A Big Win For NF1-PN Patients in Europe
The European Commission has granted conditional approval for EZMEKLY® (mirdametinib) to treat symptomatic, inoperable plexiform neurofibromas (PNs) in patients aged 2 and up with NF1. This makes it the first therapy in the EU approved for both adults and children.
Why it matters:
- In the pivotal Phase 2b ReNeu trial, tumor shrinkage (≥20%) was seen in 41% of adults and 52% of children.
- Among responders, about 90% experienced benefits for at least 12 months, with around half maintaining response at 24 months.
- Patients also reported early and sustained improvements in pain and quality of life.
- The treatment is available in capsules and a dispersible tablet, making it easier for those who have difficulty swallowing pills.
Safety & Access:
Common side effects include skin rash, diarrhea, nausea, and elevated muscle enzymes. These side effects are generally manageable. SpringWorks (now part of Merck) notes this conditional approval opens the door for wider access across Europe.
What’s next?
As a conditional authorization, further data and follow-up are required. But this is a major milestone, especially for adults, who previously had no approved options in Europe.
Read the full press release on SpringWorks Therapeutics’ website.