After Merck KGaA's recent confirmation that it's in "advanced discussions" to acquire SpringWorks Therapeutics, the Connecticut rare disease specialist has boosted its value with a new FDA nod.
Tuesday, the agency signed off on SpringWorks’ Gomekli (mirdametinib) as a treatment for the rare genetic disorder neurofibromatosis type 1 (NF1).
More specifically, Gomekli is for those age 2 and older who have NF1 accompanied by symptomatic benign tumors that can’t be removed surgically. The tumors, which are called plexiform neurofibromas (PNs), develop along the peripheral nerve sheath and can occur inside or outside of the body.
Gomekli becomes the first treatment for adults and children with the disorder. Five years ago, AstraZeneca and Merck won FDA approval for Koselugo for patients with NF1-PNs, but the nod is limited to pediatric patients.
“The NF1-PN patient community has a great need for more treatment options," Saqib Islam, SpringWorks' CEO, said in a release. "With today’s approval, we are honored to serve both adults and children with NF1-PN and provide them with a therapy that has the potential to shrink their tumors and offer meaningful symptomatic relief."
The company said Gomekli will be available within two weeks but did not reveal its price. The treatment is under review in Europe, with the company expecting a decision to come in 2025.
With the FDA endorsement, SpringWorks gained a lucrative rare pediatric disease priority review voucher, which can be used to fast-track the review process for a new drug application or can be sold.
This is the second approval in the last 15 months for SpringWorks, a spinout Pfizer established in 2017 equipped with a portfolio of rare disease candidates.
In November 2023, the FDA gave a thumbs-up to SpringWorks’ Ogsiveo as a treatment for patients with desmoid tumors who need systemic therapy.
Last month, the company reported that Ogsiveo is off to a strong launch, achieving sales of $61 million in the fourth quarter and $172 million for the year. The company also said it closed 2024 with $462 million in cash, cash equivalents and marketable securities.
Shortly before the approval, Merck KGaA confirmed it was in “advanced discussions” to buy out SpringWorks. With the M&A talk and the approval, SpringWorks’ share price has increased 34% this week, and its market cap has swollen from $2.99 billion to $4.1 billion.
Gomekli’s approval was backed by a phase 2b trial in which it achieved a 52% response rate in pediatric patients and a 41% response rate in adults. The trial enrolled 58 adult and 56 pediatric patients. Adverse events reported by more than 25% of patients included rash, diarrhea, musculoskeletal pain, nausea and vomiting.
NF1-PN affects approximately 40,000 people in the U.S. and can cause pain, severe disfigurement and functional impairment. PNs also can become malignant, SpringWorks said.