New active substance status

New active substance status is granted to a substance that has never been authorised in a medicinal product in the European Union or that, although close to a substance already authorised, differs significantly from it. It is assessed during the authorisation procedure, by the Committee for Medicinal Products for Human Use (CHMP) for centrally authorised medicinal products, according to the criteria set out in the Commission’s guidelines. It determines whether a separate period of regulatory protection opens and whether the centralised procedure is available.

Criteria for the status

A chemical, biological or radiopharmaceutical substance never authorised in the Union may qualify as a new active substance. The same applies to an isomer, mixture of isomers, complex, derivative or salt of a chemical substance already authorised, if it differs significantly in its safety or efficacy properties, and to a biological substance already authorised whose molecular structure, nature of the source material or manufacturing process differs significantly. The burden lies on the applicant, who must establish this significant difference with data.

Effects of the status

Recognition of the status prevents the medicinal product from being attached to the global marketing authorisation of the related substance and opens its own period of protection under the « 8+2+1 » rule. Conversely, a salt or isomer without a significant difference is regarded as the same active substance, which allows a generic to rely on the dossier of the reference. The status also determines mandatory access to the centralised procedure for new active substances intended for certain indications, such as cancer, diabetes or neurodegenerative diseases, and optional access for others.

Distinction from the concept of product in patent law

Regulatory status is not the same as the concept of product within the meaning of the supplementary protection certificate. The two concepts derive from distinct texts with distinct purposes, so recognition of the status does not guarantee that a certificate will be granted, particularly for a salt or enantiomer of an active ingredient already authorised. Likewise, regulatory novelty does not prejudge the novelty or inventive step of a patent claiming a salt, an enantiomer or a crystalline form, which are assessed under the rules specific to patent law.

Key points

New active substance status determines whether a medicinal product starts a new regulatory protection clock or remains attached to an earlier authorisation. Applicants should prepare, from the development stage, the data establishing the significant difference in safety or efficacy. For a generic manufacturer, reading the European public assessment report makes it possible to check whether the status has been recognised and to recalculate the expiry date of the protection.

---

This glossary entry is general in scope and does not constitute legal advice.

Dhenne Avocats acts in pharmaceutical patent disputes and on the medicines regulatory questions that accompany them, from the marketing authorisation to the supplementary protection certificate.

Pharmaceutical patents and medicines regulation · Pharma case law · Tell us about your situation