Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.SpringWorks Therapeutics has selected Onco360 as national specialty pharmacy partner for Gomekli (mirdametinib).
The study demonstrated that the product achieved the primary endpoint of confirmed overall response rate. Credit: Laurynas Me on Unsplash.
Subscribe to our email newsletter
Following its recent US Food and Drug Administration (FDA) approval, the therapy is now a treatment option for patients aged two years and older with neurofibromatosis type 1 (NF1) who suffer from symptomatic plexiform neurofibromas (PN) that cannot be fully resected.
NF1 is a genetic disorder that affects approximately 30-50% of children and adults, leading to the development of PN.
The FDA’s approval of Gomekli was based on data from the ReNeu trial, which evaluated 114 patients, including 58 adults and 56 children, with NF1 and symptomatic PN.
The study demonstrated that the product achieved the primary endpoint of confirmed overall response rate (cORR) of 41% in adults and 52% in children.
cORR is measured by the proportion of patients showing complete or partial response on MRI scans of the target PN volume from baseline to Cycle 24, as evaluated by blinded independent central review on at least two consecutive scans within a two to six-month period.
Both adult and paediatric patients experienced early and sustained improvements in pain and quality of life (QOL) from baseline following treatment with Gomekli.
The majority of adverse reactions reported were found to be mild to moderate in severity.
In adult patients, the most common adverse reactions, occurring in at least 25% of cases, included rash, nausea, diarrhoea, musculoskeletal pain, and vomiting.
Paediatric patients also most frequently experienced diarrhoea, rash, musculoskeletal pain, abdominal pain, headache and vomiting.
Onco360 chief commercial officer Benito Fernandez said: “Onco360 is grateful for the opportunity to partner on another therapy with the team at SpringWorks Therapeutics and become a specialty pharmacy provider for Gomekli.”
“We are proud to add Gomekli for the treatment of neurofibromatosis type 1 patients to our growing portfolio of rare disease therapies.”
