Drug Regulator, EMA (EMEA), Publish Concept Paper on Need for A Guideline on the Clinical Development of Thrombopoetin Receptor Agonists for the Treatment of Chronic Immune (idiopathic) Thrombocytopenic Purpura.
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Thrombopoetin agonists constitute an innovative approach for the management of patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP). Over the recent past, a number of requests for Scientific Advice and Marketing Authorisation Applications have been assessed by the CHMP, thus giving sufficient basis issuing recommendations on the clinical development of this type of drugs in ITP patients.
A number of Marketing Authorisation Applications of medicinal products for the treatment of ITP have been evaluated in the recent past by the CHMP. These drugs, being of substantially different origin and structure, share the same mechanism of action, namely the stimulation of thrombopoetin receptors. This is a completely innovative approach in the treatment of ITP, which is deemed a relevant added tool for the management of these patients. A number of important aspects dealing with the evaluation
of both the safety and efficacy of this new type of drugs have triggered the need for specific CHMP guidance relating to the clinical investigation of these products in ITP.
Considering the new therapeutic alternatives to deal with the thrombocytopenia based on the stimulation of production of platelets by megakaryocytes in the marrow, different molecules have been developed (a recombinant polypeptide and a low molecular weight, synthetic, non-peptide molecule are available which act as agonists of thrombopoetin receptor). Up to now, no formal EU guidelines on the clinical development of products for ITP were available, and the regulatory experience was limited
to classical immunoglobulin therapy for which, considering the wide clinical experience, only limited clinical data had been requested. Thrombopoetin receptor agonists constitute an innovative therapeutic approach which certainly is felt to fill an unmet medical need in chronic refractory ITP patient population. This approach has lead however to carefully reconsider which type of clinical data, in terms of both safety and efficacy, should be requested to allow a proper benefit/risk evaluation. Key relevant aspects would be the dose selection, the definition of the therapeutic goal, identification of relevant target populations and discussion particular safety aspects linked to the mechanism of action of these drugs and /or their molecular structure. Importantly, children deserve specific reflections, since disease features may be different with respect to adults.
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Damien Bové is THE Drug Development and Regulatory Consultant (pharmaceutical or biotechnology), I work with my clients to define a drug development target, define a drug development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch.
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