Kinesys Consulting Ltd is pleased to announce that the European Commission, acting on the positive recommendation from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP), has granted orphan drug designation to Soligenix’ recombinant modified ricin toxin A-chain subunit, the active pharmaceutical ingredient in RiVax®, for the prevention of ricin poisoning. RiVax® has previously been granted orphan drug designation from the US Food and Drug Administration (FDA).
Kinesys has secured this designation on behalf of its US partner Soligenix, Inc., New Jersey.
“We are extremely pleased to have received European orphan drug designation for the RiVax® program,” stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix, Inc. “This EU orphan designation, combined with our US orphan designation, positions this biodefense program for a potentially accelerated global regulatory product development pathway to address this unmet need. RiVax® has shown up to 100% protection against aerosolized ricin exposure in non-human primates and safety in humans and we look forward to further developing our thermostable formulation for human use.”
For further information regarding Soligenix, Inc., please visit the Company’s website at www.soligenix.com
The European Medicines Agency (EMA) will now be publishing “orphan maintenance assessment report” for every orphan-designated medicine which has been recommended for marketing authorisation.
To qualify for orphan designation, a medicine must target a disease that is life-threatening or chronically debilitating that affects less than 5 in 10,000 patients in the EU. If there is already another treatment available for the targeted rare disease, the developer of the new medicine must show that there is a significant benefit to patients compared to the existing options, meaning the medicine provides a clinically relevant advantage or a major contribution to patients.
Orphan designation gives access to a number of incentives to foster research and innovation, including fee reductions for scientific advice and ten year market exclusivity when the medicine is authorised in the EU.
The Committee for Orphan Medicinal Products (COMP) assesses whether a medicine fulfils the criteria for orphan designation at two points in time: early on in a medicine’s development to provide access to incentives supporting the development activities, and again at the time of marketing authorisation of the new treatment to reconfirm its eligibility for ten year market exclusivity.
In the context of increasing demand for greater transparency, these new “orphan maintenance assessment reports” will now summarise the reasoning of the COMP on whether or not a medicine designated as an orphan medicine during its development still fulfils the designation criteria at the time of its authorisation. These reports will be published as part of a medicine’s European Public Assessment Report (EPAR) once the European Commission has adopted its marketing authorisation decision. The orphan maintenance assessment reports will be published for all positive and negative COMP opinions, as well as withdrawals. They will describe the orphan condition and its seriousness, the spread of the condition at the time of maintenance of the designation, and, if applicable, the significant benefit over already authorised medicines.
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Since 2014, there has been a welcome recognition within Europe of the need for improved regulatory mechanisms whereby medicines being developed to target serious or life-threatening diseases that represent an unmet clinical need can be made available to patients at the earliest opportunity.
This has resulted in the introduction of the Early Access to Medicines Scheme (EAMS) in April 2014, to support early patient access to important medicines in the UK, the first step of which is the MHRA’s Promising Innovative Medicine (PIM) designation within the EAMS. The EMA’s PRIority MEdicines (PRIME) scheme followed in March 2016, which provides additional regulatory support to the sponsor with the aim of optimising development plans with a view to accelerating the evaluation of important medicines across the EU.
This article by Kinesys Consulting Ltd. in TOPRA‘s Regulatory Rapporteur Journal presents the various programmes available to support expedited development of medicines in areas of unmet medical need, with particular focus on applications for PIM and PRIME designation. A procedural insight into each of these schemes is provided along with recent experiences of submitting consecutive PRIME and PIM applications relating to the same medicinal product, which was at the same stage of development and had an identical data package.
To access the article, click: here