Module 6

02/10/2024

Example: Keytruda (active: pembrolizumab) for Cancer Line extension to add paediatric population and indication to the MA PIP covers a development plan: • From 3 years to less than 18 years of age • Treatment of classical Hodgkin lymphoma with incomplete early response to front-line chemotherapy • Treatment of relapsed or refractory classical Hodgkin lymphoma

Study 1

Study 2

Multi-centre, open-label , single-arm trial to evaluate pharmacokinetics, pharmacodynamics, toxicity, safety and activity of pembrolizumab in paediatric patients from 6 months to less than 18 years with an advanced melanoma or a PD-L1 positive advanced, relapsed or refractory solid tumour or lymphoma , including an expansion phase

Open-label trial to evaluate the safety and efficacy of pembrolizumab in combination with chemotherapy in paediatric patients from 3 years to less than 18 years of age (and young adults) with a classical Hodgkin with incomplete early response to front-line therapy

Indication approved: KEYTRUDA as monotherapy is indicated for the treatment of paediatric patients aged 3 years and older with relapsed or refractory classical Hodgkin lymphoma who have failed autologous stem cell transplant (ASCT) or following at least two prior therapies when ASCT is not a treatment option.

The Organisation for Professionals in Regulatory Affairs

Lecture 1: Part 2 – Maintain and Extend the Brand

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Extending the Orphan Drug Brand Opportunities to add further Orphan Indications to the MA

• During the post-MA planning phase to extend the brand to add new/other orphan indication(s) to the MA, an orphan drug designation (ODD) for the planned orphan indication must to be obtained and maintained . • Once the ODD for the planned new orphan indication is obtained the company is obliged to submit ‘annual reports’ to summarise the development of the planned new orphan indication and reconfirm the orphan product continues to maintain the ODD criteria . Annual reports are submitted to the EMA COMP until the new orphan indication is approved onto the MA via a Type II Variation. • The ‘ golden rule ’: due to the legal framework of orphan market exclusivity, orphan and non-orphan indications cannot be filed under the same brand (tradename). • Each indication with an ODD confers ten years' market exclusivity for the particular indication. • A medicine that has multiple orphan designations for different conditions will benefit from separate market exclusivity periods pertaining to its different orphan designations.

The Organisation for Professionals in Regulatory Affairs

Lecture 1: Part 2 – Maintain and Extend the Brand

62

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