Novel FDA Approvals – February 2025
February’s approvals highlight progress in underserved spaces, with therapies addressing rare diseases and previously untreated populations. From pediatric and adult access in NF1-PN to a safer TGCT alternative, this month is a powerful example of how precision and safety are shaping the future of pharma.
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The FDA’s Center for Drug Evaluation and Research (CDER) is responsible for regulating drug products in the United States and approving novel therapeutics. Over the past decade, from 2015 to 2024, the Center for Drug Evaluation and Research approved an average of 47 novel drugs per year.
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*Sourced from the FDA as of February 2025 at https://www.fda.gov/media/184967/download?attachment#:~:text=The%2010%2Dyear%20graph%20below,novel%20drug%20approvals%20per%20year.&text=CDER%20identified%2024%20of%20the,as%20first%2Din%2Dclass.
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Of the 50 novel therapeutics approved in 2024, 24 (48%) of them were identified as first-in-class treatments that provide unprecedented pharmacologic effects in their targeted disease state.
During February 2025, The Center for Drug Evaluation and Research approved 2 novel drugs to enter the pharmaceutical market:
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Gomekli (mirdametinib)
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Romvimza (vimseltinib)
This article will take a deeper look into each of these newly approved medications, exploring the pharmacologic properties and approval pathways associated with them.
Gomekli
After being granted with Priority Review for its New Drug Application, SpringWorks Therapeutics, Inc. received FDA approval for its new MEK inhibitor, mirdametinib (Gomekli), on February 11, 2025. SpringWorks Therapeutics is a biopharmaceutical company that focuses on expanding treatment options for patients with severe rare diseases and cancer. Staying on brand, Gomekli received approval for the treatment of adult and pediatric patients with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas not amenable to complete resection (NF1-PN). Gomekli is the first and only medication currently FDA-approved for the treatment of NF1-PN in children and adults.
"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.” said Annette Bakker, Ph.D., Chief Executive Officer of the Children’s Tumor Foundation, regarding this new approval.
"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.” said Annette Bakker, Ph.D., Chief Executive Officer of the Children’s Tumor Foundation, regarding this new approval.
About Neurofibromatosis Type 1
Neurofibromatosis is an autosomal dominant genetic disorder that causes benign tumors to form throughout the body. This disease encompasses three different subtypes: neurofibromatosis type 1 (NF1), neurofibromatosis type 2 (NF2), and schwannomatosis. Neurofibromatosis type 1, also known as Von Recklinghausen’s disease, is the most common form of this disease, affecting nearly 1 in 3000 people worldwide.
While most people with neurofibromatosis type 1 have an average life expectancy, there are various symptoms and problems that may develop as a result of the disease. Common clinical manifestations of neurofibromatosis type 1 include:
Cafe-au-lait spots
Tan or brown birthmarks that often present at birth. While these spots are common for anyone, it is generally understood that having six or more spots raises suspicion for neurofibromatosis.
Lisch nodules
Tiny, pigmented bumps that appear on the iris of the eye. These will often appear during teenage years and do not require any treatments or monitoring.
Freckling in the armpits or groin
Often appearing by the age of 5, freckles can be very similar to cafe-au-lait spots though they are smaller in size. The presence of freckles alone does not indicate neurofibromatosis, but they may be suggestive of the disease alongside other symptoms.
Optic pathway glioma
These slow-growing brain tumors develop in or around the optic nerve and occur in 10-15% of children with neurofibromatosis type 1. The risk of developing an optic pathway glioma is highest in children between the ages of 1 and 6, and while they often do not cause symptoms, they may alter vision or require chemotherapy.
Growth abnormalities
Children with neurofibromatosis type 1 are often shorter than most kids their age and have larger head sizes than normal (macrocephaly).
Neurofibromas
Noncancerous, rubbery lumps that form on the skin (cutaneous), under the skin (subcutaneous), or on any nerve throughout the body. While they may be painful or itchy, they do not typically cause further symptoms. These most often appear in children between the ages of 10 to 15.
Plexiform neurofibromas: as the most complex type of neurofibroma, these occur in nearly 50% of patients with neurofibromatosis type 1 and can involve multiple nerves and their surrounding tissues. These may often occur deep within the body and can only be detected via medical imaging (i.e., MRI scan). These neurofibromas can become cancerous in up to 10% of patients with neurofibromatosis type 1.
There is no cure for neurofibromatosis type 1, but there are treatments available to help manage symptoms and associated conditions.
How Does Datroway Work?
Mechanism of Action
Gomekli inhibits mitogen‐activated protein kinase kinase (MEK1/MEK2), which are key components of the cellular RAS-MAPK signal transduction pathway. Neurofibromatosis is primarily characterized by mutations in the NF1 gene, which renders the cell unable to adequately produce neurofibromin. Neurofibromin is a tumor suppressor gene that negatively regulates RAS signaling, and so patients with neurofibromatosis type 1 have hyperactive RAS signaling, driving the growth and proliferation of plexiform neurofibroma.
By inhibiting MEK1/2, Gomekli can effectively block the downstream effects of hyperactive RAS signaling, reducing the phosphorylation of extracellular signal-regulated kinase (ERK) and thus decreasing cell proliferation and tumor growth.
A depiction of this mechanism can be viewed below:
By inhibiting MEK1/2, Gomekli can effectively block the downstream effects of hyperactive RAS signaling, reducing the phosphorylation of extracellular signal-regulated kinase (ERK) and thus decreasing cell proliferation and tumor growth.
A depiction of this mechanism can be viewed below:
ERK=extracellular signal-regulated kinase; MAPK=mitogen-activated protein kinase; MEK=mitogen-activated protein kinase kinase; RAF=rapidly accelerated fibrosarcoma; RAS=rat sarcoma viral oncogene homolog.
*Sourced from SpringWorks Therapeutics as of April 2025 at https://www.gomekli.com/hcp/moa-and-reneu-study-design
Clinical Trial Evidence
The phase 2b ReNeu clinical trial was pivotal for Gomekli in obtaining FDA approval. The study included 58 adults (≥18 years) and 56 children (2 to 17 years) with symptomatic plexiform neurofibromas linked to neurofibromatosis type 1 causing significant morbidities. Enrolled patients received mirdametinib (Gomekli) at a dose of 2 mg/m² twice daily (maximum 4 mg twice daily) in a 3-week on/1-week off cycle for 24 total cycles (~22 months).
The primary endpoint in ReNeu was the confirmed overall response rate (ORR), defined as complete response (disappearance of the targeted plexiform neurofibroma) or partial response (≥ 20% reduction). The primary endpoint was met in 41% of adult patients and 52% of pediatric patients.
Entering the Current Landscape
It is approximated that 40,000 people in the United States are living with plexiform neurofibromas associated with neurofibromatosis type 1 (NF1-PN), and the majority of which are adults who have not been approved for treatment until Gomekli.
Prior to Gomekli’s approval, the mainstay of treatment for plexiform neurofibromas with neurofibromatosis type 1 was surgical resection, or Koselugo (selumetinib) for kids above the age of 2.
Koselugo
Koselugo (selumitinib) works via the same pathway as Gomekli, but it is only approved for use in pediatric patients with plexiform neurofibromas and neurofibromatosis type 1. While the approval of Koselugo in April 2020 was a breakthrough for the treatment of plexiform neurofibromas, it excluded a major portion of the patient population by not covering adults.
“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,” said Saqib Islam, Chief Executive Officer of SpringWorks.
Romvimza
After following the standard FDA approval pathway, Deciphera Pharmaceuticals received full approval for its novel drug, Romvimza (vimseltinib), on February 14, 2025. Deciphera is a commercial-stage biopharmaceutical company that aims to develop innovative treatment options for people with cancer. Romvimza is approved for treating adults with symptomatic tenosynovial giant cell tumors (TGCTs) who may experience worsening functional limitation or severe morbidity with surgical resection.
About Tenosynovial Giant Cell Tumors
Tenosynovial giant cell tumors are a group of rare, often non-life-threatening tumors that develop from the synovium of joints, bursae, or tendon sheaths. The synovium is a thin layer of connective tissue that lines the inside of the joints to aid in lubrication and protection of the joint. The bursae are small, fluid-filled sacs that act as cushions between tendons, muscles, and bones surrounding joints to reduce friction. Tendon sheaths are layers of connective tissues that cover and protect the tendons.
These giant cell tumors cause the affected synovium, bursae, or tendon sheaths to thicken and overgrow, making it difficult and/or painful to move the affected area. These tumors can develop anywhere, but are most commonly seen in the following areas:
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Fingers or thumbs
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Ankles or feet
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Knees
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Wrists
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Knees
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Elbows
While these tumors are benign, those who suffer may be more likely to develop rheumatoid arthritis or osteoarthritis in the affected joint(s).
How Does Grafapex Work?
Mechanism of Action
Romvimza is a kinase inhibitor that has high selectivity for the colony stimulating factor 1 receptor (CSF1R). In tenosynovial giant cell tumors, dysregulation of the CSF1 gene leads to overexpression of CSF1, bringing an excess of tumor-promoting, inflammatory cells to the tumor site. By inhibiting the CSF1 receptor, Romvimza decreases CSF1 signaling and inhibits tumor growth.
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*Sourced from Deciphera as of April 2025 at https://www.romvimzahcp.com/mod-and-moa
Clinical Trial Evidence
The phase 3 MOTION study of Romvimza included 123 patients with symptomatic tenosynovial giant cell tumor to evaluate its safety and efficacy profiles. The primary endpoint for efficacy was overall response rate (ORR) in tumor length at 25 weeks (6 months) of treatment. The trial was 2:1 randomized placebo-controlled, and 40% of patients receiving 30mg vimseltinib twice weekly met the overall response rate, compared to 0% for the patients in the placebo group. Compared to the placebo group, patients in the vimseltinib group also had statistically significant improvements in active range of motion, patient-reported physical function, and patient-reported pain at 25 weeks of treatment.
Hans Gelderblom, MD, from the Leiden University Medical Center in the Netherlands, one of the MOTION investigators, shared that Romvimza (vimseltinib) is “the first well-tolerated agent to demonstrate significant improvement in a number of other important quality-of-life measures without any observed liver injury as seen with other approved TGCT treatment.”
Entering the Current Landscape
Similar to neurofibromatosis, tynosynovial giant cell tumors do not have any definitive cure. While most are typically removed with surgery, tynosynovial giant cell tumors have a high rate of recurrence, even after surgery. Along with this, surgery may not be a feasible option for certain patients, as it can potentially cause worsening functional limitation or severe morbidity.
Before the approval of Romvimza, these patients were limited to the use of Turalio (pexidartinib) for the treatment of tynosynovial giant cell tumors that could not be surgically removed. While Turalio is effective in treating this condition, it is associated with an unpredictable risk of serious liver toxicity that can be potentially fatal in patients. The phase 3 MOTION trial showed no evidence of hepatotoxicity or drug-induced liver injury with vimseltinib.
A Summary of February Approvals
The pharmaceutical industry is highly variable with a growing arsenal of novel therapeutics, like Gomekli and Romvimza. By proactively exploring novel approval trends, Ceuticon strives to help pharmacists optimize patient care, promote medication safety, and offer informed guidance to their healthcare teams. At Ceuticon, we don’t just share updates—we connect them to your career. Our breakdowns help pharmacists and pharma professionals track what’s next, understand the real-world implications, and bring more strategic value to every conversation. If you’re ready to lead with knowledge, our tools and training can help you stay sharp, relevant, and prepared for the evolving landscape of life sciences.
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