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Novartis pledges 10-year commitment with Morehouse School of Medicine and other leading organizations to co-create effective, measurable solutions for health equity

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Novartis pledges 10-year commitment with Morehouse School of Medicine and other leading organizations to co-create effective, measurable solutions for health equity

Jul 20, 2021: Novartis and the Novartis US Foundation announced a planned 10-year collaboration with Coursera, the National Medical Association, Thurgood Marshall College Fund, Morehouse School of Medicine and 26 additional Historically Black Colleges, Universities and Medical Schools (HBCUs) to co-create programs that address the root causes of systemic disparities in health outcomes and create greater diversity, equity and inclusion across the research and development ecosystem.

Leaders from these companies, organizations, and learning institutions have signed a pledge to co-develop programs focused on building trust in the health care system with communities of color and making measurable progress towards health equity.

Working together with the communities they aim to impact, the collaboration will focus on improving access to high-quality education, technology, improved health outcomes, and promising jobs; increasing clinical trial and clinical trial investigator diversity; addressing inherent bias in the data standards used to diagnose and treat disease; and finding actionable solutions to environmental and climate issues that disproportionately affect health among communities of color.

“At Novartis, we envision a world with equity in health for all. Just as there are a multitude of factors and causes behind racial disparities in health and education, there is no single solution to this critical challenge.

It will take the concerted, urgent action of diverse stakeholders across the public and private sectors,” said Vas Narasimhan, MD, CEO of Novartis.

“We are honored and humbled to work together with these organizations to build enduring solutions to some of the most pressing, deeply rooted, and historic challenges in the United States, and we invite other like-minded companies and organizations to join us in creating this paradigm shift in health equity.”

Over an initial period of ten years, the collaboration will focus on four key areas:

  1. Enable the next generation of Black and African American leaders by creating equitable access to high quality education and professional development for future leaders, in health science, technology and business-related fields.
  2. Support the establishment of Digitally Enabled Clinical Trial Centers of Excellence, managed and led by clinical researchers of color, to build trust, increase diversity and inclusivity in clinical trials, and contribute to improved health outcomes for people of color.
  3. Research and validate existing data standards that drive diagnosis, clinical trial endpoints and population health policy to identify areas for increased inclusivity and ensure accurate data collection and unbiased treatment decisions.
  4. Establish Digitally Enabled Research Centers on the impact of the environment and climate change on health to identify solutions to environmental and climate issues that disproportionately affect communities of color.

All parties will spend the next six months co-creating programs with the communities, including establishing the first clinical trial, data standards, and environment, climate and health research centers at Morehouse School of Medicine. Each organization will bring its own expertise and resources to design and implement enduring solutions in these areas.

“Health equity is not only accessible healthcare for patients, but developing educational and professional opportunities to create a diverse pipeline of educators, clinicians and other professionals, as well as ensuring all are included in clinical studies,” said Valerie Montgomery Rice, MD, President and CEO, Morehouse School of Medicine.

“This is a first of its kind collaboration and Morehouse School of Medicine is excited to work with Novartis and this coalition of medical schools, colleges, universities and other leading companies and organizations to create centers of excellence for clinical trials, data standards research, and environment and health research.

We know that real change starts here, when work is done to make a significant impact on representation and inclusion.”

Health disparities affecting minority groups are endemic in the US, most recently demonstrated during the COVID-19 pandemic by the disproportionate rates of hospitalization and death in minority groups.

Compared with non-Hispanic whites, the Black and African American community has a lower life expectancy, a higher mortality rate from cancer, a greater likelihood of diseases such as asthma, and significantly increased rates of maternal and infant mortality.

Some of these health disparities are further exacerbated by increased exposure to negative environmental factors such as air pollution, excessive heat, and poorer water quality in communities of color.

Health inequity extends to Black and African American underrepresentation across medical systems – not just among clinical trial participants but also among medical school students, physicians, and clinical trial investigators.

In 2019, Black and African Americans comprised 13.4% of the US population but only 6.2% of medical school graduates,5% of practicing physicians, and an even smaller proportion of clinical trial investigators.

“Black and African American people endured education and health disparities in the United States long before the COVID-19 pandemic.

Their exclusion from the research and development ecosystem has resulted in mistrust and a delayed uptake of life-saving innovative medicines and effective care models, further exacerbating racial disparities in care and outcomes,” said Patrice Matchaba, MD, President of the Novartis US Foundation.

“We are proud to come together to take our direction from Black and African American community members and other minority groups on programs that will help achieve sustained change.”

Enabling the next generation of Black and African American leaders
As an initial step, the Novartis US Foundation plans to invest $20 million in scholarships, mentorships and research grants over the next 10 years to help create equitable access to high quality education and professional development for HBCU students in health-related fields.

Administered by The Thurgood Marshall College Fund, the program will train and prepare up to 1,200 students:

  • Scholarships: Three-year scholarships of $10,000 a year for up to 360 students at select Historically Black Colleges, Universities and Medical Schools
  • Mentorships: Novartis employee volunteers will mentor up to 400 HBCU students, including the scholarship recipients, for a period of three years each, for a total of up to 1,200 students. Students also will receive career readiness and leadership development training
  • Internships: HBCU undergraduate and medical school students will be encouraged and supported to apply for the Novartis annual internship program
  • Research grants: Competitive faculty research grant program offering up to ten grants of $25,000 each year to HBCU faculty, focusing on actionable solutions to health equity issues

“We are pleased to work with Novartis to answer the calls for urgent action to address the role that systemic racism plays in health disparities among Black people,” said Harry L. Williams, EdD, President and CEO, Thurgood Marshall College Fund. 

“This kind of investment in HBCUs is critical to ensuring that Black students have opportunities to pursue influential roles in health, science and technology by eliminating barriers and creating pathways through mentorship and training.”

https://www.novartis.com/news/media-releases/novartis-pledges-10-year-commitment-morehouse-school-medicine-26-historically-black-colleges-universities-medical-schools-and-other-leading-organizations-co

DSRU Observational Studies on the COVID19 vaccines

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DSRU Observational Studies on the COVID19 vaccines

DSRU Observational Studies on the COVID19 vaccines

“Recruitment of participants is almost complete for our post-authorisation study to monitor the safety of the AstraZeneca COVID-19 vaccine.

The study uses active surveillance methodology, meaning that we actively recruit patients at or before vaccination and remind them at intervals to inform us of any symptoms they have following vaccination.

The DSRU is also involved in European collaborative studies on the Pfizer, Moderna and AstraZeneca COVID-19 vaccines, with more studies on other COVID vaccines and treatments in the pipeline.

For some of these studies, the DSRU conducts studies on the CPRD database, while others involve collection of data from NHS patients or prescribers.

The DSRU is undertaking the UK arm of the COVID Vaccine Monitor study, a European study which monitors for side effects of all COVID vaccines, led by the Dutch national pharmacovigilance centre, Lareb.”

https://www.dsru.org/dsru-observational-studies-on-the-covid19-vaccines/

Reacta Biotech raises £2.9m to scale up food allergy clinical diagnostics portfolio

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Reacta Biotech raises £2.9m to scale up food allergy clinical diagnostics portfolio

July 20, 2021: “Deeside, North Wales-headquartered Reacta Biotech has raised £2.9m in its latest funding round to scale up its current portfolio of food allergy clinical diagnostic kits.

The latest funding round was led by Praetura Ventures with follow-on equity from the Development Bank of Wales. Other investors include venture capitalist Jon Moulton’s family office, Perscitus an Reacta’s management team.

The funding will be used to advance the manufacturing of Reacta’s current portfolio of clinical diagnostic testing kits for allergies, including peanut, egg and milk oral food challenges (OFCs), and to extend it product portfolio.

“It’s been a landmark year for Reacta, and we continue to go from strength-to-strength as a business, developing new diagnostic products and expanding our capabilities,” said Paul Abrahams, chief executive officer of Reacta.

“The number of people experiencing food allergies is increasing, so it’s vital that technologies to accurately and safely test for allergies are developed. We’re committed to providing excellence in the development of a suite of OFC products, and revolutionising the way food allergies are diagnosed worldwide,” he added.

In a statement, Reacta noted that although the ‘principle’ of OFCs is not new, its products represent the first ‘pharmaceutical grade’, standardised, dose-controlled and masked OFCs available in the market.

Approximately 250 million people globally are affected by food allergies, with more than 8.5% of the population in the UK estimated to have a food allergy.

“As a patient investor, we have backed Reacta with equity finance since 2019. Our continued support has enabled the business to develop their diagnostic products and expand their capabilities from their base here in North Wales.

Importantly, they have now secured the specialist backing of Praetura Ventures, enabling the team to scale-up for further growth,” said Mike Bakewell and Oliver Wheatley of the Development Bank of Wales.”

https://biotech.autonomoussites.com/2021/07/20/reacta-biotech-raises-2-9m-to-scale-up-food-allergy-clinical-diagnostics-portfolio/

BlueRock Therapeutics Receives FDA Fast Track Designation for DA01 in Advanced Parkinson’s Disease

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BlueRock Therapeutics Receives FDA Fast Track Designation for DA01 in Advanced Parkinson’s Disease

July 19, 2021: BlueRock Therapeutics LP, a clinical stage biopharmaceutical company and wholly owned subsidiary of Bayer AG, announced that the U.S. FDA has granted Fast Track designation for DA01 for advanced Parkinson’s disease (PD).

DA01, BlueRock’s pluripotent stem cell-derived dopaminergic neuron therapy, is under evaluation in a Phase 1 study.

The FDA’s Fast Track designation is intended to facilitate the development and review of drug candidates that treat serious conditions and address an unmet medical need.

A drug candidate that receives Fast Track designation may be eligible for more frequent interaction with the FDA to discuss the drug candidate’s development plan as well as eligibility for accelerated approval and priority review.

“Receiving Fast Track Designation from the FDA is an important step, which will help us further accelerate clinical development of our DA01 cell therapy approach for Parkinson’s disease,” says Joachim Fruebis, Ph.D., BlueRock’s Chief Development Officer.

“This is another critical step in the BlueRock mission to create authentic cellular medicines to reverse devastating diseases, with the vision of improving the human condition.”

https://media.bayer.com/baynews/baynews.nsf/id/BlueRock-Therapeutics-Receives-FDA-Fast-Track-Designation-DA01-Treatment-Advanced-Parkinsons-Disease?Open&parent=news-overview-category-search-en&ccm=020

WHO Position Statement on Innovative Clinical Trial Designs for Development of New TB Treatments

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July 19, 2021: “The World Health Organization is publishing a position statement on Innovative Clinical Trial Designs for Development of New TB Treatments.

This document aims to support TB regimen development by highlighting key clinical trial characteristics to help advance novel therapies.

It summarises key innovations in TB clinical trial designs, ranging from pharmacokinetic-pharmacodynamic (PK-PD) modelling and new advances in biomarker development to the value of novel clinical trial design methodologies and post-licensure observational studies.

The position statement targets TB drugs and regimens developers including pharmaceutical industry, TB researchers and trialists, preclinical scientists and modellers, and calls for increased collaboration as well as data sharing and integration and standardisation of tools and measurements to advance scientific discovery.

It also emphasizes engagement with national TB programmes and affected communities to facilitate the translation of evidence into national and global policies on TB treatment and care.

Innovations in TB drug development and clinical trial design are anticipated to accelerate the development and evaluation as well as facilitate approval of novel regimens to treat all forms of TB and lead to a welcome expansion of the TB drug pipeline.

“The discovery, development and rapid uptake of new tools, interventions, and strategies are critical to substantially reduce TB incidence and reach the global End TB targets” said Dr Tereza Kasaeva, Director, WHO Global Tuberculosis Programme.

“This includes shorter, safer and more effective regimens for treating drug sensitive TB and drug resistant TB that can be used in all patient populations, including children, pregnant women, people living with HIV, and other populations at-risk”.

https://www.who.int/news/item/19-07-2021-position-statement-on-clinical-trial-designs

FDA approves fexinidazole as first all-oral treatment for sleeping sickness

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FDA approves fexinidazole as first all-oral treatment for sleeping sickness

July 19 2021: “The US FDA has approved fexinidazole as the first all-oral treatment for both stages of the Trypanosoma brucei gambiense form of sleeping sickness (Human African trypanosomiasis) in patients 6 years of age and older and weighing at least 20 kg.

Fexinidazole was developed as part of an innovative partnership between the non-profit research and development organization Drugs for Neglected Diseases initiative (DNDi), which conducted the pivotal clinical trials for this treatment, in partnership with the National Sleeping Sickness Programs of the Democratic Republic of Congo (DRC) and Central African Republic (CAR), and Sanofi.

Sleeping sickness is a parasitic disease transmitted by the bite of an infected tse-tse fly. It affects mostly populations living in remote rural areas of sub-Saharan Africa, where about 65 million people are at risk of infection. Left untreated, sleeping sickness is almost always fatal.

Through Sanofi’s collaboration the number of sleeping sickness cases reported to the WHO has been reduced by ~97% between 2001 and 2020. DNDi, Sanofi and partners are deeply committed to ensuring access to fexinidazole in all sleeping sickness-endemic countries.

Current treatment options for the disease are effective, but burdensome for patients and health workers due to the need for infusion or injection, requiring hospitalization, especially challenging for people living in remote areas.

Related News: Sanofi renews partnership with the WHO to fight Neglected Tropical Diseases

“Having a simple, all-oral treatment for sleeping sickness is a dream come true for frontline clinicians,” said Dr Bernard Pécoul, DNDi Executive Director.
“We are proud of this latest milestone in our long-term partnership with Sanofi, developed in close collaboration with researchers in countries hard-hit by sleeping sickness.”

Fexinidazole is indicated as a 10-day once-a-day treatment for Trypanosoma brucei gambiense sleeping sickness, the most common form of the disease found in West and Central Africa.

Fexinidazole is the first all-oral treatment that works both for the first stage of the disease, as well as the second stage of the disease in which the parasites have crossed the blood-brain barrier, causing patients to suffer from neuropsychiatric symptoms.

“This FDA approval is a key milestone in Sanofi’s long-term commitment to fight sleeping sickness, started 20 years ago alongside the WHO through an ambitious partnership to combat Neglected Tropical Diseases” said Luc Kuykens, Senior Vice President, Sanofi Global Health unit.

“Following the positive scientific opinion granted by the European Medicines Agency end 2018, the FDA approval is an important step to revitalize efforts to support the sustainable elimination of the disease”.

As a result of FDA approval, a Tropical Disease Priority Review Voucher (PRV) has been awarded to DNDi.

The FDA Tropical Disease PRV Program was established in 2007 to incentivize development of new treatments for neglected tropical diseases, including sleeping sickness.

Any benefits from the PRV will be shared between Sanofi and DNDi, which will enable continued investments in innovating for and ensuring access to new health tools for sleeping sickness and other neglected diseases.

Sanofi commits to continue to provide the drug free-of-charge to the World Health Organization for distribution to affected countries, as part of a long-term collaboration with WHO.”

https://www.sanofi.com/en/media-room/press-releases/2021/2021-07-19-07-30-00-2264542

AZ’s Imfinzi approved in China for extensive-stage small cell lung cancer

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AZ’s Imfinzi approved in China for extensive-stage small cell lung cancer

July 19, 2021: AstraZeneca’s Imfinzi (durvalumab) has been approved in China for the 1st-line treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC), in combination with standard of care platinum chemotherapy (etoposide plus a choice of either carboplatin or cisplatin).

The approval by China’s National Medical Products Administration was based on positive results from the CASPIAN Phase III trial.

The trial showed that Imfinzi plus chemotherapy demonstrated a statistically significant and clinically meaningful improvement in overall survival (OS) versus chemotherapy alone.

In addition, results from the China cohort of patients were consistent with the global results.

Small cell lung cancer (SCLC) is a highly aggressive, fast-growing form of lung cancer that typically recurs and progresses rapidly despite initial response to chemotherapy.

Prognosis is particularly poor, as only 7% of all SCLC patients and 3% of those with extensive-stage disease will be alive five years after diagnosis.

Dave Fredrickson, Executive Vice President, Oncology Business Unit, said: “Today’s approval of Imfinzi plus chemotherapy brings an important global standard of care to patients with extensive-stage small cell lung cancer in China, who have had few treatment options and a dire prognosis. Physicians can now offer these patients a well-tolerated immunotherapy regimen with sustained overall survival and prolonged treatment response, as well as convenient dosing. This is also the first time physicians have had the choice to combine immunotherapy with cisplatin, a preferred chemotherapy in this setting in China.”

The CASPIAN Phase III trial met the primary endpoint of OS for Imfinzi plus chemotherapy in June 2019, reducing the risk of death by 27% versus chemotherapy alone (based on a hazard ratio [HR] of 0.73; 95% confidence interval [CI] 0.59-0.91; p=0.0047), with a median OS of 13.0 months versus 10.3 months for chemotherapy alone. These results were published in The Lancet in October 2019.4 Results also showed an increased confirmed objective response rate for Imfinzi plus chemotherapy (68% versus 58% for chemotherapy alone). Imfinzi added to chemotherapy delayed the time for disease symptoms to worsen.4

An updated analysis showed sustained efficacy for Imfinzi plus chemotherapy after a median follow up of more than two years (OS HR of 0.75; 95% CI 0.62-0.91; nominal p=0.0032), with median OS of 12.9 months versus 10.5 months for chemotherapy alone. An estimated 22.2% of patients treated with Imfinzi plus chemotherapy were alive at 24 months versus 14.4% for chemotherapy alone. The safety and tolerability for Imfinzi and chemotherapy were consistent with the known safety profiles of these medicines. No patients tested positive for treatment-emergent anti-drug antibodies to Imfinzi.

Safety and efficacy results in the China cohort of patients were consistent with results in the overall global trial population. Detailed results from this cohort will be presented at a forthcoming medical meeting.

The CASPIAN Phase III trial used a fixed dose of Imfinzi (1500mg) administered every three weeks for four cycles while in combination with chemotherapy and then every four weeks as a monotherapy until disease progression.

Based on the trial results, Imfinzi,in combination with etoposide and either carboplatin or cisplatin, is approved in the 1st-line setting of ES-SCLC in more than 55 countries, including the US, Japan and across the EU.

Imfinzi is also being tested following concurrent chemoradiation therapy in patients with limited-stage SCLC in the ADRIATIC Phase III trial as part of a broad development programme.

In addition, Imfinzi is also approved to treat non-small cell lung cancer (NSCLC) in the curative-intent setting of unresectable, Stage III disease after chemoradiation therapy in the US, Japan, China, across the EU and in many other countries, based on results from the PACIFIC Phase III trial.

Small cell lung cancer
Lung cancer is the leading cause of cancer death among men and women and accounts for about one-fifth of all cancer deaths

Lung cancer is broadly split into NSCLC and SCLC, with about 15% classified as SCLC.

About two-thirds of SCLC patients are diagnosed with extensive-stage disease, in which the cancer has spread widely through the lung or to other parts of the body.

CASPIAN
CASPIAN was a randomised, open-label, multi-centre, global Phase III trial in the 1st-line treatment of 805 patients with ES-SCLC.

The trial compared Imfinzi in combination with etoposide and either carboplatin or cisplatin platinum chemotherapy, or Imfinzi and chemotherapy with the addition of a second immunotherapy, tremelimumab, versus chemotherapy alone.

In the two experimental arms, patients were treated with four cycles of chemotherapy. In comparison, the control arm allowed up to six cycles of chemotherapy and optional prophylactic cranial irradiation.

The trial was conducted in more than 200 centres across 23 countries, including the US, Europe, South America, Asia and the Middle East.

The primary endpoint was OS in each of the two experimental arms. In June 2019, AstraZeneca announced the CASPIAN Phase III trial had met one primary endpoint of demonstrating OS for Imfinzi plus chemotherapy at a planned interim analysis.

In March 2020, however, it was announced that the second experimental arm with tremelimumab did not meet its primary endpoint of OS.

Imfinzi
Imfinzi (durvalumab) is a human monoclonal antibody that binds to PD-L1 and blocks the interaction of PD-L1 with PD-1 and CD80, countering the tumour’s immune-evading tactics and releasing the inhibition of immune responses.

In addition to approvals in ES-SCLC and unresectable, Stage III NSCLC, Imfinzi is approved for previously treated patients with advanced bladder cancer in several countries.

Since the first approval in May 2017, more than 100,000 patients have been treated with Imfinzi.

As part of a broad development programme, Imfinzi is being tested as a single treatment and in combinations with other anti-cancer treatments for patients with NSCLC, SCLC, bladder cancer, hepatocellular carcinoma, biliary tract cancer (a form of liver cancer), oesophageal cancer, gastric and gastroesophageal cancer, cervical cancer, ovarian cancer, endometrial cancer, and other solid tumours.”

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2021/imfinzi-approved-in-china-for-extensive-stage-sclc.html

COVID-19 pandemic leads to major backsliding on childhood vaccinations

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COVID-19 pandemic leads to major backsliding on childhood vaccinations

July 15, 2021: 23 million children missed out on basic vaccines through routine immunization services in 2020 – 3.7 million more than in 2019 – according to official data published today by WHO and UNICEF.

This latest set of comprehensive worldwide childhood immunization figures, the first official figures to reflect global service disruptions due to COVID-19, show a majority of countries last year experienced drops in childhood vaccination rates.

Concerningly, most of these – up to 17 million children – likely did not receive a single vaccine during the year, widening already immense inequities in vaccine access.

Most of these children live in communities affected by conflict, in under-served remote places, or in informal or slum settings where they face multiple deprivations including limited access to basic health and key social services.

“Even as countries clamour to get their hands on COVID-19 vaccines, we have gone backwards on other vaccinations, leaving children at risk from devastating but preventable diseases like measles, polio or meningitis,” said Dr Tedros Adhanom Ghebreyesus, WHO Director-General.

“Multiple disease outbreaks would be catastrophic for communities and health systems already battling COVID-19, making it more urgent than ever to invest in childhood vaccination and ensure every child is reached.”

In all regions, rising numbers of children miss vital first vaccine doses in 2020; millions more miss later vaccines

Disruptions in immunization services were widespread in 2020, with the WHO Southeast Asian and Eastern Mediterranean Regions most affected. 

As access to health services and immunization outreach were curtailed, the number of children not receiving even their very first vaccinations increased in all regions.

As compared with 2019, 3.5 million more children missed their first dose of diphtheria, tetanus and pertussis vaccine (DTP-1) while 3 million more children missed their first measles dose. 

“This evidence should be a clear warning – the COVID-19 pandemic and related disruptions cost us valuable ground we cannot afford to lose – and the consequences will be paid in the lives and wellbeing of the most vulnerable,” said Henrietta Fore, UNICEF Executive Director.

“Even before the pandemic, there were worrying signs that we were beginning to lose ground in the fight to immunize children against preventable child illness, including with the widespread measles outbreaks two years ago.

The pandemic has made a bad situation worse. With the equitable distribution of COVID-19 vaccines at the forefront of everyone’s minds, we must remember that vaccine distribution has always been inequitable, but it does not have to be.”

Table 1: Countries with the greatest increase in children not receiving a first dose of diphtheria-tetanus-pertussis combined vaccine (DTP-1)

 20192020
India1’403’0003’038’000
Pakistan567’000968’000
Indonesia472’000797’000
Philippines450’000557’000
Mexico348000454’000
Mozambique97’000186’000
Angola399’000482’000
United Republic of Tanzania183’000249’000
Argentina97’000156’000
Venezuela (Bolivarian Republic of)75’000134’000
Mali136’000193’000

The data shows that middle-income countries now account for an increasing share of unprotected children – that is, children missing out on at least some vaccine doses. India is experiencing a particularly large drop, with DTP-3 coverage falling from 91% to 85%.

Fuelled by funding shortfalls, vaccine misinformation, instability and other factors, a troubling picture is also emerging in WHO’s Region of the Americas, where vaccination coverage continues to fall. Just 82% of children are fully vaccinated with DTP, down from 91% in 2016.

Countries risk resurgence of measles, other vaccine-preventable diseases

Even prior to the COVID-19 pandemic, global childhood vaccination rates against diphtheria, tetanus, pertussis, measles and polio had stalled for several years at around 86%.

This rate is well below the 95% recommended by WHO to protect against measles –often the first disease to resurge when children are not reached with vaccines – and insufficient to stop other vaccine-preventable diseases.

With many resources and personnel diverted to support the COVID-19 response, there have been significant disruptions to immunization service provision in many parts of the world.

In some countries, clinics have been closed or hours reduced, while people may have been reluctant to seek healthcare because of fear of transmission or have experienced challenges reaching services due to lockdown measures and transportation disruptions.

“These are alarming numbers, suggesting the pandemic is unravelling years of progress in routine immunization and exposing millions of children to deadly, preventable diseases”, said Dr Seth Berkley, CEO of Gavi, the Vaccine Alliance. “This is a wake-up call – we cannot allow a legacy of COVID-19 to be the resurgence of measles, polio and other killers.

We all need to work together to help countries both defeat COVID-19, by ensuring global, equitable access to vaccines, and get routine immunization programmes back on track.

The future health and wellbeing of millions of children and their communities across the globe depends on it.” 

Concerns are not just for outbreak-prone diseases. Already at low rates, vaccinations against human papillomavirus (HPV) – which protect girls against cervical cancer later in life – have been highly affected by school closures.

As a result, across countries that have introduced HPV vaccine to date, approximately 1.6 million more girls missed out in 2020. Globally only 13% girls were vaccinated against HPV, falling from 15% in 2019.

Agencies call for urgent recovery and investment in routine immunization

As countries work to recover lost ground due to COVID-19 related disruptions, UNICEF, WHO and partners like Gavi, the Vaccine Alliance are supporting efforts to strengthen immunization systems by:

  • Restoring services and vaccination campaigns so countries can safely deliver routine immunization programmes during the COVID-19 pandemic;
  • Helping health workers and community leaders communicate actively with caregivers to explain the importance of vaccinations;
  • Rectifying gaps in immunization coverage, including identifying communities and people who have been missed during the pandemic.
  • Ensuring that COVID-19 vaccine delivery is independently planned for and financed and that it occurs alongside, and not at the cost of childhood vaccination services.
  • Implementing country plans to prevent and respond to outbreaks of vaccine-preventable diseases, and strengthen immunization systems as part of COVID-19 recovery efforts

The agencies are working with countries and partners to deliver the ambitious targets of the global Immunization Agenda 2030, which aims to achieve 90% coverage for essential childhood vaccines; halve the number of entirely unvaccinated, or ‘zero dose’ children, and increase the uptake of newer lifesaving vaccines such as rotavirus or pneumococcus in low and middle-income countries.”

https://www.who.int/news/item/15-07-2021-covid-19-pandemic-leads-to-major-backsliding-on-childhood-vaccinations-new-who-unicef-data-shows

Status on FDA Advisory Committee vote on AZ’s roxadustat in anaemia of chronic kidney disease

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Status on FDA Advisory Committee vote on AZ’s roxadustat in anaemia of chronic kidney disease

July 16, 2021: The FDA Cardiovascular and Renal Drugs Advisory Committee (CRDAC) has voted 13 to 1 that the benefit-risk profile of roxadustat does not support approval for the treatment of anaemia in chronic kidney disease (CKD) in non-dialysis dependent (NDD) adult patients, and 12 to 2 that the benefit-risk profile of roxadustat does not support approval for the treatment of anaemia in CKD in dialysis-dependent (DD) adult patients.

The FDA will consider the vote, independent opinions and recommendations from experts as it reviews the new drug application (NDA) and is not bound by the Committee’s recommendation.

The safety and efficacy of roxadustat, an oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor, have been demonstrated in the Phase III programme including more than 8,000 patients and published in five peer-reviewed journal articles.

Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, said: “New solutions are needed for the six million people in the US affected by anaemia of chronic kidney disease.

Although we are disappointed by today’s outcome, we will continue to work closely with our partner FibroGen and the FDA to determine the path forward for roxadustat.”

Earlier this year, the FDA confirmed it would convene a meeting of the CRDAC to review the NDA for roxadustat and requested further clarifying analyses of clinical data to support the assessment.

The FDA has not announced when it will make its final decision for the roxadustat NDA.

The CRDAC provides the FDA with independent, expert advice and reviews and evaluates available data concerning the safety and efficacy of marketed and potential new medicines for use in the treatment of cardiovascular (CV) and renal disorders.

Roxadustat is approved in a number of countries, including China, Japan, Chile and South Korea for the treatment of anaemia in CKD in NDD and DD adult patients.

It is under regulatory review in other jurisdictions, including in the European Union, where it has recently received a positive CHMP opinion.

Anaemia
Anaemia can be a serious medical condition in which patients have insufficient RBCs and low levels of haemoglobin, a protein in RBCs that carries oxygen to cells throughout the body.

Anaemia of CKD frequently causes significant fatigue, cognitive dysfunction and decreased quality of life, and is associated with increased risk of hospitalisation, CV complications and death.

Severe anaemia is common in patients with CKD, cancer, myelodysplastic syndrome (MDS), inflammatory diseases and other serious illnesses. Anaemia is particularly prevalent in patients with CKD.

CKD affects 840 million patients worldwide and is generally progressive, characterised by gradual loss of kidney function that may eventually lead to kidney failure.

Phase III programme
The Phase III programme included more than 8,000 patients and was conducted by AstraZeneca, FibroGen and Astellas Pharma Inc. (Astellas).

The OLYMPUS, ALPS and ANDES trials evaluated roxadustat compared to placebo in NDD-CKD patients.

ROCKIES, SIERRAS and HIMALAYAS evaluated roxadustat compared to epoetin alfa in DD-CKD and incident dialysis (ID) patients. 

HIMALAYAS evaluated roxadustat compared to epoetin alfa in ID patients; ROCKIES and SIERRAS included ID and prevalent dialysis patients.

Roxadustat
Roxadustat, an oral medicine, could be the first in a new class of treatments called oral HIF-PH inhibitors that promotes erythropoiesis, or RBC production, through increased endogenous production of erythropoietin, improved iron absorption and mobilisation, and reduction of hepcidin.

Roxadustat is also in clinical development for anaemia associated with MDS and for chemotherapy-induced anaemia.

Roxadustat is approved in China, Japan, Chile and South Korea (under the name Evrenzo), for the treatment of anaemia in CKD in NDD and DD adult patients.

In Europe, the Marketing Authorisation Application for Evrenzo for the treatment of anaemia in CKD in NDD and DD patients was submitted by Astellas and accepted by the European Medicines Agency for review in May 2020 and is under final regulatory review following a positive EU CHMP opinion in June 2021.

AstraZeneca and FibroGen are collaborating on the development and commercialisation of roxadustat for the potential treatment of anaemia in the US, China and other countries in the Americas, Australia and New Zealand, as well as Southeast Asia.

Astellas and FibroGen are collaborating on the development and commercialisation of roxadustat for the potential treatment of anaemia in Japan, Europe, Turkey, Russia and the Commonwealth of Independent States, the Middle East and South Africa.

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2021/status-on-us-fda-advisory-committee-for-roxadustat.html

FDA Grants First Full Approval for Treatment of Lymphoma in Dogs

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FDA Grants First Full Approval for Treatment of Lymphoma in Dogs

July 15, 2021: The U.S. FDA fully approved Tanovea (rabacfosadine injection) to treat lymphoma in dogs. Lymphoma, also called lymphosarcoma, is a type of cancer that can affect many species, including dogs.

Tanovea is the first conditionally approved new animal drug for dogs to achieve the FDA’s full approval.

Tanovea was approved using the new animal drug approval process after initially receiving conditional approval under statutory provisions related to drugs intended for uncommon diseases or conditions in major animal species (minor uses) or for use in the minor species of animals under the FDA’s Minor Use and Minor Species (MUMS) program (i.e., MUMS drugs).

“The FDA is dedicated to making treatment options available for all patients – including animals suffering from rare conditions.

Today’s first ever full approval of a new animal drug for treating lymphoma in dogs demonstrates the positive impact that the FDA’s Minor Use and Minor Species program can have on the availability of novel animal treatments,” said Acting FDA Commissioner Janet Woodcock, M.D.

“We’re committed to continue using all our authorities to help make limited-demand treatment options available to our animal companions.”

The FDA’s MUMS program is intended to make more medications legally available to veterinarians and animal owners for the treatment of minor animal species and uncommon diseases in the major animal species.

This program provides innovative ways to bring products to market for these small populations and is designed to help pharmaceutical companies overcome the financial roadblocks they face in providing limited-demand animal drugs.

Before the passage of legislation establishing the program in 2004, pharmaceutical companies would rarely attempt to bring MUMS drugs to market, because the markets were too small to generate an adequate financial return.

“Today’s approval shows that drugs to treat rare animal diseases, like canine lymphoma, can go through the FDA’s conditional approval pathway to reach full approval.

This gives veterinarians another important tool to help extend the quality of life for dogs with lymphoma, and potentially give them and their owners more time together,” said Steven M. Solomon, M.P.H., D.V.M., director of the FDA’s Center for Veterinary Medicine.

“While canine lymphoma affects fewer than 70,000 dogs in the U.S. annually, it accounts for up to 24 percent of all cancers in dogs, making it one of the most significant canine cancers.

For the first time, dog owners have the assurance of a treatment that has fully met the FDA’s standards for effectiveness in dogs.”

Lymphoma originates from white blood cells called lymphocytes. These cells are a normal part of the immune system and protect the body from infection, but they grow abnormally if impacted by lymphoma.

Although lymphoma can affect virtually any organ in the body, it most commonly starts in organs that function as part of the immune system, such as the lymph nodes, spleen, and bone marrow.

The signs of lymphoma in dogs vary depending on which organs are affected. The cause of canine lymphoma is unknown.

Chemotherapy drugs often have potential side effects, but, unlike in human medicine in which patients may be willing to tolerate uncomfortable side effects in exchange for a potential cure, the primary purpose of chemotherapy in pets is to extend survival, with an emphasis on the pet’s quality of life and comfort.

The active ingredient in Tanovea is rabacfosadine, a substance that kills rapidly growing cancer cells.

Tanovea must be prescribed by a licensed veterinarian because professional expertise is needed to correctly diagnose lymphoma in dogs, determine the best treatment, and manage potential side effects.

Tanovea comes in a concentrated form and, once diluted, is injected into a vein over a period of 30 minutes.

The treatment is repeated every three weeks for up to a total of five administrations.

Because Tanovea is a chemotherapeutic drug, owners should take extra care when handling and cleaning up after their dogs for a period of five days following each treatment. Veterinarians should discuss these precautions with dog owners.

Tanovea received conditional approval External Link Disclaimer under the MUMS program in December 2016, meaning that the agency had found the drug was safe for the intended purpose and had a “reasonable expectation of effectiveness” for treating lymphoma in dogs. 

Conditional approval External Link Disclaimer allowed the drug manufacturer to market Tanovea for a period of time while collecting additional study data to meet the “substantial evidence” standard of effectiveness for full approval.

These additional studies have now been reviewed as the basis for the full approval.

The effectiveness and safety of Tanovea was demonstrated in a well-controlled clinical field study involving a total of 158 dogs that had been diagnosed with multicentric lymphoma with at least one enlarged peripheral lymph node.

The study was open to dogs of any breed, except West Highland White Terriers.

West Highland White Terriers were not enrolled due to the breed’s tendency to develop pulmonary fibrosis, which is a known potential side effect of Tanovea that was identified prior to conditional approval.

All 158 dogs were evaluated for safety (120 in the Tanovea group and 38 in the placebo group), and 148 dogs were evaluated for effectiveness (112 in the Tanovea group and 36 in the placebo group).

Dogs ranged from one to 15 years in age in the Tanovea group and from three to 16 years in age in the placebo group.

The study found that Tanovea extended the median survival rate by 61 days and for dogs with a complete response to the drug, the median progression-free survival was extended to 168 days.

The most common side effects seen in dogs treated with Tanovea included diarrhea, decreased appetite, vomiting, lethargy, weight loss and neutropenia, a decrease in a type of white blood cell.

The most serious adverse events included pulmonary fibrosis and skin issues, including infection, ulceration and skin peeling in some cases.

Veterinarians should advise owners about the possible adverse events and side effects before using the drug and share the client information sheet developed for the product.

The FDA encourages dog owners to work with their veterinary team to report any adverse events or side effects potentially related to the use of any drug, including Tanovea.

The FDA granted approval for Tanovea to VetDC Inc.

https://www.fda.gov/news-events/press-announcements/fda-grants-first-full-approval-treatment-lymphoma-dogs

FDA approves Bayer’s finerenone for chronic kidney disease associated with type 2 diabetes

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FDA approves Bayer’s finerenone for chronic kidney disease associated with type 2 diabetes

July 9, 2021: “Bayer announced that the U.S.FDA has approved finerenone, the first non-steroidal, selective mineralocorticoid receptor (MR) antagonist, under the brand name Kerendia®.

Finerenone 10 mg or 20 mg is indicated to reduce the risk of sustained estimated glomerular filtration rate (eGFR) decline, end-stage kidney disease, cardiovascular death, non-fatal myocardial infarction, and hospitalization for heart failure in adult patients with chronic kidney disease (CKD) associated with type 2 diabetes (T2D).

The approval of finerenone by the FDA is based on the positive results of the pivotal Phase III FIDELIO-DKD study, presented at the American Society of Nephrology’s (ASN) Kidney Week Reimagined 2020 and simultaneously published in the New England Journal of Medicine in October 2020, and follows Priority Review designation granted by the FDA in January 2021.

“There are more than 160 million patients living with CKD and T2D worldwide.

Even when blood glucose levels and blood pressure are well-controlled, patients still remain at risk of CKD progression.

This means there is a high unmet medical need for early intervention to prevent further end-organ damage and premature death by slowing patients’ rate of decline in kidney function as well as reducing cardiovascular risk,” said Professor George L. Bakris, MD, Department of Medicine, American Heart Association Comprehensive Hypertension Center, University of Chicago Medicine, USA and principal investigator of the FIDELIO-DKD trial.

“The approval of finerenone offers a new path to protect patients from further kidney damage through addressing MR overactivation, a key driver of CKD progression, which is unaddressed by currently available therapies.”

Chronic kidney disease (CKD) is a common and potentially deadly condition that is generally underrecognized. CKD can shorten life expectancy of patients with type 2 diabetes by up to 16 years, relative to the general population living without either disease.

Up to 40% of all patients with type 2 diabetes develop chronic kidney disease. In T2D, mineralocorticoid receptor (MR) overactivation is thought to contribute to CKD progression which can be driven by metabolic, haemodynamic or inflammation and fibrosis factors.

“The Phase III FIDELIO-DKD trial is the first large contemporary positive outcomes study in patients with chronic kidney disease (CKD) and type 2 diabetes (T2D) with a primary composite endpoint consisting exclusively of kidney-specific outcomes.

It is also part of the largest global Phase III clinical trial program to date in CKD and T2D,” said Dr. Michael Devoy, Chief Medical Officer and Head of Medical Affairs and Pharmacovigilance at Bayer’s Pharmaceuticals Division.

“With our longstanding expertise in innovative science in the cardiovascular space, today’s approval of finerenone marks an important milestone in Bayer’s commitment to improving the lives of patients with kidney and cardiovascular diseases.”

Finerenone has also been submitted for marketing authorization in the European Union (EU) and in China, as well as multiple other countries worldwide and these applications are currently under review.

About Finerenone
Finerenone (BAY 94-8862) is a novel, non-steroidal, selective antagonist of the mineralocorticoid receptor (MR) that has been shown to block harmful effects of mineralocorticoid receptor (MR) overactivation.

In T2D, MR overactivation is thought to contribute to CKD progression and cardiovascular damage which can be driven by metabolic, haemodynamic or inflammation and fibrosis factors.

Having randomized more than 13,000 patients with CKD and T2D around the world, the Phase III programme with finerenone in CKD and T2D comprises two studies, evaluating the effect of finerenone versus placebo on top of standard of care on both renal and cardiovascular outcomes.

FIDELIO-DKD (FInerenone in reducing kiDnEy faiLure and dIsease prOgression in Diabetic Kidney Disease) investigated the efficacy and safety of finerenone in comparison to placebo in addition to standard of care on the reduction of kidney failure and kidney disease progression in approximately 5,700 patients with CKD and T2D.

Based on the positive data from FIDELIO-DKD, the U.S. FDA has approved finerenone for marketing authorization in the U.S. Finerenone has been submitted for marketing authorization in the EU and other countries worldwide based on the positive data from FIDELIO-DKD.”

https://media.bayer.com/baynews/baynews.nsf/id/US-FDA-approves-finerenone-treatment-patients-chronic-kidney-disease-associated-2-diabetes?OpenDocument&sessionID=1626328619

Novartis announces positive Phase III REACH3 data for Jakavi in chronic GvHD

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Novartis announces positive Phase III REACH3 data for Jakavi in chronic GvHD

Jul 14, 2021: Novartis announced that The New England Journal of Medicine (NEJM) published positive results from the Phase III REACH3 trial demonstrating Jakavi® (ruxolitinib) significantly improved outcomes in patients with steroid-refractory/dependent chronic graft-versus-host disease (GvHD) compared to best available therapy (BAT).

The study’s main findings, which had been previously presented at the 62nd American Society of Hematology (ASH) Annual Meeting, were published along with new subgroup analyses showing favorable overall response rate (ORR) at week 24 for Jakavi across all major subgroups, including baseline individual organ involvement.

REACH3 is jointly sponsored by Novartis and Incyte.

“Patients with chronic GvHD can experience severe and life-threatening symptoms in different organs around the body, which makes the disease more difficult to treat and increases the risk of poor outcomes,” said Dr. Robert Zeiser, University Hospital Freiburg, Department of Haematology, Oncology and Stem Cell Transplantation, Freiburg, Germany.

“With these new results from REACH3, we can see more clearly the potential benefits of what may become a new standard of care for chronic GvHD patients who have not adequately responded to first-line steroids.”

The study found that treatment with Jakavi led to significant improvements in ORR at week 24 (49.7% vs. 25.6%; odds ratio [OR], 2.99; P<0.001i), which was the trial’s primary endpoint.

Also, best overall response (BOR) rate at any time up to week 24 was achieved in 76.4% of patients in the Jakavi arm compared to 60.4% in the BAT arm (OR, 2.17; 95% CI, 1.34-3.52).

Jakavi also demonstrated statistically significant and clinically meaningful improvements in key secondary endpoints:

  • Patients on Jakavi achieved longer failure-free survival (FFS) than patients receiving BAT (median FFS not yet reached vs. 5.7 months; hazard ratio, 0.37; 95% CI, 0.27 to 0.51; P<0.001).
  • Jakavi-treated patients also had greater improvements in self-reported symptoms compared to BAT1 (24.2% vs. 11.0%; OR, 2.62; P=0.001)ii.

In addition, a new subgroup analysis included in the publication found that patients on Jakavi had better outcomes regardless of the individual organs affected at baseline1.

“These new Jakavi data underscore its potential benefits and the importance of making it available to patients at risk for an all-too-common and life-threatening complication of stem cell transplants,” said Susanne Schaffert, PhD, President, Novartis Oncology.

“We are pleased that regulatory submissions are underway and will continue to work toward wide accessibility of this important new medicine for GvHD.”

No new safety signals were observed in REACH3, and adverse events (AEs) attributable to treatment were consistent with the mechanism of action and the known safety profile of Jakavi.

The most common AEs of grade 3 or higher in the Jakavi vs. BAT arms were thrombocytopenia (15.2% vs. 10.1%), anemia (12.7% vs. 7.6%), neutropenia (8.5% vs. 3.8%) and pneumonia (8.5% vs. 9.5%).

While 37.6% and 16.5% of patients required Jakavi and BAT dose modifications due to AEs, respectively, the number of patients who discontinued treatment due to AEs was low (16.4% Jakavi vs. 7% BAT).

Mortality rates were similar across treatment arms (18.8% Jakavi vs. 16.5% BAT).

Deaths reported as primarily due to chronic GvHD were slightly higher for Jakavi.

In 2019, the US Food and Drug Administration (FDA) approved ruxolitinib (marketed by Incyte Corporation in the US as Jakafi®) for the treatment of steroid-refractory acute GvHD in adult and pediatric patients 12 years and older, based on results of the single-arm Phase II REACH1 trial.

The Incyte filing in the US for Jakafi in steroid-refractory chronic GvHD is currently undergoing review by the FDA.

Outside the US, Novartis regulatory submissions for acute and chronic GvHD are underway worldwide.

The NEJM publication of the REACH3 results is available online at www.NEJM.org.

About Jakavi® (ruxolitinib) 
Jakavi® (ruxolitinib) is an oral inhibitor of the JAK 1 and JAK 2 tyrosine kinases. Jakavi is approved by the European Commission for the treatment of adult patients with polycythemia vera (PV) who are refractory to or intolerant of hydroxyurea and for the treatment of disease- related splenomegaly or symptoms in adult patients with primary myelofibrosis (MF) (also known as chronic idiopathic MF), post-polycythemia vera MF or post-essential thrombocythemia MF.

Jakavi is approved in over 100 countries for patients with MF, including EU countries, Switzerland, Canada, Japan and in more than 85 countries for patients with PV, including EU countries, Switzerland, Japan and Canada.

The exact indication for Jakavi varies by country. Additional worldwide regulatory filings are underway in MF and PV. 

Novartis licensed ruxolitinib from Incyte Corporation for development and commercialization outside the United States.

Ruxolitinib is marketed in the United States by Incyte Corporation as Jakafi® for patients with PV who have had an inadequate response to or are intolerant of hydroxyurea, for patients with intermediate or high-risk MF, and steroid-refractory acute GvHD in adult and pediatric patients 12 years and older. 

The recommended starting dose of Jakavi in PV is 10 mg given orally twice daily.

The recommended starting dose of Jakavi in MF is 15 mg given orally twice daily for patients with a platelet count between 100,000 cubic millimeters (mm) and 200,000 mm, and 20 mg twice daily for patients with a platelet count of >200,000 mm.

Doses may be titrated based on safety and efficacy.

There is limited information to recommend a starting dose for MF and PV patients with platelet counts between 50,000/mm and <100,000/mm.

-The maximum recommended starting dose in these patients is 5 mg twice daily, and patients should be titrated cautiously. 

Jakavi is a registered trademark of Novartis AG in countries outside the United States. Jakafi is a registered trademark of Incyte Corporation.

The safety and efficacy profile of Jakavi has not yet been established outside of its approved indications.”

https://www.novartis.com/news/media-releases/novartis-announces-nejm-publication-positive-phase-iii-reach3-data-jakavi-chronic-gvhd

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