SpringWorks Gains FDA Green Light For Genetic Disorder Drug Gomekli

Following the recent statement by Merck KGaA that it is in advanced talks about buying SpringWorks Therapeutics, the rare illness specialist from Connecticut has increased its worth by receiving a fresh approval from the FDA.

On Tuesday, the regulatory agency gave its authorization for the uncommon genetic condition known as neurofibromatosis type 1 (NF1) to be treated with the drug Gomekli (mirdametinib), developed by SpringWorks.

To be more precise, Gomekli is directed at individuals who are at least 2 years old and have NF1 in conjunction with benign tumors that are symptomatic and cannot be eliminated surgically. The tumors, which are known as plexiform neurofibromas (PNs), grow along the sheath of the peripheral nerve and may take form either within or outside of the body.

Gomekli is the first therapy for adults as well as kids with the disease. Five years earlier, AstraZeneca and Merck received FDA clearance for Koselugo for kids with NF1-PNs; however, that approval is restricted to pediatric patients.

“The NF1-PN patient community has a great need for more treatment options,” Saqib Islam, SpringWorks’ CEO, stated 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 firm said that Gomekli would be accessible within two weeks, but did not disclose its pricing. The medicine is now undergoing evaluation in Europe, with the organization anticipating a decision in 2025.

Following the FDA backing, SpringWorks acquired a valuable priority review voucher for rare pediatric diseases, which may expedite the review procedure for a new medicine application or be sold.

This is the second clearance for SpringWorks in the last 15 months, a spinout from Pfizer founded in 2017, which has a portfolio of prospects for rare diseases.

In November 2023, the FDA approved SpringWorks’ Ogsiveo as a therapy for those with desmoid tumors requiring systemic treatment.

The firm announced last month that Ogsiveo is off to a great start with $61 million in Q4 and $172 million in annual sales. The company reported concluding last year with $462 million in cash, cash equivalents, and marketable securities.

Merck KGaA said it was in advanced negotiations to acquire SpringWorks shortly before the clearance. Due to the M&A discussions and subsequent acceptance, SpringWorks’ share price has risen by 34% this week, resulting in an increase in its market capitalization from just under $3 billion to $4.1 billion.

Gomekli’s recognition was supported by a phase 2b study demonstrating a 52% response rate in juvenile patients and 42% in adults. The study had 58 adult and 56 pediatric participants. Adverse effects documented by over 25% of individuals included diarrhea, rash, musculoskeletal discomfort, nausea, and vomiting.

NF1-PN impacts over 40,000 individuals in the U.S. and may result in discomfort, significant deformity, and functional limitations. According to SpringWorks, PNs may potentially develop malignancy.