SpringWorks Therapeutics Secures FDA Approval for Gomekli, Boosting Value Amid Acquisition Talks

SpringWorks Therapeutics has scored a significant victory with the U.S. Food and Drug Administration's (FDA) approval of Gomekli (mirdametinib) for the treatment of neurofibromatosis type 1 (NF1). This development comes as the Connecticut-based rare disease specialist finds itself in the midst of acquisition discussions with Merck KGaA.

Gomekli: A Breakthrough for NF1 Patients

Gomekli has been approved for patients aged 2 and older with NF1 accompanied by symptomatic benign tumors, known as plexiform neurofibromas (PNs), that cannot be surgically removed. This approval marks a significant milestone as Gomekli becomes the first treatment available for both adults and children with this disorder.

The FDA's decision was based on impressive results from a phase 2b trial, which demonstrated a 52% response rate in pediatric patients and a 41% response rate in adults. The study involved 58 adult and 56 pediatric patients, with common adverse events including rash, diarrhea, musculoskeletal pain, nausea, and vomiting.

Saqib Islam, CEO of SpringWorks, emphasized the importance of this approval, stating, "The NF1-PN patient community has a great need for more treatment options. 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."

Market Impact and Acquisition Talks

The FDA approval has had a significant impact on SpringWorks' market position. The company's share price has surged by 34% this week, with its market capitalization growing from $2.99 billion to $4.1 billion. This boost comes at a crucial time, as Merck KGaA recently confirmed it is in "advanced discussions" to acquire SpringWorks.

In addition to the market value increase, SpringWorks has gained a rare pediatric disease priority review voucher, which can be used to expedite the review process for a future drug application or be sold for a substantial sum.

SpringWorks' Growing Portfolio

Gomekli's approval adds to SpringWorks' recent successes. In November 2023, the company received FDA approval for Ogsiveo, a treatment for patients with desmoid tumors requiring systemic therapy. Ogsiveo has shown strong initial performance, with sales reaching $61 million in the fourth quarter and $172 million for the year 2024.

SpringWorks, originally a spinout from Pfizer established in 2017, has quickly built a valuable portfolio of rare disease treatments. The company reported a solid financial position, closing 2024 with $462 million in cash, cash equivalents, and marketable securities.

As SpringWorks prepares to make Gomekli available within two weeks, the pharmaceutical industry awaits further developments in the potential acquisition by Merck KGaA, which could significantly reshape the rare disease treatment landscape.