16^th European Biosimilars Congress April 03-04, 2023 Barcelona, Spain

Biography:

Adriana Kiedzierska-Mencfeld has more than 15 years’ experience in recombinant protein chemistry and over 10 years’ experience in biosimilars development and manufacturing. Working in Polpharma Biologics S.A. for over 8 years she has actively participated in development of biosimilars. Her career in Biosmilars she started from development than moving into production for clinical trials and currently being in commercial phase. As a part of the site leadership team, she has participated in several GMP inspections, customer audits and successfully passing the FDA-PLI (Pre-License Inspection) inspection, gaining experience in a regulated environment. Going through various phases of the product life cycle, she participated in the preparation of registration dossiers for various agencies, as well as in the product approval process. Currently, as a Member of the Management Board and Head of the Polpharma Biologics Branch in GdaÅ„sk, she is responsible for all operations in GdaÅ„sk, managing over 500 employees. Her focus is team building, people development and knowledge sharing.

Abstract:

Approval of FYB201- ranibizumab product (Lucentis biosimilar) is key milestone for Polpharma Biologics, which is an international biotechnology company with integrated operations in the European Union (EU), developing and manufacturing biosimilar medicines. Using state-of-the-art platform technologies, Polpharma Biologics develops biosimilar products to treat a range of conditions in major therapeutic areas. Programs at Polpharma Biologics start in cell line development and transition through technical and clinical development to commercial-scale production, preparing drugs for future commercial partnerships with global pharmaceutical organizations. The company pipeline contains more than six biosimilars in different stages. FYB201 was developed by Bioeq, a Joint Venture between Polpharma Biologics and Formycon. The production of biosimilars is a process with high levels of scientific rigor and the approval of FYB201 (Ranivisio®, CIMERLI™ (ranibizumab-eqrn), is the culmination of years of dedication by Polpharma Biologics. Starting from technology transfer and process optimization, method transfer and then process validation, Polpharma Biologics actively participated in development of ranibizumab biosimilar. Currently being commercial manufacturer of drug substance ranibizumab, experienced transformation from development to commercial organization. FYB201 is highly similar to the reference product Lucentis® in terms of comparable efficacy, safety, pharmacokinetics and immunogenicity in patients with age-related neovascular (wet) macular degeneration. FDA-approval and interchangeability designation are based on a totality of evidence including analytical, nonclinical, clinical and manufacturing data. Manufacturing of product for various markets (e.g. UK, US, Europe) requires expertise in many areas, high level of GMP awareness and quality culture.

Biography:

Bertrand Merot is Founder & CEO of Bio-Sourcing. He has done his PhD in Genetics and MBA; he worked previously for LFB Biotechnologies, Oncodesign, Meristem Therapeutics, Limagrain where he held several managerial positions (CEO, CSO, Vice President). He has also been Board Member of France Biotech.

Abstract:

The biopharmaceutical industry faces huge production limitations in terms of volume and cost. The strong growth rate of biosimilars, the need to serve the Global Southern markets (LMIC countries) and the occurrence of pandemics, require production of large volumes of biotherapeutics, at ever lower costs than currently available biosimilars and in compliance with sustainable development objectives. The investments needed to create new bioproduction units using mammalian cell cultures (CHO) cannot meet this global health challenge.

As the ultimate objective is to give access to essential medicines to larger populations of patients, the production paradigm has to drastically evolve, using what nature has given us as the best bioreactor, namely leveraging the natural secretion of large amount of proteins in the milk of dairy animals.

Bio-Sourcing changes the Bioproduction design by developing a disruptive technology platform (BioMilk®) using dedicated and specialized transgenic goats and thanks to the company expertise in several breakthrough technologies which includes genome editing (CRISPR) and Nuclear Transfer (SCNT).

The Bio-Sourcing innovative approach leads to a drastic reduction in capital requirements and production costs, especially for large volumes (over several hundred Kg), as well as an improvement in the quality/efficiency of biopharmaceuticals through better glycosylation.

The technological approach developed by Bio-Sourcing has already given rise to biotherapeutics approved by regulatory bodies (FDA, EMA).

An additional benefit of this technology is a strong contribution to sustainable development, drastically reducing the carbon footprint.

Bio-Sourcing is already currently in the industrialization phase and has the potential to offer producers of biosimilars the possibility, in a context of cost pressure, to produce essential bioproteins for global unmet medical needs.

Biography:

Francois Xavier Frapaise, MD, has over 40 years of international drug development, strategic planning and marketing experience at major pharmaceutical companies including Sanofi, Bayer and Abbott, has hold multiple C-level positions (CSO, CMO, CEO) in different Pharmacos in the US and Europe. He is currently heading a clinical/Regulatory Consulting Company; he has extensive experience of biosimilars development (Merck KGaA, Boehringer-Ingelheim, Pfenex). He held an academic position at the Thrombosis Research Center at the Loyola Medical Center in Maywood (IL). He holds an MD degree from Faculté de Médecine René Descartes, Paris France, and is an INSEAD alumni.

Abstract:

Background: Many patients still have limited or no access to life-changing therapeutic proteins in the treatment of their cancer or autoimmune disorders. The current clinical development model of biosimilars is expensive and in many cases, large phase 3 trials do not provide meaningful information on the clinical equivalence (efficacy/safety/immunogenicity) between biosimilars and reference compounds. At the same time, the development of state-of-the-art orthogonal analytical methods has enabled a better understanding of the structure and structure–function relationship of biotherapeutics.

Observations: In the recent years many agencies such as EMA and the FDA accept applications for compounds such as peg-filgrastim based on a state-of-the-art Chemistry, Manufacturing and Controls (CMC) package and phase 1 study. At the same time, some other regulators, hospitals drug committees, clinicians continue to request evidence of efficacy/safety in patients before approving/prescribing biosimilars. From a clinicians’ perspective, this is understandable, however considering the objectives of biosimilarity assessment, namely finding a suitable, sensitive model to assess differences if they exist, this approach of testing equivalence in patients is more difficult to justify. Indeed, in some indications, validated surrogate markers of efficacy are available (neutrophils count for peg-filgrastim); furthermore, safety is virtually impossible to compare in a population that has spontaneously around 90% Adverse Events, such as cancer patients; immunogenicity is also difficult to compare in subject who are immuno compromised and/or receive immunosuppressant therapy. Running patient’s trials that are not likely to provide the expected evidence also raised ethical concerns.

Conclusion: We believe that increased regulatory harmonization, better understanding of biosimilar development objectives and methods, education of all stakeholders on biosimilars would help ensuring patients have early access to life-saving compounds many of them currently cannot afford.

Biography:

Luis Gerardo Alcalá is a regulatory affairs expert based in Mexico City. During his career, he has shown a strong devotion to bringing into the market medical supplies of the best quality that are safe and effective for the patients. He has built differently proven regulatory strategies for national and international companies, having been able to collaborate with companies from around the World, such as the USA, Brazil, Argentina, Chile, Colombia, France, Germany, The UK, Italy, China, Japan, South Korea, among others. His expertise line includes medical devices, medical drugs and biotechnology drugs, which include generic and innovative drugs. He has worked with new registers, variations and renewals. Having been able to collaborate directly with Big Pharma Company, he also specializes in newly formed companies, always focusing on the best possible regulatory path and having in mind the safety of the patients, as he always says “We are all potential patients”.

Abstract:

Biological products, as well as other medical supplies, are regulated in each country by the competent Health Authority (HA). According to the FDA, they are used to diagnose, prevent, treat and cure diseases and medical conditions. The difference with a more traditional drug is that the Active Pharmaceutical Ingredient (API), or APIs, is produced by processing chemical compounds, whereas in a biological drug, the active ingredient is known as Bulk Process Intermediate (BPI) and it is made by biotechnology in a living system, such as a microorganism, plant cell, or animal cell.

The first biopharmaceutical drug was approved in 1982 by FDA, recombinant human insulin. By 2022, FDA reports that there are over 621 FDA-licensed biologics products and the global biotechnology market size was estimated at USD 1, 023.92 billion in 2021, with an expected growth during the next years. With this in mind and considering that old patents are coming off, other companies can offer new biological products or biosimilar products. A biosimilar is defined by FDA as “A biological product that is highly similar to and has no clinically meaningful differences from an existing FDA-approved reference product” (a biological product previously approved).

Biosimilars can be seen as new therapeutic alternatives, as a way of reducing costs for the patients or the insurance and able to provide the patients with a faster product, as biosimilars typically have a timeline for approval of 8 years vs. <12 years of the reference or innovator drug, not to mention the reduce on the development budget of around 10-15%. Biosimilars look attractive for any of the involved parties, but they must be safe, of quality and effective. To make sure of this, HA had made different changes in their legislations. In this abstract, we will review recent changes and the current global situation of biosimilars with an emphasis on FDA & EMA.

Biography:

Moh'd Alqahtani has done B. Pharm, M.S. in Pharmacoeconomics, PhD Candidate in Social and Administrative Pharmacy/Consultant and expert in regulatory affairs for pharmaceutical products in the US, Europe, Middle East and North Africa (MENA) region.

Abstract:

It has been observed that spending on biological drugs has increased significantly in the United States (1). Around $2.0 billion was spent on biologic agents, nonfederal hospital costs and three of the top five most expensive drugs. Biological medications have also enhanced the quality of life and altered the progression of several life-threatening diseases or conditions (2). However, many reasons prevent the use of biological medicines, including the high manufacturing cost. Biological drugs are more expensive than conventional drugs (3); many patients cannot afford those (4). Recently, biosimilars have been offered as cost-effective and cost-saving alternatives (5). Nevertheless, studies have found that many healthcare providers have different perceptions that result in biosimilars not being used for their patients. Most physicians prefer originator products for biologic-naive patients and hesitate to switch from biologics to biosimilars.