With Merck circling, SpringWorks gets new drug approval
Hard on the heels of the disclosure that Merck KGaA is talking to SpringWorks about a possible takeover, a new FDA approval could have sweetened the deal.
Stamford, Connecticut-based SpringWorks' Gomekli (mirdametinib) has become the first medicine approved for both adults and children with plexiform neurofibromas (PN) in patients with the genetic disorder neurofibromatosis type 1 (NF1), a rare genetic condition.
NFI causes tumours to develop on the covering of nerve cells, leading to disfigurement and other debilitating complications like pain, motor dysfunction, and malignant peripheral nerve sheath cancers. It affects approximately 100,000 children and adults in the US.
The MEK1 and MEK2 inhibitor showed that it was able to reduce NF1-PN lesions in the 114-patient phase 2b ReNeu trial, with an overall response rate (ORR) of 41% in adults and 52% in children. It has been cleared by the US regulator to treat NF1-PN in patients aged two and over with PN that cannot be treated with surgery.
German chemical and pharma group Merck confirmed earlier this week that it is interested in acquiring SpringWorks, which also has another FDA-approved therapy – Ogsiveo (nirogacestat) for desmoid tumours – that made sales of $172 million last year and by some estimates could grow to become a $800 million-plus product.
FDA approval means that Gomekly becomes only the second approved treatment for NF-1 patients in the US after AstraZeneca's MEK1/2 inhibitor Koselgo/Koselugo (selumetinib), co-marketed by MSD, which was approved for NF1-PN in paediatric patients aged two and older in 2020. That product brought in sales of $631 million last year, almost double its turnover in 2023.
"The NF1-PN patient community has a great need for more treatment options," said Saqib Islam, SpringWorks' chief executive.
"We are honoured to serve both adults and children with NF1-PN and provide them with a therapy that has the potential to shrink their tumours and offer meaningful symptomatic relief," he added.
Along with a second revenue stream, SpringWorks also gets a priority review voucher (PRV) from the FDA, awarded as an incentive to carry out drug development for a rare paediatric disease, that can be used to reduce the review time for any drug to six months. PRVs can be transferred to other companies and can command sales prices of $100 million or more.
The company said Gomekli should be available through a specialty pharmacy and specialty distributor network in the US within two weeks, but has not revealed its price. The drug is also under review in Europe, with regulatory decisions expected around the middle of 2025 and Germany slated to be the first EU market.
