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European Union has, at least by territory and population, expanded as the European Council overwhelmingly decided to grant Moldova and Ukraine, with the possibility of Georgia, candidates status to join the bloc. Current, the European Union consists of 27 members and has an estimated total population of about 447 million. Moldova, Ukraine and Georgia, all former Soviet republics, will together add approximately 50.8 million to the current population of the European Union.

As former Soviet republics, the three attained their political independence and within the international laws, must be considered with respect based on the principles of their territorial integrity and national sovereignty. While the granting them their new status after official requests from them, it has indeed sparked debates especially in the Russian Federation.

European Union leaders have formally agreed to grant candidate status to Ukraine, as well as Moldova, although the two former Soviet republics face a long path before joining the bloc. Ukraine applied to join the bloc just days after the Russian invasion on 24 February, and the process from application to candidacy has gone through at record speed.

Undoubtedly the new status has opened wide, most possibly, better doors and a platform to spring up with economic development through integration into European Union. President of the European Council, Charles Michel, noted: it is a historic moment, today marks a crucial step on your path towards the European Union. Our future is together.

The official congratulated the leaders of Ukraine and Moldova. Regarding Georgia, the European Council decided to recognize the European perspective of Georgia and is ready to grant candidate status once the outstanding priorities are addressed, Michel said. Congratulations to the Georgian people, he said. A historic moment in EU-Georgia relations: Georgias future lies within the EU.

The European Commission on June 17 recommended that the summit grant a candidate status to Ukraine and Moldova. It is a symbol of hope to support the Ukrainians while the country had a long way to go before actual accession. A few days later, Speaker of Moldova Parliament, Igor Grosu, announced that Moldova ready to join new sanctions, mostly in finance and banking, against Russia.

We will show solidarity with the EU, as our status and European aspirations oblige us. Of course, we will join [any new sanctions] meant to stop the military operation. We are seeking to contribute to this goal by any diplomatic means, Grosu said following a decision by the EU.

Moldovas Deputy Prime Minister and Minister of Foreign Affairs, Nicu Popescu earlier said the East European nation could not fully join anti-Russian sanctions due to its weak economy. European Union candidate status now provides Moldova with access to worlds most developed market. It offers similar new economic opportunities to bothe Ukraine and Georgia.

In one of her warm-hearted illuminating speeches at a media briefing, President Maia Sandu emphasized: Candidate country status gives us a clear direction of our development, support on this path, and most importantly, hope. We are a small and vulnerable country, which would feel more secure when it becomes part of the European family, in which we could count on support from all members and institutions. Belonging to the EU also means access to the richest and the most developed market in the world.

Moldova, however, expects more support from the European Union to improve the wellbeing of its people and provide preconditions for developing the business environment. The situation will not change overnight after candidate status has been granted, as a lot of hard work is still ahead, Sandu said, attributing the current hardships in Moldova to the conflict in Ukraine that began late February.

Ukraines President Volodymyr Zelensky hailed the news as a unique and historic moment, adding Ukraines future is within the EU while the French President Emmanuel Macron said that the decision by EU leaders sent a very strong signal to Russia that Europeans support Ukraines pro-Western aspirations.

At least, they have joined the European family that offers practical warmth for sustainable development. Ukraine has already signed an agreement with the European Union on joining its LIFE Program, an international funding instrument for the environment and climate action, whose budget on environment protection projects for 2021-2027 amounts to 5.43 billion, Ukrainian media reported with reference to the Environment Protection and Natural Resources Ministry.

Ukrainian Environment Protection Minister Ruslan Strilets and European Commissioner for Environment, Oceans, and Fisheries Virginijus Sinkevicius signed the agreement.The ministry has over 15 concrete proposals to be transformed into relevant projects to be presented for consideration under LIFE Program.

Ukraine has received great support and colossal capabilities from the European Union for restoring not only the environment but also live nature in Ukraine. This is something for which there has always been a lack of funding. LIFE is a powerful financial tool of the participating countries. This means great confidence in Ukraine, Strilets said. This should help us develop more new projects which local businesses could be engaged with. Therefore, weve made a very important step today.

In the near future Moldova, Ukraine and Georgia have the possibility to access the benefits from the Global Gateway, a new European strategy directed at boosting smart, clean and secure links in digital, energy and transport sectors and to strengthen health, education and research systems across the world.

It is in line with the commitment of the G-7 leaders from June 2021 to launch a values-driven, high-standard and transparent infrastructure partnership to meet global infrastructure development needs. The Global Gateway is also fully aligned with the UNs Agenda 2030 and its Sustainable Development Goals, as well as the Paris Agreement.

In addition, late June the he Group of Seven economic powers the U.S., Germany, France, the U.K., Italy, Canada and Japan made some progress in bringing their counterparts from their five guest countries closer to Western views on sanctions against Russia. The G-7 is committing themselves to support the new members especially Ukraine.

Ahead of his trip, Biden authorized another US$450 million in weaponry to be sent to Ukraine, bringing the total U.S. commitment to US$6.1 billion since the start of the war. Offering a concrete template, the G-7 combined are aiming to invest US$600 billion in public and private capital for infrastructure projects over the next five years, with US$200 billion of that total coming from the United States.

According to European lawmakers interviewed by local Russian media Izvestia, this step has broad support from the EU. Meanwhile, Russia views the move ambiguously. On the one hand, it sees EU membership as tantamount to striving for NATO, on the other hand, European integration is a purely economic issue and does not raise any concerns.

Well see, well analyze the consequences, former Deputy Foreign Minister Grigory Karasin told Izvestia. The context is important; it is not as harmless as it might have seemed three years ago. Decisions are being made amid a sanctions offensive and against everything Russian, he added.

That being said, the European Union noted that obtaining candidate status is only the first step towards membership. Engin Eroglu, a member of the Committee on Foreign Affairs in the European Parliament, in an interview with Izvestia said that the process of gaining membership to the EU does not mean automatic entry, but it means that the country has started pro-European processes and reforms, which are partially financed by Brussels.

The granting of candidate status to Ukraine and Moldova has angered other countries that have been striving to join the European Union for several years now. For example, the European Commission has so far denied this status to Georgia, the newspaper writes.

Tbilisi, to put it mildly, was not happy about the refusal, but this will not be a reason for any deterioration in relations between the European Union and Georgia, Head of the Department of Integration Studies at Moscow State University of International Relations (MGIMO) Nikolay Kaveshnikov told Izvestia.

Russia consistently expresses fierce opposition to this European membership over the past several years. President Vladimir Putin had declared Ukraine to be part of Moscows sphere and insisted he was acting due to attempts to bring the country into NATO, the Western alliance that comes with security guarantees.

Granting Ukraine and Moldova candidate status to join the European Union looks like nothing more than a scam by the West, according to Russian Foreign Ministry Spokeswoman Maria Zakharova. Scam is such a wonderful word, seeing that the numerous decisions taken by the West are more like combination of a destructive, provocative nature, rather than well-thought-out steps, the diplomat said, speaking to the Sputnik Radio.

I think thats certainly their case, she added, Given these maneuvers, these zigzags that we now are witnessing from the West with regards to Moldova, Ukraine, and Georgia, it is no longer necessary to prove anything in terms of market conditions. There is a direct link between economics and politics. And this is exactly what they have always stood against. She described the actions by the European Union as infringement of Russias territorial integrity, and as encroachment on former Soviet space and territory.

On the distinctive opposite side, Russia sees no risks for itself in the fact that Ukraine and Moldova have been granted EU candidate status, Russian Foreign Minister Sergey Lavrov said at a press conference following talks with his Azerbaijani counterpart Jeyhun Bayramov on June 24 in Baku.

Our position has always been that the European Union is not a political bloc, unlike NATO. The development of its relations with any countries that wish to do so does not create any threats and risks for us, Lavrov said in reply to a media question. Of course, we will realistically consider the European Unions behavior and monitor the real steps it takes and how the candidate countries act: whether they comply with these requirements or still try to show their independence.

These new European Union members have some strategic significance. Moldova is a landlocked country in Eastern Europe. It shares borders with Romania to the west and Ukraine to the north. Ukraine, with a coastline along the Black Sea and the Sea of Azov to the south and southeast, respectively could be used for economic benefits by the European Union.

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European Union Could Share Its Solid Economic Benefits With Moldova, Ukraine And Georgia OpEd - Eurasia Review

Iranian foreign minister Hossein Amirabdollahian and European Union (EU) representative for foreign affairs Josep Borrellannounced during a joint press conference in Tehran over the weekend that Iran would renew negotiations over its nuclear program with the United States, which have stalled since March following animpasse over political disputes between Washington and Tehran.

The two diplomatsindicated that they had held a long but positive discussion on Saturday, after which Amirabdollahian agreed to the resumption of indirect discussions between Iran and the United States, emphasizing that Irans primary concern was returning to the sanctions relief promised under the original deal.

Whatever issue that can impact Irans economic benefits will not be agreeable, Abdollahian said. We hope that the American side will this time realistically and fairly engage in committed and responsible acts towards reaching the final point of an agreement.

Borrell emphasized that the resumed discussions were intended to resolv[e] the last outstanding issues, without describing what those issues were.

In addition to his meeting with Amirabdollahian, Borrell and his deputy Enrique Mora met deputy foreign minister and chief nuclear negotiator Ali Bagheri Kani and Supreme National Security Council Secretary Ali Shamkhani.

Iran and the P5+1 nations fully cooperated in the Joint Comprehensive Plan of Action (JCPOA) from its inception in 2015 until 2018, when President Donald Trump unilaterally withdrew from the deal and imposed maximum pressure sanctions on Iran in an attempt to win better concessions. Although Iran initially continued to adhere to its obligations under the deal, it gradually withdrew from them as tensions rose between Iran and the West. Iran withdrew from the deal altogether after the assassination of Islamic Revolutionary Guard Corps (IRGC) Quds Force commander Qassem Soleimani in January 2020.

President Joe Biden and Iranian president Ebrahim Raisi have voiced willingness to return to the nuclear agreement and negotiations to this end have continued in Vienna, Austria, but political issues haveobstructed the finalization process as Irans nuclear program draws closer to completion.

In particular, the two sides have clashed over the U.S. designation of the IRGC, the branch of Irans military devoted to preserving the Iranian governments theocratic character, as a foreign terrorist organization, obstructing its ability to engage in international commerce. Because the designation was part of Trumps maximum pressure campaign, Iranian negotiators have pressed Biden to undo it. However, Biden has refused, partly due to powerful political opposition to the move on Capitol Hill.

Trevor Filseth is a current and foreign affairs writer for the National Interest.

Image: Reuters.

Link:
Iran and the European Union Agree to Resume Nuclear Talks - The National Interest Online

LONDON

British Prime Minister Boris Johnson signaled Monday that a plan to rip up parts of the post-Brexit trade deal he signed with the European Union could become law by the end of the year.

Lawmakers began debating legislation that rewrites trade rules for Northern Ireland on Monday, the first step on what could be a rocky journey through Parliament.

If approved by lawmakers, the legislation would remove checks on goods entering Northern Ireland from the rest of the U.K., thereby scrapping parts of a trade treaty that Johnson signed before Britain left the EU in 2020.

Johnson said he thought the plan could be approved fairly rapidly if Parliament cooperates. Asked if the measures could be in place this year, he said: Yes, I think we could do it very fast, Parliament willing.

The British government says the rules, known as the Northern Ireland Protocol, are burdening businesses and undermining peace in Northern Ireland. It argues the unilateral move is justified under international law because of the genuinely exceptional situation.

Johnsons opponents, however, say the move is illegal.

Many of us are extremely concerned that the bill brazenly breaks a solemn international treaty, it trashes our international reputation, it threatens a trade war at a time when our economy is flat and it puts us at odds with our most important ally, Conservative Andrew Mitchell said in Parliament Monday.

Foreign Secretary Liz Truss replied that the governments plans are the only solution to resolve trade problems in Northern Ireland because the EU has been unwilling to reopen negotiations.

The plans have caused concern among some of Johnsons fellow Conservatives, already worried about his judgment and popularity following a series of ethics scandals and two special election defeats.

But Johnson said Monday that questions about his leadership are no longer relevant.

We settled that a couple of weeks ago, he told reporters in Germany, referring to his survival in a no-confidence vote three weeks ago.

The EU has threatened to retaliate against the U.K. if it goes ahead with its plan to rewrite the rules of the post-Brexit deal, raising the specter of a trade war between the two major economic partners.

Irish Foreign Affairs Minister Simon Coveney said he was hugely disappointed that the U.K. government is still pursuing its unlawful approach to the Northern Ireland Protocol.

This is not the way to find sustainable solutions to the genuine concerns of people and business in Northern Ireland and only adds to uncertainty, he said.

The blocs ambassador to Britain, Joao Vale de Almeida, said Britains plan was illegal because it is a breach of international law, a breach of EU law, U.K. law and international law.

It is a treaty that we signed, ratified and even went through a general election in this country, he told Times Radio.

Northern Ireland is the only part of the U.K. that shares a border with an EU country, Ireland. When Britain left the European Union and its borderless free-trade zone, the two sides agreed to keep the Irish land border free of customs posts and other checks because an open border is a key pillar of the peace process that ended decades of violence in Northern Ireland.

Instead, to protect the EUs single market, there are checks on some goods, such as meat and eggs, entering Northern Ireland from the rest of the U.K.

Johnsons Conservative government claims overzealous EU implementation means the rules are not working as expected and are causing a political crisis in Northern Ireland.

You have got one tradition, one community, that feels that things really arent working in a way that they like or understand youve got unnecessary barriers to trade between Great Britain and Northern Ireland, Johnson said.

All we are saying is you can get rid of those whilst not in any way endangering the EU single market, he said.

British unionists there say the checks are fraying the bonds between Northern Ireland and the rest of the U.K., seen by some unionists as a threat to their British identity. Northern Irelands main unionist party is blocking the formation of a new power-sharing government in Belfast, saying it wont take part until the Brexit trade rules are scrapped.

I want to see the reestablishment of the Northern Ireland Assembly and the Executive, and the protocol is getting in the way of that, Lewis told Sky News. We have got to resolve that. Thats what this legislation will do.

Ultimately, we want to do this by agreement with the EU, he added. But to do that, they need to show some flexibility and actually come and negotiate in a flexible way.

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U.K. presses on with rewrite of Brexit rules while EU calls it illegal - Los Angeles Times

Sanofi - Aventis Groupe

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.

Notes:

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