New advanced therapy medicinal products in treatment of autoimmune diseases

2022 ◽  
pp. 319-359
Author(s):  
Shahrbanoo Jahangir ◽  
Sareh Zeydabadinejad ◽  
Zhila Izadi ◽  
Mahdi Habibi-Anbouhi ◽  
Ensiyeh Hajizadeh-Saffar
2020 ◽  
Vol 21 (14) ◽  
pp. 4982 ◽  
Author(s):  
Pietro Gentile ◽  
Aris Sterodimas ◽  
Jacopo Pizzicannella ◽  
Laura Dionisi ◽  
Domenico De Fazio ◽  
...  

Stromal vascular fraction (SVF) containing adipose stem cells (ASCs) has been used for many years in regenerative plastic surgery for autologous applications, without any focus on their potential allogenic role. Allogenic SVF transplants could be based on the possibility to use decellularized extracellular matrix (ECM) as a scaffold from a donor then re-cellularized by ASCs of the recipient, in order to develop the advanced therapy medicinal products (ATMP) in fully personalized clinical approaches. A systematic review of this field has been realized in accordance with the Preferred Reporting for Items for Systematic Reviews and Meta-Analyses-Protocols (PRISMA-P) guidelines. Multistep research of the PubMed, Embase, MEDLINE, Pre-MEDLINE, PsycINFO, CINAHL, Clinicaltrials.gov, Scopus database, and Cochrane databases has been conducted to identify articles and investigations on human allogenic ASCs transplant for clinical use. Of the 341 articles identified, 313 were initially assessed for eligibility on the basis of the abstract. Of these, only 29 met all the predetermined criteria for inclusion according to the PICOS (patients, intervention, comparator, outcomes, and study design) approach, and 19 have been included in quantitative synthesis (meta-analysis). Ninety-one percent of the studies previously screened (284 papers) were focused on the in vitro results and pre-clinical experiments. The allogenic use regarded the treatment of perianal fistulas, diabetic foot ulcers, knee osteoarthritis, acute respiratory distress syndrome, refractory rheumatoid arthritis, pediatrics disease, fecal incontinence, ischemic heart disease, autoimmune encephalomyelitis, lateral epicondylitis, and soft tissue defects. The information analyzed suggested the safety and efficacy of allogenic ASCs and ECM transplants without major side effects.


2020 ◽  
pp. 1-7

Advanced Therapy Medicinal Products (ATMPs) embody innovative therapies that have created great hope for patients suffering from previously untreatable diseases. Unfortunately, the pharaonic cost to produce and authorise ATMPs is a challenge for both patients and public health care systems, ultimately reducing patients’ access to treatment. Over the last 11 years, only 15 ATMP marketing authorisation applications received a positive draft opinion from the European Medicines Agency’s (EMA’s) Committee for Advanced Therapies (CAT). Moreover, due to poor return on investment, several ATMPs have already been removed from the market. In addition to the centralised procedure to obtain a marketing authorisation, the legislator foresees an alternative route for authorising ATMPs, the so-called “ATMP Hospital Exemption”. However, such ATMPs must be produced on a limited scale, on a non-routine basis. As a result, valuable ATMP therapies that have been used for years in hospitals may disappear. To avoid this, we propose, in this paper, an additional possibility to regularise ATMPs: the “Magistral Preparation of ATMPs”. It is a feasible pathway, which was already proposed for bacteriophage therapy, and which is particularly suitable for personalised therapies and considerably decreases the cost of the final products. We also discuss the practical impact of the ATMP regulation for (for-profit) industries and for (non-profit) hospitals. Two practical examples, the cultured human chondrocytes and the cultured human keratinocytes, are discussed.


2012 ◽  
Vol 3 (3) ◽  
pp. 421-424
Author(s):  
Audrey Chenesseau ◽  
Anna Pavlou

The present Flash News Report sets outs recent EU regulatory law developments (as of 25 June 2012) which pharmaceutical companies should be aware of in the areas of Pharmacovigilance, Information on Medicines, Pricing and Reimbursement, EU Data Protection and Advanced Therapy Medicinal Products.


Author(s):  
Karolina Jagiello ◽  
Anita Sosnowska ◽  
Alicja Mikolajczyk ◽  
Tomasz Puzyn

This article gives a brief description of the existing regulations related to biomaterials safety that need to be considered before it is introduced into EU market. According to these regulations, the risk analysis should include two characteristics: probability of occurrence of harm, and severity. Identified user-related harm should be reduced by managing the risk. Additionally, the review presents an overview of engineered biomaterials (EBMs), which in combination with nanoscale components (NPs) have shown promises in Advanced Therapy Medicinal Products (ATMP) and Medical Devices (MD). In this article, recent challenges, objectives and perspectives in risk assessment and risk management of ATMP and MD composed of nanobiomaterials were also highlighted.


Bioanalysis ◽  
2020 ◽  
Vol 12 (15) ◽  
pp. 1043-1048
Author(s):  
Sophie Tourdot ◽  
Carolina B Quaglia ◽  
Paul Chamberlain ◽  
Anne S De Groot ◽  
Nikki Dellas ◽  
...  

Given the expanding number of complex therapeutic protein drugs and advanced therapy medicinal products that are being developed, improving our ability to assess the potential immunogenicity of biologics is critical to ensuring treatment efficacy and patient safety. In this context, the European Immunogenicity Platform annual meeting provides opportunities for experts from industry and academia, regulators and clinicians to convene and discuss immunogenicity assessment methods and tools. This report summarizes the key messages on immunogenicity testing, prediction, clinical relevance and advanced therapy medicinal products discussed at the 11th Open Scientific European Immunogenicity Platform Symposium on Immunogenicity of Biopharmaceuticals, Lisbon, Portugal, 17–19 February 2020.


2015 ◽  
Vol 18 (7) ◽  
pp. A517-A518
Author(s):  
E Hanna ◽  
C Rémuzat ◽  
P Auquier ◽  
M Toumi

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