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WHO: People living longer and healthier lives but COVID-19 threatens to throw progress off track

May 13, 2020: “All over the world, the COVID-19 pandemic is causing significant loss of life, disrupting livelihoods, and threatening the recent advances in health and progress towards global development goals highlighted in the 2020 World Health Statistics published by the World Health Organization (WHO) today.

“The good news is that people around the world are living longer and healthier lives.

The bad news is the rate of progress is too slow to meet the Sustainable Development Goals and will be further thrown off track by COVID-19,” said Dr Tedros Adhanom Ghebreyesus, WHO Director-General.

“The pandemic highlights the urgent need for all countries to invest in strong health systems and primary health care, as the best defense against outbreaks like COVID-19, and against the many other health threats that people around the world face every day. Health systems and health security are two sides of the same coin.”

WHO’s World Health Statistics — an annual check-up on the world’s health — reports progress against a series of key health and health service indicators, revealing some important lessons in terms of progress made towards the Sustainable Development Goals and gaps to fill.

Life expectancy and healthy life expectancy have increased, but unequally.

The biggest gains were reported in low-income countries, which saw life expectancy rise 21% or 11 years between 2000 and 2016 (compared with an increase of 4% or 3 years in higher income countries).

One driver of progress in lower-income countries was improved access to services to prevent and treat HIV, malaria and tuberculosis, as well as a number of neglected tropical diseases such as guinea worm.

Another was better maternal and child healthcare, which led to a halving of child mortality between 2000 and 2018.

But in a number of areas, progress has been stalling. Immunization coverage has barely increased in recent years, and there are fears that malaria gains may be reversed.

And there is an overall shortage of services within and outside the health system to prevent and treat noncommunicable diseases (NCDs) such as cancer, diabetes, heart and lung disease, and stroke. In 2016, 71 per cent of all deaths worldwide were attributable to NCDs, with the majority of the 15 million premature deaths (85%) occurring in low and middle-income countries.

This uneven progress broadly mirrors inequalities in access to quality health services. Only between one third and one half the world’s population was able to obtain essential health services in 2017.

Service coverage in low- and middle-income countries remains well below coverage in wealthier ones; as do health workforce densities. In more than 40% of all countries, there are fewer than 10 medical doctors per 10 000 people. Over 55% of countries have fewer than 40 nursing and midwifery personnel per 10 000 people. 

The inability to pay for healthcare is another major challenge for many. On current trends, WHO estimates that this year, 2020, approximately 1 billion people (almost 13 per cent of the global population) will be spending at least 10% of their household budgets on health care.

The majority of these people live in lower middle-income countries.

“The COVID-19 pandemic highlights the need to protect people from health emergencies, as well as to promote universal health coverage and healthier populations to keep people from needing health services through multisecotral interventions like improving basic hygiene and sanitation,” said Dr Samira Asma, Assistant Director General at WHO.

In 2017, more than half (55%) of the global population was estimated to lack access to safely-managed sanitation services, and more than one quarter (29%) lacked safely-managed drinking water. In the same year, two in five households globally (40%) lacked basic handwashing facilities with soap and water in their home. 

The World Health Statistics also highlight the need for stronger data and health information systems.

Uneven capacities to collect and use accurate, timely, and comparable health statistics, undermining countries’ ability to understand population health trends, develop appropriate policies, allocate resources and prioritize interventions. 

For almost a fifth of countries, over half of the key indicators have no recent primary or direct underlying data, another major challenge in enabling countries to prepare for, prevent and respond to health emergencies such as the ongoing COVID-19 pandemic.

WHO is therefore supporting countries in strengthening surveillance and data and health information systems so they can measure their status and manage improvements.” 

“The message from this report is clear: as the world battles the most serious pandemic in 100 years, just a decade away from the SDG deadline, we must act together to strengthen primary health care and focus on the most vulnerable among us in order to eliminate the gross inequalities that dictate who lives a long, healthy life and who doesn’t,” added Asma. “We will only succeed in doing this by helping countries to improve their data and health information systems.” 

https://www.who.int/news-room/detail/13-05-2020-people-living-longer-and-healthier-lives-but-covid-19-threatens-to-throw-progress-off-track

FDA Acknowledges Receipt of Bronchitol NDA Resubmission – Decision Review Date 1 November 2020

May 13, 2020: ” Pharmaceutical research company Pharmaxis Ltd announced its US licensee Chiesi Farmaceutici S.p.A. (Chiesi) has received acknowledgement of receipt of the Bronchitol NDA resubmission from the US Food and Drug Administration (FDA).

The receipt of this communication from the FDA indicates that the submission responds to all issues raised in the complete response letter received in June 2019.

However, the FDA have classified the resubmission as Class 2 which changes the FDA review period from an expected two months to six months and sets a Goal Action Date of 1 November 2020.

Pharmaxis CEO Gary Phillips said, “While I am pleased that the FDA has confirmed Chiesi’s submission to be complete, I am disappointed the FDA has exercised its discretion to set a six month review timetable.

The US$10 million launch milestone payable by Chiesi subsequent to an approval of Bronchitol by the FDA therefore moves from the fourth quarter of 2020 to the first quarter of 2021.” Chiesi Group is responsible for the regulatory approval and commercialisation of Bronchitol in the United States.

If Bronchitol is approved by the FDA, Pharmaxis will receive the US$10 million milestone payment on the supply of Bronchitol for the US commercial launch and additional mid to high teen percentage royalties on in‐market net sales.

Pharmaxis will manufacture and be the exclusive supplier of Bronchitol for the US market.

Bronchitol is approved and marketed in Europe, Russia, Australia and several other countries.”

http://www.pharmaxis.com.au/assets/Documents/pdf/02020/ASX/2020-05-13-Bronchitol-FDA-Goal-Date.pdf

Coronavirus (COVID-19) Update: Daily Roundup May 12, 2020

May 12, 2020: “The U.S. Food and Drug Administration today announced the following actions taken in its ongoing response effort to the COVID-19 pandemic:
  • The FDA took important actions to help accelerate the development of prevention and treatment options for COVID-19 by providing new guidance with recommendations for innovators and researchers conducting work in this area.

    These guidance documents aim to make the process for submitting applications to initiate studies for new drugs and biological products more efficient by outlining recommendations for ways to design clinical trials to evaluate safety and effectiveness of these medical products for COVID-19.
  • The FDA provided an update on surveillance inspections during the COVID-19 pandemic.

    In the update, the agency stated it will continue to utilize and implement additional alternative inspection tools and approaches while postponing domestic and foreign routine surveillance inspections.

    This will continue as local, national and international conditions warrant, with the exception of certain mission-critical inspections. 
  • The FDA issued a warning letter to one company that was selling fraudulent COVID-19 products, as part of the agency’s effort to protect consumers.

    The seller warned, Fusion Health and Vitality LLC, recently offered products, including “CORE” and “IMMUNE SHOT,” for sale in the U.S. with claims that misleadingly represented the products as safe and/or effective for the prevention and treatment of COVID-19.

    There are currently no FDA-approved products to prevent or treat COVID-19. Consumers concerned about COVID-19 should consult with their health care provider.
  • To date, the FDA has posted 48 COVID-19 related warning letters.
  • The FDA posted updated information for blood establishments regarding COVID-19, which aligns with the agency’s guidance on convalescent plasma to allow donation 14 days after complete resolution of symptoms.

    The posting also provides updated information blood establishments may wish to consider when evaluating prospective blood donors.
  • Yesterday, the FDA issued an Emergency Use Authorization (EUA) for the Ascom teleCARE IP Nurse Call System for use by healthcare providers and patients in healthcare environments, including temporary hospital facilities, to facilitate remote communication between patients and healthcare providers.

    The remote communication and monitoring capabilities of the teleCARE IP Nurse Call System may reduce the amount of contact by healthcare providers with patients who are in isolation rooms, thereby reducing healthcare provider risk of exposure to SARS-CoV-2, the virus that causes COVID-19.
  • Yesterday, FDA issued a EUA for the emergency use of the Eko electrocardiogram (ECG) Low Ejection Fraction Tool (“ELEFT”) to be used by healthcare professionals to provide an assessment of Left Ventricular Ejection Fraction as a diagnostic aid to screen for potential cardiac complications associated with COVID-19 or underlying cardiac conditions that may affect clinical management of COVID-19, in adult patients having or suspected of having COVID-19.

    The EUA was issued to Manatt, Phelps & Philips.
  • Testing updates to date:
    • During the COVID-19 pandemic, the FDA has worked with more than 500 test developers who have said they will be submitting EUA requests to the FDA for tests that detect the virus.
    • To date, the FDA has authorized 93 tests under EUAs, which includes 12 antibody tests and 1 antigen test.

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

The agency also is responsible for the safety and security 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/coronavirus-covid-19-update-daily-roundup-may-12-2020

New data from 18 approved and investigational pfizer medicines to be showcased at asco20 virtual scientific program

May 12, 2020: “Pfizer announced that new data from clinical trials of 18 approved and investigational medicines will be presented virtually at the ASCO20 Virtual Scientific Program, from May 29-May 31.

The data that will be presented build on Pfizer’s strong track record in oncology by providing new insights in areas like breast, colorectal and genitourinary cancers, which include bladder, prostate, and kidney cancer.

Data from Pfizer’s early stage pipeline, including a novel anti-HER2 antibody-drug conjugate, will also be presented as Pfizer aims to transform the cancer treatment landscape well into the future.

“Our data presentations will highlight the depth and breadth of our cancer portfolio, including our current medicines and new generation of potential therapies,” said Chris Boshoff, M.D., Ph.D., Chief Development Officer, Oncology, Pfizer Global Product Development.

“We are particularly excited to share the first presentation of detailed overall survival results from the JAVELIN Bladder 100 trial of BAVENCIO and the final overall survival data from the PROSPER trial of XTANDI.

These data will support our rapidly expanding efforts in bladder cancer and add to the growing body of clinical evidence generated with XTANDI in prostate cancer.”

New data will be featured in nine oral presentations, including a Plenary Session presentation of data from the JAVELIN Bladder 100 trial evaluating BAVENCIO® (avelumab) as a first-line maintenance treatment for locally advanced or metastatic urothelial carcinoma (UC).

Additional data provide insights on Pfizer’s medicines, including IBRANCE® (palbociclib), BRAFTOVI® (encorafenib), XTANDI® (enzalutamide) and lorlatinib, as well as its cutting-edge, investigational compounds, including a HER2-targeted antibody-drug conjugate in patients with solid tumors.

BAVENCIO is being developed and commercialized in collaboration with Merck KGaA, Darmstadt, Germany. As part of a global agreement, Pfizer and Astellas jointly develop and commercialize XTANDI.

To help interested non-scientists better understand the latest research, Pfizer has also developed summaries in non-technical language for results of company-sponsored studies being presented in the ASCO20 Virtual Scientific Program called “abstract plain language summaries (APLS).”

https://www.pfizer.com/news/press-release/press-release-detail/new_data_from_18_approved_and_investigational_pfizer_medicines_to_be_showcased_at_asco20_virtual_scientific_program

Pfizer invites the public to listen to the webcast of Pfizer discussion at healthcare conference

May 12, 2020: “Pfizer Inc. invites investors and the general public to listen to a webcast of a discussion with Charles Triano, Senior Vice President, Investor Relations, at the RBC Capital Markets 2020 Global Healthcare Conference on Tuesday, May 19, 2020, at 11:30 a.m. EDT.

To listen to the webcast, visit our web site at www.pfizer.com/investors. Information on accessing and pre-registering for the webcast will be available at www.pfizer.com/investors beginning today.

About Pfizer: Breakthroughs That Change Patients’ Lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines.

Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time.

Consistent with our responsibility as one of the world’s premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world.

For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.Pfizer.com.

Disclosure Notice: 

The webcast may include forward-looking statements about, among other things, our anticipated future operating and financial performance, business plans and prospects, expectations for our product pipeline, in-line products and product candidates, strategic reviews, capital allocation objectives, plans for and prospects of our acquisitions and other business-development activities, including our proposed transaction with Mylan N.V. (Mylan) to combine Upjohn and Mylan to create a new global pharmaceutical company, Viatris, our acquisition of Array BioPharma Inc. and our transaction with GSK which combined our respective consumer healthcare businesses into a new consumer healthcare joint venture, benefits anticipated from the reorganization of our commercial operations in 2019, our ability to successfully capitalize on growth opportunities or prospects, manufacturing and product supply, our efforts to respond to COVID-19, our expectations regarding the impact of COVID-19 on our business, and plans relating to share repurchases and dividends, among other things, that are subject to substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements.

A description of these risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2019 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors” and “Forward-Looking Information and Factors That May Affect Future Results”, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at www.sec.gov and www.pfizer.com.

The forward-looking statements in the webcast speak only as of the original date of the webcast. Pfizer assumes no obligation to update forward-looking statements contained in the webcast as the result of new information or future events or developments.

ADDITIONAL INFORMATION

This communication shall not constitute an offer to sell or the solicitation of an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such jurisdiction.

No offer of securities shall be made except by means of a prospectus meeting the requirements of Section 10 of the Securities Act of 1933, as amended. In connection with the proposed combination of Upjohn Inc. (“Newco”), a wholly owned subsidiary of Pfizer Inc. (“Pfizer”) and Mylan N.V. (“Mylan”), which will immediately follow the proposed separation of the Upjohn business (the “Upjohn Business”) from Pfizer (the “proposed transaction”), Newco and Mylan have filed certain materials with the SEC, including, among other materials, the Form S-4, Form 10 and Prospectus filed by Newco and the Proxy Statement filed by Mylan.

The Form S-4 was declared effective on February 13, 2020 and the Proxy Statement and the Prospectus were first mailed to shareholders of Mylan on or about February 14, 2020 to seek approval of the proposed transaction.

Form 10 has not yet become effective. After the Form 10 is effective, a definitive information statement will be made available to the Pfizer stockholders relating to the proposed transaction.

Newco and Mylan intend to file additional relevant materials with the SEC in connection with the proposed transaction. INVESTORS AND SECURITY HOLDERS ARE URGED TO READ DOCUMENTS FILED WITH THE SEC CAREFULLY AND IN THEIR ENTIRETY BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION ABOUT MYLAN, NEWCO AND THE PROPOSED TRANSACTION.

The documents relating to the proposed transaction (when they are available) can be obtained free of charge from the SEC’s website at www.sec.gov.

These documents (when they are available) can also be obtained free of charge from Mylan, upon written request to Mylan or by contacting Mylan at (724) 514-1813 or [email protected] or from Pfizer on Pfizer’s internet website at https://investors.Pfizer.com/financials/sec-filings/default.aspx or by contacting Pfizer’s Investor Relations Department at (212) 733-2323, as applicable.

PARTICIPANTS IN THE SOLICITATION

This communication is not a solicitation of a proxy from any investor or security holder. However, Pfizer, Mylan, Newco and certain of their respective directors and executive officers may be deemed to be participants in the solicitation of proxies in connection with the proposed transaction under the rules of the SEC.

Information about the directors and executive officers of Pfizer may be found in its Annual Report on Form 10-K filed with the SEC on February 27, 2020, and its definitive proxy statement relating to its 2020 Annual Meeting filed with the SEC on March 13, 2020, as supplemented by its supplement to proxy statement filed with the SEC on April 7, 2020.

Information about the directors and executive officers of Mylan may be found in its Annual Report on Form 10-K filed with the SEC on February 28, 2020, and its definitive proxy statement relating to its 2019 Annual Meeting filed with the SEC on May 24, 2019.

Additional information regarding the interests of these participants can also be found in the Form S-4, the Proxy Statement and the Prospectus.

These documents can be obtained free of charge from the sources indicated above.

https://www.pfizer.com/news/press-release/press-release-detail/pfizer_invites_public_to_listen_to_webcast_of_pfizer_discussion_at_healthcare_conference-27

Coronavirus (COVID-19) Update: Daily Roundup May 11, 2020

May 11, 2020: The U.S. Food and Drug Administration today announced the following actions taken in its ongoing response effort to the COVID-19 pandemic:
  • The FDA issued an Emergency Use Authorization (EUA) for emergency use of the Fresenius Propoven 2% Emulsion to maintain sedation via continuous infusion in patients older than 16 who require mechanical ventilation in an ICU during the COVID-19 public health emergency.

    Fresenius Propoven 2% Emulsion has important differences in its formulation compared to FDA-approved propofol drugs; providers should consult the Health Care Provider Fact Sheet for more information before administering it.

  • The virus that causes COVID-19 has led to an increased number of people with severe respiratory illness. As a result, there is a shortage of FDA-approved drugs such as propofol that are used for sedation of mechanically ventilated patients.
  • The FDA issued warning letters to two companies for selling fraudulent COVID-19 products, as part of the agency’s effort to protect consumers.

    There are currently no FDA-approved products to prevent or treat COVID-19. Consumers concerned about COVID-19 should consult with their health care provider.
    • The first seller warned in a letter issued jointly with The Federal Trade Commission (FTC) , Seanjari Preeti Womb Healing, L.L.C., offers a honey product for sale in the U.S. with misleading claims that the products are safe and/or effective for the prevention and treatment of COVID-19.
    • The second seller warned, Plum Dragon Herbs, Inc., offers traditional Chinese medicine products for sale in the U.S. with misleading claims that the products are safe and/or effective for the prevention of COVID-19. 
    • To date, the FDA has posted 46 COVID-19 related warning letters.
  • Today, the FDA added content to the question-and-answer appendix in its guidance titled “Conduct of Clinical Trials of Medical Products during COVID-19 Public Health Emergency.”

    The updated guidance includes new content with considerations for using alternate laboratories or imaging centers, holding trial participant visits via video conference, and conducting required postmarketing clinical trials.

    The guidance also includes updated information about managing protocol deviations and amendments to ongoing trials, and about consulting with the FDA regarding administering investigational product infusions at home rather than at the clinical trial site.
  • The FDA approved two Abbreviated New Drug Applications:
    • Cisatracurium besylate injection USP 20 mg/10 mL is indicated to facilitate tracheal intubation and to provide skeletal muscle relaxation during surgery or mechanical ventilation. Side effects of cisatracurium include bradycardia, hypotension, flushing, bronchospasm, and rash.
    • Azithromycin Tablets USP, 600 mg, is indicated for mild to moderate infections caused by designated, susceptible bacteria that cause certain sexually transmitted diseases and mycobacterial infections.

      Side effects of azithromycin tablets include hypersensitivity, QT prolongation, diarrhea, nausea, abdominal pain, and vomiting.
    • Both ANDAs are relevant to COVID-19, and both medicines are listed in the FDA Drug Shortage Database.

      FDA recognizes the increased demand for certain products during the novel coronavirus pandemic and we remain deeply committed to facilitating access to medical products to help address critical needs of the American public.
  • Diagnostics update to date:
    • On Friday, the FDA issued the first EUA for a COVID-19 antigen test, a new category of tests for use in the ongoing pandemic.

      These diagnostic tests quickly detect fragments of proteins found on or within the virus by testing samples collected from the nasal cavity using swabs.

      The EUA was issued to Quidel Corporation for the Sofia 2 SARS Antigen FIA test.
    • Today, the FDA updated the Policy for Coronavirus Disease-2019 Tests During the Public Health Emergency.

      The update includes EUA submission templates for molecular, antigen, and serology tests.

      Developers may choose to use these templates to facilitate the preparation, submission, and authorization of a EUA for various types of COVID-19 tests.
    • During the COVID-19 pandemic, the FDA has worked with more than 385 test developers who have said they will be submitting EUA requests to the FDA for tests that detect the virus.
    • To date, the FDA has issued 92 EUAs, which includes 12 antibody tests and 1 antigen test.
    • The FDA has been notified that more than 245 laboratories have begun testing under the policies set forth in our COVID-19 Policy for Diagnostic Tests for Coronavirus Disease-2019 during the Public Health Emergency Guidance.
    • The FDA also continues to keep its COVID-19 Diagnostics FAQ up to date.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security 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/coronavirus-covid-19-update-daily-roundup-may-11-2020

Coronavirus (COVID-19) Update: FDA updates on surveillance inspections during COVID-19

May 11, 2020: “During COVID-19, the U.S. Food and Drug Administration will continue to utilize and implement additional alternative inspection tools and approaches while postponing domestic and foreign routine surveillance inspections.

This will continue as local, national and international conditions warrant, with the exception of certain mission critical inspections.

Mission critical inspections are identified on a case-by-case basis and conducted with appropriate safety measures in place.

In this unprecedented time, we are working hard to help ensure the safety and integrity of the products we all need, while striking an appropriate balance between a scaled back surveillance inspection program and the continuation of providing robust regulatory oversight.

While this pandemic has added new complexities to our normal operations, we have implemented alternative approaches to on-site surveillance inspections that provide us with useful information toward fulfilling the agency’s mission.

The FDA is collaborating with the CDC to develop a process that would govern how and where to return to on-site facility surveillance inspections in accordance with the gating criteria outlined in the White House Guidelines for Opening Up America Again.

We expect this to be a phased approach driven by scientific data. Our priority and commitment are to first protect the health and well-being of not only our own highly skilled workforce and state contract inspectors, but also the health of workers in the important industries we regulate.

Although inspections are critical, they are one part of a robust and multi-pronged approach to overseeing the safety and quality of FDA regulated products.

Safety and quality must be part of the daily routine at any regulated facility for their products to be high quality and reliably suitable for the U.S. consumer.

Based on decades of experience with our diverse regulated industries, we believe most FDA-regulated firms understand and appreciate their responsibility to ensure the safety of the products they manufacture or produce.

We consider these firms to be our partners in public health. The FDA’s regulatory oversight of crucial industry sectors is vital to the long-term health of America, but product safety and quality are ultimately the establishment’s responsibility.

Most firms strive to reliably provide quality products and maintain the integrity of the supply chain.

We are in steady communication with our industry partners as well as our domestic and foreign regulatory counterparts to assist them during this difficult time.

Our leadership team meets daily to discuss the urgent issues facing us as we facilitate efforts to diagnose, treat and prevent the disease; survey the medical and food product supply chains for potential shortages or disruptions and help to mitigate such impacts, as necessary; and leverage the full breadth of our public health tools.

We continue to closely monitor the global situation and remain in contact with our domestic and foreign regulatory counterparts to inform our assessment of the feasibility of a return to routine on-site surveillance inspections as conditions improve.

The FDA is confident in our ability to maintain oversight during this pandemic by leveraging all available authorities, alternative tools, and scientific methods to ensure the integrity and availability of safe and quality products for the American people.

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

The agency also is responsible for the safety and security 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/coronavirus-covid-19-update-fda-updates-surveillance-inspections-during-covid-19

Coronavirus (COVID-19) Update: FDA Takes New Actions to Accelerate Development of Novel Prevention, Treatment Options for COVID-19

May 11, 2020: “The U.S. Food and Drug Administration took important actions to help accelerate the development of prevention and treatment options for COVID-19 by providing new guidance with recommendations for innovators and researchers conducting work in this area.

These guidance documents aim to make the process for submitting applications to initiate studies for new drugs and biological products more efficient and outline recommendations for ways to design clinical trials to evaluate safety and effectiveness of these medical products for COVID-19.

“Our staff continues to work around the clock with the world’s best innovators and researchers to help expedite the development of prevention and treatment options for COVID-19,” said FDA Commissioner Stephen M. Hahn, M.D. “Accelerating the investigation of safe and effective therapies that could benefit people affected by the COVID-19 pandemic is one of the FDA’s highest priorities.

We are committed to maximizing our regulatory flexibility and using every tool at our disposal to speed the development and availability of these medical products and believe these new guidances will help innovators and researchers do just that.”

Quickly after the emergence of this virus, the FDA began working directly with federal health partners, academia and industry to advance medical countermeasures against COVID-19.

Given the urgent nature of the pandemic, the FDA launched a new program called the Coronavirus Treatment Acceleration Program to help move new medical products to patients as soon as possible, while at the same time determine whether they are effective and if the benefits outweigh the risks.

So far, more than 130 clinical trials of potential COVID-19 related drugs and biological products are underway with FDA oversight and additional development programs for other agents are in the planning stages. T

hese include studies on therapies such as antiviral drugs to keep viruses from multiplying, as well as therapies called immunomodulators aimed at tamping down the body’s own immune reaction to the virus, in cases where the body starts attacking the patient’s own organs.

Studies like these, which are conducted by the pharmaceutical industry and academic researchers, assess the products’ toxicity and help to find out if they work against COVID-19.

The FDA has started to receive data from clinical studies and expects to receive more data regarding investigational products soon.

The agency intends to engage with pharmaceutical sponsors and other government partners to facilitate patient access as soon as possible when favorable results are observed. 

The guidances issued today build on steps the agency has taken to streamline its review and advice process to get studies started as quickly as possible, and they incorporate information provided to individual innovators or researchers over the past several months.

The first guidance, “COVID-19 Public Health Emergency: General Considerations for Pre-IND (Investigational New Drug application) Meeting Requests for COVID-19 Related Drugs and Biological Products,” outlines a more efficient process for developers to receive agency feedback on their supporting data with the goal of starting clinical trials as soon as possible.

The FDA is committed to helping sponsors get potentially effective products into study quickly, while protecting the safety of patients.

To that end, the guidance provides sponsors clarity on the types of data and information they should provide to address clinical, nonclinical and quality considerations before submitting an application to initiate studies.

The second guidance, “COVID-19: Developing Drugs and Biological Products for Treatment or Prevention,” provides the FDA’s current recommendations on later stage clinical trials intended to establish safety and effectiveness for COVID-19 products.

The guidance outlines critical sponsor considerations such as appropriate patient selection, including the evaluation of therapies in patients at high risk of complications from COVID-19 (e.g., the elderly).

In addition, the guidance helps sponsors to understand how to design their trials, including considerations of study duration, assessment of efficacy and monitoring for safety.

The FDA anticipates this guidance will help sponsors to efficiently design studies that may lead to the review and potential approval of safe and effective drugs and biological products to address the COVID-19 pandemic.

These guidance documents are supplemented by a variety of ongoing activities and broad agency guidance to support innovative study designs, including master protocols, to test multiple products and multiple populations simultaneously and get answers on safety and efficacy as quickly as possible.

In addition to the FDA’s work to expedite preventative and therapeutic options, the agency is engaged in a public-private partnership with the National Institutes of Health and others to speed the development of COVID-19 vaccine and treatment options.

The Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) partnership, which also includes other U.S. and European government partners and more than a dozen biopharmaceutical companies, will develop a collaborative framework for prioritizing vaccine and drug candidates, streamlining clinical trials, coordinating regulatory processes and/or leveraging assets among all partners to rapidly respond to COVID-19 and future pandemics.

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

The agency also is responsible for the safety and security 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/coronavirus-covid-19-update-fda-takes-new-actions-accelerate-development-novel-prevention-treatment

Sarclisa® (isatuximab) Phase 3 IKEMA trial meets primary endpoint early in patients with relapsed multiple myeloma

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May 12, 2020: “The Phase 3 IKEMA clinical trial evaluating Sarclisa® (isatuximab) added to carfilzomib and dexamethasone met the primary endpoint at its first planned interim analysis, demonstrating significantly prolonged progression-free survival compared to standard of care carfilzomib and dexamethasone alone in patients with relapsed multiple myeloma.
There were no new safety signals identified in this study.
“When Sarclisa was added to standard-of-care treatment carfilzomib and dexamethasone in this phase 3 trial, results clearly demonstrated a significant reduction in risk of disease progression or death,” said John Reed, M.D., Ph.D., Global Head of Research and Development at Sanofi. 
“This is the second positive phase 3 trial for Sarclisa, further supporting the potential our medicine has to improve outcomes for patients struggling with relapsed multiple myeloma.”
Results will be submitted to an upcoming medical meeting and are anticipated to form the basis of regulatory submissions planned for later this year.
The randomized, multi-center, open label Phase 3 IKEMA clinical trial enrolled 302 patients with relapsed multiple myeloma across 69 centers spanning 16 countries.
All study participants received one to three prior anti-myeloma therapies. During the trial, Sarclisa was administered through an intravenous infusion at a dose of 10mg/kg once weekly for four weeks, then every other week for 28-day cycles in combination with carfilzomib twice weekly at the 20/56mg/m2 dose and dexamethasone at the standard dose for the duration of treatment. The primary endpoint of IKEMA is progression-free survival.
Secondary endpoints include overall response rate, the rate of very good partial response or greater, minimal residual disease, complete response rate, overall survival and safety.
The use of Sarclisa in combination with carfilzomib and dexamethasone in relapsed multiple myeloma is investigational and has not been fully evaluated by any regulatory authority.
Sarclisa is a monoclonal antibody that binds to a specific epitope on the CD38 receptor on multiple myeloma cells.
It is designed to work through many mechanisms of action including programmed tumor cell death (apoptosis) and immunomodulatory activity.
CD38 is highly and uniformly expressed on the surface of multiple myeloma cells, making it a potential target for antibody-based therapeutics such as Sarclisa.
Sarclisa is approved in the U.S. in combination with pomalidomide and dexamethasone for the treatment of adults with relapsed refractory multiple myeloma who have received at least two prior therapies including lenalidomide and a proteasome inhibitor.
In the U.S., the generic name for Sarclisa is isatuximab-irfc, with irfc as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the U.S. Food and Drug Administration.
Sarclisa has also received positive CHMP opinion in combination with pomalidomide and dexamethasone for the treatment of adults with relapsed and refractory multiple myeloma who have received at least two prior therapies including lenalidomide and a proteasome inhibitor and have demonstrated disease progression on the last therapy.
A final decision on the Marketing Authorisation Application for Sarclisa in the E.U. is expected in the coming months.
The safety and efficacy of Sarclisa has not been fully evaluated by any regulatory authority outside of the U.S., Switzerland, Canada and Australia.
Sarclisa continues to be evaluated in multiple ongoing Phase 3 clinical trials in combination with current standard treatments for people with multiple myeloma.
It is also under investigation for the treatment of other blood cancer types (hematologic malignancies) and solid tumors.
For more information on Sarclisa clinical trials please visit www.clinicaltrials.gov.
Multiple myeloma is the second most common hematologic malignancy, with more than 138,000 new diagnoses of multiple myeloma worldwide yearly.
Despite available treatments, multiple myeloma remains an incurable malignancy and is associated with a significant patient burden. Since multiple myeloma does not have a cure, most patients will relapse.
Relapsed multiple myeloma is the term for when cancer returns after treatment or a period of remission.
Refractory multiple myeloma refers to when cancer does not respond or no longer responds to therapy.”

https://www.sanofi.com/en/media-room/press-releases/2020/2020-05-12-07-00-00

For More News: CHMP gives Sanofi positive opinion for Sarclisa for the treatment of relapsed and refractory multiple myeloma

ABBOTT RECEIVES FDA EMERGENCY USE AUTHORIZATION FOR COVID-19 ANTIBODY BLOOD TEST ON ALINITY™ I SYSTEM

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May 11, 2020: “Abbott announced that the U.S. FDA issued Emergency Use Authorization (EUA) for the company’s SARS-CoV-2 IgG lab-based serology blood test on the Alinity i system.

Abbott plans to ship nearly 30 million antibody tests globally in May across its ARCHITECT® and Alinity i platforms and will have capacity for 60 million tests in June.
Antibody testing will help provide a greater understanding of the virus, including how long antibodies stay in the body and how much of the population has been infected.
Last month, Abbott received FDA EUA and CE Mark for its SARS-CoV-2 IgG antibody blood test on the ARCHITECT system.* 
Abbott has already begun shipping antibody tests for use on the ARCHITECT systems globally including in the U.S., UK, Italy, Spain and India.
“Having more options of highly reliable tests across our platforms will help healthcare workers and health officials as they conduct broad scale testing for COVID-19,” said Robert B. Ford, president and chief executive officer, Abbott.
“Abbott is a leader in providing antibody testing at large scale on multiple systems, which is helping meet the needs of laboratories as they look to build testing capacity.”
The Alinity i system is Abbott’s next-generation immunoassay instrument, which was designed to offer greater efficiencies to lab clinicians running tests.
Alinity i systems are in use around the world in hospital and academic centers as well as reference laboratories.
Abbott also expects to submit this week for CE Mark to the IVD Directive (98/79/EC) in the European Union for the Alinity i SARS-CoV-2 IgG test.
Our contributions to bring broad scale access to reliable testing
As a leader in infectious disease testing, Abbott’s goal is to bring highly reliable tests for customers and patients around the world.
 A study to determine the clinical performance of Abbott’s SARS-CoV-2 IgG assay found it had greater than 99.6% specificity (ability to exclude false positives) and 100% sensitivity (ability to exclude false negatives) in patients tested 14 days after symptoms began.
Abbott is collaborating with leading virology labs to validate test results and is working to quickly install additional instruments to help contribute to large scale testing.
This is Abbott’s fourth COVID-19 test to receive FDA EUA, helping to provide hospitals and labs across the U.S. with broad, reliable molecular and antibody testing during this pandemic

Abbott’s Alinity family of harmonized solutions is unprecedented in the diagnostics industry, working together to address the challenges of using multiple diagnostic platforms and simplifying diagnostic testing.
Alinity systems are designed to be more efficient – running more tests in less space and minimizing human errors – while continuing to provide quality results.
The availability of the Alinity systems and tests varies by geography. More information is available at abbott.com/alinity.”

https://abbott.mediaroom.com/2020-05-11-Abbott-Receives-FDA-Emergency-Use-Authorization-for-COVID-19-Antibody-Blood-Test-on-Alinity-TM-i-System

NICE draft guidance recommends new treatment option for people with early breast cancer

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May 07, 2020: “Roche’s Kadcyla (trastuzumab emtansine)should soon be available on the NHS as a treatment option for patients with HER2-positive early breast cancer had won the backing of the National Institute for Health and Care Excellence (NICE).

Around 7000 people diagnosed each year in England with early breast cancer have HER2-positive disease. HER2, which stands for human epidermal growth factor receptor 2, is a protein on the surface of cancer cells which makes them grow and divide.
Trastuzumab emtansine is a type of targeted cancer drug.
Trastuzumab attaches to the HER2 receptor allowing the emtansine to go into the cancer cell where it becomes active and kills the cancer cell.
Clinical trial evidence shows that in people who still have some cancer cells remaining after chemotherapy to shrink their tumour and HER2-targeted treatment, trastuzumab emtansine increases the time people remain free of disease compared with trastuzumab alone.
It is not known if trastuzumab emtansine increases the length of time people live because the final trial results are not yet available.
Trastuzumab emtansine costs on average £51,000 per patient for a course of treatment at its full list price. 
The company has agreed a commercial arrangement which makes trastuzumab emtansine available to the NHS with a confidential discount.
Around 800 people will be eligible for treatment with trastuzumab emtansine under this guidance.
Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE, said: “Additional treatment options that can increase the amount of time in which people remain free of disease after surgery, and perhaps stop it from coming back all together, are particularly welcome. “
 We are therefore pleased to be able to recommend that trastuzumab emtansine is made available routinely for people with HER2-positive early breast cancer after surgery.”

https://www.nice.org.uk/news/article/nice-draft-guidance-recommends-new-treatment-option-for-people-with-early-breast-cancer

Lynparza approved in the US as 1st-line maintenance treatment with bevacizumab for HRD-positive advanced ovarian cancer

May 11,2020: AstraZeneca and MSD announced that Lynparza (olaparib) in combination with bevacizumab has been approved in the US for the maintenance treatment of adult patients with advanced epithelial ovarian, fallopian tube or primary peritoneal cancer who are in complete or partial response to 1st-line platinum-based chemotherapy and whose cancer is associated with homologous recombination deficiency (HRD) positive status defined by either a deleterious or suspected deleterious BRCA mutation, and/or genomic instability.

Patients will be selected for therapy based on an FDA-approved companion diagnostic test.

The approval by the US Food and Drug Administration (FDA) was based on a biomarker subgroup analysis of the Phase III PAOLA-1 trial which showed that Lynparza in combination with bevacizumab maintenance treatment reduced the risk of disease progression or death by 67% (equal to a hazard ratio of 0.33).

The addition of Lynparza improved progression-free survival (PFS) to a median of 37.2 months versus 17.7 months with bevacizumab alone in patients with HRD-positive advanced ovarian cancer.

Approximately one in two women with advanced ovarian cancer has an HRD-positive tumour. For patients with advanced ovarian cancer, the primary aim of 1st-line treatment is to delay disease progression for as long as possible with the intent to achieve long-term remission.

Isabelle Ray-Coquard, principal investigator of the PAOLA-1 trial and medical oncologist, Centre Léon Bérard and President of the GINECO group, said: “Ovarian cancer is a devastating disease.

The magnitude of benefit in HRD-positive patients in the PAOLA-1 trial is impactful.

The combination of Lynparza and bevacizumab now provides women with HRD-positive advanced ovarian cancer with a new standard of care and I look forward to seeing this translate into clinical practice.”

Dave Fredrickson, Executive Vice President, Oncology Business Unit, said: “This approval represents another milestone for Lynparza in patients with ovarian cancer.

The median progression-free survival of more than three years offers new hope for more women to delay relapse in this difficult-to-treat disease. These results further establish that HRD-positive is a distinct subset of ovarian cancer, and HRD testing is now a critical component for the diagnosis and tailoring of treatment for women with advanced ovarian cancer.”

Roy Baynes, Senior Vice President and Head of Global Clinical Development, Chief Medical Officer, MSD Research Laboratories, said: “Advances in understanding the role of biomarkers and PARP inhibition have fundamentally changed how physicians treat this aggressive type of cancer.

Today’s approval based on the PAOLA-1 trial highlights the importance of HRD testing at diagnosis to identify those who may benefit from Lynparza in combination with bevacizumab as a 1st-line maintenance treatment.”

The full results from the Phase III PAOLA-1 trial were published in The New England Journal of Medicine.

Regulatory reviews are currently underway in the EU, Japan and other countries for Lynparza based on results from the PAOLA-1 trial.

As part of a broad development programme, Lynparza is being tested as a monotherapy and in combination across multiple tumour types including as a potential adjuvant treatment of patients with germline BRCA-mutated high-risk HER2-negative primary breast cancer in the Phase III OlympiA trial.

Financial considerations

Following this approval for Lynparza in the US, AstraZeneca will receive from MSD $100m in Collaboration Revenue, anticipated to be booked by the Company during the second quarter of 2020.

Ovarian cancer

Ovarian cancer is the eighth most common cause of death from cancer in women worldwide. In 2018, there were nearly 300,000 new cases diagnosed and around 185,000 deaths.

Most women are diagnosed with advanced (Stage III or IV) ovarian cancer and have a five-year survival rate of approximately 30%. 

Approximately 50% of ovarian cancers are HRD-positive including BRCA1/2 mutation.  Some 22% of ovarian cancers have a BRCA1/2 mutation.

For patients with advanced ovarian cancer, the primary aim of 1st-line treatment is to delay the progression of the disease for as long as possible and maintain the patient’s quality of life with the intent of achieving complete remission.

In the US, bevacizumab was approved for use in combination with chemotherapy for the 1st-line treatment of advanced ovarian cancer in 2018.

Within two years nearly half of all patients with advanced ovarian cancer are receiving this combination treatment.

PAOLA-1

PAOLA-1 is a double-blind Phase III trial testing the efficacy and safety of Lynparza in combination with bevacizumab vs. bevacizumab alone, as a 1st-line maintenance treatment for newly diagnosed advanced FIGO Stage III-IV high-grade serous or endometroid ovarian, fallopian tube, or peritoneal cancer patients who had a complete or partial response to 1st-line treatment with platinum-based chemotherapy and bevacizumab.

AstraZeneca and MSD announced in August 2019 that the trial met its primary endpoint of PFS.

Simultaneously, the Myriad Genetics myChoice CDx test has been approved in the US as a companion diagnostic for Lynparza in this new indication.

Homologous recombination deficiency

HRD, which defines a sub-group of ovarian cancer, encompasses a wide range of genetic abnormalities, including BRCA mutations and beyond.

As with BRCA gene mutations, HRD interferes with normal cell DNA repair mechanisms and confers sensitivity to PARP inhibitors including Lynparza.

Lynparza

Lynparza (olaparib) is a first-in-class PARP inhibitor and the first targeted treatment to block DNA damage response (DDR) in cells/tumours harbouring a deficiency in homologous recombination repair, such as mutations in BRCA1 and/or BRCA2.

Inhibition of PARP with Lynparza leads to the trapping of PARP bound to DNA single-strand breaks, stalling of replication forks, their collapse and the generation of DNA double-strand breaks and cancer cell death. 

Lynparza is being tested in a range of PARP-dependent tumour types with defects and dependencies in the DDR pathway.

Lynparza is currently approved in a number of countries, including those in the EU, for the maintenance treatment of platinum-sensitive relapsed ovarian cancer.

It is approved in the US, the EU, Japan, China, and several other countries as 1st-line maintenance treatment of BRCA-mutated advanced ovarian cancer following response to platinum-based chemotherapy.

It is also approved in the US, Japan, and a number of other countries for germline BRCA-mutated, HER2-negative, metastatic breast cancer, previously treated with chemotherapy; in the EU, this includes locally advanced breast cancer.

Lynparza is approved in the US and several other countries for the treatment of germline BRCA-mutated metastatic pancreatic cancer. Regulatory reviews are underway in several jurisdictions for ovarian, breast, pancreatic and prostate cancers.

Lynparza, which is being jointly developed and commercialised by AstraZeneca and MSD, has been used to treat over 30,000 patients worldwide. 

Lynparza has the broadest and most advanced clinical trial development programme of any PARP inhibitor, and AstraZeneca and MSD are working together to understand how it may affect multiple PARP-dependent tumours as a monotherapy and in combination across multiple cancer types. 

Lynparza is the foundation of AstraZeneca’s industry-leading portfolio of potential new medicines targeting DDR mechanisms in cancer cells