Specialists in orphan drug designation applications
Advanced Therapy Medicinal Products (ATMPs)
Integrated scientific and regulatory services to ensure the quality, safety and compliance of advanced-therapy medicinal products.
Advanced therapy medicinal products (ATMPs), such as cell therapy, gene therapy, and tissue engineering products, offer enormous potential for the treatment of serious diseases.
However, these innovative solutions face multiple challenges during development and approval, including uncertainty about the product's benefit-risk balance, limitations of nonclinical testing, practical and ethical challenges of clinical development, complex quality considerations of highly individualised therapies, and specific regulatory requirements.
We have in-depth knowledge and expertise in the EU, UK and US regulatory approval processes for advanced therapies, including those that target orphan diseases and high unmet medical needs.
Our specialist team can advise you on the following:
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ATMP classification
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Legislative framework applicable to ATMPs
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Clinical Trials Regulation
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Paediatric Regulation
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Pharmacovigilance
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Genetically Modified Organisms (GMO) legislation
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Marketing Authorisation
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Orphan Drug Designation
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Combination ATMPs
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Early access and fast-track procedures