The European Commission has approved EZMEKLY (mirdametinib) for treatment of symptomatic, inoperable plexiform neurofibromas in patients with neurofibromatosis type 1 (NF1) aged two and older.
It is the first authorised therapy in the EU for both adult and paediatric NF1-PN patients.
Merck announced the decision, which grants conditional marketing authorisation to SpringWorks Therapeutics, a Merck healthcare company. NF1-PN is a rare genetic disorder that affects an estimated 135,000 people in the EU, causing disfigurement, pain and disability.
Ignacio Blanco, MD PhD, Chairman of the National Reference Center for Adult Patients with Neurofibromatosis in Spain, said: “This approval represents an important advance, especially for adults who previously did not have an approved treatment.”
The EC’s approval is supported by the phase 2b ReNeu trial, which enrolled 114 NF1-PN patients aged two and above. The trial showed confirmed objective response rates of 41% in adults and 52% in children, with significant reductions in tumour size and improvements in quality of life.
Annette Bakker, PhD, CEO of the Children’s Tumor Foundation, and Dariusz Adamczewski, MD, Director of CTF Europe, said: “This European Commission approval is an important milestone for NF patients and caregivers.”
EZMEKLY is available in tablet form that dissolves in water, helping those unable to swallow pills. Adverse reactions in adults included dermatitis acneiform, diarrhoea, nausea and musculoskeletal pain. In children, reactions included elevated blood creatine phosphokinase, abdominal pain, vomiting and headache.
Jan Kirsten, Global Head of Rare Tumor Business at Merck, added: “We are taking a major step toward improving care for this underserved community.”
