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EMA, European Medicines Agency (CHMP position on CJD and plasma-derived and urine-derived medicinal products) logo

EMA, European Medicines Agency (CHMP position on CJD and plasma-derived and urine-derived medicinal products)

The European Medicines Agency is the decentralised EU agency that runs the scientific evaluation, supervision and safety monitoring of medicines, set up in 1995 and based in Amsterdam since 2019. Its committees, including the CHMP for human medicines and the COMP for orphan medicines, are staffed by experts from national regulators such as the French ANSM. For prion disease it matters in three ways. Since 1998 its human medicines committee (first the CPMP, now the CHMP) has kept a position on CJD and medicines made from human plasma or urine, revised in 2003, 2004 and 2011 and replaced in May 2024 by a reflection paper (Revision 3). Since 2004 it has published guidance on keeping animal TSE agents such as BSE out of medicines, last revised in 2011, plus a 2004 guideline on testing plasma product manufacturing for vCJD risk. And through the COMP it gave a positive orphan opinion in 2017 for a thiophene compound in CJD, the only prion disease orphan designation found. Information only, not medical, legal or regulatory advice.

Key facts

  • EMA describes itself as a decentralised EU agency for the scientific evaluation, supervision and safety monitoring of medicines, with a 36 member Management Board and seven scientific committees whose members are experts put forward by the national competent authorities of member states, working with EMA as the European medicines regulatory network.
  • EMA was set up in 1995 to harmonise the work of national medicines regulators; the Committee for Orphan Medicinal Products dates from 2000, and EMA moved from London to Amsterdam in 2019.
  • The first EU level text on prions and blood products was the CPMP position statement CPMP/201/98 on new variant CJD and plasma-derived medicinal products, with a legal effective date of 25 February 1998, later superseded by the CJD position statement EMEA/CPMP/BWP/2879/02 (effective 20 February 2003) and its Revision 1 (effective 23 June 2004).
  • Revision 2, the CHMP position statement EMA/CHMP/BWP/303353/2010, was published on 28 June 2011 after public consultation from June to September 2010. A search excerpt of its text says that recalling plasma-derived products is not justified when a donor is later confirmed to have sporadic, genetic or iatrogenic CJD, and that UK transfusion cases are strong evidence that vCJD can pass through blood.
  • The current text is the CHMP reflection paper on CJD and plasma-derived and urine-derived medicinal products, Revision 3 (EMA/CHMP/BWP/303353/2010 Rev 3), first published on 31 May 2024 together with an overview of comments, after a draft published on 16 November 2018 and consulted on until 31 October 2019. A search excerpt says it was agreed by the Biologics Working Party on 17 April 2024 and adopted by the CHMP that month. Its content is covered in regulation-eu-chmp-cjd-plasma-reflection-paper.
  • EMA's guideline on investigating plasma product manufacturing processes for vCJD risk, CPMP/BWP/CPMP/5136/03, has had a legal effective date of 1 October 2004 and no later revision is listed. A separate guideline on Plasma Master File certification, CPMP/BWP/4663/03, was adopted in February 2004, per a search excerpt only.
  • The note for guidance on minimising the risk of transmitting animal spongiform encephalopathy agents via human and veterinary medicinal products covers both kinds of medicine and lists Directive 2001/83/EC among its related legislation. Its Revision 3, EMA/410/01 Rev. 3, was published in the Official Journal (C 73) on 5 March 2011, replaced Rev. 2 of January 2004, proposed 1 July 2011 as its date of application, switched country BSE risk to the OIE classification and added criteria for bovine gelatin and peptones.
  • In 2017 the European Commission granted orphan designation EU/3/17/1883, after a positive COMP opinion of 12 May 2017, to NeuroScios GmbH of Austria for pentamer formyl thiophene acetic acid (NSC500) in CJD. At the time the compound had only been tested in experimental models, no patient trial had started, and CJD prevalence was put below 0.1 per 10,000 in the EU. This was the only prion disease orphan designation found; web searches for fatal familial insomnia and other prion conditions found no other EU designation, which does not rule one out.
  • EMA held an expert meeting on human TSEs and medicines made from human blood and plasma on 1 December 2000 and published a summary report; only its title was seen.
  • In the EU Clinical Trials Information System, the Ionis ION717 prion trial (2023-503355-98-00) is flagged as a rare disease trial with no orphan designation stated; it has one site each in France, Germany, Italy and Spain with Italy as reference member state, as shown on a third party mirror of the register.

Timeline

  1. 1995EMA set up to harmonise national medicines regulation.
  2. 1998CPMP position statement on new variant CJD and plasma-derived medicinal products takes effect.
  3. 2000EMA expert meeting on human TSEs and medicines made from blood and plasma.
  4. 2003CPMP position statement on CJD and plasma-derived and urine-derived products takes effect.
  5. 2004Revision 1 of the position statement takes effect.
  6. 2004Legal effective date shown for the animal TSE note for guidance.
  7. 2004Guideline on investigating plasma product manufacturing for vCJD risk takes effect.
  8. 2011Revision 3 of the animal TSE note for guidance (EMA/410/01 Rev. 3) published in the Official Journal.
  9. 2011CHMP position statement Revision 2 published.
  10. 2017European Commission grants orphan designation EU/3/17/1883 for NSC500 in CJD.
  11. 2018Draft Revision 3 published for consultation (to 31 October 2019).
  12. 2019EMA relocates from London to Amsterdam.
  13. 2024CHMP reflection paper Revision 3 published, replacing the position statement.
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