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CDC recommends Moderna COVID 19 Vaccine for childern and adolescent

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CDC recommends Moderna COVID 19 Vaccine for childern and adolescent

 June 24, 2022: “CDC Director Rochelle P. Walensky, M.D., M.P.H., endorsed the CDC Advisory Committee on Immunization Practices’ (ACIP) recommendation that Moderna’s COVID-19 vaccine be used as an option for children ages 6 through 17 years, in addition to its already recommended use in children 6 months through 5 years and adults 18 years and older.

This recommendation reinforces the use of Moderna’s COVID-19 vaccine as an important tool in the pandemic and provides another vaccine option for children and adolescents.

The ACIP recommendation comes after a thorough review of the scientific evidence demonstrating safety and efficacy, and supports the use of the vaccine among those 6 through 17 years of age.

The following is attributable to CDC Director Dr. Rochelle P. Walensky:

“It is critical that we protect our children and teens from the complications of severe COVID-19 disease.

Today, we have expanded the options available to families by recommending a second safe and effective vaccine for children ages 6 through 17 years.

Vaccinating this age group can provide greater confidence to families that their children and adolescents participating in childcare, school, and other activities will have less risk for serious COVID-19 illness.”

https://www.cdc.gov/media/releases/2022/s0623-moderna-children.html

New Novartis extension phase data show nearly 80% of RMS patients treated with Kesimpta had NEDA-3

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New Novartis extension phase data show nearly 80% of RMS patients treated with Kesimpta had NEDA-3

June 27, 2022: “Novartis announced new data from the Phase 3 ASCLEPIOS I/II trials and the ALITHIOS open-label extension showing continuous treatment with Kesimpta® (ofatumumab) significantly increased the odds of achieving no evidence of disease activity (NEDA-3) versus switching from teriflunomide.

These data were presented at the European Academy of Neurology (EAN) Annual Meeting being held in Vienna, Austria and virtually on June 25–28, 2022.

These data show that after four years of treatment, 78.8% of those who continuously received Kesimpta achieved NEDA-3 (defined as having no MS relapses, no disability worsening and no MRI activity) versus only 51.8% of those who switched from teriflunomide to Kesimpta in the extension phase (odds ratio: 3.89; p<0.001).

These data build on the previously presented efficacy data from ASCLEPIOS I/II and ALITHIOS showing sustained differences in cumulative relapses, MRI lesion activity and the risk of disability worsening between those who were continuously treated with Kesimpta versus those who switched at a later date.

“Early initiation of high-efficacy therapies for the treatment of relapsing multiple sclerosis has been shown to improve long-term outcomes versus escalating from lower efficacy therapies,” said Professor Ludwig Kappos, University Hospital Basel.

“NEDA-3 is an important endpoint for physicians to consider when deciding to initiate high efficacy therapy, with this latest data from ALITHIOS we can clearly see the benefit of starting Kesimpta early versus switching to it later from teriflunomide.”

About Multiple Sclerosis
Multiple sclerosis (MS) is a chronic inflammatory disease of the central nervous system characterized by myelin destruction and axonal damage in the brain, optic nerves and spinal cord.

MS, which affects approximately 2.3 million people worldwide, can be characterized into four main types: clinically isolated syndrome (CIS), relapsing-remitting (RRMS), secondary progressive (SPMS) and primary progressive (PPMS).

The various forms of MS can be distinguished based on whether a patient experiences relapses (clearly defined acute inflammatory attacks of worsening neurological function), and/or whether they experience progression of neurologic damage and disability from the onset of the disease.

About Kesimpta® (ofatumumab)
Kesimpta is a targeted, precisely dosed and delivered B-cell therapy that provides the flexibility of self-administration for adults with relapsing forms of multiple sclerosis (RMS).

It is an anti-CD20 monoclonal antibody (mAb) self-administered by a once-monthly injection, delivered subcutaneously.

Initial doses of Kesimpta are at Weeks 0, 1 and 2, with the first injection performed under the guidance of a healthcare professional.

As shown in preclinical studies, Kesimpta is thought to work by binding to a distinct epitope on the CD20 molecule inducing potent B-cell lysis and depletion.

The selective mechanism of action and subcutaneous administration of Kesimpta allows precise delivery to the lymph nodes, where B-cell depletion in MS is needed, and preclinical studies have shown that it may preserve the B-cells in the spleen.

Once-monthly dosing of Kesimpta differs from other anti-CD20 therapies as it allows faster repletion of B-cells, offering more flexibility in MS management.

Ofatumumab was originally developed by Genmab and licensed to GlaxoSmithKline. Novartis obtained rights for ofatumumab from GlaxoSmithKline in all indications, including RMS, in December 2015.

Ofatumumab has been approved for the treatment of relapsing forms of multiple sclerosis in the United States, European Union, United Kingdom, Canada, China, Switzerland, Singapore, Australia, Japan, Argentina, United Arab Emirates, Albania, and India etc.”

https://www.novartis.com/news/media-releases/new-novartis-extension-phase-data-show-nearly-80-rms-patients-treated-kesimpta-ofatumumab-had-no-evidence-disease-activity-neda-3

FDA Provides Update on Efforts to Increase Supply and Availability of Safe and Nutritious Infant Formula

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FDA Provides Update on Efforts to Increase Supply and Availability of Safe and Nutritious Infant Formula

June 22, 202: “The U.S. Food and Drug Administration is providing an update on the steps it has taken, and will continue to take, to ensure that American consumers have access to safe and nutritious infant formula in the coming weeks and months. 

“The FDA is working night and day to ensure that parents and caregivers can readily find safe and nutritious formula products for any child who needs it,” said FDA Commissioner Robert M. Califf, M.D.

“I have personally spoken with infant formula manufacturers over the past several weeks and all have significantly increased their production efforts, which is resulting in more supply that will be available on stores shelves moving forward.”

FDA Flexibilities Have Resulted in Approximately 365 Million Bottles Worth of Infant Formula

In the past month, the FDA has issued enforcement discretion letters for the importation of the infant formula products from six countries, with a total estimated quantity of 17 million cans, or about 365 million full-size, 8-ounce bottles.

These products have already started to hit the U.S. market and more will appear on store shelves over the coming weeks and months.

Consumers should have confidence that the infant formula that is being imported to the U.S. through this process involved a thorough review of the information provided by the companies, including details about the product’s nutritional adequacy and safety, microbiological testing results, labeling information, and importantly, details about the manufacturing facility’s food safety production practices and inspection history.

Agency Continues Regular Inspections of Infant Formula Facilities

The FDA takes its responsibility seriously to ensure the foods we eat are safe and meet our rigorous standards for quality and safety – this is particularly true for infant formula.

While the FDA Food Safety Modernization Act (FSMA) requires the agency to inspect domestic food facilities at least once every 3 to 5 years, the FDA has a policy of annually inspecting infant formula manufacturers because the products serve as the sole source of nutrition for some of our most vulnerable consumers. 
 
This goal of annual surveillance inspections of these facilities has consistently been met over the years with the exception of 2020 during the COVID-19 pandemic. However, even throughout the COVID-19 pandemic, the FDA continued to inspect facilities, including infant formula facilities.

In fact, we inspected six firms in fiscal year 2020 (Oct. 1, 2019 – Sept. 30, 2020), including several during the height of the pandemic in the summer and fall of 2020.

In order to develop a prioritized list of firms to inspect throughout 2021, the FDA evaluated the firms that we did not inspect in 2020 to focus on firms that met certain risk criteria, including a firm’s inspection history, hazard signals such as consumer complaints, and other factors. 

While the agency postponed certain food surveillance inspections during the pandemic, the FDA resumed its annual surveillance inspection schedule for infant formula facilities beginning in July 2021.

In fiscal year 2021 (Oct. 1, 2020 – Sept. 30, 2021), we prioritized inspections of infant formula firms, conducting 16 domestic inspections throughout the year; including Abbott, Mead Johnson, Gerber, and Nestle. Since the start of fiscal year 2022 (Oct. 1, 2021), we have conducted 10 domestic infant formula inspections and one foreign, including Abbott, Mead Johnson, and Gerber. 

Importantly, it is a firm’s responsibility to ensure the consistent quality and safety of the products they produce.

In addition to our oversight work, the FDA stresses the importance of a company’s quality systems and culture. Ultimately, when problems are found it is the responsibility of the firm to correct those issues to keep consumers safe.

Latest Update on Certain Specialty, Metabolic Infant Formula Being Released by Abbott on a Case-by-Case Basis

The FDA is continuing to work to ensure infants and individuals with medical conditions who rely on certain specialty, amino acid-based and metabolic infant formula products have access to these life-sustaining products.

Some of these products have been available on a case-by-case basis from Abbott Nutrition over the last several months because the risk of not having access to them could significantly worsen underlying medical conditions and in some cases pose life-threatening risks.

To date, this case-by-case release has provided life-sustaining products to more than 2,411 infants and individuals in need, and more than 280,000 containers of additional product remain accessible to those in need.

The products are available on a case-by-case basis to patients, hospitals and institutions by calling Abbott Nutrition at 1-800-881-0876. 

Abbott Nutrition tested certain product lots that Abbott has held in storage since the FDA’s Feb. 17 warning and the company’s voluntary recall due to concerns that they were manufactured under insanitary conditions.

These products have now undergone enhanced batch testing and the company reports none of the batches tested positive for Cronobacter, a bacterium that can cause severe foodborne illness primarily in infants.

The enhanced testing provides further safety assurances to those seeking access to these products.

It’s important to note that even with the enhanced batch product testing being completed, the agency continues to recommend that parents and caregivers seeking access to these products first work with their child’s medical provider to determine whether comparable products or other changes to feeding practices may be an appropriate substitute. 

If comparable, alternative products are not available or appropriate, parents and caregivers using these products should consider following the FDA and CDC’s most current advice on how to reduce the possibility of a Cronobacter infection.

Because powdered formula is not sterile and can also be contaminated in homes, it is advised to wash hands with soap and water, especially before preparing bottles and feeding.

Ensuring all surfaces and feeding items are clean when preparing infant formula will also reduce the potential for possible contamination. 

Abbott’s Sturgis Facility Update and Prior Inspections at the Facility

Abbott recently announced that recent severe weather and rainfall resulted in flooding in areas of the Sturgis facility on Monday, June 13.

While this is an unfortunate setback and a reminder that natural weather events can also cause unforeseen supply chain disruptions, Abbott is working quickly to assess the damage and will be reporting its progress to the agency in the days ahead.

We will return to the facility and work closely with Abbott so that the Sturgis facility can restart producing safe and quality formula products as quickly as possible.
 
Making sure that parents and caregivers have access to both safe and available infant formula remains a top priority for the FDA, and our teams are working night and day to help make that happen.

Separately, given the overwhelming public interest in the FDA’s prior inspections of the Sturgis facility, the FDA is also releasing establishment inspection reports for several previous Abbott inspections.

As previously reported, during our 2019 and 2021 inspections, the agency collected and tested samples of finished products.

These samples tested negative for pathogens. During these inspections, it was determined the firm had found batches of Cronobacter contaminated finished product and had taken the appropriate action to destroy three batches, two in 2019 and one in 2020, before distribution. 

Our most recent inspection in January 2022 was a for cause inspection due to consumer complaints of infant illnesses following consumption of infant formula produced at Abbott’s Sturgis facility. 

Agency Review of Abbott Infant Formula Consumer Complaints  

The work the FDA does to make sure the foods we eat are safe is constant. This work includes ongoing surveillance of every consumer complaint it receives about the products the agency regulates.

To date, the FDA has reviewed and investigated a total of 129 complaints associated with Abbott Nutrition formula products. Of these, 119 complaints were reported after Abbott voluntarily recalled product on February 17.

The FDA has previously reported its review of complaints related to nine infant deaths.

Only two were associated with the Abbott Nutrition Sturgis plant investigation, and despite extensive investigation the evidence does not rule in or rule out a definitive link between these infant deaths and the product produced at Abbott Nutrition’s Sturgis plant.

The agency was notified of one additional consumer complaint on June 10, 2022, that resulted in an infant death in January 2022.

The agency has initiated an investigation, given that the complaint referenced that the infant had consumed an Abbott product. However, the investigation of this most recent consumer complaint is in its preliminary stages and the agency will provide an update as it learns more.”

https://www.fda.gov/news-events/press-announcements/fda-provides-update-efforts-increase-supply-and-availability-safe-and-nutritious-infant-formula

FDA Denies Authorization to Market JUUL Products

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FDA Denies Authorization to Market JUUL Products

June 23, 2022: “The U.S. Food and Drug Administration issued marketing denial orders (MDOs) to JUUL Labs Inc. for all of their products currently marketed in the United States.

As a result, the company must stop selling and distributing these products. In addition, those currently on the U.S. market must be removed, or risk enforcement action.

The products include the JUUL device and four types of JUULpods: Virginia tobacco flavored pods at nicotine concentrations of 5.0% and 3.0% and menthol flavored pods at nicotine concentrations of 5.0% and 3.0%.

Retailers should contact JUUL with any questions about products in their inventory.

“Today’s action is further progress on the FDA’s commitment to ensuring that all e-cigarette and electronic nicotine delivery system products currently being marketed to consumers meet our public health standards,” said FDA Commissioner Robert M. Califf, M.D.

“The agency has dedicated significant resources to review products from the companies that account for most of the U.S. market.

We recognize these make up a significant part of the available products and many have played a disproportionate role in the rise in youth vaping.”

These MDOs only pertain to the commercial distribution, importation and retail sales of these products, and do not restrict individual consumer possession or use—the FDA cannot and will not enforce against individual consumer possession or use of JUUL products or any other tobacco products. 

After reviewing the company’s premarket tobacco product applications (PMTAs), the FDA determined that the applications lacked sufficient evidence regarding the toxicological profile of the products to demonstrate that marketing of the products would be appropriate for the protection of the public health.

In particular, some of the company’s study findings raised concerns due to insufficient and conflicting data – including regarding genotoxicity and potentially harmful chemicals leaching from the company’s proprietary e-liquid pods – that have not been adequately addressed and precluded the FDA from completing a full toxicological risk assessment of the products named in the company’s applications. 

To date, the FDA has not received clinical information to suggest an immediate hazard associated with the use of the JUUL device or JUULpods.

However, the MDOs issued today reflect FDA’s determination that there is insufficient evidence to assess the potential toxicological risks of using the JUUL products.

There is also no way to know the potential harms from using other authorized or unauthorized third-party e-liquid pods with the JUUL device or using JUULpods with a non-JUUL device.

The FDA recommends against modifying or adding substances to tobacco products. JUUL users are encouraged to report any unexpected health problems or product problems to the FDA through the Safety Reporting Portal and to seek medical attention as necessary.

“The FDA is tasked with ensuring that tobacco products sold in this country meet the standard set by the law, but the responsibility to demonstrate that a product meets those standards ultimately falls on the shoulders of the company,” said Michele Mital, acting director of the FDA’s Center for Tobacco Products.

“As with all manufacturers, JUUL had the opportunity to provide evidence demonstrating that the marketing of their products meets these standards.

However, the company did not provide that evidence and instead left us with significant questions.

Without the data needed to determine relevant health risks, the FDA is issuing these marketing denial orders.” 

Any products subject to an MDO may not be offered for sale or distributed in the United States, or the FDA may take enforcement action. 

In addition to ensuring that JUUL complies with this order, as with unauthorized products generally, the FDA intends to ensure compliance by distributors and retailers.

Specifically, the FDA notes that all new tobacco products on the market without the statutorily required premarket authorization are marketed unlawfully and are subject to enforcement action.  

As the FDA has stated in the past, unauthorized electronic nicotine delivery system (ENDS) products for which no application is pending, including for example, those with an MDO, are among our highest enforcement priorities.

Therefore, the FDA encourages retailers to discuss products in their inventory with their suppliers including the current status of any particular tobacco product’s marketing application or marketing authorization.

Manufacturers will be the best source of that information and retailers should rely on manufacturers directly to inform decisions about which products to continue selling.

There are many resources to help smokers who want to quit. Quitting all tobacco products is the best possible path to good health.

Some current JUUL users who will not have access to JUUL products following this action or current smokers who want to transition away from cigarettes and cigars may decide to switch to other ENDS products that have been reviewed and authorized by the FDA based on their potential to benefit adult smokers. 

To date, the FDA has authorized 23 ENDS products. Under the PMTA pathway, applicants must demonstrate to the agency, among other things, that permitting the marketing of the new tobacco product would be appropriate for the protection of the public health. 

The FDA continues to work to complete its review of the remaining pending applications for deemed products submitted by the Sept. 9, 2020, deadline.

Related Information

  • Tobacco Products Marketing Orders
  • Premarket Tobacco Product Applications
  • Quitting Smoking and Other Tobacco Public Health Resources
  • Safety Reporting Portal for Tobacco Products

https://www.fda.gov/news-events/press-announcements/fda-denies-authorization-market-juul-products

FDA Releases Action Plan for Rare Neurodegenerative Diseases, Including ALS

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FDA Releases Action Plan for Rare Neurodegenerative Diseases, Including ALS

June 23, 2022: “The U.S. Food and Drug Administration unveiled its Action Plan for Rare Neurodegenerative Diseases including Amyotrophic Lateral Sclerosis (ALS) – a five-year strategy for improving and extending the lives of people living with rare neurodegenerative diseases by advancing the development of safe and effective medical products and facilitating patient access to novel treatments. 

“The effects of rare neurodegenerative diseases are devastating, with very few effective therapeutic options available to patients.

We recognize the urgent need for new treatments that can both improve and extend the lives of people diagnosed with these diseases,” said FDA Commissioner Robert M. Califf, M.D.

“To face that challenge and to accelerate drug development, we need innovative approaches to better understand these diseases while also building on current scientific and research capabilities.

This action plan, especially including the use of public-private partnerships and direct involvement of patients, will ensure the FDA is working toward meeting the task set forth by Congress to enhance the quality of life for those suffering by facilitating access to new therapies.” 
 
The action plan is a blueprint for how the agency will move forward in aggressively tackling challenges in drug development for rare neurodegenerative diseases, including ALS, in order to improve patients’ health.

Specific actions include regulatory science initiatives, enhancements to existing programs and new policy initiatives.

The plan has been developed in accordance with the provisions of the Accelerating Access to Critical Therapies for ALS Act, or ACT for ALS, that President Biden signed into law on December 23, 2021. 

Within a five-year span, the plan will focus on bolstering scientific advancement and promoting innovation for rare neurodegenerative diseases by engaging in targeted activities including: 

  • Establishing the FDA Rare Neurodegenerative Diseases Task Force (FY 22).
  • Establishing the public-private partnership for rare neurodegenerative diseases (FY 22).
  • Developing disease-specific science strategies (FY 22 – FY 26).
  • Leveraging ongoing FDA regulatory science efforts.

The ALS Science Strategy is an element of the plan focused specifically on ALS.

It provides a forward leaning framework for FDA activities to assess key regulatory science priorities. The focus areas of the ALS Science Strategy are to:

  • Improve characterization of disease pathogenesis and natural history —including quantifying disease progression, predictive and prognostic biomarkers, and efficient translation and implementation of basic science discovery is needed.
  • Facilitate patient access to new drugs whenever possible and promote greater participation in clinical trials by reducing barriers and burdens faced by diverse populations; utilizing digital health technologies and decentralized trial designs; and ensuring mechanisms for expanded access (generally outside of a clinical trial) are appropriately integrated into development programs.
  • Enhance clinical trial infrastructure and agility to enable early selection of promising therapeutic candidates for further development, optimize clinical trial design, improve access to the trials, streamline clinical trial operations, and reduce the time and cost of drug development.

Key to the success of the FDA’s implementation of the ALS Science Strategy will be patient engagement, public workshops, research projects, coordination across FDA centers and offices and collaboration with the National Institutes of Health (NIH).

ACT for ALS also requires the Department of Health and Human Services (HHS) to implement a Public-Private Partnership for Neurodegenerative Diseases between NIH, FDA and one or more outside entities through cooperative agreements, contracts or other appropriate mechanisms to advance the understanding of neurodegenerative diseases and foster development of treatments for ALS and other rare neurodegenerative diseases.

It also directs the agency to award grants and contracts to public and private entities to cover the costs of research and development of interventions that are intended to prevent, diagnose, mitigate, treat or cure ALS and other rare neurodegenerative diseases.”

https://www.fda.gov/news-events/press-announcements/fda-releases-action-plan-rare-neurodegenerative-diseases-including-als

For Wilson disease, ALXN1840 shows rapid and sustained improvement in copper mobilisation from tissues

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For Wilson disease, ALXN1840 shows rapid and sustained improvement in copper mobilisation from tissues

June 23, 2022: “Detailed results from the positive FoCus Phase III trial in Wilson disease showed that ALXN1840, an investigational once-daily, oral medicine, met its primary endpoint demonstrating three-times greater copper mobilisation from tissues compared to the standard of care (SoC) arm (Least Square Mean Difference [LSM Diff] 2.18 µmol/L; p< 0.0001), including in patients who had been treated previously for an average of 10 years.1

In the trial, people taking ALXN1840 experienced rapid copper mobilisation, with a response at four weeks and sustained through 48 weeks.

Results from the trial will be presented on 23 June at the 2022 International Liver Congress (ILC) in London.

Wilson disease is a rare and progressive genetic condition in which the body’s pathway for removing excess copper is compromised.

This may result in the accumulation of copper in a person’s liver, brain or other vital organs. Damage from excess copper build-up in tissues and organs may lead to symptoms of liver, neurological and psychiatric diseases, which may be irreversible.

Even after SoC treatment is initiated, some patients experience worsening of disease, especially of neurologic symptoms.

Change in neurological scale scores and clinician-reported functional assessments with ALXN1840 treatment were also evaluated in a post-hoc analysis as secondary endpoints in the Phase III trial.  

In patients who were symptomatic at baseline, there were greater improvements in neurological scores for those treated with ALXN1840 compared to SoC (Unified Wilson Disease Rating Scale [UWDRS] part II symptomatic ALXN1840 -1.7, SoC -0.8; UWDRS Part III symptomatic ALXN1840 -2.91, SoC -1.31). However, there were no significant differences between treatment groups observed at 48 weeks.1  

Most patients in the trial had low symptom scores at baseline, so there was minimal room for total score improvement (UWDRS Part II ALXN1840 -0.6, SoC -0.3; UWDRS Part III ALXN1840 -2.20, SoC -1.02).

As people with Wilson disease experience a highly varied degree of symptoms4, this total score may not reflect the extent of disease severity.

ALXN1840 was well tolerated and the long-term safety and efficacy of ALXN1840 is being assessed in an up to 60-month extension period.1

Professor Karl Heinz Weiss, MD, Director of the Department of Internal Medicine at Salem Medical Centre Heidelberg and investigator in the FoCus Phase III trial, said: “These data from the largest global trial in Wilson disease to date show significant copper mobilisation from the tissues with ALXN1840, even in patients who were on standard of care for over a decade on average.

These results have the potential to reframe the way doctors can think about the disease given that current therapies focus on removing copper from the blood. 

We are also encouraged by initial neurological improvement with ALXN1840 in those who were symptomatic and believe that assessing individual patient experiences may provide a better understanding of the impact on daily life.”

Marc Dunoyer, Chief Executive Officer, Alexion, said: “Many people with Wilson disease continue to experience symptoms even after years of intervention with current therapies, illuminating an urgent need to re-evaluate the standard of care.

Applying our 30 years of experience in rare disease clinical development, Alexion has conducted rigorous scientific research to bring fresh thinking to Wilson disease around the importance of copper mobilisation from the tissues.

These data further our efforts to potentially introduce a novel treatment for patients who have gone decades without meaningful innovation.”

Summary of efficacy and safety results

The primary endpoint gauged the daily mean Area Under the Effect Curve (AUEC) for directly measured non-ceruloplasmin-bound copper (dNCC)ii over 48 weeks.

The dNCC parameter includes copper bound in an inert complex with ALXN1840.”

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2022/alxn1840-shows-rapid-and-sustained-improvement-in-copper-mobilisation-from-tissues.html

Pfizer Invites Public to View and Listen to Webcast of July 28 Conference Call with Analysts

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Pfizer Invites Public to View and Listen to Webcast of July 28 Conference Call with Analysts

June 24, 2022: “Pfizer Inc. invites investors and the general public to view and listen to a webcast of a conference call with investment analysts at 10 a.m. EDT on Thursday, July 28, 2022.

The purpose of the call is to provide an update on Pfizer’s results, as reflected in the company’s Second Quarter 2022 Performance Report, to be issued that morning.

To view and listen to the webcast and view the Performance Report, visit our web site at www.pfizer.com/investors.

Information on accessing and registering for the webcast will be available at www.pfizer.com/investors beginning today. Participants are advised to register in advance of the conference call.

You can also listen to the conference call by dialing either 800-456-4352 in the United States and Canada or 785-424-1086 outside of the United States and Canada. The password is “PFEQ222”.

The transcript and webcast replay of the call will be made available on our web site atwww.pfizer.com/investors within 24 hours after the end of the live conference call and will be accessible for at least 90 days.

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 170 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.

In addition, to learn more, please visit us on www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Disclosure Notice:The webcast may include forward-looking statements about, among other things, our anticipated operating and financial performance, reorganizations, business plans, strategy and prospects; expectations for our product pipeline, in-line products and product candidates, including anticipated regulatory submissions, data read-outs, study starts, approvals, clinical trial results and other developing data, revenue contribution, growth, performance, timing of exclusivity and potential benefits; strategic reviews; capital allocation objectives; dividends and share repurchases; plans for and prospects of our acquisitions, dispositions and other business development activities, and our ability to successfully capitalize on these opportunities; manufacturing and product supply; and our efforts to respond to COVID-19, including the Pfizer-BioNTech COVID-19 vaccine (Comirnaty) and our oral COVID-19 treatment (Paxlovid), 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, 2021 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.

Sanofi-GSK first to report successful efficacy study against Omicron

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Sanofi-GSK first to report successful efficacy study against Omicron

June 24, 2022: “Sanofi and GSK announce positive data from their vaccine trial which evaluated an adjuvanted bivalent D614 and Beta (B.1.351) vaccine candidate. Sanofi-GSK’s vaccine is the first candidate to demonstrate efficacy in a placebo-controlled trial in an environment of high Omicron variant circulation.

The vaccine candidate showed a favorable safety and tolerability profile.

Earlier this month Sanofi reported positive data from two trials conducted with its new next-generation COVID-19 booster vaccine candidate modelled on the Beta variant antigen and including GSK’s pandemic adjuvant.

The data supporting this next-generation booster vaccine will be submitted to regulatory authorities and indicate the potential of Sanofi-GSK’s next-generation Beta-based booster to be a relevant response to public health needs.

Thomas Triomphe
Executive Vice President Vaccines, Sanofi
“Today’s results reinforce the strong potential for the Beta antigen to confer broad protection against multiple strains that cause COVID-19.

With the immunogenicity data from our Beta-booster vaccine, they support our belief that, in a largely seropositive world, a next-generation Beta booster vaccine could provide protection against variants like Omicron.

mRNA has proven speed to market; we are demonstrating here the efficacy that our recombinant protein platform can provide to the world. We look forward to completing our submissions to regulatory authorities and are ready to contribute to ongoing vaccination campaigns with our next-generation booster.”

Roger Connor
President of GSK Vaccines
“These positive data show efficacy of our protein-based, bivalent adjuvanted vaccine candidate in an environment of high Omicron variant circulation.

Our vaccine candidate has the potential to make an important contribution to public health as the pandemic evolves further.

We are looking forward to the discussions with regulatory authorities with the aim of making our vaccine candidate available later this year.”

In Stage 2 of the Phase 3 COVID-19 vaccine trial VAT08 of more than 13,000 participants 18 and above years of age, the Sanofi-GSK Beta-containing vaccine candidate demonstrated an efficacy of 64.7% (95% confidence interval [CI, 46.6, 77.2]) against symptomatic COVID-19 and 72% efficacy (95% confidence interval [CI, 45.8, 86.6]) in Omicron-confirmed symptomatic cases (sequencing was performed for 71 cases out of 121 total cases to date).

In previously seropositive populations, the Sanofi-GSK vaccine candidate demonstrates an overall efficacy of 75.1% (95% confidence interval [CI, 56.3, 86.6]) against symptomatic infection, and 93.2% (95% confidence interval [CI, 73.2, 99.2]) in Omicron-confirmed symptomatic cases, according to the sequencing analysis performed to date.

Throughout Stage 1 and Stage 2 of the VAT08 trial (~23,000 participants in total), the Sanofi-GSK vaccine demonstrated a favorable safety and tolerability profile.

These efforts are supported by federal funds from the Biomedical Advanced Research and Development Authority, part of the office of the Assistant Secretary for Preparedness and Response at the U.S. Department of Health and Human Services in collaboration with the U.S. Department of Defense Joint Program Executive Office for Chemical, Biological, Radiological and Nuclear Defense under Contract # W15QKN-16-9-1002 and the National Institute of Allergy and Infectious Diseases (NIAID).

About the Sanofi and GSK partnership
In the collaboration between the two companies, Sanofi provides its recombinant antigen and will be the marketing authorization holder.

GSK contributes with its pandemic adjuvant, both established vaccine platforms that have proven successful against influenza.”

https://www.sanofi.com/en/media-room/press-releases/2022/2022-06-24-05-29-02-2468538

Novartis receives positive CHMP opinion for Scemblix® for chronic myeloid leukemia

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Novartis receives positive CHMP opinion for Scemblix® for chronic myeloid leukemia

June 24, 2022: “Novartis announced that the Committee for Medicinal Products for Human Use (CHMP) of the EMA has adopted a positive opinion and recommended granting marketing authorization for Scemblix® (asciminib) for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP), previously treated with two or more tyrosine kinase inhibitors (TKIs). 

If approved, Scemblix will be the first CML treatment in Europe that works by specifically targeting the ABL myristoyl pocket (also known as a STAMP inhibitor in scientific literature), representing an important therapeutic advancement for patients who experience intolerance and/or resistance to currently available TKI therapies.

It is estimated that, every year, more than 6,300 people will be diagnosed with CML in Europe.

While many patients will benefit from available TKI therapies, a significant proportion may experience intolerance or resistance to these treatments.

In an analysis of patients with CML treated with two prior TKIs, approximately 55% reported intolerance to previous treatment; and a pooled analysis of patients in the second-line setting revealed that up to 70% are unable to achieve major molecular response (MMR) within two years of follow-up.

“Although CML treatments have advanced over the last 20 years, many patients continue to experience side effects and resistance to treatment, affecting their quality of life and putting them at risk of disease progression or even death,” says Dr. Andreas Hochhaus, Head of the Department of Hematology and Medical Oncology at Jena University Hospital in Germany.

“If approved, the novel mechanism of action of Scemblix brings us another option to combat these challenges faced by patients — offering new hope in the management of their disease.”

The positive CHMP opinion for Scemblix is based on results from the pivotal Phase III ASCEMBL trial, which showed a near doubling of MMR rate for patients treated with Scemblix vs. Bosulif®* (bosutinib) (25.5% vs. 13.2%) at 24-weeks, with a more than three times lower discontinuation rate due to adverse reactions (5.8% vs. 21.1%).

The most common (incidence ≥ 20%) adverse reactions reported in this analysis were thrombocytopenia (29.5%) and neutropenia (23.1%) in the Scemblix arm; and diarrhea (71.1%), nausea (46.1%), increased ALT (28.9%), vomiting (26.3%), rash (23.7%), increased AST (21.1%) and neutropenia (21.1%) in the Bosulif arm.

These results were confirmed in longer-term follow-up, where the MMR rate at week 96 was more than double with Scemblix (37.6%, 95% CI: 29.99-45.65) compared with Bosulif (15.8%, 95% CI: 8.43-25.96).

This data was shared at oral presentations during the  American Society for Clinical Oncology (ASCO) and the European Hematology Association (EHA) annual meetings in June 2022.

“We are pleased with the recommendation of Scemblix and hope to offer patients living with CML in Europe timely access to this innovative therapy, if approved,” said Haseeb Ahmad, President, Europe, Novartis.

“We’ve worked relentlessly to improve CML care over the past two decades, and must seize this opportunity to help patients in need achieve better outcomes.

With the strong clinical results seen to-date, we believe we have the potential to transform the standard of care in CML yet again with Scemblix.”

The CHMP recommended approval of Scemblix in CML will be referred to the European Commission (EC). The EC will review the CHMP recommendations and deliver a final decision in the coming months.

About Scemblix® (asciminib)
Scemblix is the first CML treatment that acts as a STAMP inhibitor, specifically targeting the ABL myristoyl pocket.

This novel mechanism of action may help address resistance in patients with CML previously treated with two or more TKIs and overcome mutations at the defective BCR::ABL1 gene, which is associated with the over-production of leukemic cells

Scemblix represents an important development for patients who experience resistance and/or intolerance to currently available TKI therapies, and it is being studied across multiple treatment lines for CML-CP, both as a monotherapy and in combination.

Specifically, the ASC4FIRST Phase III study (NCT04971226) evaluates Scemblix in newly diagnosed adult patients with Ph+ CML-CP vs. an investigator-selected TKI, with recruitment proceeding ahead of plan.

Novartis has initiated regulatory filings for Scemblix in multiple countries and regions across the globe.

In October 2021, the US FDA granted accelerated approval of Scemblix for adult patients with Ph+ CML-CP, previously treated with two or more TKIs based on MMR rate at 24 weeks, and full approval for adult patients with Ph+ CML-CP with the T315I mutation.

In accordance with the Accelerated Approval Program, continued approval for the first indication may be contingent upon verification and description of clinical benefit from confirmatory evidence. Further data has been shared with the FDA for evaluation.

Scemblix has received approval in several countries outside the US for adult patients with Ph+ CML-CP with resistance or intolerance to at least two or more previous therapies.”

https://www.novartis.com/news/media-releases/novartis-receives-positive-chmp-opinion-scemblix-novel-treatment-adult-patients-chronic-myeloid-leukemia

Novartis Tabrecta receives EC approval for METex14 skipping advanced nSCLC

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Novartis Tabrecta receives EC approval for METex14 skipping advanced nSCLC

June 22, 2022: “Novartis announced that the EC approved Tabrecta® (capmatinib) as a monotherapy for treatment of adults with advanced non-small cell lung cancer (NSCLC) harboring alterations leading to mesenchymal-epithelial-transition factor gene (MET) exon 14 (METex14) skipping who require systemic therapy following prior treatment with immunotherapy and/or platinum-based chemotherapy.

The approval follows a positive opinion issued in April by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) and is applicable to all 27 European Union member states plus Iceland, Norway and Liechtenstein.

“Patients with METex14 skipping alterations typically have a more advanced form of lung cancer that is often associated with a poor prognosis and limited response to standard therapy, including immunotherapy,” said Juergen Wolf, MD, from the Center for Integrated Oncology, University Hospital Cologne, Germany, and lead investigator of the GEOMETRY mono-1 trial.

“With the approval of Tabrecta in Europe, supported by advances in biomarker testing that can help doctors direct treatment more precisely, patients with this specific genomic profile have a new targeted treatment option that can lead to improved outcomes.”

The approval is based on results from the Phase II GEOMETRY mono-1 trial that demonstrated positive overall response rates (ORR) among adult patients with advanced NSCLC whose tumors had alterations leading to METex14 skipping.

In the study, among 31 patients who received Tabrecta as second- (n=30) or later-line (n=1) therapy in the METex14 skipping pretreated population, a confirmed ORR of 51.6% (95% CI, 33.1-69.8) was achieved, and the ORR across all 100 previously-treated patients, which included patients who received one or more prior lines of systemic therapy, was 44.0% (95% CI, 34.1-54.3).

The most common treatment-related adverse events (AEs) (incidence ≥20%) were peripheral oedema, nausea, fatigue, increased blood creatinine, vomiting, dyspnea, decreased appetite and back pain.

“As the leading cause of cancer-related deaths worldwide, lung cancer can be a devastating diagnosis for patients and their families,” said Marie-France Tschudin, President, Innovative Medicines International & Chief Commercial Officer, Novartis.

“With this new targeted therapy that treats a specific mutation driving cancer growth, we are delivering a much-needed treatment option and bringing hope to patients with this challenging disease.”

In the European Union, there are an estimated 291,000 patients with locally advanced or metastatic NSCLC.

METex14 skipping, a recognized oncogenic driver, occurs in approximately 3-4% of NSCLC cases.

About Tabrecta (capmatinib)
Tabrecta (capmatinib) is approved in several countries including the EU, US, Switzerland and Japan.

It is the number one prescribed targeted therapy for patients with advanced NSCLC with alterations leading to METex14 skipping globally.

Tabrecta is a kinase inhibitor that targets MET. Tabrecta was discovered by Incyte and licensed to Novartis in 2009.

Under the agreement, Incyte granted Novartis worldwide exclusive development and commercialization rights to capmatinib and certain back-up compounds in all indications.”

https://www.novartis.com/news/media-releases/novartis-receives-european-commission-approval-tabrecta-treatment-metex14-skipping-advanced-non-small-cell-lung-cancer

AZ’s NDA filing anticipated based on positive data from interim analysis

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AZ’s NDA filing anticipated based on positive data from interim analysis

FDA Announces Plans for Proposed Rule to Reduce Addictiveness of Tobacco Products

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FDA Announces Plans for Proposed Rule to Reduce Addictiveness of Tobacco Products

June 21, 2022: “The Biden-Harris Administration published plans for future potential regulatory actions that include the U.S. Food and Drug Administration’s plans to develop a proposed product standard that would establish a maximum nicotine level to reduce the addictiveness of cigarettes and certain other combusted tobacco products.

The goal of the potential rule would be to reduce youth use, addiction and death. 

Each year, 480,000 people die prematurely from a smoking-attributed disease, making tobacco use the leading cause of preventable disease and death in the United States.

Additionally, tobacco use costs nearly $300 billion a year in direct health care and lost productivity. 

While nicotine is not what makes smoking cigarettes so toxic, it’s the ingredient that makes it very hard to quit smoking.

Addiction to nicotine in combusted products is the main driver of sustained use of these products.

In fact, more than half of adult cigarette smokers make a serious quit attempt each year (quitting for at least a day), but most do not succeed due to the addictive nature of cigarettes.

Such a product standard, if proposed and then finalized after a thorough process, would make those products minimally- or non-addictive.

“Nicotine is powerfully addictive,” said FDA Commissioner Robert M. Califf, M.D.

“Making cigarettes and other combusted tobacco products minimally addictive or non-addictive would help save lives.

The U.S. Surgeon General has reported that 87 percent of adult smokers start smoking before age 18, and about two-thirds of adult daily smokers began smoking daily by 18 years of age.

Lowering nicotine levels to minimally addictive or non-addictive levels would decrease the likelihood that future generations of young people become addicted to cigarettes and help more currently addicted smokers to quit.” 

A paper published by the FDA in the New England Journal of MedicineExternal Link Disclaimer in 2018 projected that by year 2100, a potential nicotine product standard could result in more than 33 million people not becoming regular smokers, a smoking rate of only 1.4%, and more than 8 million fewer people dying from tobacco-related illnesses. The current smoking rate is 12.5%.

The Spring 2022 Unified Agenda of Regulatory and Deregulatory actions published today provides a report on the actions administrative agencies are considering issuing in the near and long term and currently lists several planned potential regulatory actions related to tobacco products; however, the dates in the Unified Agenda are not intended to be a precise estimate of when the work necessary to complete a proposed rule will be finished nor a final decision regarding whether a rule will be proposed.  

The FDA also remains focused on its regulatory oversight of e-cigarettes and other electronic nicotine delivery systems (ENDS).

Thus far, FDA has taken action on approximately 99% of the nearly 6.7 million products for which applications were received by the Sept. 9, 2020, deadline, including issuing marketing denial orders for more than 1 million ENDS products.

The FDA has also issued warning letters to ENDS product manufacturers and retailers who continue to sell products that are illegally on the market. 

The agency is focused on expeditiously completing the review of the remaining applications we received by the Sept. 9 deadline with a focus on those products with large market share.

In addition, the FDA has made a significant investment in a multimedia e-cigarette public education campaign aimed at the nearly 10.7 million youth aged 12-17 who have ever used e-cigarettes or are open to trying them highlighting information about the potential risks of e-cigarette use.”

https://www.fda.gov/news-events/press-announcements/fda-announces-plans-proposed-rule-reduce-addictiveness-cigarettes-and-other-combusted-tobacco

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