The FDA has approved GOMEKLI™(mirdametinib) for the treatment of NF1-PN in both adult and pediatric patients, according to SpringWorks Therapeutics

The U.S. Food and Drug Administration (FDA) has approved GOMEKLI™ (mirdametinib), SpringWorks Therapeutics, Inc.’s MEK inhibitor, for the treatment of adult and pediatric patients with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) that are not amenable to complete resection. SpringWorks Therapeutics, Inc. is a commercial-stage biopharmaceutical company that focuses on cancer and severe rare diseases.One Following the clearance, the FDA issued a rare pediatric condition priority review voucher (PRV) to SpringWorks.

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,

We are grateful to each clinical trial participant, their families, the investigators, and the patient advocacy groups involved in the journey towards making GOMEKLI™ available in the U.S. I am proud that we are delivering on our commitment to patients with devastating diseases with our company’s second FDA approval in less than 18 months.

Saqib Islam

About 100,000 children and adults in the US today suffer with NF1, a genetic condition. Plexiform neurofibromas, or PNs, are tumors that grow infiltratively along the peripheral nerve sheath and can cause significant deformity, discomfort, and functional impairment. Patients with NF1 have a 30–50% lifetime risk of acquiring PNs. In the US, there are about 40,000 patients with NF1-PN, most of whom are adults without access to an approved medication prior to GOMEKLI™. Malignant peripheral nerve sheath tumors, an aggressive and sometimes lethal condition, can develop from plexiform neurofibromas.

The infiltrative tumor growth pattern of plexiform neurofibromas along nerves makes surgical excision difficult, and up to 85% of plexiform neurofibromas are thought to be resistant to full resection.

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Patients with NF1-PN often face significant challenges with their health and have had limited treatment options to manage this devastating condition,

It was very encouraging in the ReNeu trial to see that GOMEKLI™ provided deep and durable responses, with a manageable safety profile that enabled patients to stay on therapy. This approval represents an important advance, especially for adults who previously did not have an approved treatment.

Christopher Moertel

The FDA granted SpringWorks a rare pediatric condition priority review voucher, and GOMEKLI was authorized under Priority Review. For the treatment of NF1-PN, GOMEKLI™ has already received Fast Track and Orphan Drug designations.

Based on findings from the Phase 2b ReNeu trial, which included 114 patients with NF1-PN ≥2 years of age (58 adults and 56 pediatric patients), the FDA approved GOMEKLI™.10 According to a blinded independent central review, GOMEKLI™ achieved a confirmed objective response rate (ORR) of 41% (N=24/58) in adults and 52% (N=29/56) in children, meeting the primary aim. Deep and long-lasting tumor volume reductions were seen; the median best percentage change in target PN volume was -41% in adults (range: -90 to 13%) and -42% in children (range: -91 to 48%). Of those with a confirmed response, 90% of children and 88% of adults had a response that lasted at least 12 months, while 48% and 50%, respectively, had a response that lasted at least 24 months. As measured by a variety of patient-reported outcome measures, patients in both groups also saw early and long-lasting improvements in their quality of life and pain compared to baseline.

A controllable safety and tolerability profile was displayed by GOMEKLI™. In individuals receiving GOMEKLI™, rash, diarrhea, nausea, musculoskeletal discomfort, vomiting, and exhaustion were the most frequently reported side effects (>25%). In children, rash, diarrhea, musculoskeletal discomfort, abdominal pain, vomiting, headache, paronychia, left ventricular dysfunction, and nausea were the most frequent adverse effects (>25%). Additional Important Safety Information is included below, along with Warnings & Precautions regarding embryo-fetal toxicity, dermatologic adverse reactions, left ventricular dysfunction, and ocular toxicity.

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GOMEKLI™ showed a controllable profile of safety and tolerability. Adults receiving GOMEKLI™ most frequently had rash, diarrhea, nausea, musculoskeletal pain, vomiting, and exhaustion (>25%). Rash, diarrhea, musculoskeletal discomfort, abdominal pain, headache, vomiting, paronychia, left ventricular dysfunction, and nausea were the most frequent adverse effects (>25%) that affected children. Additional Important Safety Information, such as Warnings & Precautions about eye toxicity, left ventricular dysfunction, adverse reactions in the skin, and embryo-fetal damage, is provided below.

We are excited to celebrate the extraordinary milestone of our partners and long-term friends at SpringWorks for the NF1-PN community. This FDA approval shows the power of collaboration to advance innovative science for drugs that may otherwise not have been taken forward,

When industry, researchers, and organizations like ours driving treatment innovation join forces, scientific progress moves faster, and patients gain access to the therapies they need. Every treatment approval is hard-won, built on research, persistence, and partnership. Today, that work delivers a critical new option for NF patients of all ages.

Annette Bakker

Also Read: Search for Novel Tuberculosis Drug Targets Accelerated by Artificial Intelligence

NF1-PN is a complex, devastating disease that affects not only individual patients, but entire families. Treatment advances are crucial to achieving better outcomes for patients and this FDA approval offers hope for NF patients and their families.

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Kim Bischoff

Source: SpringWorks Therapeutics – Press Release


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Graduated from the University of Kerala with B.Sc. Botany and Biotechnology. Attained Post-Graduation in Biotechnology from the Kerala University of Fisheries and Ocean Science (KUFOS) with the third rank. Conducted various seminars and attended major Science conferences. Done 6 months of internship in ICMR – National Institute of Nutrition, Hyderabad. 5 years of tutoring experience.

Ajmal Aseem

Graduated from the University of Kerala with B.Sc. Botany and Biotechnology. Attained Post-Graduation in Biotechnology from the Kerala University of Fisheries and Ocean Science (KUFOS) with the third rank. Conducted various seminars and attended major Science conferences. Done 6 months of internship in ICMR – National Institute of Nutrition, Hyderabad. 5 years of tutoring experience.

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