Gomekli Shortage: What Providers and Prescribers Need to Know in 2026

Since Gomekli (mirdametinib) received FDA approval on February 11, 2025, NF1 specialists and oncology practices have been navigating the practical challenges of getting this new specialty drug into patients' hands. While there is no official FDA drug shortage, the structural access barriers for a newly approved specialty drug treating a rare disease are significant — and prescribers need to be prepared to help their patients navigate them.

Current Availability Status

Gomekli is not listed on the FDA's Drug Shortage Database as of 2026. SpringWorks Therapeutics distributes the medication through a specialty pharmacy and distributor network. The drug is available in two dosage forms: capsules (1 mg and 2 mg) and tablets for oral suspension (1 mg), the latter being particularly valuable for pediatric patients who cannot swallow capsules.

However, "available" in the specialty pharmacy context is not the same as "accessible." The typical access pathway for Gomekli involves multiple steps — PA submission, specialty pharmacy enrollment, financial assistance assessment — any of which can introduce delays. For providers, anticipating and addressing these barriers proactively is key to minimizing treatment delays for NF1-PN patients.

Common Prior Authorization Criteria to Document

Most commercial and government insurance plans require prior authorization for Gomekli. The PA criteria typically align with the FDA-approved indication. Ensure your documentation clearly addresses all of the following:

Confirmed genetic or clinical diagnosis of neurofibromatosis type 1 (NF1)

Presence of symptomatic plexiform neurofibromas causing significant morbidity (pain, functional impairment, or disfigurement)

Documentation that PNs are not amenable to complete surgical resection (with clinical or surgical rationale)

Patient age 2 years or older (FDA-approved population)

ECOG or Lansky performance status, or documentation of patient's functional status

MRI or volumetric imaging documenting target PN lesions

Using SpringWorks CareConnections as a Practice Resource

SpringWorks CareConnections is an important practice resource for providers prescribing Gomekli. Field Access Managers (FAMs) from SpringWorks can assist your practice in person or virtually to facilitate access, provide regional payor education, and respond promptly to coverage questions.

From the HCP side, CareConnections offers:

Benefits investigation to determine coverage and out-of-pocket costs before submitting PA

PA support and appeals assistance

Bridge supply for eligible patients experiencing insurance delays

Patient Assistance Program (PAP) for uninsured or underinsured patients — medication at no cost

Nurse Advocate support to help patients stay on therapy and manage adherence

To enroll a patient in CareConnections, download the enrollment form from springworkstxcares.com and submit it with patient authorization. You can also call 844-CARES-55 (844-227-3755), Monday through Friday, 8 AM to 10 PM ET.

Clinical Safety Monitoring Requirements for Providers

Gomekli carries important safety monitoring requirements that providers must be prepared to manage. These include:

Ocular toxicity: Conduct comprehensive ophthalmic assessments before initiating therapy, at regular intervals, and for any new visual changes. Retinal vein occlusion occurred in 2.7% of adults; retinal pigment epithelium detachment in 1.3%.

Left ventricular dysfunction: Obtain echocardiogram before treatment, every 3 months during the first year, and periodically thereafter. The risk is higher in children.

Dermatologic reactions: Rash occurred in >25% of patients. Initiate supportive care at first signs. Withhold, reduce dose, or permanently discontinue based on severity.

Embryo-fetal toxicity: Verify pregnancy status before initiating therapy. Counsel females on contraception during treatment and for 6 weeks after last dose; males with female partners of reproductive potential should use contraception during treatment and for 3 months after last dose.

Who Is Qualified to Prescribe Gomekli?

Gomekli is not a controlled substance and does not require a special DEA registration to prescribe. However, given the monitoring requirements and the complexity of NF1 management, it should ideally be prescribed by or in consultation with providers experienced in NF1, oncology, or rare disease medicine. These include:

Medical oncologists and neuro-oncologists

Pediatric hematologists/oncologists

Neurologists and pediatric neurologists (including those credentialed in adult neurology who also see NF patients)

Rare disease and genetics specialists at NF multidisciplinary clinics

NPs and PAs working under oncology or rare disease supervision

How medfinder Can Support Your Patients' Access

medfinder for providers is a service that calls pharmacies — including specialty pharmacies — on your patients' behalf to identify which ones can fill their Gomekli prescription. This eliminates the time your office staff would otherwise spend on the phone and ensures patients are connected to an available dispenser quickly.

For a complete step-by-step guide on supporting patient access, see our article on how to help your patients find Gomekli in stock.