Adnkronos (Adnkronos) – The Italian Medicines Agency (AIFA) has approved the reimbursement in Italy of selumetinib, an oral, selective MEK inhibitor, for the monotherapy treatment of inoperable, symptomatic plexiform neurofibromas (Pn) in adult patients with neurofibromatosis type 1 (NF1). Alexion, AstraZeneca Rare Diseases, announced in a statement that AIFA's approval follows the European Commission's approval of the drug's expanded indication, based on the results of the Komet study, a large, international, placebo-controlled Phase 3 study that enrolled 145 adult patients from 13 different countries across North America, South America, Europe, Asia, and Australia, published in The Lancet.
In the primary analysis of the Komet study, selumetinib demonstrated a statistically significant and clinically relevant objective response rate (ORR) compared to placebo. The safety profile was consistent with the known safety profile and established use in pediatric patients.
Nf1 is a rare, progressive genetic disease usually diagnosed in early childhood but often progresses into adulthood and can affect any organ system – the note explains – Up to 50% of people with Nf1 may develop a type of non-malignant tumor called plexiform neurofibroma that can affect the brain, spinal cord and nerves.
Pn tumors can appear throughout life and grow to a large size, causing pain, deformity, and muscle weakness, among other debilitating symptoms.
“The approval of selumetinib for the treatment of adults with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas represents an important step forward, extending to this patient population a therapeutic option previously available only to pediatric patients,” explains Chiara Leoni, head of the Pediatric Rare Diseases and Innovative Therapies Unit at the Agostino Gemelli University Hospital Foundation in Rome.
“The data confirm the value of selumetinib across the entire treatment continuum in terms of clinical, radiological, and overall improvement in quality of life, offering a new therapeutic opportunity both for patients diagnosed in adulthood and for those making the transition from childhood.”
The joint comment from the associations representing adult patients with neurofibromatosis in Italy – Ananas Aps; National Association for Help for Neurofibromatosis, Friendship and Solidarity; Anf–Associazione Neurofibromatosi; and Linfa-Lottiamo insieme contro le neurofibromatosi Aps – is also positive. "The availability of selumetinib to adults with plexiform neurofibromas represents a significant milestone for the entire NF1 community," they stated. "This result offers new perspectives to people living with the disease and their families, and it recognizes a long-unmet need for treatment. It also demonstrates what can be achieved when associations, clinicians, institutions, and the industry work together with a common goal: improving the lives of people with NF1 and continuing to advance treatment options."
"The expansion of selumetinib's indication for the treatment of plexiform neurofibromas in adults with NF1 marks a significant advancement for these patients, who until now had limited treatment options, and also helps ensure continuity of care during the delicate transition from adolescence to adulthood," commented Anna Chiara Rossi, Vice President and General Manager of Alexion, AstraZeneca Rare Disease. "This result confirms Alexion's ongoing commitment to rare diseases and the search for solutions that address unmet needs, with the aim of concretely improving the care of people living with these diseases." Selumetinib, the statement concludes, has been approved in the United States, Europe, Japan, and other countries for the treatment of adult patients with NF1 who have symptomatic, inoperable PN, and further regulatory reviews are ongoing.
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