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Nexviadyme (avalglucosidase alfa) approved by European Commission as a potential new standard of care for the treatment of Pompe Disease

Approved for the treatment of the full spectrum of both late-onset Pompe disease and infantile-onset Pompe disease

First new treatment option approved for the Pompe community in Europe in more than 15 years

Paris, June 28, 2022 The European Commission has granted marketing authorization for Nexviadyme (avalglucosidase alfa), an enzyme replacement therapy (ERT) for the long-term treatment of both late-onset and infantile-onset Pompe disease, a rare, progressive and debilitating muscle disorder. Nexviadyme is the first and only newly approved medicine for Pompe disease in Europe since 2006, when the European Commission authorized the marketing of alglucosidase alfa, branded Myozyme.

Benedikt Schoser, MD.Senior Consultant and Professor, Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University MunichThe approval of Nexviadyme in Europe to treat Pompe disease is backed by a robust body of evidence showing clinically meaningful improvements that can impact quality of life. The totality and rigor of the data is particularly noteworthy given the complexities of research and development for such a rare and progressive condition. Nexviadymes demonstrated clinical benefit and molecular innovation bring a new treatment option to people living with Pompe disease who continue to face unmet needs.

Addressing an unmet need for people living with Pompe disease

Pompe disease can present as infantile-onset Pompe disease (IOPD), the most severe form of the disease with rapid onset in infancy, or late-onset Pompe disease (LOPD), which progressively damages muscles over time. If left untreated, IOPD can lead to heart failure and death within the first year of life, while people with LOPD may require mechanical ventilation to help with breathing or a wheelchair to assist with mobility as the disease progresses.

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Nexviadyme now approved in many countries around the world

Nexviadyme is approved in multiple markets around the world for the treatment of certain people living with Pompe disease, including the European Union, the United States, Japan, Canada, Switzerland, Australia, Brazil, Taiwan and the United Arab Emirates. Outside of Europe, the treatment is marketed under the brand name Nexviazyme. In the U.S. and Japan, the majority of the Myozyme (alglucosidase alfa)-treated population has started, or is in the process of starting, treatment with Nexviazyme (avalglucosidase alfa).

In November 2021, Sanofi announced that as part of the European Medicines Agencys (EMA) review of Nexviadyme, the Committee for Medicinal Products for Human Use (CHMP) issued an opinion that the therapy does not qualify as a New Active Substance (NAS). In April 2022, the Committee for Orphan Medicinal Product (COMP) also recommended Nexviadyme be removed from the Community Register of Orphan Medicinal Products (OMP).

Sanofi strongly disagrees with both opinions and believes these conclusions were the result of an erroneous and very narrow interpretation of the NAS and OMP principles that demonstrate molecular innovation and clinical benefit. Sanofi stands by the totality of data in support of Nexviadyme as a potential new standard of care and is concerned that such narrow interpretation will undermine rare disease incentive mechanisms in Europe. We believe withholding these distinct designations could negatively impact patient health in Europe by restricting access to innovative advancements in care.

Bill SiboldExecutive Vice President, Specialty Care, SanofiFor more than two decades, weve been working with the community and leveraging our scientific expertise to improve care for people living with Pompe disease. We strongly believe in the meaningful clinical benefits of this medicine as a new standard of care and will work hard to ensure the broadest possible access in Europe despite the European Commissions failure to recognize Nexviadymes NAS and OMP designations. We call on patient advocacy groups, policymakers, clinicians and patients to join us in our efforts to ensure innovative treatments are appropriately recognized and made available to patients in Europe and beyond.

Nexviadyme, a new ERT for late-onset Pompe disease and infantile-onset Pompe Disease

Positive outcomes in key disease burden measures

In a robust clinical development program, Nexviadyme demonstrated clinically meaningful differences in key areas of disease burden for people living with late-onset Pompe disease and infantile-onset Pompe disease.

Results from the COMET study comparing Nexviadyme to alglucosidase alfa in LOPD at 49 weeks included:

Patients treated with Nexviadyme showed a 2.9% improvement from baseline (SE=0.9) in forced vital capacity (FVC) percent-predicted, a key measure of respiratory function and the studys primary endpoint, which was 2.4% points greater as compared to the change with alglucosidase alfa. This difference exceeded the non-inferiority margin (p=0.0074; 95% CI, -0.13, 4.99). Statistical superiority was narrowly missed (p=0.06).

Patients treated with Nexviadyme walked 32.2 meters farther (SE=9.9) compared to baseline in the 6-minute walk test (6MWT), a key secondary endpoint, which was 30 meters farther (p=0.040; 95% CI, 1.33, 58.69) than the change with alglucosidase alfa. Formal statistical testing for all secondary endpoints was not conducted.

Results from the Mini-COMET study evaluating Nexviadyme in IOPD patients showed improvement or stabilization at six months in efficacy outcomes, the trials secondary objective, of gross motor function measure (GMFM-88), quick motor function test (QMFT), pediatric evaluation of disability index (Pompe-PEDI), left ventricular mass z-score (LVMZ), and eyelid position measurements in patients previously declining or insufficiently controlled with alglucosidase alfa.

A pooled safety analysis from four clinical studies found serious adverse reactions reported in patients treated with Nexviadyme included chills (1.4%), headache, dyspnoea, respiratory distress, nausea, skin discoloration, chest discomfort, pyrexia, blood pressure increased, body temperature increased, heart rate increased, and oxygen saturation decreased (0.7% each). Additionally, hypersensitivity reactions (43.5%), anaphylaxis (1.4%) and infusion-associated reactions (26.1%) were reported. The most frequently reported adverse drug reactions (ADRs) (>5%) were pruritus (9.4%), rash (8%), headache (7.2%), urticaria (6.5%), fatigue (6.5%), nausea (5.8%), and chills (5.1%).

Mechanism of action designed for increased uptake

People living with Pompe disease have low levels of the enzyme acid alpha-glucosidase (GAA), which results in build-up of glycogen, leading to irreversible damage to skeletal and cardiac muscles. Nexviadyme is specifically designed to target the mannose-6-phosphate (M6P) receptor, the key pathway for cellular uptake of ERT and transport to the lysosome, and has an average 15-fold higher level of M6P moieties as compared to alglucosidase alfa. Nexviadyme aims to help improve uptake and enhance glycogen clearance in target tissues as compared to alglucosidase alfa, which was used as the comparator arm in the pivotal Phase 3 COMET study.

About SanofiWe are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve peoples lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions.

Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY

Media RelationsSandrine Guendoul | + 33 6 25 09 14 25 | sandrine.guendoul@sanofi.comSally Bain|+ 1 617 834 6026 |sally.bain@sanofi.comKate Conway|+ 1508364 4931 |kate.conway@sanofi.com

Investor RelationsEva Schaefer-Jansen | + 33 7 86 80 56 39 | eva.schaefer-jansen@sanofi.comArnaud Delpine | + 33 06 73 69 36 93 | arnaud.delepine@sanofi.comCorentine Driancourt | + 33 06 40 56 92 | corentine.driancourt@sanofi.comFelix Lauscher | + 1 908 612 7239 | felix.lauscher@sanofi.comPriya Nanduri |+1 617 764 6418 | priya.nanduri@sanofi.comNathalie Pham | + 33 07 85 93 30 17 | nathalie.pham@sanofi.com

Sanofi Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates regarding the marketing and other potential of the product, or regarding potential future revenues from the product. Forward-looking statements are generally identified by the words expects, anticipates, believes, intends, estimates, plans and similar expressions. Although Sanofis management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, unexpected regulatory actions or delays, or government regulation generally, that could affect the availability or commercial potential of the product, the fact that product may not be commercially successful, the uncertainties inherent in research and development, including future clinical data and analysis of existing clinical data relating to the product, including post marketing, unexpected safety, quality or manufacturing issues, competition in general, risks associated with intellectual property and any related future litigation and the ultimate outcome of such litigation, and volatile economic and market conditions, and the impact that COVID-19 will have on us, our customers, suppliers, vendors, and other business partners, and the financial condition of any one of them, as well as on our employees and on the global economy as a whole. Any material effect of COVID-19 on any of the foregoing could also adversely impact us. This situation is changing rapidly and additional impacts may arise of which we are not currently aware and may exacerbate other previously identified risks. The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under Risk Factors and Cautionary Statement Regarding Forward-Looking Statements in Sanofis annual report on Form 20-F for the year ended December 31, 2021. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements.

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Press Release: Nexviadyme (avalglucosidase alfa) approved by European Commission as a potential new standard of care for the treatment of Pompe...

EMA has recommended granting a conditional marketing authorisation in the European Union (EU) for Roctavian (valoctocogene roxaparvovec) for the treatment of severe haemophiliaA in adults who do not have factorVIII inhibitors (auto-antibodies produced by the immune system which make factor VIII medicines less effective) and no antibodies to adeno-associated virus serotype 5 (AAV5).

Patients with haemophiliaA cannot produce factorVIII (an essential protein required for blood to clot and stop bleeding); they are more prone to bleeding and have prolonged bleeding, e.g. after injury or surgery. Haemophilia A is a rare debilitating disease affecting approximately 0.7 in 10,000 people in the EU. It is life long and may be life threatening when bleeding occurs in the brain, the spinal cord or the gut.

Medicines currently authorised for treating haemophilia A mostly contain factor VIII, to replace the missing protein. Available treatments require one or more injections per week or per month and are lifelong. Therefore, there is an unmet medical need for new therapeutic approaches that might free patients from frequent injections.

Roctavian is the first gene therapy to treat haemophilia A. The active substance in Roctavian, valoctocogene roxaparvovec, is based on a virus (adeno-associated virus or AAV) which has been modified to not cause disease in humans. The virus contains the gene for factor VIII; once given to a patient as a one-off infusion, it is expected to carry the factor-VIII gene into the liver cells, enabling them to produce the missing factor VIII. This helps the blood to clot more easily and prevents bleeding or reduces bleeding episodes. It is yet unknown how long the treatment effect from this single infusion will last in an individual patient. A sustained positive treatment effect of up to two years following a single infusion has been reported in approximately one hundred patients in the main study and up to five years in a few patients in a supportive trial conducted by the applicant. Longer-term follow-up tests may be required to verify a continued safe and effective response to the medicine.

EMAs recommendation is based on the results ofa Phase 3 single-arm (main study), non-randomised study in 134 male patients with haemophilia A without a history of factor VIII inhibitor and without detectable pre-existing antibodies to AAV5. Two years after the administration, efficacy data showed that the therapy significantly increased factor VIII activity levels in the majority of patients. Bleeding rates were reduced by 85% and most patients (128) no longer needed factor VIII replacement therapy.

Hepatotoxicity (liver damage), a common side effect due to immune reaction induced by these AAV-based gene therapies and characterised so far by an increase in the levels of a liver enzyme called alanine aminotransferase (ALT), has been reported with Roctavian. The condition can be treated successfully with corticosteroids. Other common side effects include headache, joint pain and nausea.

Patients treated with Roctavian will be monitored for 15 years, to ensure the long-term efficacy and safety of this gene therapy.

Roctavian was supported through EMA's PRIority MEdicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support to medicines that have a particular potential to address patients' unmet medical needs.

In its overall assessment of the available data, the Committee for Advanced Therapies (CAT), EMA's expert committee for cell- and gene-based medicines, found that the benefits of Roctavian outweighed the possible risks in patients with haemophilia A.

The CHMP, EMAs human medicines committee, agreed with the CATs assessment and positive opinion, and recommended approval of this medicine.

The opinion adopted by theCHMPis an intermediary step on Roctavians path to patient access. The opinion will now be sent to the European Commission for the adoption of a decision on an EU-widemarketing authorisation. Once amarketing authorisationhas been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role or use of this medicine in the context of the national health system of that country.

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First gene therapy to treat severe haemophilia A | European Medicines Agency - European Medicines Agency |

A villager transporting food aid in Kandahar province, Afghanistan, in April.Credit...Bryan Denton for The New York Times

Road blocks, roadside bombs, ambushes and kidnapping were the kinds of threats aid workers operating in Afghanistan faced for two decades during the Talibans insurgency. Parts of the country were off limits entirely as the conflict ebbed and flowed, and even on open roads the risk of gun battles remained real.

Last August, however, the Taliban stormed to power, effectively ending the war and easing security for millions of residents as well as for humanitarian workers.

There is no active conflict going on now, said Babar Baloch, a spokesman for the U.N. refugee agency who started working in the country more than two decades ago. We do have more access.

That access should make it easier to bring relief after the deadliest earthquake in two decades killed at least 1,000 people and injured more than 1,600 others.

We are rushing in supplies for the earthquake and for other humanitarian emergencies, Mr. Baloch said.

But even as access has improved, other developments since last August have sent humanitarian needs soaring and brought unforeseen problems, aid workers say.

Most notable is the loss of financial, technical and security assistance from Western governments that were desperate to see the country emerge as a democracy after the Taliban was toppled in 2001.

That flood of aid more than doubled the nations annual per capita gross domestic product, from around $200 in 2001 to around $500 last year, according to World Bank figures. But those gains were not spread evenly rural areas saw less change than cities such as the capital, Kabul, and the southern city of Kandahar.

The economy has contracted sharply since the Taliban took power. Investors and foreign governments pulled back and many skilled workers fled, while the United States and other Western countries froze government bank accounts now controlled by the Taliban.

As the country struggles with drought and hunger, half of Afghanistans roughly 40 million people need humanitarian aid, the United Nations said in January as it asked member nations for more than $5 billion for the people of Afghanistan, as governments struggle with how to support a population in need while avoiding helping the Taliban itself.

Part of the Afghanistan population is already in a humanitarian crisis, with people buying expired bread that is normally fed to animals, the U.N. Resident Coordinator in Afghanistan, Ramiz Alakbarov, said on Wednesday, referring to trends in the country over the past year. This does add to the burden of dealing with the earthquake.

In addition, aid workers face an institutional challenge rarely seen in other countries: Coordinating operations with officials who were long part of an insurgent force, but are now attempting to transition into a full-fledged government.

In one measure of the dynamic, Abdul Qahar Balkhi, the spokesman for the Afghan Ministry of Foreign Affairs, asked those wishing to help from any part of the world to donate to the countrys arm of the Red Crescent, which would deliver aid to victims in full transparency.

See the article here:
At Least 1,000 Killed in Afghanistan Earthquake, Officials Say

The military ranks of Afghanistan were the military insignia used by militaries of Afghanistan throughout history.

Following the return of the Taliban into power, the Islamic Emirate Armed Forces continue to use the rank insignia of the Islamic Republic Armed Forces.

The rank insignia of commissioned officers.

The rank insignia of non-commissioned officers and enlisted personnel.

The rank insignia of commissioned officers.

The rank insignia of non-commissioned officers and enlisted personnel.

The rank insignia of commissioned officers.

The rank insignia of non-commissioned officers and enlisted personnel.

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Military ranks of Afghanistan - Wikipedia

KABUL, June 25 (Reuters) - Vital medical supplies reached hospitals on Saturday in the remote area of Afghanistan hit by an earthquake that killed more than 1,000 people this week, as the country's Taliban government appealed for more international aid.

Authorities have called off the search for survivors in the mountainous southeastern region near the Pakistani border following's Wednesday's 6.1-magnitude quake, which also injured about 2,000 people and damaged or destroyed 10,000 homes.

Aftershocks on Friday killed at least five more people in the area some 160 km (100 miles) southeast of the capital Kabul, and medical staff said rudimentary healthcare facilities were hampering their efforts to help the injured.

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"Those injured that were in a bad condition and needed operations, (which) we can't do here, have been sent to Kabul," said Abrar, who goes by one name, the manager of a hospital in Paktika, the worst-affected province.

In Kabul, hospitals more used to treating victims of war have opened their wards to earthquake victims.

Usually we admit only war related patients or patients in life threatening conditions, but in this case we decided to make an exception in order to support the Afghan people, said Stefano Sozza, the country director for Emergency Hospital, an Italian-funded surgical centre for war victims.

One of the patients, a woman from Gayan district of Paktika, whose name Reuters is withholding for security reasons, said nine members of her family had died in the earthquake.

Afghan men try to retrieve a car from the debris of damaged houses after the recent earthquake in Wor Kali village in the Barmal district of Paktika province, Afghanistan, June 25, 2022. REUTERS/Ali Khara

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"Just I remain," she said. "My legs are broken, we have nothing; we eat what the Taliban give us."

The disaster is a major test for Afghanistan's hardline Taliban rulers, who have been shunned by many foreign governments due to concerns about human rights since they seized control of the country last year.

Afghanistan has been cut off from much direct international assistance because of Western sanctions, deepening a humanitarian crisis in swaths of the country even before this week's earthquake.

The United Nations and several other countries have rushed aid to the affected areas, with more due to arrive over the coming days, and the Taliban appealed on Saturday for further aid shipments to help quake victims.

"We call on all humanitarian organizations to help the people," said Mohammad Amen Hozifa, a spokesperson for the Paktika provincial government.

China's Foreign Ministry said on Saturday the nation would provide humanitarian aid worth 50 million yuan ($7.5 million) to Afghanistan including tents, towels, beds and other materials to help those affected by the earthquake. read more

The UN's migration agency said on Saturday it had begun distributing thousands of emergency shelters and hygiene kits in affected areas.

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Reporting by Mohammad Yunus Yawar and Charlotte Greenfield in KabulAdditional reporting by Emma Farge in GenevaWriting by Alasdair PalEditing by Helen Popper

Our Standards: The Thomson Reuters Trust Principles.

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Taliban appeal for more aid after deadly Afghanistan earthquake