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On 1 Aug. 2024, the European Artificial Intelligence Act (AI Act) came into force (1). The AI Act is designed to ensure that artificial intelligence (AI) developed and used in the European Union (EU) is trustworthy, and that there are safeguards in place to protect people’s fundamental rights. In January 2024, the European Commission (EC) launched a package of measures to support European small and medium enterprises (SMEs) and start-ups in the development of trustworthy AI, and in May, the EC established an AI Office (2,3). In July 2024, the amended European High Performance Computing Joint Undertaking (EuroHPC JU) Regulation came into force to further develop, deploy, extend, and maintain the EU supercomputing and data infrastructure (4). This paved the way for the establishment of AI Factories, dedicated AI supercomputers, that can be used for training General Purpose AI (GPAI) models (5).

This article was published in the September 2024 issue of 

The AI Act aims to harmonize AI across the EU to encourage the uptake of this technology and support innovation and investment. The AI Act also prohibits certain AI practices and sets out regulations on “high-risk” AI systems and those that pose transparency risks (

Figure 1. Definition of artificial intelligence (AI)-based product safety and risk-based approach. (Figure courtesy of the author)

The AI Act also introduces rules to reduce the systemic risk of systemic GPAI models that are capable of performing a wide range of distinct tasks such as the generation of human-like text (6). The new legislation imposes stricter regulation and fines on tech companies operating generative AI models (i.e., DeepMind and OpenAI will fall under the general-use AI category and would be classed as minimal risk). However, developers of high-risk models would be asked to evaluate models, assess and mitigate systemic risks, conduct adversarial testing, report serious incidents to the EC, ensure cybersecurity, and report on their energy efficiency (6).

The European Federation of Pharmaceutical Industries and Associations (EFPIA) welcomes the AI Act and “believes in the potential of applying AI to deliver benefit for patients, life-science companies, and society” (7). However, it stresses that “the regulatory frameworks governing the use of AI in research, development, and manufacture must be fit-for-purpose, risk-based, non-duplicative, globally aligned, and adequately tailored” (7). When AI-enabled tools are used in pharma R&D, they are exempt from the requirements of the EU AI Act; however, developers and deployers of AI-enabled digital health tools (DHTs) and AI-enabled digital medicine would be impacted by the AI Act, but they would not be considered high risk (8).

Enforcement of the AI rules will be phased in

EU Member States have a year to designate national competent authorities to oversee the application of the AI rules. The Commission’s AI Office will oversee the implementation of the AI Act at the EU level, and be responsible for enforcing the rules for GPAI models (1).

The EC will establish the following three advisory bodies to support the implementation of the rules:

European Artificial Intelligence Board—

to ensure the uniform application of the AI Act across EU Member States and to cooperate between the EC and the Member States.

A scientific panel of independent experts—

to offer technical advice and input on enforcement. The panel can issue alerts to the AI Office about risks associated with GPAI models.

a diverse set of stakeholders to offer guidance to the AI Office (1).

The AI Office has the power to enforce these policies. Companies that do not comply with the rules will be fined 1.5–7% of their global annual turnover depending on the violation (e.g., companies can be fined up to 1.5% for supplying incorrect information) (1).

The AI Act’s regulations will take effect in phases. Rules regarding prohibited practices will apply as of 2 Feb. 2025; obligations on GPAI models will apply as of 2 Aug. 2025; and both transparency obligations and obligations on high-risk AI systems will apply as of 2 Aug. 2026 (9).

In the interim, the EC launched the AI Pact to encourage and support organizations before implementing the AI Act (10). The EC has urged the industry to voluntarily commit to the AI Act requirements before the legal deadline. In November 2023, it obtained responses from more than 550 organizations, and the AI Office initiated the development of the AI Pact, based upon two pillars:

establish a gateway to engage the AI Pact network, encourage the exchange of best practices, and provide practical information on the AI Act implementation process

encourage AI system providers and deployers to prepare early and take actions towards compliance with requirements and obligations set out in the legislation.

The AI Pact has enabled the EC to work with stakeholders to support them in understanding the objectives of the AI Act, to adapt and prepare for the implementation of the AI Act, and to share knowledge and build trust in AI technologies (10).

Meanwhile, the Joint Research Centre continues to undertake independent, evidence-based research to support EU policies and determine the societal impact of the AI Act across Europe.

The EU AI Act sends a clear message to the rest of the world that Europe is taking AI very seriously and emphasises the importance of trust, transparency, and accountability. The EC is the first governmental body to create a legal infrastructure on AI and has paved the way for others to emulate and replicate.During the coming months, rules will be phased in and European standards will be refined. Therefore, companies need to work closely with the AI Office and other relevant authorities to ensure the safe deployment of AI solutions that will be beneficial to society and drive future innovation in Europe.

European Artificial Intelligence Act Comes into Force

Commission Launches AI Innovation Package to Support Artificial Intelligence Startups and SMEs

Commission Establishes AI Office to Strengthen EU Leadership in Safe and Trustworthy Artificial Intelligence

Setting Up of AI Factories is Now Possible after EuroHPC Regulation Amendment

The EU Passes the AI Act and its Implications for Digital Medicine Are Unclear

International Bar Association. The Regulation of Foundation Models in the EU AI Act. 

EFPIA Statement on the Use of AI in the Medicinal Product Lifecycle in the Context of the AI Act

Cheryl Barton, PhD,is founder and director of PharmaVision, Pharmavision.co.uk.


Vol. 36, No. 8
September 2024
Pages: 9-10

When referring to this article, please cite it as Barton, C. European Artificial Intelligence Act Comes into Force. 

Articles

What impact will the AI Act have on pharma and digital medicine?

Thermo Scientific’s DNAPac RP HPLC columns | Image courtesy of Thermo Fisher Scientific.

The Thermo Scientific SurePac Bio 550 SEC MDi columns enable fast separations of adeno-associated virus (AAV) monomers and aggregates. The columns are designed with 3 µm monodisperse particles and inert (MDi) column hardware aimed at providing superior AAV monomer retention and higher resolution of aggregates in cell and gene therapies. These columns offer fast run times, optimal separation efficiency, and reliable results with the goal of accelerating time-to-market for novel therapeutics. In addition, Thermo’s DNAPac RP HPLC columns are offered in semi-preparative formats to enable scalability in the development of DNA/RNA oligonucleotides. The DNAPac RP semi-preparative columns are designed so that laboratories can navigate the expanding field of oligonucleotide therapies and precision medicine. The DNAPac RP semi-preparative column utilizes unique chemistry for enhanced performance across wide ranges of pH, temperature, and mobile-phase compositions, offering precise separations and sample purification.

Thermo Scientific’s columns offer fast run times, optimal separation efficiency, and reliable results.

Separation and Purification Columns for Enhanced Retention

Freudenberg Medical’s Y-connector assemblies | Image courtesy of Freudenberg Medical.

Freudenberg Medical has added custom single-use assemblies to its product portfolio. The new offerings consist of, but are not limited to, single-use Y-connector manifolds, tubing assemblies, and bottle cap assemblies with multiple inlets/outlets. Bottlecap assemblies with Y-connector tubing are commonly used in labs and in current good manufacturing practice (CGMP) bioprocesses during critical fluid transfer applications. The single-use assemblies are cleanroom manufactured. The Y-connector overmolds offer unobstructed flow within a critical fluid path and can prevent leaks from occurring. Leaks are commonly seen with standard fluid couplings, fittings, and connectors. The expanded portfolio complements the company’s existing PharmaFocus Premium product line, which contains high purity, platinum-cured silicone tubing and sanitary ends for single-use systems. The company’s tubing, molded components, and systems are used in bioprocessing, biotechnology, lab processes, GMP manufacturing, and small batch processes as well as for the production of cell and gene therapies, messenger RNA, biosimilars, and regenerative proteins.

Freudenberg Medical’s new offerings include single-use Y-connector manifolds, tubing assemblies, and bottlecap assemblies, among others.

Custom Single-Use Assemblies

The ROSS CDA-300 Dual Shaft Mixer | Image courtesy of Charles Ross & Son Company.

The ROSS CDA-300 Dual Shaft Mixer includes two agitators: one is a two-wing anchor agitator that is driven by a 20 horsepower (HP) totally enclosed fan cooled (TEFC) high efficiency, inverter duty motor; the second agitator is a high-speed disperser that is powered by a 60 HP TEFC high efficiency, inverter duty motor. The mixer is designed with a disperser that has an open disc blade, capable of traveling roughly 5000 feet per minute to induce vigorous turbulent flow. This vigorous flow creates a vortex into which dry ingredients can be added for fast wetting. The change can on the CDA-300 features an air/oil hydraulic lift that allows the use of multiple mix cans constructed of stainless steel type 304. The mix cans include heating/cooling jackets on the sidewalls and bottom for temperature control. Easy discharge is enabled by a three-inch manually operated stainless-steel flush bottom ball valve with tri-clamp connection. The mixer can be easily moved around the production floor on casters. The mixer is designed for vacuum operation to produce air-free products with highly repeatable density, viscosity, composition, and dispersion quality. The NEMA 12 control panel includes a seven-inch color touch screen for user-friendly operation, while providing independent agitator speed controls, load display, cycle timer, and other functions as well as ensuring safety with limit switches that prevent operation in the absence of the mix can or when the agitators are not in the mixing position.

Charles Ross & Son Company has introduced the ROSS CDA-300 Dual Shaft Mixer, consisting of two agitators and a disperse with an open disc blade. 

Dual Shaft Mixer for High-Performance Applications

Asahi Kasei Medical’s virus removal filters | Image courtesy of Asahi Kasei Group

Asahi Kasei Medical’s Planova virus removal filters are used in the manufacturing processes of biopharmaceuticals and plasma derivatives. The filters are also used in biosafety testing services and biopharmaceutical contract development and manufacturing operations. Planova comprises cellulose hollow-fiber membrane filters that were developed to specifically remove viruses from biotherapeutic products. The line was launched in 1989 and was followed by Planova BioEX hydrophilic polyvinylidene fluoride hollow-fiber membrane filters in 2009. Both product lines have been adopted worldwide. The latest offering is Planova S20N, a next-generation line of cellulose hollow-fiber membrane filters, which was launched in 2022. This latest addition features robust virus removal capability and simplified operation.

Asahi Kasei Medical now offers its Planova virus removal filters for use in biopharma manufacturing and plasma derivatives manufacturing.

Virus Removal Filters

Patrick Lavery is an Editor for the MJH Life Sciences brands 

. Previously at MJH, he filled the same role for 

Science laboratory test tubes , laboratory equipment | Image Credit: © BillionPhotos.com - stock.adobe.com

Several academic departments at the University of Oxford have collaborated on a new and multifaceted way to combat substandard or falsified vaccines, the risk of which these researchers say is growing and poses a threat to global public health. The method they propose uses matrix-assisted laser desorption/ionization–mass spectrometry (MALDI-MS), which identifies components of a sample by giving the constituent molecules a charge and then separating them, combined with open-source machine learning to create a multi-component model that can differentiate between authentic and fake vaccines (1).

In a press release on Aug. 28, 2024, which summarized research recently published in the journal 

, the University of Oxford said the method could be carried out by clinical mass spectrometers already in use in hospitals worldwide, which would help to address what the researchers said was an “urgent need” for better monitoring of the global vaccine supply chain (1,2).

“This innovative research provides compelling evidence that MALDI-MS techniques could be used in accessible systems for screening for vaccine falsification globally, especially in centers with hospital microbiology laboratories, enhancing public health and confidence in vaccines,” said Paul Newton, study co-author and professor of tropical medicine at the Centre for Tropical Medicine and Global Health, also a part of the University of Oxford, in the press release.

The university’s Vaccine Identity Evaluation collaboration, which began during the early stages of the COVID-19 pandemic, was created to help crack down on both counterfeit vaccines and those considered substandard, usually due to improper temperature storage (3). Raman spectroscopy and rapid diagnostic testing are some of the other approaches the collective has considered in the last several years in addition to MALDI.

"We are thrilled to see the method’s effectiveness and its potential for deployment into real-world vaccine authenticity screening,” James McCullagh, study co-leader and professor of biological chemistry at Oxford, said in the press release. “This is an important milestone for the Vaccine Identity Evaluation consortium which focuses on the development and evaluation of innovative devices for detecting falsified and substandard vaccines, supported by multiple research partners including the World Health Organization, medicine regulatory authorities, and vaccine manufacturers."

Billions of vaccine doses are used every year in immunization campaigns across the world, according to the Oxford press release, and although the overwhelming majority are of excellent quality, those that are not can have serious health impacts not only for failing to treat the disease for which they were intended, but also causing death in some cases, and eroding public confidence in vaccines (1). The MALDI-MS method, the researchers said, was able to distinguish between legitimate vaccines for the likes of influenza, hepatitis B, and meningococcal disease, and solutions often used in falsified vaccines, such as sodium chloride.

“This latest research will bring the world community one step closer to being able to tell apart falsified, ineffective vaccines from the real thing, making us all safer,” said Nicole Zitzmann, study co-author and professor of virology, in the release.

1. University of Oxford. Scientists Develop New Method to Detect Fake Vaccines. Press Release. Aug. 28, 2024.
2. Clarke, R.; Bharucha, T.; Arman, B. Y.; et al. Using Matrix Assisted Laser Desorption Ionisation Mass Spectrometry Combined with Machine Learning for Vaccine Authenticity Screening. 


3. Centre for Global Health Research. Vaccine Identity Evaluation. 

The approach makes use of clinical mass spectrometers that are already available in hospitals around the world.

University of Oxford Chemists Develop Method for Vaccine Authenticity Screening

Text sign showing Industry News. Business photo text delivering news to the general public or a target public | Image Credit: © Artur - stock.adobe.com

AstraZeneca is making an investment of approximately US $135 million, or the equivalent of 1.375 billion Swedish krona, in its Sweden Biomanufacturing Center in Södertälje, as announced in a press release* on Aug. 26, 2024 (1).

The money will help expand the functional area of the Södertälje campus by 50%, adding 2700 square meters to an existing building. Once the completion of the expanded facility is finalized, AstraZeneca said, manufacturing of products for patients will begin in the new section, which could be as early as the fourth quarter of 2027 (1).

This additional investment is the largest singular tranche of funding for the facility since it opened in 2021, according to AstraZeneca.

With biological drugs accounting for more than 50% of the projects in AstraZeneca’s current research portfolio, the Sweden Biomanufacturing Center was designed with an eye toward matching the rapidly growing share of biological medicines on the market, the press release said, as well as meeting increased internal production capacity needs (1). One of the areas the money from this investment will address is installing new equipment for filling drugs into prefilled syringes.

Per Alfredsson, CEO of AstraZeneca AB and senior vice president for global biologics operations, said in the press release that the company was happy about its investment in Södertälje, which he said demonstrated AstraZeneca's commitment to innovation and manufacturing of biological medicines in Sweden, adding that the planned expansion would strengthen the company’s ability to deliver high-quality, life-changing medicines to patients worldwide (1).

The announcement comes amid a busy few months for the Cambridge, UK-based company on the international stage. In May 2024, AstraZeneca said it was planning a new, $1.5 billion USD antibody drug conjugate facility in Singapore, for which construction would begin by the end of 2024 with the goal of full operation sometime in 2029 (2). And in July 2024, the company completed its $1.05 billion USD purchase originally announced in March 2024 of Amolyt Pharma, a clinical-stage biotechnology company which had developed treatments for rare endocrine diseases, strengthening a rare disease late-stage pipeline in tandem with its 2021 acquisition of Alexion (3).

AstraZeneca said the new investment in the Sweden Biomanufacturing Center not only stands to eventually benefit millions of patients worldwide, but also aligns with the company’s commitment to job creation and economic growth in the Södertälje region—yet while the press release said new jobs would be created as a result of the expansion made possible by this round of funding, no estimate was given as to when or how many job opportunities would be made available.

*Ed. note: The Aug. 26 AstraZeneca press release was originally written in Swedish, and was translated to English for purposes of this report.

1. AstraZeneca. AstraZeneca is Investing in the Expansion of the Sweden Biomanufacturing Center in Södertälje. Press Release. Aug. 26, 2024.
2. AstraZeneca. AstraZeneca Plans $1.5 Billion Manufacturing Facility for Antibody Drug Conjugates (ADCs) in Singapore. Press Release. May 20, 2024.
3. AstraZeneca. AstraZeneca to Acquire Amolyt Pharma, Expanding Late-Stage Rare Disease Pipeline. Press Release, March 14, 2024.

This is the company’s largest single investment in the Sweden Biomanufacturing Center, in Södertälje, which opened in 2021.

AstraZeneca Makes Additional $135 Million Investment in Swedish Biologics Facility

Feliza Mirasol is the science editor for 

AstraZeneca’s Imfinzi (durvalumab), a human monoclonal antibody (mAb), was approved by FDA earlier this month in combination with chemotherapy for treating adult patients with resectable early-stage (IIA-IIIB) non-small cell lung cancer (NSCLC) and no known epidermal growth factor receptor mutations or anaplastic lymphoma kinase rearrangements. Following this regimen, patients are treated with Imfinzi in combination with neoadjuvant chemotherapy before surgery and as adjuvant monotherapy after surgery, according to an Aug. 16, 2024 company press release.

Also in August 2024, Imfiniza and Lynparza (olaparib) were approved throughout the 

 (EU) for treating certain patients with primary advanced or recurrent endometrial cancer (1). With this EU approval, Imfinzi plus chemotherapy can be used as first-line treatment, followed by treatment with Imfiniza and Lynparza (olaparib). In addition, Imfinzi, may be used to treat patients with mismatch repair proficient disease. A regimen using Imfinzi plus chemotherapy, followed by Imfinzi alone, can also be used for treating patients with mismatch repair deficient (dMMR) (1).

FDA’s latest approval was based on positive results from a pivotal Phase III trial (AEGEAN), which showed a statistically significant and clinically meaningful 32% reduction in the risk of recurrence, progression events, or death before and after surgery in patients treated with Imfinzi in combination with chemotherapy versus chemotherapy alone. These results were published in 

“This approval brings an important new treatment option that should become a backbone combination approach for patients with resectable non-small cell lung cancer, who have historically faced high rates of recurrence even after chemotherapy and surgery. When added both before and after surgery, durvalumab delivered a significant and meaningful improvement in outcomes in this curative-intent setting,” said John V. Heymach, MD, PhD, professor and chair Thoracic/Head and Neck Medical Oncology, The University of Texas MD Anderson Cancer Center, in the AstraZeneca press release.

Imfinzi was shown to be well tolerated, AstraZeneca stated in the press release. Furthermore, no new safety signals were observed in the neoadjuvant and adjuvant settings. Adding Imfinzi to neoadjuvant chemotherapy showed consistency with the known profile for this combination and did not compromise patients' ability to complete surgery, compared to chemotherapy alone.

“Today’s approval of Imfinzi in resectable early-stage lung cancer builds on its strong foundation of changing clinical practice in unresectable Stage III disease. We remain committed to bringing novel approaches [such as] AEGEAN to early lung cancer settings where cure is the goal of treatment,” said Dave Fredrickson, executive vice-president, Oncology Business Unit, AstraZeneca, in the press release.

In addition to the United States, Imfinzi is approved in the United Kingdom, Switzerland, and Taiwan (China) for this indication, also based on the Phase III (AEGEAN) results. Furthermore, the company has regulatory applications under review in the EU, China, and several other countries in this indication.

Further to its application in the treatment of lung cancers, Imfinzi is also approved in combination with chemotherapy (gemcitabine plus cisplatin) for treating locally advanced or metastatic biliary tract cancer and in combination with AstraZeneca’s Imjudo (tremelimumab), a mAb that blocks the activity of cytotoxic T-lymphocyte-associated protein 4 to aid in activating T-cells, for treating unresectable hepatocellular carcinoma (HCC). Imfinzi is also approved in Japan and the EU as a monotherapy for unresectable HCC as well as in the US in combination with chemotherapy (carboplatin plus paclitaxel), followed by Imfinzi monotherapy, for treating primary advanced or recurrent endometrial cancer that is dMMR.

1. AstraZeneca. Lynparza and Imfinzi Combination Approved in the EU for Patients with Mismatch Repair Proficient Advanced or Recurrent Endometrial Cancer. Press Release. Aug. 14, 2024.
2. Heymach, J. V.; Harpole, D.; Mitsudomi, T.; et al. Perioperative Durvalumab for Resectable Non–Small-Cell Lung Cancer. 

 (18),1672–1684. DOI: 10.1056/NEJMoa2304875

Phase III trial results showed that Imfinzi (durvalumab in combination with chemotherapy reduced the risk of recurrence, progression, or death by 32% in patients with resectable non-small cell lung cancer before and after surgery.

AstraZeneca’s Imfinzi Gets Expanded Indication in Lung Cancer on FDA Approval

With the completion of its acquisition of Morphic Holding, a biopharmaceutical company developing oral integrin therapies for serious chronic diseases, earlier this month, Eli Lilly and Company (Lilly) boosts its immunology pipeline. Through the acquisition, Lilly gains MORF-057, Morphic’s selective oral small-molecule inhibitor of α4β7 integrin. The drug candidate is in development for treating inflammatory bowel disease (IBD), Lilly said in an Aug. 16, 2024 company press release.

The acquisition’s completion comes a month after Lilly first announced its intention in July 2024 to acquire Morphic in a strategic move to improve outcomes for patients with IBD (1). Under the agreement, Lilly began a tender offer to acquire all outstanding shares of Morphic for $57 per share in cash, or an aggregate amount of approximately $3.2 billion, payable at the close of the transaction. The transaction was approved by the boards of directors of both companies (1).

"We are committed to exploring innovative approaches for immunologic diseases and believe Morphic's pipeline holds promise in improving outcomes and expanding treatment options for people with devastating conditions [such as] IBD," said Daniel Skovronsky, MD, PhD, chief scientific officer of Lilly and president, Lilly Research Laboratories and Lilly Immunology, in the company press release.

MORF-057, Morphic's lead program, has the potential to improve outcomes and expand treatment options for IBD patients. MORF-057 is currently being evaluated in two Phase II studies in ulcerative colitis and another Phase II study in Crohn's disease. Morphic is additionally developing a preclinical pipeline of other molecules for treating autoimmune diseases, pulmonary hypertensive diseases, fibrotic diseases, and cancer (1).

"Acquiring Morphic reinforces our growing capabilities in gastroenterology, building on the strong foundation of Omvoh, our first-in-class molecule already approved and launched around the world for ulcerative colitis and under regulatory review for Crohn's disease. Further, the acquisition allows Lilly to research potential combination treatments that could better serve people beyond what is possible with currently available medicines," Skovronsky also said in the release. Lilly’s Omvoh (mirikizumab-mrkz) is indicated in the United States for treating moderately to severely active ulcerative colitis in adults.

In an earlier move this month, Lilly opened its Seaport Innovation Center (LSC) in Boston, Mass., which serves as a R&D facility in the Boston Seaport district for advancing the company’s efforts in RNA and DNA-based therapies. The center will also conduct R&D for discovering new drug targets for developing therapeutics across several disease areas, including diabetes, obesity, cardiovascular diseases, neurodegeneration, and chronic pain (2).

LSC is housed in a 12-story building and totals 346,000 square feet. The new site includes laboratories and office space. It will also house the first Lilly Gateway Labs location on the East Coast of the US. Lilly’s goal is to foster a culture of shared expertise and real-time learning in the hopes of accelerating the development of novel medicines. The center is expected to accommodate approximately 500 Lilly scientists and researchers as well as 200 people from companies within Lilly Gateway Labs.

1. Eli Lilly and Company. Lilly to Acquire Morphic to Improve Outcomes for Patients with Inflammatory Bowel Disease. Press Release. July 8, 2024.
2. Eli Lilly and Company. Lilly Opens State-of-the-Art Research and Development Center in the Boston Seaport. Press Release. Aug. 13, 2024.

The completed acquisition of Morphic expands Lilly's immunology pipeline with Morphic's oral integrin therapies for treating inflammatory bowel disease.

Completion of $3.2 Billion Morphic Acquisition Expands Lilly’s IBD Portfolio

The European Commission (EC) has approved Roche’s PiaSky (crovalimab) for certain patients with paroxysmal nocturnal hemoglobinuria (PNH), Roche announced in a press release on Aug. 27, 2024 (1). PiaSky is a recycling monoclonal antibody that inhibits the complement protein C5; the EC approval was granted for adults, and adolescents aged 12 and older with a body weight of at least 40 kg (approximately 88 pounds), who are either new to or have been previously treated with C5 inhibitors.

PNH is a rare, life-threatening blood condition typified by the complement system, part of the innate immune system, working to destroy red blood cells. This can result in symptoms such as anemia, fatigue, and blood clots and can lead to kidney disease (1).

Roche said that the recycling technology used in the development of PiaSky allows for monthly, subcutaneous administration in which a smaller volume of medicine is able to bind and inhibit the C5 protein multiple times and act longer in the body (1). It is the first monthly subcutaneous treatment for PNH approved in the European Union (giving patients the option to self-administer, following adequate training) and has been approved for similar use in the United States and Japan.

A Phase III study (COMMODORE 2) in people with PNH who had not been treated with C5 inhibitors showed that the rate of adverse events in patients treated with crovalimab (78%) was similar to that of an existing standard of care C5 inhibitor, eculizumab (80%) (2–3).

“People with PNH are often burdened with lifelong, frequent intravenous infusions with time-consuming clinic visits, meaning that their lives, as well as their caregivers’ and families’ lives, may revolve around the demands of their treatment,” Alexander Röth, MD, head of classical hematology and hemostasis at the West German Cancer Centre, University Hospital Essen, Germany, and lead investigator of the study, said in the press release. “More flexible treatment options such as PiaSky, which are just as effective but less frequent and can be given more quickly at home, are essential to give people with PNH greater control over their treatment and more independence.”

“The PiaSky approval brings a new option to the PNH treatment landscape, combining the disease control achievable through C5 inhibition with a cutting-edge recycling technology that enables monthly subcutaneous administration,” said Levi Garraway, MD, PhD, Roche chief medical officer and head of global product development. “We are pleased to bring this new treatment to people with PNH in Europe with the hope it may lessen the treatment burden faced by many living with this condition.”

1. Roche. Roche’s PiaSky Approved in the EU as the First Monthly Subcutaneous Treatment for People with PNH. Press Release. Aug. 27, 2024.
2. Röth, A.; He, G.; Brodsky, A.; et al. The Phase III, Randomized COMMODORE 2 Trial: Results from a Multicenter Study of Crovalimab vs. Eculizumab in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Naïve to Complement Inhibitors. 


3. National Library of Medicine. A Phase III Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants with Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors. (COMMODORE 2). 

, updated Aug. 13, 2024 (accessed Aug. 27, 2024).

PiaSky (crovalimab) has previously been approved in countries such as the United States and Japan for monthly, subcutaneous use that may be self-administered with adequate training.

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