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AstraZeneca and Ionis sign deal to develop and commercialise eplontersen

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AstraZeneca and Ionis sign deal to develop and commercialise eplontersen

December 07, 2021: “Liver-targeted antisense therapy in Phase III development for the treatment of transthyretin amyloidosis, a systemic, progressive and fatal condition
 

AstraZeneca has entered into a new global development and commercialisation agreement with Ionis Pharmaceuticals, Inc. (Ionis) for eplontersen, formerly known as IONIS-TTR-LRX. 

Eplontersen is a ligand-conjugated antisense investigational medicine currently in Phase III clinical trials for amyloid transthyretin cardiomyopathy (ATTR-CM) and amyloid transthyretin polyneuropathy (ATTR-PN).

It is designed to reduce the production of transthyretin (TTR protein) to treat both hereditary and non-hereditary forms of TTR amyloidosis (ATTR).

The companies will jointly develop and commercialise eplontersen in the US, while AstraZeneca will develop and commercialise it in the rest of the world, except in Latin America.

ATTR-CM is a systemic, progressive and fatal condition that leads to progressive heart failure and death within four years from diagnosis.

It remains underdiagnosed and its prevalence is thought to be underestimated due to a lack of disease awareness and the heterogeneity of symptoms.

Hereditary ATTR-PN is a debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade.

Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca, said: “Eplontersen has the potential to halt the progression of TTR-mediated amyloidosis, irrespective of whether it’s caused by genetic mutations or aging.

Thanks to its precise liver-targeting properties, it also has the potential to be a best-in-class treatment for patients suffering from this devastating disease and who currently have limited options.”

Hereditary ATTR-PN is expected to be the first indication for which the companies will seek regulatory approval for eplontersen, with the potential to file a new drug application with the US Food and Drug Administration by the end of 2022.

Financial considerations
AstraZeneca will pay Ionis an upfront payment of $200m and additional conditional payments of up to $485m following regulatory approvals.

It will also pay up to $2.9bn of sales-related milestones based on sales thresholds between $500m and $6bn, plus royalties in the range of low double-digit to mid-twenties percentage depending on the region.

The collaboration includes territory-specific development, commercial and medical affairs cost-sharing provisions.

The transaction will be funded with cash and is expected to be neutral to Core earnings in 2021.

It will be accounted for as an intangible asset acquisition, recognised initially at the upfront amount, with any potential future milestone payments capitalised into the intangible asset as they are recognised.

Ionis will continue to manufacture and supply eplontersen for the existing clinical studies and process qualification.

AstraZeneca will be responsible for commercial supply, with transition timing to be agreed by both parties. AstraZeneca will book all sales generated under the agreement.

The transaction is expected to close in the fourth quarter of 2021, subject to customary closing conditions and regulatory clearances. The transaction does not impact the AstraZeneca’s financial guidance for 2021.”

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2021/astrazeneca-ionis-to-collaborate-on-eplontersen.html

EMA accepts regulatory submission for olipudase alfa, for ASMD

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EMA accepts regulatory submission for olipudase alfa, for ASMD

December 06 2021: “The European Medicines Agency has accepted for review under an accelerated assessment procedure the Marketing Authorization Application (MAA) for olipudase alfa, Sanofi’s investigational enzyme replacement therapy which is being evaluated for the treatment of acid sphingomyelinase deficiency (ASMD).

Historically referred to as Niemann-Pick disease (NPD) type A and type B, ASMD is a rare, progressive, and potentially life-threatening disease for which no treatments are currently approved.

The estimated prevalence of ASMD is approximately 2,000 patients in the U.S., Europe (EU5 Countries) and Japan. If approved, olipudase alfa will become the first and only therapy for the treatment of ASMD.


“Today’s milestone has been decades in the making and our gratitude goes to the ASMD community who has stood by us with endless patience while olipudase alfa advanced through clinical development,” said Alaa Hamed, MD, MPH, MBA, Global Head of Medical Affairs, Rare Diseases, Sanofi. 

“Olipudase alfa represents the kind of potentially life-changing innovation that is possible when industry, medical professionals and the patient community work together toward a common goal.”

The MAA is based on positive results from two separate clinical trials (ASCEND and ASCEND-Peds) evaluating olipudase alfa in adult and pediatric patients with non-central nervous system (CNS) manifestations of ASMD type A/B and ASMD type B.

Olipudase alfa has received special designations from regulatory agencies worldwide, recognizing the innovation potential of the investigational therapy.

“Scientific innovation is the greatest source of hope for people living with diseases like ASMD where there are no approved treatments and is a critical component for ensuring a viable healthcare ecosystem,” said Bill Sibold, Executive Vice President of Sanofi Genzyme. 

“At Sanofi, we have a long history of pioneering scientific innovation, and we remain committed to finding solutions to address unmet medical needs, including those of the rare disease community.”

The EMA awarded olipudase alfa the PRIority MEdicines designation, also known as PRIME, intended to aid and expedite the regulatory process for investigational medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options.

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to olipudase alfa.

This designation is intended to expedite the development and review of drugs intended to treat serious or life-threatening diseases and conditions.

The criteria for granting Breakthrough Therapy designation include preliminary clinical evidence indicating that the molecule may demonstrate substantial improvement on a clinically significant endpoint over available therapies.

In Japan, olipudase alfa was awarded the SAKIGAKE designation, which is intended to promote research and development in Japan for innovative new medical products that satisfy certain criteria, such as the severity of the intended indication.

In September, Sanofi filed the J-NDA submission for olipudase alfa.

About ASMD

ASMD results from a deficient activity of the enzyme acid sphingomyelinase (ASM), which is found in special compartments within cells called lysosomes and is required to breakdown lipids called sphingomyelin.

If ASM is absent or not functioning as it should, sphingomyelin cannot be metabolized properly and accumulates within cells, eventually causing cell death and the malfunction of major organ systems.

The deficiency of the lysosomal enzyme ASM is due to disease-causing variants in the sphingomyelin phosphodiesterase 1 gene (SMPD1). The estimated prevalence of ASMD is approximately 2,000 patients in the U.S., Europe (EU5 Countries) and Japan.

ASMD represents a spectrum of disease caused by the same enzymatic deficiency, with two types that may represent opposite ends of a continuum sometimes referred to as ASMD type A and ASMD type B.

ASMD type A is a rapidly progressive neurological form of the disease resulting in death in early childhood due to central nervous system complications.

ASMD type B is a serious and potentially life-threatening disease that predominantly impacts the lungs, liver, and spleen, as well as other organs.

ASMD type A/B represents an intermediate form that includes varying degrees of neurologic involvement.

Patients with ASMD type A/B or ASMD type B were studied in the ASCEND trial program. Another type of NPD is NPD type C, which is unrelated to ASMD.

About olipudase alfa

Olipudase alfa is an investigational enzyme replacement therapy designed to replace deficient or defective ASM, allowing for the breakdown of sphingomyelin.

Olipudase alfa is currently being investigated to treat non-CNS manifestations of ASMD. Olipudase alfa has not been studied in ASMD type A patients.

Olipudase alfa is an investigational agent and the safety and efficacy have not been evaluated by the FDA, EMA, or any other regulatory authority worldwide.”

https://www.sanofi.com/en/media-room/press-releases/2021/2021-12-06-14-00-00-2346501#

FDA Expands Authorization of Two Monoclonal Antibodies for Treatment and Post-Exposure Prevention of COVID-19

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FDA Expands Authorization of Two Monoclonal Antibodies for Treatment and Post-Exposure Prevention of COVID-19

December 03, 2021: “The U.S. Food and Drug Administration revised the emergency use authorization (EUA) of bamlanivimab and etesevimab (previously authorized for pediatric patients 12 years of age and older weighing at least 40 kilograms, or about 88 pounds), to additionally authorize bamlanivimab and etesivimab administered together for the treatment of mild to moderate COVID-19 in all younger pediatric patients, including newborns, who have a positive COVID-19 test and are at high risk for progression to severe COVID-19, including hospitalization or death.

This revision also authorizes bamlanivimab and etesevimab, to be administered together, for post-exposure prophylaxis for prevention of COVID-19 in all pediatric patients, including newborns, at high risk of progression to severe COVID-19, including hospitalization or death. 

“Now all patients at high risk of severe COVID-19, including children and newborn babies, have an option for treatment and post-exposure prevention. Children under one year of age who are exposed to the virus that causes COVID-19 may be at particularly high risk for severe COVID-19 and this authorization addresses the medical needs of this vulnerable population,” said Patrizia Cavazzoni, M.D., director of the FDA’s Center for Drug Evaluation and Research.

“While today’s authorization includes post-exposure prevention of COVID-19 in children, this therapeutic option is not a substitute for vaccination. Vaccines remain our best tool in the fight against the virus and there is a COVID-19 vaccine authorized for children 5 years of age and above.”

Monoclonal antibodies are laboratory-made proteins that mimic the immune system’s ability to fight off harmful pathogens, such as viruses.

Bamlanivimab and etesevimab are monoclonal antibodies that are specifically directed against the spike protein of SARS-CoV-2, designed to block the virus’ attachment and entry into human cells.

Bamlanivimab and etesevimab bind to different but overlapping sites on the spike protein of the virus.

In February, the FDA originally authorized bamlanivimab and etesevimab administered together to treat mild-to moderate COVID-19 in adults and pediatric patients (12 years of age or older weighing at least 40 kg) with positive results of direct SARS-CoV-2 viral testing, and who are at high-risk for progressing to severe COVID-19 and/or hospitalization.

In September, the agency authorized its use for post-exposure prevention of COVID-19 in certain adults and pediatric individuals (12 years of age and older weighing at least 40 kg) who are at high-risk for progression to severe COVID-19, including hospitalization or death.

To support today’s action, bamlanivimab and etesevimab, administered together, were studied in a clinical trial of 125 pediatric patients (14 adolescent patients received placebo), all with at least one risk factor for severe COVID-19, to evaluate the safety and pharmacokinetics of treatment in pediatric patients.

Patients weighing less than 40 kg (88 pounds) received doses of bamlanivimab and etesevimab adjusted for their body weight, to achieve comparable exposures to adults and adolescents receiving the authorized dose.

Given the similar course of COVID-19 disease, the authorization of bamlanivimab and etesevimab in younger pediatric patients, including neonates, is supported by safety and efficacy data in adolescents and adults, together with additional pharmacokinetic and safety data from the clinical trial in pediatric patients. 

Serious adverse events including hypersensitivity, anaphylaxis, and infusion-related reactions have been observed with bamlanivimab with and without coadministration of etesevimab. Possible side effects of bamlanivimab and etesevimab administered together include nausea, dizziness, pruritus, and rash. 

The FDA is working with sponsors of all currently authorized therapeutics to assess the activity against any global SARS-CoV-2 variant(s) of interest and is committed to communicating with the public as we learn more.

Under the EUA, fact sheets that provide important information about the emergency use of bamlanivimab and etesevimab, to be administered together, must be made available to health care providers and to patients, parents and caregivers.

These fact sheets include dosing instructions, potential side effects and drug interactions. 

The EUA was issued to Eli Lilly and Co.”

https://www.fda.gov/news-events/press-announcements/fda-expands-authorization-two-monoclonal-antibodies-treatment-and-post-exposure-prevention-covid-19

NIHR supported study publishes COVID-19 booster data

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NIHR supported study publishes COVID-19 booster data

December 03, 2021: “Six COVID-19 vaccines are safe and boost immunity for people who have had two doses of AstraZeneca or Pfizer-BioNTech, results from the UK-wide COV-BOOST trial show.

The world-first study was key to shaping the UK booster programme and gives vital evidence for global vaccination efforts.

The study, led by University Hospital Southampton, has had its latest results published in the Lancet.

COV-BOOST looked at the safety, immune responses and side-effects of seven vaccines when used as a third, booster jab. 

Run at 18 National Institute for Health Research-supported sites, the study saw 2,878 people aged 30 or over recruited.

Participants received one of these boosters 10-12 weeks after their initial two-dose vaccination with either AstraZeneca or Pfizer-BioNTech.

A control group was given a meningitis vaccine, to account for reactions not specific to the COVID-19 jabs. [1]

The seven vaccines trialled were:

  • AstraZeneca-Oxford
  • Pfizer-BioNTech
  • Moderna
  • Novavax
  • Valneva
  • Janssen
  • CureVac

Of these, only AstraZeneca, Pfizer-BioNTech, Janssen and Moderna are currently licensed for use in the UK. Half-doses of Pfizer-BioNtech, Novavax and Valneva were also tested.

Professor Saul Faust, trial lead and Director of the NIHR Clinical Research Facility, University Hospital Southampton NHS Foundation Trust (UHS), said:

“Our side effect data shows all seven vaccines are safe to use as a third dose, with acceptable levels of ‘reactogenicity’ – inflammatory side effects like injection site pain, muscle soreness, fatigue.

All seven boosted levels of spike protein antibodies significantly after two doses of AstraZeneca. However only six also did so after two doses of Pfizer-BioNTech (AstraZeneca, Pfizer-BioNTech, Moderna, Novavax, Janssen and CureVac).

There were also large variations in response with different boosters.

“It’s really encouraging that a wide range of vaccines, using different technologies, show benefits as a booster dose to either of these vaccines.

That gives confidence and flexibility in developing booster programmes here and globally, with other factors like supply chain and logistics also in play.”

There were large differences in spike protein antibody levels after 28 days across the vaccines. In people who had received two initial doses of AstraZeneca, these ranged from 1.8 times higher to 32.3 times higher with different booster vaccines.

For those who had received Pfizer-BioNTech initially, the range was 1.3 times higher to 11.5 times higher. Booster results were similar for those aged 30-69 years and those aged 70 years or older.

The study also looked at immune T-cell responses. T-cells are likely to be important in controlling disease severity, although their impact on overall protection or longevity of immunity is not yet known.

COV-BOOST reported T-cell responses in several combinations of initial and booster vaccines, however these were not predictable based on spike protein antibody levels.

Reactions to all seven vaccines were similar, with fatigue, headache, and injection site pain most often reported. These were more commonly reported by those aged 30-69. 912 of the 2,878 participants experienced a total of 1036 adverse events, only 24 of which were severe.

Prof Faust added: “It’s important to note two things about these results. First, they only relate to these vaccines as boosters to the two primary vaccinations, not how well they work as first and second doses.

Secondly, the data describe the immune response at 28 days, not vaccine effectiveness.

The relationship between that response and long-term protection is still poorly understood. We will be looking at the longer-term immune responses in COV-BOOST, conducting further tests at three months and one year after receiving boosters.

“We are also looking at whether a longer period between second and third doses improves response to the two of the booster vaccines. Several studies have shown this effect between first and second doses.

We’ve done that by giving some of our original control participants the booster at a later point, and we expect those results to be available in the new year.”

Professor Andrew Ustianowski, National Clinical Lead for the UK NIHR COVID Vaccine Research Programme said:

“Heading into the winter, and due to the emergence of the Omicron, the results from the COV-BOOST study are extremely timely and of national and international importance.

“Since the beginning of the pandemic the National Institute for Health Research and the NHS have been supported by the efforts and selflessness of study participants – helping us to identify the most effective vaccines and how they can be used flexibly to protect more people.

“We welcome the latest results from the study, and continue to support the COV-BOOST team with the further analysis of data which will help us understand the use of these vaccines as boosters long term.”

COV-BOOST was designed so that stored samples can used in evaluating these vaccines’ effectiveness in neutralising any new variants of concern, and COV-BOOST samples have been made available to UKHSA for testing against omicron.”

https://www.nihr.ac.uk/news/nihr-supported-study-publishes-covid-19-booster-data/29454

WHO welcomes historic decision by Gavi to fund the first malaria vaccine

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WHO welcomes historic decision by Gavi to fund the first malaria vaccine

December 06, 2021: “WHO welcomes the historic decision by the Gavi Alliance Board to invest in the first malaria vaccine programme.

The decision secures investment in the long-awaited malaria vaccine and assures that many more children at risk will benefit from this life-saving vaccine and additional malaria prevention.

The Gavi Board  approved an investment to support the malaria vaccine introduction, procurement and delivery for Gavi-eligible countries in sub-Saharan Africa in 2022-2025. 

An initial investment of US$ 155.7 million for 2022-2025 will initiate the implementation of this additional tool to “help drive down child mortality in Africa,” according to Gavi.

In response to the decision, Minister of Health of Ghana and Gavi Board member Hon. Kwaku Agyeman-Manu said in the announcement: “Ghana, together with several countries on the continent, is proud to have been involved in the pilot program and the development of the first approved malaria vaccine, and today we welcome the decision made by the Gavi board to invest in the malaria vaccine programme.

We must now work together to ensure children across the continent can benefit from this additional malaria intervention.”

“This global investment is another milestone for the first malaria vaccine, which will boost child survival and extend the reach of malaria prevention through the existing platform of childhood vaccination,” said Dr Kate O’Brien, Director of the Department of Immunization, Vaccines and Biologicals.

“This international financing of malaria vaccine doses for country implementation is a concrete step forward to increase access to the RTS,S malaria vaccine.”

The WHO recommendation for the RTS,S malaria vaccine and this follow-on decision was achieved through the support and contributions of WHO across many departments and all levels of the Organization, the Ministries of Health in the pilot countries of Ghana, Kenya and Malawi, African-based evaluation partners, UNICEF, PATH, GSK, the Malaria Vaccine Implementation Programme (MVIP) funders (Gavi, the Global Fund and Unitaid) and other international and country-level public and private partners.

Interest in the malaria vaccine in endemic countries is high and demand for the vaccine is expected to outpace the currently limited supply.

Current vaccine production estimates are for up to 15 million doses per year; however, demand is estimated at more than 80 million doses annually.

“The significance of these two announcements – first, the WHO recommendation, and second, Gavi’s decision to open a funding window for the vaccine – is truly historic,” said Dr Pedro Alonso, Director of the Global Malaria Programme.

“We need new tools to reach our global malaria targets. And now, for the first time, we have a malaria vaccine that we estimate can save an additional 40 000 to 80 000 lives of African children each year.

This represents a scientific and public health breakthrough.”

WHO and partners are committed to finding approaches and taking actions to accelerate vaccine availability to increase vaccine access and reduce child illness and deaths.”

https://www.who.int/news/item/06-12-2021-who-welcomes-historic-decision-by-gavi-to-fund-the-first-malaria-vaccine

FDA Proposes Changes to Food Safety Modernization Act Rule to Enhance Safety of Agricultural Water Used on Produce

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FDA Proposes Changes to Food Safety Modernization Act Rule to Enhance Safety of Agricultural Water Used on Produce

December 02, 2021: “The U.S. Food and Drug Administration issued a proposed rule that aims to enhance the safety of produce.

It proposes to require farms to conduct comprehensive assessments that would help them identify and mitigate hazards in water used to grow produce.

This is the latest step in the agency’s implementation of the FDA Food Safety Modernization Act (FSMA), and it proposes to replace some of the existing requirements for agricultural water in the Produce Safety Rule (PSR).

“There have been far too many foodborne illness outbreaks possibly linked to pre-harvest agricultural water in recent years, including water coming from lands nearby produce farms.

As a federal government agency charged with protecting public health, the FDA is committed to implementing effective modern, science-based measures designed to prevent these outbreaks from occurring in the future,” said Frank Yiannas, FDA Deputy Commissioner for Food Policy and Response.

“The proposed rule is the latest action taken by the FDA to continue working towards implementation of key provisions of FSMA.

If finalized, we’re confident this proposal would result in fewer outbreaks in the U.S. related to produce, protecting public health and saving lives.

This proposed rule is a monumental step towards further improving the safety of the fruits and vegetables Americans serve their families every day, and the FDA looks forward to engaging with stakeholders on the proposed changes.”

The proposed rule, if finalized, would change certain pre-harvest agricultural water requirements for produce and farms subject to the PSR, other than sprouts operations. Key provisions in the proposed rule include:

  • A requirement for farms to manage their agricultural water quality based on the results of a comprehensive systems assessment (“agricultural water assessment”) that is adaptable to the wide variety of water sources and uses and future scientific advancements. 
  • An annual assessment by farms of their pre-harvest agricultural water to identify any conditions likely to introduce hazards into, or onto, covered produce or food contact surfaces.
    Based on these assessments, farms would then determine whether corrective or mitigation measures are reasonably necessary to reduce the potential for contamination.
    The assessment would include an evaluation of the farm’s water system, agricultural water use practices, crop characteristics, environmental conditions and other relevant factors, such as the results of any testing conducted to inform the assessment. 
  • A requirement that farms implement expedited mitigation measures for hazards related to certain activities associated with adjacent and nearby lands, to protect the quality of the water used on produce.

    This is being included following several recent outbreak investigations on produce that revealed potential routes of contamination including activities and conditions, such as animal grazing and the presence of livestock and wildlife on land adjacent to, or near, produce farms or their water sources. 
  •  The removal of certain testing requirements for pre-harvest agricultural water and replacing them with the agricultural water assessments identified above.

    The proposed revisions are intended to address stakeholder concerns about complexity and practical implementation challenges while protecting public health. 

The FDA intends to continue working closely with stakeholders and our state and tribal partners to provide necessary training, technical assistance, education and outreach.

The agency will hold two virtual public meetings to discuss the proposal and hear feedback, and more details will be announced in a forthcoming Federal Register notice.

In addition, the agency is also developing an online tool to assist growers in understanding agricultural water assessments.

Recognizing that the current agricultural water compliance dates for covered produce other than sprouts under the PSR are set to begin in January 2022, the agency intends to exercise enforcement discretion for those agricultural water requirements while pursuing another proposed rule to extend the compliance dates for all of the agricultural water requirements in the PSR for such covered produce.

More information on the proposed compliance date extension will be announced in a forthcoming Federal Register notice.

Today’s proposal is one of the critical remaining pieces of working towards FSMA implementation.

The FDA has taken many important steps to achieve the food safety goals envisioned by Congress when FSMA was established in 2011, such as implementing seven foundational rules.

The FDA also has developed multiple action plans to address specific food safety issues and has further built on the foundation under FSMA through the New Era of Smarter Food Safety initiative.”

https://www.fda.gov/news-events/press-announcements/fda-proposes-changes-food-safety-modernization-act-rule-enhance-safety-agricultural-water-used

EMA accepts regulatory submission for olipudase alfa, the first potential therapy for ASMD

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EMA accepts regulatory submission for olipudase alfa, the first potential therapy for ASMD

December 06 2021: “The European Medicines Agency (EMA) has accepted for review under an accelerated assessment procedure the Marketing Authorization Application (MAA) for olipudase alfa, Sanofi’s investigational enzyme replacement therapy which is being evaluated for the treatment of acid sphingomyelinase deficiency (ASMD).

Historically referred to as Niemann-Pick disease (NPD) type A and type B, ASMD is a rare, progressive, and potentially life-threatening disease for which no treatments are currently approved. The estimated prevalence of ASMD is approximately 2,000 patients in the U.S., Europe (EU5 Countries) and Japan.

If approved, olipudase alfa will become the first and only therapy for the treatment of ASMD.

“Olipudase alfa represents the kind of potentially life-changing innovation that is possible when industry, medical professionals and the patient community work together toward a common goal.”

The MAA is based on positive results from two separate clinical trials (ASCEND and ASCEND-Peds) evaluating olipudase alfa in adult and pediatric patients with non-central nervous system (CNS) manifestations of ASMD type A/B and ASMD type B.

Olipudase alfa has received special designations from regulatory agencies worldwide, recognizing the innovation potential of the investigational therapy.

“Scientific innovation is the greatest source of hope for people living with diseases like ASMD where there are no approved treatments and is a critical component for ensuring a viable healthcare ecosystem,” said Bill Sibold, Executive Vice President of Sanofi Genzyme. 

“At Sanofi, we have a long history of pioneering scientific innovation, and we remain committed to finding solutions to address unmet medical needs, including those of the rare disease community.”

The EMA awarded olipudase alfa the PRIority MEdicines designation, also known as PRIME, intended to aid and expedite the regulatory process for investigational medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options.

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to olipudase alfa.

This designation is intended to expedite the development and review of drugs intended to treat serious or life-threatening diseases and conditions.

The criteria for granting Breakthrough Therapy designation include preliminary clinical evidence indicating that the molecule may demonstrate substantial improvement on a clinically significant endpoint over available therapies.

In Japan, olipudase alfa was awarded the SAKIGAKE designation, which is intended to promote research and development in Japan for innovative new medical products that satisfy certain criteria, such as the severity of the intended indication.

In September, Sanofi filed the J-NDA submission for olipudase alfa.

About ASMD

ASMD results from a deficient activity of the enzyme acid sphingomyelinase (ASM), which is found in special compartments within cells called lysosomes and is required to breakdown lipids called sphingomyelin.

If ASM is absent or not functioning as it should, sphingomyelin cannot be metabolized properly and accumulates within cells, eventually causing cell death and the malfunction of major organ systems.

The deficiency of the lysosomal enzyme ASM is due to disease-causing variants in the sphingomyelin phosphodiesterase 1 gene (SMPD1).

The estimated prevalence of ASMD is approximately 2,000 patients in the U.S., Europe (EU5 Countries) and Japan.

ASMD represents a spectrum of disease caused by the same enzymatic deficiency, with two types that may represent opposite ends of a continuum sometimes referred to as ASMD type A and ASMD type B.

ASMD type A is a rapidly progressive neurological form of the disease resulting in death in early childhood due to central nervous system complications.

ASMD type B is a serious and potentially life-threatening disease that predominantly impacts the lungs, liver, and spleen, as well as other organs.

ASMD type A/B represents an intermediate form that includes varying degrees of neurologic involvement.

Patients with ASMD type A/B or ASMD type B were studied in the ASCEND trial program. Another type of NPD is NPD type C, which is unrelated to ASMD.

About olipudase alfa

Olipudase alfa is an investigational enzyme replacement therapy designed to replace deficient or defective ASM, allowing for the breakdown of sphingomyelin.

Olipudase alfa is currently being investigated to treat non-CNS manifestations of ASMD. Olipudase alfa has not been studied in ASMD type A patients.

Olipudase alfa is an investigational agent and the safety and efficacy have not been evaluated by the FDA, EMA, or any other regulatory authority worldwide.”

https://www.sanofi.com/en/media-room/press-releases/2021/2021-12-06-14-00-00-2346501

AstraZeneca reinforces leadership in breast cancer at SABCS 2021

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AstraZeneca reinforces leadership in breast cancer at SABCS 2021

December 03, 2021: “AstraZeneca will underscore its ambition to redefine care with new data from across its portfolio of innovative medicines at the 2021 San Antonio Breast Cancer Symposium (SABCS) 7-10 December 2021.

Fourteen AstraZeneca medicines and potential new medicines from the pipeline will be featured across 33 abstracts showcasing the Company’s leadership across different types and stages of breast cancer, including three oral presentations.

Breast cancer is now the most diagnosed cancer worldwide with an estimated 2.3 million people diagnosed in 2020.

Susan Galbraith, Executive Vice President, Oncology R&D, AstraZeneca, said: “Continuing our year of breakthroughs in breast cancer, our data at SABCS will reinforce the practice-changing potential of Enhertu with new analyses from the DESTINY-Breast03 trial.

Early data from the BEGONIA and TROPION-PanTumor01 trials demonstrate great promise in treating patients who have limited treatment options.

These data build on our decades of experience in pioneering medicines to redefine care for patients.”

Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said: “Progress in breast cancer increasingly involves more personalised approaches to treating patients across subtypes and stages of disease, which is a key focus of our data at this year’s SABCS.

Our extensive knowledge of breast cancer disease biology and the patient experience fuel our ambition to deliver medicines that can truly revolutionise and reshape treatment for every type of breast cancer patient.”

Transforming the treatment of advanced breast cancers with antibody drug conjugates (ADCs)
An oral presentation will share further results from a range of patient subgroups from the DESTINY-Breast03 Phase III trial, including those with stable brain metastases and patients characterised by hormone receptor status, number of prior lines of therapy or status of visceral metastasis.

Results from DESTINY-Breast03 demonstrated superior progression-free survival (PFS) for Enhertu (trastuzumab deruxtecan) versus trastuzumab emtansine (T-DM1) in patients with HER2-positive breast cancer previously treated with trastuzumab and a taxane.

In another oral presentation, updated results from the TROPION-PanTumor01 Phase I trial will continue to build promising evidence of the anti-tumour activity of datopotamab deruxtecan in patients with triple-negative breast cancer (TNBC).

Treating breast cancer early where there is more opportunity for cure
New data on patient quality of life from the OlympiA Phase III trial of Lynparza (olaparib) will be presented as an oral presentation.

These patient-reported outcomes data will provide compelling evidence that further supports Lynparza as a potential treatment option for the adjuvant treatment of patients with germline BRCA-mutated (gBRCAm) high-risk HER2-negative early breast cancer.

The supplemental New Drug Application of Lynparza for this indication was recently granted Priority Review by the US Food and Drug Administration.

Changing the treatment landscape with next-generation medicines and novel combinations
A poster and spotlight poster discussion will share results from the BEGONIA Phase Ib/II trial testing Imfinzi (durvalumab) combinations in advanced/metastatic TNBC with data from arm 1 (Imfinzi plus paclitaxel), arm 2 (Imfinzi, paclitaxel and capivasertib) and arm 5 (Imfinzi, paclitaxel and oleclumab), which will further demonstrate the benefits of combining immune checkpoint inhibitors with other novel molecules.

Additionally, ongoing trials posters will share information about the ongoing SERENA-4 Phase III trial which evaluates our next-generation oral selective oestrogen receptor degrader (SERD) camizestrant (AZD9833) in combination with CDK4/6 inhibitors in the 1st-line treatment of patients with oestrogen receptor (ER)-positive, HER2-negative advanced breast cancer, and the SERENA-6 Phase III trial of camizestrant with CDK4/6 inhibitors in patients with hormone receptor (HR)-positive, HER2-negative metastatic breast cancer with an ESR1 mutation.

A further ongoing trial poster will share information on the CAPItello-292 Phase III trial, evaluating the benefit of adding capivasertib (an AKT inhibitor) to the treatment regimen of Faslodex (fulvestrant) and palbociclib in patients with HR-positive, HER2-negative locally advanced, unresectable or metastatic breast cancer.

Enhertu and datopotamab deruxtecan are developed and commercialised in collaboration with Daiichi Sankyo worldwide, except in Japan where Daiichi Sankyo maintains exclusive rights. Lynparza is developed and commercialised in collaboration with MSD (Merck & Co., Inc. in the US and Canada).

Key AstraZeneca presentations during SABCS 2021

Lead authorAbstract titlePresentation details
Enhertu (trastuzumab deruxtecan)
Hurvitz STrastuzumab deruxtecan (T-DXd; DS-8201a) vs. trastuzumab emtansine (T-DM1) in patients with HER2+ metastatic breast cancer (mBC): results of the randomized phase 3 study DESTINY-Breast03Presentation GS3-01Oral – General Session 39 December, 202108:45 – 11:30 CT14:45 – 17:30 GMT
Vaz Batista MTrastuzumab deruxtecan in patients with HER2[+] or HER2-low–expressing advanced breast cancer and central nervous system involvement: Preliminary results from the DEBBRAH phase 2 study [IIS]Publication PD4-06Spotlight Poster Discussion 48 December, 202117:00 – 18:30 CT23:00 – 00:30 (+1) GMT
Datopotamab deruxtecan (Dato-DXd)
Krop IDatopotamab deruxtecan (Dato-DXd) in Advanced/Metastatic Human Epidermal Growth Factor Receptor 2 Negative (HER2−) Breast Cancer: Results From the Phase 1 TROPION-PanTumor01 Study [J101 TNBC prelim results]Presentation GS1-05Oral presentation – General Session 17 December, 202108:00 – 10:45 CT14:00 – 16:45 GMT
Lynparza (olaparib)
Ganz PAQuality of life results from OlympiA: A phase III, multicenter, randomized, placebo-controlled trial of adjuvant olaparib after (neo)-adjuvant chemotherapy in patients with germline BRCA1/2 mutations and high risk HER-2 negative early breast cancerPresentation GS4-09Oral – General Session 410 December, 202108:45 – 11:15 CT14:45 – 17:15 GMT
Imfinzi (durvalumab)
Schmid PBEGONIA: Phase 1b/2 study of durvalumab (d) combinations in locally advanced/metastatic triple-negative breast cancer (TNBC): Results from arm 1 d + paclitaxel (p), arm 2 d + p + capivasertib (c), and arm 5 d + p + oleclumab (o)Publication PD10-03Spotlight Poster Discussion 109 December, 202117:00 – 18:30 CT23:00 – 00:30 (+1) GMT
Camizestrant (AZD9833)
André FSERENA-4: A Phase III comparison of AZD9833 (camizestrant) plus palbociclib, versus anastrozole plus palbociclib, for patients with ER-positive/HER2-negative advanced breast cancer who have not previously received systemic treatment for advanced diseasePublication OT2-11-06Ongoing Trials Poster Session 29 December, 202117:00 – 18:30 CT23:00 – 00:30 (+1) GMT
Bidard FCSERENA-6: A Phase III study to assess the efficacy and safety of AZD9833 (camizestrant) compared with aromatase inhibitors when given in combination with palbociclib or abemaciclib in patients with HR+/HER2- metastatic breast cancer with detectable ESR1m who have not experienced disease progression on first-line therapyPublication OT2-11-05Ongoing Trials Poster Session 29 December, 202117:00 – 18:30 CT23:00 – 00:30 (+1) GMT
Faslodex (fulvestrant) and capivasertib
Rugo HSCAPItello-292: A phase Ib/III study of capivasertib, palbociclib and fulvestrant, versus placebo, palbociclib and fulvestrant, for endocrine therapy-resistant HR+/HER2− advanced breast cancerPublication OT2-14-01Ongoing Trials Poster Session 29 December, 202117:00 – 18:30 CT23:00 – 00:30 (+1) GMT

Notes

AstraZeneca in breast cancer
Driven by a growing understanding of breast cancer biology, AstraZeneca is starting to challenge, and redefine, the current clinical paradigm for how breast cancer is classified and treated to deliver even more effective treatments to patients in need – with the bold ambition to one day eliminate breast cancer as a cause of death.

AstraZeneca has a comprehensive portfolio of approved and promising compounds in development that leverage different mechanisms of action to address the biologically diverse breast cancer tumour environment.

AstraZeneca aims to continue to transform outcomes for HR-positive breast cancer with foundational medicines Faslodex and Zoladex and the next-generation oral SERD and potential new medicine camizestrant.

PARP inhibitor Lynparza is a targeted treatment option for metastatic breast cancer patients with an inherited BRCA mutation.

AstraZeneca with MSD continue to research Lynparza in metastatic breast cancer patients with an inherited BRCA mutation and are exploring new opportunities to treat these patients earlier in their disease.

Building on the first approval of Enhertu, a HER2-directed ADC, in previously treated HER2-positive metastatic breast cancer, AstraZeneca and Daiichi Sankyo are exploring its potential in earlier lines of treatment and in new breast cancer settings.

To bring much needed treatment options to patients with triple-negative breast cancer, an aggressive form of breast cancer, AstraZeneca is testing immunotherapy Imfinzi in combination with other oncology medicines, including Lynparza and Enhertu, evaluating the potential of AKT kinase inhibitor, capivasertib, in combination with chemotherapy, and collaborating with Daiichi Sankyo to explore the potential of TROP2-directed ADC, datopotamab deruxtecan.”

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2021/astrazeneca-reinforces-leadership-in-breast-cancer-at-sabcs-2021-with-new-data-underscoring-ambition-to-redefine-cancer-care.html

Novartis focused on medicines company delivering strong operational performance

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Novartis focused on medicines company delivering strong operational performance

December 2, 2021: “Novartis holds an investor event to provide a comprehensive overview of the company’s progress in advancing its industry-leading R&D engine.

Vas Narasimhan, CEO of Novartis, said “Novartis has transformed to become a focused medicines company, building depth in our core therapeutic areas and strength across key technology platforms.

We expect to continue delivering strong operational performance, with 4%+ CAGR through to 2026*, driven by the momentum of our multi-billion dollar in-market growth drivers.

Up to 20 new assets with significant sales potential could be approved by 2026, which will fuel the next phase of growth and address major unmet needs.

We are building the foundation for long-term differential growth by investing in advanced technology platforms and data science.

Novartis remains disciplined and shareholder focused in its capital allocation priorities, as we continue to deliver on our strategy”.

New announcements at R&D Day 2021:

Cosentyx, our largest medicine by sales, showed topline results in moderate to severe hidradenitis suppurativa (HS), a potential new indication. 

Two Phase 3 studies (SUNRISE and SUNSHINE) met their primary endpoint, with more patients treated with Cosentyx achieving a HS Clinical Response (HiSCR), compared with placebo, at week 16.

The safety of Cosentyx in HS was consistent with the therapy’s known safety profile. The trials are ongoing to 52 weeks and are expected to complete in H2 2022. Regulatory filings are planned for 2022.

Novartis presents T-Charge™, a next generation CAR-T cell therapy platform, expected to increase CAR-T potency and have important process efficiencies to reduce turnaround time.

In first-in-human trials to be presented at ASH 2021, lead candidates YTB323 and PHE885 showed 75% Complete Response in Diffuse Large B-Cell Lymphoma (DLBCL) at three months and 100% Best Overall Response (BOR) in multiple myeloma, respectively.

Novartis is developing T-Charge™ as the foundational platform for a wave of potentially transformative CAR-T cell therapies.2,3

Phase 3 study starts planned or ongoing across 5 core therapeutic areas include:

  • Cardio-Renal: Leqvio (CVRR-LDL-C), pelacarsen (CVRR-Lp(a)), iptacopan (C3G; IgAN) 
  • IHD: Cosentyx (HS; GCA; lupus nephritis), ligelizumab (CSU; food allergy; CINDU), ianalumab (Sjögren’s syndrome), remibrutinib (CSU)
  • Neuroscience: Zolgensma (SMA IT), remibrutinib (MS)
  • Oncology: Kisqali (HR+/HER2- BC adjuvant), 177Lu-PSMA-617 (mCRPC, pretaxane; mHSPC), canakinumab (adjuvant NSCLC), NIS793 (PDAC), JDQ443 (NSCLC, 2/3L)
  • Hematology: Iptacopan (aHUS; PNH), Scemblix® (CML 1L), sabatolimab (HR-MDS), and YTB323 (2L DLBCL)

(for abbreviations, see below)

Novartis announces a global co-development and co-commercialization agreement with UCB to bring disease-modifying therapies to people living with Parkinson’s Disease. 

The agreement covers UCB0599, a potential first-in-class, small molecule, alpha-synuclein misfolding inhibitor currently in Phase 2 clinical development.

In addition, upon completion of the ongoing Phase 1 program, there is an opt-in to co-develop UCB7853, an anti-alpha-synuclein antibody.

Both assets could transform care for 10 million people living with Parkinson’s Disease worldwide given the lack of disease-modifying therapies.4,5

Novartis also provides a comprehensive overview of its mid- and late-stage pipeline assets in five core therapeutic areas, highlighting: Leqvio, pelacarsen, iptacopan, Cosentyx, ligelizumab, remibrutinib, ianalumab, LNA043, branaplam, Zolgensma, Kisqali, 177Lu-PSMA-617, sabatolimab, JDQ443, TNO155, Scemblix and NIS793.

Additionally, Novartis highlights the continued expansion of its pipeline and capabilities in advanced technology platforms that are expected to drive multiple waves of biopharmaceutical innovation. These include: T-Charge™, Targeted Protein Degradation, Cell Therapy, Gene Therapy, Radioligand Therapy and xRNA.”

https://www.novartis.com/news/media-releases/novartis-rd-day-spotlights-attractive-growth-profile-underpinned-strong-market-brands-20-potential-high-value-pipeline-assets-and-technology-platforms

FDA Proposes Changes to Food Safety Modernization Act Rule to Enhance Safety of Agricultural Water Used on Produce

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FDA Proposes Changes to Food Safety Modernization Act Rule to Enhance Safety of Agricultural Water Used on Produce

December 02, 2021: “The U.S. Food and Drug Administration issued a proposed rule that aims to enhance the safety of produce.

It proposes to require farms to conduct comprehensive assessments that would help them identify and mitigate hazards in water used to grow produce.

This is the latest step in the agency’s implementation of the FDA Food Safety Modernization Act (FSMA), and it proposes to replace some of the existing requirements for agricultural water in the Produce Safety Rule (PSR).

“There have been far too many foodborne illness outbreaks possibly linked to pre-harvest agricultural water in recent years, including water coming from lands nearby produce farms.

As a federal government agency charged with protecting public health, the FDA is committed to implementing effective modern, science-based measures designed to prevent these outbreaks from occurring in the future,” said Frank Yiannas, FDA Deputy Commissioner for Food Policy and Response.

“The proposed rule is the latest action taken by the FDA to continue working towards implementation of key provisions of FSMA.

If finalized, we’re confident this proposal would result in fewer outbreaks in the U.S. related to produce, protecting public health and saving lives.

This proposed rule is a monumental step towards further improving the safety of the fruits and vegetables Americans serve their families every day, and the FDA looks forward to engaging with stakeholders on the proposed changes.”

The proposed rule, if finalized, would change certain pre-harvest agricultural water requirements for produce and farms subject to the PSR, other than sprouts operations.

Key provisions in the proposed rule include:

  • A requirement for farms to manage their agricultural water quality based on the results of a comprehensive systems assessment (“agricultural water assessment”) that is adaptable to the wide variety of water sources and uses and future scientific advancements. 
  • An annual assessment by farms of their pre-harvest agricultural water to identify any conditions likely to introduce hazards into, or onto, covered produce or food contact surfaces.

    Based on these assessments, farms would then determine whether corrective or mitigation measures are reasonably necessary to reduce the potential for contamination.

    The assessment would include an evaluation of the farm’s water system, agricultural water use practices, crop characteristics, environmental conditions and other relevant factors, such as the results of any testing conducted to inform the assessment. 
  • A requirement that farms implement expedited mitigation measures for hazards related to certain activities associated with adjacent and nearby lands, to protect the quality of the water used on produce.

    This is being included following several recent outbreak investigations on produce that revealed potential routes of contamination including activities and conditions, such as animal grazing and the presence of livestock and wildlife on land adjacent to, or near, produce farms or their water sources. 
  •  The removal of certain testing requirements for pre-harvest agricultural water and replacing them with the agricultural water assessments identified above.

    The proposed revisions are intended to address stakeholder concerns about complexity and practical implementation challenges while protecting public health. 

The FDA intends to continue working closely with stakeholders and our state and tribal partners to provide necessary training, technical assistance, education and outreach.

The agency will hold two virtual public meetings to discuss the proposal and hear feedback, and more details will be announced in a forthcoming Federal Register notice.

In addition, the agency is also developing an online tool to assist growers in understanding agricultural water assessments.

Recognizing that the current agricultural water compliance dates for covered produce other than sprouts under the PSR are set to begin in January 2022, the agency intends to exercise enforcement discretion for those agricultural water requirements while pursuing another proposed rule to extend the compliance dates for all of the agricultural water requirements in the PSR for such covered produce.

More information on the proposed compliance date extension will be announced in a forthcoming Federal Register notice.

Today’s proposal is one of the critical remaining pieces of working towards FSMA implementation.

The FDA has taken many important steps to achieve the food safety goals envisioned by Congress when FSMA was established in 2011, such as implementing seven foundational rules.

The FDA also has developed multiple action plans to address specific food safety issues and has further built on the foundation under FSMA through the New Era of Smarter Food Safety initiative.”

https://www.fda.gov/news-events/press-announcements/fda-proposes-changes-food-safety-modernization-act-rule-enhance-safety-agricultural-water-used

MHRA grants conditional marketing authorisation for COVID-19 treatment

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MHRA grants conditional marketing authorisation for COVID-19 treatment

December 02, 2021: “GlaxoSmithKline and Vir Biotechnology announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted a conditional marketing authorisation for Xevudy (sotrovimab) for the treatment of symptomatic adults and adolescents (aged 12 years and over and weighing at least 40 kg) with acute COVID-19 infection who do not require oxygen supplementation and who are at increased risk of progressing to severe COVID infection. 

It is recommended that Xevudy is administered within 5 days of onset of symptoms of COVID-19.

In the coming weeks, UK patients at increased risk of severe complications of COVID-19 will be able to access sotrovimab following a supply agreement reached earlier with the UK government.  

The Conditional Marketing Authorisation covers England, Scotland, and Wales.  Supply of sotrovimab in Northern Ireland is enabled under the regulation 174 of the Human Medicines Regulations 2012,

George Katzourakis, Senior Vice President, Europe, GSK said: “The conditional marketing authorisation in Great Britain, coupled with the supply agreement, is a testament to the critical need to make sotrovimab available in the UK as the pandemic continues to progress.  

We believe it is important to ensure that we have treatments ready and available, especially early treatment options, for a broad group of patients at increased risk of progressing to severe COVID-19.”

George Scangos, PhD, Chief Executive Officer of Vir, said: “We are pleased with the UK Government’s recognition of the role that monoclonal antibodies, like sotrovimab, have to play in addressing this pandemic.

With clinical data demonstrating a 79% reduction in hospitalisations for more than 24 hours or death in non-hospitalised patients, we are confident sotrovimab will continue to be a critical tool in the fight against COVID-19.

We look forward to making sotrovimab available to the NHS and patients in the UK and to continuing our efforts to increase access worldwide.”

The design of sotrovimab, a monoclonal antibody, is based on the natural antibodies humans already make.

In clinical trials sotrovimab was shown to significantly reduce the risk of hospitalisation or death among high-risk adult outpatients with mild to moderate COVID-19.

The final analysis from the COMET-ICE trial demonstrated a 79% reduction (adjusted relative risk reduction  95% CI, p<0.001; sotrovimab (6/528 [1%]) vs placebo (30/529 [6%]) in hospitalisation for more than 24 hours or death due to any cause (by Day 29) compared to placebo.

Sotrovimab has been well tolerated in clinical studies conducted to date. The most common adverse reactions are hypersensitivity reactions, seen in approximately 2% of cases.

GSK and Vir are committed to ongoing evaluation of sotrovimab as the COVID-19 landscape continues to evolve at different rates across the globe and new variants of concern and interest emerge.

Sotrovimab binds to an epitope on SARS-CoV-2 that is shared with SARS-CoV-1 (the virus that causes SARS), indicating that the epitope is conserved, which may make it more difficult for resistance to develop.

This indicates potential for sotrovimab to be effective across multiple variants of concern.

Updated in vitro data, published in bioRxiv (the pre-print server for biology) demonstrate that sotrovimab retains activity against all current tested variants of concern and interest of the SARS-CoV-2 virus as defined by the World Health Organisation (WHO), plus others, including, but not limited to, Delta (B.1.617.2), Delta Plus (AY.1 or AY.2) and Mu (B.1.621).

Based on the sequence of the Omicron variant, we believe sotrovimab is likely to maintain activity and potency against this variant

This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. “

https://www.gsk.com/en-gb/media/press-releases/mhra-grants-conditional-marketing-authorisation1-for-covid-19-treatment-xevudy-sotrovimab/

GSK welcomes Gavi decision to fund the roll out of malaria vaccines for children

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GSK welcomes Gavi decision to fund the roll out of malaria vaccines for children

 December 02, 2021: “GlaxoSmithKline welcomed the decision of the Gavi Board to provide funding for the procurement and introduction of malaria vaccines into routine child immunisation programmes in Gavi eligible countries.

Today’s announcement follows shortly after the World Health Organization (WHO) recommendation for the broader use of GSK’s RTS,S/AS01e to reduce childhood illness and deaths from malaria in children living in sub-Saharan Africa and other regions with moderate to high transmission as defined by WHO.

Africa continues to bear the heaviest malaria burden and African children are at highest risk of dying of malaria: more than 260,000 African children under the age of five died from malaria in 2019. Covid has increased this disease burden and made the need for new tools to tackle malaria even more urgent.

GSK’s RTS,S/AS01e is the first and only malaria vaccine to have been shown in pivotal long-term clinical trials to significantly reduce malaria in children.

The vaccine is the result of over 35 years of research led by GSK, with PATH and other partners.

Thomas Breuer, Chief Global Health Officer, GSK, said: “Our malaria vaccine is a new and important tool that is urgently needed given rising numbers of malaria cases in African children. The vaccine has the potential to have a significant impact on the burden of malaria in Sub-Saharan Africa.

Gavi’s funding decision is a critical next step in ensuring access to the malaria vaccine for children who need it, by creating visibility around potential demand for the vaccine, as well as local implementation needs.

We have been a proud partner of Gavi since it was founded in 2000 and look forward to continuing to work with Gavi and other partners to develop solutions to ensure sustainable access to the malaria vaccine.”

Malaria vaccine implementation programmes (MVIP) started in 2019 in 3 countries (Ghana, Kenya and Malawi) and have already shown high impact in real-life childhood vaccination settings and strong community demand, with more than 800,000 children having received at least 1 dose of the RTS,S/AS01e vaccine.

In August, new data from a study of 6,000 children by the London School Hygiene and Tropical Medicine, indicated that the use of the RTS,S/AS01e vaccine in combination with seasonal malaria chemoprevention (SMC) can further reduce mortality in settings with highly seasonal malaria transmission.

GSK has committed to supply up to 15 million RTS,S/AS01e doses annually, in addition to the 10 million doses already produced and donated for use in the pilot programmes in Malawi, Kenya and Ghana.

GSK has also partnered with Gavi and MedAccess to develop an innovative financing solution to ensure continued manufacturing of the vaccine antigen ahead of key decisions by WHO and Gavi.

To secure long term supply, a product transfer is also underway with Bharat Biotech of India, which will become the sole supplier of the vaccine in 2029.

GSK will continue to supply the adjuvant (AS01e) to Bharat.”

https://www.gsk.com/en-gb/media/press-releases/gsk-welcomes-gavi-decision-to-fund-the-roll-out-of-malaria-vaccines-for-children/

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