) was observed in 41 percent of patients treated with ADUHELM 10 mg/kg compared to 10 percent of patients on placebo.

Clinical symptoms were present in 24 percent of patients treated with ADUHELM 10 mg/kg who had an observation of ARIA (-E and/or -H), compared to 5 percent of patients on placebo.

The most common symptom in patients with ARIA was headache. Other symptoms associated with ARIA included confusion, dizziness, visual disturbances, and nausea.

Adverse reactions that were reported in at least 2 percent of patients treated with ADUHELM and at least 2 percent more frequently than in patients on placebo were ARIA-E, headache, ARIA-H microhemorrhage, ARIA-H superficial siderosis, fall, diarrhea, and confusion/delirium/altered mental status/disorientation.

As part of the accelerated approval, Biogen will conduct a controlled trial to verify the clinical benefit of ADUHELM in patients with Alzheimer’s disease.

Dr. Stephen Salloway, Director of Neurology and the Memory and Aging Program at Butler Hospital, said, “This approval represents a major advance in the treatment of Alzheimer’s disease. By reducing amyloid beta plaques in the brain, we are addressing one of the defining pathologies of the disease.

People with Alzheimer’s disease, together with their doctors, can now decide if the treatment is right for them.”

“Today’s approval of ADUHELM is a transformational breakthrough in the fight to stop this horrible disease.

After years of disappointment and despair, this decision offers new hope for many families and a trigger for future investment and innovation,” said George Vradenburg, Chairman and Co-Founder of UsAgainstAlzheimer’s.

“Because ADUHELM was studied in people with early-stage Alzheimer’s disease, it will be important for our nation’s healthcare system—patients, providers and payers—to be ready for earlier detection, diagnosis and intervention in the treatment of the disease.”

Investor Webcast Information

Biogen will host a live webcast to discuss the approval of ADUHELM on June 8, 2021 at 8:00 a.m. ET. To access the webcast, please go to the Investors section of Biogen’s website at investors.biogen.com.

An archived version of the webcast will be available following the presentation.

INDICATION and IMPORTANT SAFETY INFORMATION

INDICATION
ADUHELM is a prescription medicine used to treat people with Alzheimer’s disease.

IMPORTANT SAFETY INFORMATION
What is the most important information a patient should know about ADUHELM?
ADUHELM can cause serious side effects including: Amyloid Related Imaging Abnormalities or “ARIA”. ARIA is a common side effect that does not usually cause any symptoms but can be serious. 

It is most commonly seen as temporary swelling in areas of the brain that usually resolves over time.

Some people may also have small spots of bleeding in or on the surface of the brain with the swelling.

Although most people with swelling in areas of the brain do not have symptoms, some people may have symptoms such as: headache, confusion, dizziness, vision changes, and nausea.

The patient’s healthcare provider will do magnetic resonance imaging (MRI) scans before and during treatment with ADUHELM to check for ARIA. 

Patients should call their healthcare provider or go to the nearest hospital emergency room right away if they have any of the symptoms listed above.

Before receiving ADUHELM, patients should tell their healthcare provider about all of their medical conditions, including if: they are pregnant or plan to become pregnant or are breastfeeding or plan to breastfeed.

It is not known if ADUHELM will harm their unborn baby or if aducanumab-avwa (the active ingredient in ADUHELM) passes into breast milk.

What are the possible side effects of ADUHELM? ADUHELM can cause serious side effects, including: See above “What is the most important information a patient should know about ADUHELM?”

Serious allergic reactions. Swelling of the face, lips, mouth, or tongue and hives have happened during an ADUHELM infusion.

Patients should tell their healthcare provider if they have any of the symptoms of a serious allergic reaction during or after an ADUHELM infusion.

The most common side effects of ADUHELM include: swelling in areas of the brain, with or without small spots of bleeding in or on the surface of the brain (ARIA); headache and fall.

Patients should call their healthcare provider for medical advice about side effects. Patients may report side effects to FDA at 1-800-FDA-1088.”

https://investors.biogen.com/news-releases/news-release-details/fda-grants-accelerated-approval-aduhelmtm-first-and-only

Sanofi launches its new global employee share ownership plan

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Sanofi launches its new global employee share ownership plan

June 7, 2021: “Sanofi launches Action 2021, its global employee share ownership plan, open to 92,000 employees in 73 countries.

The program, similar to programs carried out since 2013, clearly demonstrates the ongoing commitment of Sanofi and its Board of Directors to involve all employees, across all its territories, in the future development and results of the company.

“The record level of employee participation in last year’s share plan demonstrated our employee’s commitment to the company and our long-term strategy and growth.

With the continued support of the Board of Directors, we want our employees to have these opportunities to be increasingly involved in Sanofi’s success by sharing in its performance,” said Paul Hudson, Chief Executive Officer of Sanofi.

As of today, the shares will be offered at a subscription price of 69,38€, which is equal to a 20% discount off the average of the 20 opening prices of Sanofi shares from May 6 to June 2, 2021.

In addition, for every five shares subscribed, employees will be able to receive one free share (up to a maximum of four free shares per employee).

And finally, each employee will be able to purchase up to 1,500 Sanofi shares within the legal limit of a maximum payment amount that does not exceed 25% of their gross annual salary, minus any voluntary deductions already made under employee savings schemes (Group Savings Plan and/or Group Retirement Savings Plan) during the year 2021.

In 2020, the employee share ownership plan, open to around 90,000 employees in 72 countries, saw its overall uptake rate exceed 36%. More than 33,000 Sanofi employees chose to invest in the company.

Today, nearly 90,000 current or former employees of the company are direct or indirect shareholders of Sanofi, and hold approximately 2.8% of its capital.”

https://www.sanofi.com/en/media-room/press-releases/2021/2021-06-07-18-00-00-2242990

Novartis announces Phase II data for iptacopan in rare kidney disease C3G

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Jun 07, 2021: “Novartis announced positive new interim Phase II data showing investigational iptacopan (LNP023) – a first-in-class, oral, targeted factor B inhibitor – improved estimated glomerular filtration rate (eGFR) slope and stabilized kidney function in patients with C3 glomerulopathy (C3G) treated with iptacopan.

The data were presented at the 58th ERA-EDTA Congress held virtually from June 5–8, 2021.

The new interim analysis data from the open-label Phase II study (NCT03832114) in patients with C3G showed twice-daily 200mg iptacopan stabilized kidney function, as measured by change in eGFR slope: a key measure of kidney clearance function that estimates the rate of blood passing through and being filtered by the kidneys.

The kidney function of 12 patients treated with iptacopan for 12 weeks was compared with their historical kidney function data for the two-year period before they entered the study (or since diagnosis was made where this was less than two years).

Furthermore, seven C3G patients who received iptacopan for up to 25 weeks in a long-term extension study (NCT03955445) showed ongoing eGFR stabilization, suggesting extended iptacopan treatment may prolong the time to, or even potentially prevent, the development of kidney failure.

Related News: https://lifepronow.com/2021/06/07/novartis-iptacopan-2/

About the study
NCT03832114 is a Phase II, open-label, two cohort, non-randomized study evaluating the efficacy, safety and pharmacokinetics of iptacopan in patients with C3 glomerulopathy (C3G) (Cohort A) and patients who have undergone kidney transplantation and have subsequent C3G recurrence in the transplanted organ (Cohort B).

The aim of the analysis presented at the 2021 ERA-EDTA Congress was to determine whether iptacopan treatment altered eGFR slope in Cohort A.

On completion of the study, all patients had the option to receive ongoing iptacopan in a long-term extension study (NCT03955445)1.

It has the potential to become the first targeted therapy to delay progression to dialysis in C3G.

About iptacopan
Iptacopan is an investigational, first-in-class, orally administered factor B inhibitor of the alternative complement pathway, targeting one of the key drivers of these diseases.

Discovered at the Novartis Institutes for BioMedical Research, iptacopan is currently in development for a number of CDRDs where significant unmet needs exist, including C3G, IgA nephropathy (IgAN), atypical hemolytic uremic syndrome (aHUS), and membranous nephropathy (MN), as well as the blood disorder PNH.

While Novartis has a 35-year history in kidney transplantation treatments, iptacopan is the first treatment in the nephrology pipeline addressing CDRDs.

Our aim is to transform treatment by targeting one of the key drivers of these rare and often progressive diseases and, in doing so, potentially extend dialysis-free life for people with CDRDs.

https://www.novartis.com/news/media-releases/novartis-announces-new-interim-analysis-phase-ii-data-iptacopan-rare-kidney-disease-c3-glomerulopathy-c3g

WHO steps up action to improve food safety and protect people from disease

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WHO steps up action to improve food safety and protect people from disease

June 07, 2021: “Every year 600 million cases of foodborne illnesses are reported. 

In 2010, 420 000 people died due to such diseases as salmonella and E.coli infection, a third of them children under five years of age.

It is estimated that this figure is increasing year after year, but it is difficult to get a clear picture of the real impact foodborne diseases are having around the world.

To address this problem the World Health Organization has developed a handbook to help countries measure their foodborne disease burden and identify food safety system needs and data gaps so they can strengthen national infrastructure and better protect people’s health.

“Food should sustain and support human health, not harm it,” said WHO Director-General Dr Tedros Adhanom Ghebreyesus.

“WHO’s new handbook will help countries to collect and analyze data to inform sustained investments in food safety.

The COVID-19 pandemic has demonstrated the intimate links between the health of humans, animals and the planet that sustains us. WHO will continue to work with partners with a One Health approach to keep communities safe from foodborne disease.”

In 2020, the World Health Assembly adopted a new resolution mandating WHO to monitor the global burden of foodborne and zoonotic diseases at national, regional and international levels and to report on the global burden of foodborne diseases with up-to-date estimates of global foodborne disease incidence, mortality and disease burden by 2025.

The Organization is reconvening its foodborne disease burden epidemiology reference group (WHO FERG) with 26 new international experts.

The group’s main functions are to advise WHO on methodologies to estimate the global burden of foodborne diseases, to monitor global food safety indicators and measure progress being made in food safety.

There are over 250 different food hazards that cause various health issues such as acute or long-term illness or even death.

In 2015, the previous FERG helped WHO publish a historic report that revealed, for the first time ever, the global public health burden of foodborne diseases based on 31 foodborne hazards. 

The report showcased the massive health impact of unsafe food and highlighted the need for strong and sustained action.

https://www.who.int/news/item/07-06-2021-who-steps-up-action-to-improve-food-safety-and-protect-people-from-disease

FDA Grants Accelerated Approval for Alzheimer’s Drug

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FDA Grants Accelerated Approval for Alzheimer’s Drug

June 07, 2021: “The U.S. FDA approved Aduhelm (aducanumab) for the treatment of Alzheimer’s, a debilitating disease affecting 6.2 million Americans.

Aduhelm was approved using the accelerated approval pathway, which can be used for a drug for a serious or life-threatening illness that provides a meaningful therapeutic advantage over existing treatments.

Accelerated approval can be based on the drug’s effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients, with a required post-approval trial to verify that the drug provides the expected clinical benefit. 

“Alzheimer’s disease is a devastating illness that can have a profound impact on the lives of people diagnosed with the disease as well as their loved ones,” said Patrizia Cavazzoni, M.D., director of the FDA’s Center for Drug Evaluation and Research.

“Currently available therapies only treat symptoms of the disease; this treatment option is the first therapy to target and affect the underlying disease process of Alzheimer’s.

As we have learned from the fight against cancer, the accelerated approval pathway can bring therapies to patients faster while spurring more research and innovation.”

Alzheimer’s is an irreversible, progressive brain disorder that slowly destroys memory and thinking skills, and eventually, the ability to carry out simple tasks.

While the specific causes of Alzheimer’s disease are not fully known, it is characterized by changes in the brain—including amyloid plaques and neurofibrillary, or tau, tangles—that result in loss of neurons and their connections.

These changes affect a person’s ability to remember and think.

Aduhelm represents a first-of-its-kind treatment approved for Alzheimer’s disease. It is the first new treatment approved for Alzheimer’s since 2003 and is the first therapy that targets the fundamental pathophysiology of the disease.

Researchers evaluated Aduhelm’s efficacy in three separate studies representing a total of 3,482 patients.

The studies consisted of double-blind, randomized, placebo-controlled dose-ranging studies in patients with Alzheimer’s disease. Patients receiving the treatment had significant dose-and time-dependent reduction of amyloid beta plaque, while patients in the control arm of the studies had no reduction of amyloid beta plaque.

These results support the accelerated approval of Aduhelm, which is based on the surrogate endpoint of reduction of amyloid beta plaque in the brain—a hallmark of Alzheimer’s disease.

Amyloid beta plaque was quantified using positron emission tomography (PET) imaging to estimate the brain levels of amyloid beta plaque in a composite of brain regions expected to be widely affected by Alzheimer’s disease pathology compared to a brain region expected to be spared of such pathology. 

The prescribing information for Aduhelm includes a warning for amyloid-related imaging abnormalities (ARIA), which most commonly presents as temporary swelling in areas of the brain that usually resolves over time and does not cause symptoms, though some people may have symptoms such as headache, confusion, dizziness, vision changes, or nausea.

Another warning for Aduhelm is for a risk of hypersensitivity reactions, including angioedema and urticaria. The most common side effects of Aduhelm were ARIA, headache, fall, diarrhea, and confusion/delirium/altered mental status/disorientation.  

Under the accelerated approval provisions, which provide patients suffering from the disease earlier access to the treatment, the FDA is requiring the company, Biogen, to conduct a new randomized, controlled clinical trial to verify the drug’s clinical benefit.

If the trial fails to verify clinical benefit, the FDA may initiate proceedings to withdraw approval of the drug.

Aduhelm was granted Fast Track designation, which seeks to expedite the development and review of drugs that are intended to treat serious conditions where initial evidence showed the potential to address an unmet medical need.”

Aduhelm is made by Biogen of Cambridge, Massachusetts.

https://www.fda.gov/news-events/press-announcements/fda-grants-accelerated-approval-alzheimers-drug

Novartis iptacopan met Phase II study primary endpoint in rare kidney disease IgAN

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Novartis iptacopan met Phase II study primary endpoint in rare kidney disease IgAN

June 06, 2021: “Novartis announced Phase II primary endpoint data showing investigational iptacopan (LNP023) – a first-in-class, oral, targeted factor B inhibitor – reduced protein in the urine (proteinuria), an increasingly recognized surrogate marker correlating with progression to kidney failure, and showed promise in stabilizing kidney function in patients with IgA nephropathy (IgAN).

The data were presented at the 58th ERA-EDTA Congress held virtually from June 5–8, 2021.

In the Phase II study (NCT03373461), patients (n=112) with IgAN were randomized to placebo or different doses of iptacopan.

The primary endpoint was met with a statistically significant (p=0.038) dose response effect on reduction in proteinuria (as measured by 24-hour urinary protein to creatine ratio [UPCR 24h]) with iptacopan vs. placebo, at 90 days.

At the highest dose of 200mg twice daily a 23% reduction in proteinuria was predicted, compared with placebo, at 90 days.

“IgAN is a devastating disease with no currently approved treatments.

These efficacy data, seen after 90 days of treatment, along with the safety profile, offer hope that inhibition of the alternative complement pathway with iptacopan may be an effective way to delay IgAN disease progression,” said study lead author Jonathan Barratt, Professor of Renal Medicine, University of Leicester and nephrology consultant, Leicester General Hospital.

“These data highlight the ability of iptacopan to address one of the key drivers for this disease and its potential to provide a much-needed, targeted treatment for people living with IgAN.”

Iptacopan also demonstrated a trend towards stabilizing kidney function, as assessed by estimated glomerular filtration rate (eGFR): a key measure of kidney clearance function that estimates the rate of blood passing through and being filtered by the kidneys.

Additionally, iptacopan showed a favorable safety and tolerability profile1.

“Complement-driven renal diseases, such as IgAN, are devastating and mostly affect young adults, imposing a high disease burden.

These new data in IgAN add to the growing body of evidence around the potential of iptacopan to target a key driver in these rare renal diseases,” said John Tsai, Head of Global Drug Development and Chief Medical Officer at Novartis.

“Conscious of the significant patient need for disease-modifying treatment options, we are rapidly advancing clinical development of iptacopan with the Phase III IgAN trial APPLAUSE already underway.”

Iptacopan is the most advanced asset in the company’s nephrology pipeline and targets the alternative complement pathway, a key driver of complement-driven renal diseases (CDRDs).

New data from an interim analysis of a Phase II study of iptacopan in C3 glomerulopathy (C3G) – another CDRD – will also be presented at the congress on Monday 07 June 2021 at 12:30 p.m. CEST.

Novartis has plans to initiate additional Phase III studies in other renal indications.

Iptacopan is also in development for a life-threatening blood disorder, paroxysmal nocturnal hemoglobinuria (PNH).

Based on disease prevalence and positive data from Phase II studies, iptacopan has received EMA orphan drug designation in IgAN, orphan drug designations from the FDA and EMA in C3G and PNH, FDA Breakthrough Therapy Designation in PNH, and EMA PRIME designation for C3G.

About the studies

NCT03373461 is a Phase II randomized, double-blind, placebo-controlled, dose-ranging, parallel-group adaptive design study to investigate the efficacy and safety of iptacopan in primary IgAN.

It is the first study to report the efficacy and safety of selective inhibition of the alternative complement pathway in IgAN.

The primary endpoint, and the primary aim of the interim analysis presented at the 2021 ERA-EDTA Congress, was to evaluate the dose response effect of iptacopan versus placebo on the reduction in urinary protein to creatinine ratio (UPCR 24h) at 90 days of treatment.

Secondary endpoints include safety and tolerability of iptacopan, eGFR, and biomarkers reflecting activity of the alternative complement pathway.

About iptacopan
Iptacopan is an investigational, first-in-class, orally administered factor B inhibitor of the alternative complement pathway, targeting one of the key drivers of these diseases.

It has the potential to become the first targeted therapy to delay progression to dialysis in IgAN.

Discovered at the Novartis Institutes for BioMedical Research, iptacopan is currently in development for a number of CDRDs where significant unmet needs exist, including IgAN, C3G, atypical hemolytic uremic syndrome (aHUS), and membranous nephropathy (MN), as well as the blood disorder PNH.

While Novartis has a 35-year history in kidney transplantation treatments, iptacopan is the first treatment in the nephrology pipeline addressing CDRDs.

Our aim is to transform treatment by targeting one of the key drivers of these rare and often progressive diseases and, in doing so, potentially extend dialysis-free life for people with CDRDs.”

https://www.novartis.com/news/media-releases/novartis-announces-iptacopan-met-phase-ii-study-primary-endpoint-rare-kidney-disease-iga-nephropathy-igan

FDA Approves First Treatment for Patients with Plasminogen Deficiency

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FDA Approves First Treatment for Patients with Plasminogen Deficiency

June 04, 2021: “The U.S. FDA approved Ryplazim (plasminogen, human-tmvh) for the treatment of patients with plasminogen deficiency type 1, also referred to as hypoplasminogenemia, a disorder that can impair normal tissue and organ function and may lead to blindness.

“Until now, there were no FDA-approved treatment options for patients with plasminogen deficiency type 1,” said Peter Marks, M.D., Ph.D., director of FDA’s Center for Biologics Evaluation and Research.

“Today’s approval helps address an unmet medical need for individuals affected by this rare genetic disease.”

Individuals with this disease lack a protein called plasminogen, which is responsible for the ability of the body to break down fibrin clots.

Plasminogen deficiency leads to an accumulation of fibrin, causing the development of growths (lesions) that can impair normal tissue and organ function and may lead to blindness when these lesions affect the eyes. 

The active ingredient in Ryplazim is plasminogen, purified from human plasma. Treatment with Ryplazim helps to increase the plasma level of plasminogen – enabling a temporary correction of the plasminogen deficiency and reduction or resolution of the lesions.

The effectiveness and safety of Ryplazim is primarily based on one single-arm, open-label (unblinded) clinical trial enrolling 15 adult and pediatric patients with plasminogen deficiency type 1.

All patients received Ryplazim administered every two to four days for 48 weeks.

The effectiveness of Ryplazim was demonstrated by at least 50% improvement of their lesions in all 11 patients who had lesions at baseline, and absence of recurrent or new lesions in any of the 15 patients through the 48 weeks of treatment.

The most common side effects reported by patients who received Ryplazim were abdominal pain, bloating, nausea, bleeding, limb pain, fatigue, constipation, dry mouth, headache, dizziness, joint pain, and back pain.

The FDA granted Ryplazim Orphan Drug designation, which provides incentives to assist and encourage drug development for rare diseases.

The application also received Fast Track designation, Priority Review, and a Rare Pediatric Disease Priority Review Voucher.

The FDA’s rare pediatric disease priority review voucher program is intended to encourage development of new drugs and biologics to prevent and/or treat rare diseases in children. 

Patients with plasminogen deficiency type 1 may bleed from active disease-related lesions. The use of Ryplazim may prolong or worsen active bleeding.

Tissue sloughing (peeling/shedding) has also been observed. Because Ryplazim is derived from human plasma, it carries a risk of transmitting infectious agents.

Based on effective donor screening procedures and product manufacturing processes, the risk of infectious disease transmission is remote.

The FDA granted approval to ProMetic Biotherapeutics Inc.”

https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-patients-plasminogen-deficiency-rare-genetic-disorder

Novartis tislelizumab met primary endpoint for esophageal cancer

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Novartis tislelizumab met primary endpoint for esophageal cancer

Jun 04, 2021: Novartis announced results from the pivotal Phase III RATIONALE 302 trial showing the investigational anti-PD-1 immune checkpoint inhibitor tislelizumab improved overall survival (OS) versus chemotherapy (median 8.6 months vs. 6.3 months, p=0.0001).

The study evaluated tislelizumab in patients with unresectable recurrent locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) who had received prior systemic therapy.

Results were presented at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting.

Results from RATIONALE 302 in ESCC showed tislelizumab extended median OS by 2.3 months compared to chemotherapy with a 30% reduction in the risk of death (HR=0.70, 95% CI: 0.57-0.85, p=0.0001).

In PD-L1 positive patients, tislelizumab extended median OS by 3.5 months with a 46% reduction in the risk of death (HR=0.54, 95% CI: 0.36-0.79, p=0.0006).1

“These data show that tislelizumab has the potential to help patients with esophageal squamous cell carcinoma – one of the deadliest types of cancers – live longer,” said Jeff Legos, Ph.D., MBA, Senior Vice President and Head of Oncology Drug Development.

“We are excited about these results from the newest asset in our portfolio of transformational medicines and look forward to sharing these data with regulatory authorities, as we continue to explore the full potential of this uniquely designed anti-PD-1 antibody.”

Treatment with tislelizumab demonstrated median progression-free survival (PFS) of 1.6 months compared to 2.1 months (HR=0.83, 95% CI: 0.67–1.01).

Tislelizumab demonstrated a higher and more durable anti-tumor activity than chemotherapy (objective response rate [ORR], 20.3% vs. 9.8%; median duration of response [DoR], 7.1 months vs. 4.0 months).

The discontinuation rate due to treatment-related adverse events (TRAEs) was lower in patients who received tislelizumab (6.7%) compared to chemotherapy (13.8%).

The most common all-grade TRAEs (≥10%) with tislelizumab were increased aspartate aminotransferase (11.4%), anemia (11%) and hypothyroidism (10.2%). No new safety signals were identified.

“Most patients with this type of esophageal cancer are diagnosed with advanced disease, resulting in a poor prognosis for this difficult-to-treat cancer,” said Jaffer Ajani, M.D., professor of Gastrointestinal Medical Oncology at The University of Texas MD Anderson Cancer Center.

“The impact tislelizumab had on survival compared to chemotherapy in this study is highly meaningful and encouraging news for patients, caregivers and treating oncologists.”

Esophageal squamous cell carcinoma is the most common type of esophageal cancer globally and the sixth leading cause of cancer-related death worldwide.

Each year, esophageal cancer claims nearly as many lives as colon cancer across the globe.

More than two-thirds of patients with ESCC have advanced or metastatic disease at the time of diagnosis.4 The average five-year survival rate is only five percent.5

RATIONALE 302 is a randomized, global Phase III study assessing tislelizumab versus chemotherapy in patients with advanced unresectable/metastatic ESCC after prior systemic therapy. The primary endpoint is OS in the intent-to-treat (ITT) population.

The key secondary endpoint is OS in PD-L1 positive patients (vCPS ≥10%). Additional secondary endpoints included PFS, ORR, DoR and safety endpoints.

Data on tislelizumab in MSI-H cancers presented
The RATIONALE 209 study reported that tislelizumab showed durable anti-tumor activity in patients with previously treated, locally advanced, unresectable or metastatic microsatellite instability-high (MSI-H) and mismatch repair deficient (dMMR) cancers, which are known to be more responsive to immune checkpoint modulation.

Treatment with tislelizumab demonstrated an ORR of 45.9% among all tumor types, including four complete responses (CR) and 30 partial responses (PR). No disease progression was reported in the 34 responders (CR + PR), with a 12-month DoR rate of 100%).

Five percent of patients treated with tislelizumab discontinued treatment due to TRAEs, and no new safety signals were identified. Grade ≥3 TRAEs occurred in 42.5% of patients.

Related News: Novartis expands Oncology pipeline with in-licensing of tislelizumab from BeiGene
Novartis to unveil new data at ASCO and EHA from its robust portfolio

MSI-H cancer cells have a defect in the ability to correct mistakes that occur when DNA is copied, leading to mutations that contribute to cancerous growth.

Many types of cancer may have a high level of microsatellite instability, but it is seen most often in CRC, gastric cancer and endometrial cancer.

RATIONALE 209 is a single-arm, open-label Phase II study evaluating the efficacy and safety of tislelizumab monotherapy in adult patients with previously treated, locally advanced, unresectable or metastatic histologically confirmed MSI-H/dMMR solid tumors.

Radiological imaging was performed at nine weeks, then every six weeks for the first year of therapy and every 12 weeks thereafter.

The primary endpoint was IRC-assessed ORR. Secondary endpoints included time to response, DoR, disease control rate, PFS, OS and safety.”

https://www.novartis.com/news/media-releases/novartis-investigational-checkpoint-inhibitor-tislelizumab-met-primary-endpoint-overall-survival-pivotal-phase-iii-trial-esophageal-cancer-after-systemic

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