Ocular Melanoma - Market Insight, Epidemiology and Market Forecast - 2034

Key Highlights

• Ocular melanoma, while rare, is the most common primary cancer of the eye in adults, primarily affecting the uveal tract. The condition originates from melanocytes, pigment-producing cells, and can potentially metastasize, commonly to the liver.
• Ocular melanoma can be divided into uveal and non-uveal melanoma. Uveal melanoma (~95%) comprises the larger group of ocular melanoma and consists of choroidal, ciliary body, and iris melanoma. Non-uveal melanoma includes conjunctival melanoma and ocular melanoma from other sites.
• Ocular melanoma predominantly affects older individuals, with a higher incidence in fair-skinned individuals and those with lighter eye colors. Risk factors include dysplastic nevus syndrome, certain cutaneous nevi, and a family history of systemic and ocular cancers, possibly linked to genetic mutations like BAP1, GNAQ, and GNA11 alterations.
• Ocular melanoma may manifest with varied symptoms, including blurred vision, photopsia, and metamorphopsia, or remain asymptomatic for years. Diagnosis involves a comprehensive clinical evaluation, specialized tests like ocular ultrasound, fluorescein angiography, optical coherence tomography, and, in some cases, fine needle aspiration biopsy for genetic analysis.
• In 2023, the United States accounted for the highest number of diagnosed incident cases of ocular melanoma in the 7MM.
• In the United States, ocular melanoma primarily affects individuals aged 40-70 years, constituting approximately ~60% of the total cases.
• Among the EU4 and the UK, out of all diagnosed incident cases of uveal melanoma, choroidal cases were highest, followed by ciliary body and iris melanoma in 2023.
• The treatment landscape for ocular melanoma is characterized by a personalized approach using radiation and surgery, considering factors such as tumor size, location, patient health, and individual preferences.
• Metastatic uveal melanoma poses a significant challenge, having limited approved therapies. Current interventions, including chemotherapy and immunotherapy, show limited efficacy.
• In the 7MM, the United States accounted for the highest market size, with nearly 60% of the market share of the ocular melanoma market as compared to EU4 and the UK and Japan in 2023.
• In 2023, among EU4 and the UK, the UK accounted for the largest market size, while Spain accounted for the lowest share.
• Recent FDA approvals of KIMMTRAK and HEPZATO KIT offer new options for patients with metastatic uveal melanoma. Emerging therapies in the pipeline, including Darovasertib + Crizotinib and Belzupacap Sarotalocan, are expected to bring a positive shift in the ocular melanoma treatment landscape during the forecast period (2024-2034).
• Key players like Aura Biosciences, IDEAYA Biosciences, and TriSalus Life Sciences are expected to transform the treatment landscape for ocular melanoma patients.
• Lack of adequate and personalized treatment options and standardized diagnostic and sampling procedures are critical unmet needs of ocular melanoma patients. Increased patient participation in clinical trials can also help in expanding the limited treatment landscape of ocular melanoma.

Report Summary

• The report offers extensive knowledge regarding the epidemiology segments (by region, diagnosed incident cases of ocular melanoma, type-specific cases, mutation-specific cases, age-specific cases, stage-specific cases, and total treated cases) and predictions, presenting a deep understanding of the potential future growth in diagnosis rates, disease progression, and treatment guidelines. It provides comprehensive insights into these aspects, enabling a thorough assessment of the subject matter.
• Additionally, an all-inclusive account of the current management techniques and emerging therapies such as Darovasertib in combination with Crizotinib, Belzupacap sarotalocan, and the elaborative profiles of late and mid-stage (Phase III and Phase II) and prominent therapies that would impact the current treatment landscape and result in an overall market shift has been provided in the report.
• The report also encompasses a comprehensive analysis of the Ocular melanoma market, providing an in-depth examination of its historical and projected market size (2020-2034). It also includes the market share of therapies, detailed assumptions, and the underlying rationale for our methodology. The report also includes drug outreach coverage in the 7MM region.
• The report includes qualitative insights that provide an edge while developing business strategies by understanding trends through SWOT analysis and expert insights/KOL views, including experts from various hospitals and prominent universities, patient journey, and treatment preferences that help shape and drive the 7MM Ocular melanoma market.

Market

Various key players, such as Aura Biosciences, IDEAYA Biosciences, TriSalus Life Sciences, and others, are involved in developing therapies for Ocular Melanoma. The expected launch of emerging therapies and other treatments will lead to a significant increase in the market size during the forecast period [2024-2034].

• In 2023, the total market size of ocular melanoma was around USD 340 million, which is expected to increase by 2034 during the study period (2020-2034) in the 7MM.
• Among the 7MM, the United States accounted for the highest market size in 2023, followed by the United Kingdom for ocular melanoma.
• During the forecast period (2024-2034), pipeline candidates such as Darovasertib + Crizotinib and Belzupacap Sarotalocan are expected to drive the rise in the ocular melanoma market size.
• By 2034, KIMMTRAK (tebentafusp) is expected to garner the highest market share in the 7MM.

Ocular Melanoma Drug Chapters

The section dedicated to drugs in the Ocular melanoma report provides an in-depth evaluation of pipeline drugs (Phase III and Phase II) related to ocular melanoma.

The drug chapters section provides valuable information on various aspects related to clinical trials of ocular melanoma, such as the pharmacological mechanisms of the drugs involved, designations, approval status, patent information, and a comprehensive analysis of the pros and cons associated with each drug. Furthermore, it presents the most recent news updates and press releases on drugs targeting ocular melanoma.

Marketed Therapies

KIMMTRAK (tebentafusp-tebn): Immunocore

KIMMTRAK is a novel bispecific protein comprised of a soluble T-cell receptor fused to an anti-CD3 immune-effector function. The drug explicitly targets gp100, a lineage antigen expressed in melanocytes and melanoma. This is the first molecule developed using Immunocore’s ImmTAC technology platform designed to redirect and activate T cells to recognize and kill tumor cells.

KIMMTRAK is indicated for treating adult patients with unresectable or metastatic uveal melanoma. Immunocore is planning to expand access to KIMMTRAK to more patients in the United States, Europe, and globally as it continues to establish the therapy as the standard of care for the first-line treatment for metastatic uveal melanoma in countries where it is launched.

In November 2023, Immunocore signed an agreement for a European Organization for Research and Treatment of Cancer (EORTC)-sponsored trial to study KIMMTRAK as adjuvant therapy for uveal (or ocular) melanoma (ATOM).

HEPZATO KIT (melphalan for Injection/Hepatic Delivery System): Delcath Systems

HEPZATO KIT (melphalan for injection/hepatic delivery system) is a combination drug/device product that administers HEPZATO (melphalan) directly to the liver through the HDS, which permits higher drug exposure in target tissues while limiting systemic toxicity.

Melphalan is an alkylating drug of the bischloroethylamine type. As a result, its cytotoxicity appears to be related to the extent of its interstrand cross-linking with DNA, probably by binding at the N7 position of guanine. It is active against both resting and rapidly dividing tumor cells.

In August 2023, the FDA approved HEPZATO KIT (melphalan for injection/hepatic delivery system) containing melphalan as a liver-directed treatment for adult patients with uveal melanoma with unresectable hepatic metastases affecting less than 50% of the liver and no extrahepatic disease, or extrahepatic disease limited to the bone, lymph nodes, subcutaneous tissues, or lung that is amenable to resection or radiation.

Efficacy was evaluated in the FOCUS study (NCT02678572), a single-arm, multicenter, open-label trial in 91 patients with uveal melanoma with unresectable hepatic metastases.

Emerging Therapies

Darovasertib (IDE196): IDEAYA Biosciences

Darovasertib is a potent, orally available selective small molecule inhibitor of PKC that is being developed for genetically defined cancers having GNAQ or GNA11 gene mutations. PKC is a protein kinase that functions downstream of the GTPases GNAQ and GNA11.

The company is evaluating darovasertib in a Phase II/III trial as a combination therapy with Pfizer’s investigational cMET inhibitor, crizotinib, in patients with metastatic uveal melanoma, a population in which >90% of patients have tumors harboring GNAQ or GNA11 mutations, and separately as a single-agent in Phase II clinical trial as neoadjuvant and adjuvant therapy in patients having primary uveal melanoma.

In December 2022, IDEAYA Biosciences announced that the FDA granted Fast Track designation (FTD) to IDEAYA’s development program investigating darovasertib for use in combination with crizotinib, an investigational cMET inhibitor, for the treatment of adult patients with metastatic uveal melanoma (IDEAYA Biosciences, 2022a).

Belzupacap Sarotalocan (AU-011): Aura Biosciences

Belzupacap Sarotalocan (Bel-sar) is a novel VDC that consists of an HPV-derived VLP conjugated to hundreds of infrared laser-activated molecules. VDCs are a novel class of drugs with a dual mechanism of action that promotes cancer cell death by both the delivery of the cytotoxic payload to generate acute necrosis and by activating a secondary immune-mediated response. A simple suprachoroidal injection delivers the drug and has the potential to be the first approved therapy in primary choroidal melanoma.

Belzupacap sarotalocan has received ODD for the treatment of uveal melanoma from the US FDA and the EMA. The drug has also received FTD from the US FDA for the treatment of choroidal melanoma (Aura Biosciences, 2023c).

In December 2023, Aura Biosciences announced that the first patient had been dosed in the global Phase III CoMpass trial, evaluating the safety and efficacy of bel-sar for the first-line treatment of adult patients with early-stage choroidal melanoma (Aura Biosciences, 2023a).

Ocular Melanoma Market Outlook

The treatment scenario for ocular melanoma includes a variety of treatments, depending on the size and location of the tumor, whether it has spread, and the patient’s general health. The purposes of uveal melanoma treatment are to avoid metastatic dissemination and to preserve the eye with useful vision.

Treatment options for primary lesions include radiation (brachytherapy or proton beam therapy) or surgical enucleation. Approximately half of the patients will develop metastatic disease, and patients remain at lifelong risk of relapse. Most commonly, relapse occurs in the liver (~90%) with less common sites for metastatic spread, including the lung, bone, and skin (Howlett et al., 2023).

Local therapies, such as surgical interventions, play a significant role in addressing ocular melanoma, particularly for smaller tumors. Surgical removal, employing techniques like resection, laser therapy, or thermotherapy, is a common approach to managing the disease at its early stages. Additionally, localized radiation, often administered through methods like plaque brachytherapy or proton therapy, can be employed to target and reduce the size of the tumor. In cases where the tumor is large or causing severe complications, the recommendation might be enucleation, which involves the complete removal of the eye.

Adjuvant therapies, complementing primary treatments, further enhance the therapeutic arsenal. Transpupillary Thermotherapy (TTT) utilizes heat to treat the tumor and may be used in conjunction with other therapies. Photodynamic Therapy (PDT) involves the administration of a light-sensitive drug followed by laser therapy to destroy cancer cells selectively.

There has been a dearth of systemic therapies for the treatment of uveal melanoma for a long time.

Approval of KIMMTRAK (tebentafusp) and HEPZATO KIT (melphalan/hepatic delivery system; HDS) has led to a positive shift in the treatment landscape of ocular melanoma.

The emergence of therapies such as darovasertib in combination with crizotinib and belzupacap sarotalocan is expected to improve the treatment scenario of ocular melanoma further. An uptick in the diagnosis of ocular melanoma has been observed in recent years, indicating a growing awareness and improved diagnostic capabilities for this rare form of eye cancer. Additionally, ongoing research and clinical trials in the field of ocular melanoma contribute to a deeper understanding of the disease and may lead to further improvements in diagnostic and treatment strategies.

Ocular Melanoma Disease Understanding and Treatment

Ocular Melanoma Overview

Ocular melanoma is an extremely rare form of cancer that affects the eye. Although rare, it is the most common primary cancer of the eye in adults. Primary means that the cancer began at that site (in this case, the eye) and did not spread to another part of the body.

In most people, this cancer arises in a part of the eye known as the uveal tract. The uveal tract is the colored (pigmented) layer of tissue that is found beneath the white of the eye (sclera) and is composed of normally pigmented cells and blood vessels. In the front of the eye, the uvea is made up of the colored part of the eye (iris) and a circle of muscle tissue (ciliary body) that releases a transparent fluid (aqueous humor) into the eye and helps control the shape of the lens. The largest area of the uveal tract is in the back part of the eye (choroid), which is located beneath the retina, the vision-sensing portion of the eye. In most instances, ocular melanomas arise within the choroid.

Typically asymptomatic, ocular melanoma is often identified by optometrists during routine eye examinations. Some individuals, however, may manifest signs such as diminished or blurred vision in one eye, peripheral vision loss, the presence of brown or dark patches on the eye’s white surface, a dark spot on the iris, the perception of small specks or wavy lines (referred to as ‘floaters’) in their vision, occasional flashes in the vision, and alterations in the shape of the pupil.

Ocular melanoma arises from cells called melanocytes, which are the cells of the body that produce pigment. Ocular melanoma is a cancerous (malignant) tumor that can potentially spread (metastasize) to other parts of the body, most often to the liver.

The exact cause of ocular melanoma remains largely unidentified in most cases. Nevertheless, various risk factors have been associated with the development of this eye cancer. These include having fair or pale skin, possessing light-colored eyes, having a family history of melanoma, the presence of growths on or within the eye, advancing age, and certain skin conditions that lead to the formation of abnormal moles, thereby elevating the risk of ocular melanoma.

Ocular Melanoma Diagnosis

The diagnosis of ocular melanoma involves various tests conducted by a doctor or optometrist, followed by specialized assessments by an ophthalmologist with expertise in ocular oncology.

The tests include ophthalmoscopy to examine the inside of the eye, color fundus photography to capture images of the eye’s back, ultrasound to create pictures of the eye’s interior, trans illumination to identify abnormal areas, and CT and MRI scans for detailed imaging. In some cases, a biopsy is used to examine the tissue under a microscope. The choice of diagnostic tests depends on the suspicion of ocular melanoma and aims to determine the tumor’s location, size, and potential spread.

After someone is diagnosed with eye cancer, doctors will try to figure out if it has spread and, if so, how far. This process is called staging. The stage of cancer describes how much cancer is in the body. It helps determine how serious the cancer is and how best to treat it. Doctors also use a cancer’s stage when talking about survival statistics. The cancer stage is determined from the results of eye exams, imaging tests (ultrasound, CT or MRI scan, etc.), and other tests. A staging system is a standard way for the cancer care team to describe how far a cancer has spread. The most common systems used to describe the stages of eye melanomas are the American Joint Committee on Cancer (AJCC) TNM system and the system used by the Collaborative Ocular Melanoma Study (COMS) group.

Ocular Melanoma Treatment

The therapeutic management of individuals with ocular melanoma may require the coordinated efforts of a team of medical professionals, such as specialists in the diagnosis and treatment of eye disorders (ocular oncologists who are specially trained ophthalmologists), eye surgeons, physicians who specialize in the diagnosis and treatment of cancer (medical oncologists), physicians who use radiation to treat cancer (radiation oncologists), and other healthcare specialists.

Specific therapeutic procedures and interventions may vary, depending upon numerous factors, such as disease stage, tumor size, specific location of the tumor within the eye, the presence or absence of certain symptoms, an individual’s age and general health, and/or other elements. Historically, the main therapeutic options have been radiation therapy or surgery.

The most common method of treating the eye with radiation therapy is a process called brachytherapy, which may also be known as “plaque therapy,” endocurietherapy, or sealed source radiotherapy. During brachytherapy, radioactive material (implant) is placed on a small disk called a plaque. This disk is inserted into the eye socket next to or near the base of a tumor and secured to the outside of the eye. The disk is left in place for several days and then removed.

External beam radiotherapy may be used with a specialized technique called proton beam radiation. This procedure uses a machine outside of the body that delivers laser beams to the eye to destroy cancer cells. There are different types of external beam radiotherapy. Both plaque brachytherapy and proton therapy are effective treatments for ocular melanoma.

In 2022, the US FDA approved KIMMTRAK (tebentafusp-tebn) for adult patients with uveal melanoma that has spread (metastasized) or cannot be surgically removed. In order to receive this therapy, a patient must have a blood test to determine if they have a specific human leukocyte antigen (HLA) called HLA-A*02:01.

A variety of surgical techniques are also used to treat ocular melanoma. Sometimes, physicians will recommend surgical removal (resection) of the entire affected eye (enucleation). Other times, physicians may recommend surgical removal of the tissue that is affected by the disease (local resection).

Ocular Melanoma Epidemiology

The Ocular Melanoma epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by diagnosed incident cases,type-specific cases, mutation-specific cases, age-specific cases, stage-specific and treated cases in the United States, EU4 countries (Germany, France, Italy, Spain) and the United Kingdom, and Japan from 2020 to 2034.

• Among the 7MM, the United States accounted for the highest number of diagnosed incident cases of ocular melanoma, which is 49.0% of the diagnosed incident cases of ocular melanoma in 2023.
• In the US, out of all diagnosed incident cases of uveal melanoma, choroidal cases were highest, followed by ciliary body and iris melanoma in 2023.
• Among the EU4 and the UK, the United Kingdom accounted for the highest number of ocular melanoma cases, followed by France, whereas Spain accounted for the lowest number of diagnosed incident cases of ocular melanoma.
• In Japan, the highest mutation type-specific cases of ocular melanoma were for GNA11, followed by GNAQ, BAP1, and SF3B1 mutation in 2023.

KOL Views

To stay abreast of the latest trends in the market, we conduct primary research by seeking the opinions of Key Opinion Leaders (KOLs) and Subject Matter Experts (SMEs) who work in the relevant field. This helps us fill any gaps in data and validate our secondary research.

We have reached out to industry experts to gather insights on various aspects of Ocular melanoma, including the evolving treatment landscape, patients’ reliance on conventional therapies, their acceptance of therapy switching, drug uptake, and challenges related to accessibility. The experts we contacted included medical/scientific writers, professors, and researchers from prestigious universities in the US, Europe, the UK, and Japan.

Our team of analysts connected with more than 10 KOLs across the 7MM. We contacted institutions such as the Melanoma Research Foundation, Harvard Medical School, Massachusetts General Hospital Cancer Center, etc., among others. By obtaining the opinions of these experts, we gained a better understanding of the current and emerging treatment patterns in the Ocular melanoma market, which will assist our clients in analyzing the overall epidemiology and market scenario.

Qualitative Analysis

We perform Qualitative and Market Intelligence analysis using various approaches, such as SWOT analysis and Conjoint Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst’s discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.

Conjoint Analysis analyzes multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, designation, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy. In efficacy, the trial’s primary and secondary outcome measures are evaluated. Based on these, the overall efficacy is evaluated.

Further, the therapies’ safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the route of administration, order of entry and designation, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.

Market Access and Reimbursement

Because newly authorized drugs are often expensive, some patients escape receiving proper treatment or use off-label, less expensive prescriptions. Reimbursement plays a critical role in how innovative treatments can enter the market. The cost of the medicine, compared to the benefit it provides to patients who are being treated, sometimes determines whether or not it will be reimbursed. Regulatory status, target population size, the setting of treatment, unmet needs, the number of incremental benefit claims, and prices can all affect market access and reimbursement possibilities.

The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Ocular Melanoma Report Insights

• Patient Population
• Therapeutic Approaches
• Ocular Melanoma Market Size and Trends
• Existing Market Opportunity

Ocular Melanoma Report Key Strengths

• Eleven-year Forecast
• The 7MM Coverage
• Ocular Melanoma Epidemiology Segmentation
• Key Cross Competition

Ocular Melanoma Report Assessment

• Current Treatment Practices
• Reimbursements
• Market Attractiveness
• Qualitative Analysis (SWOT, Conjoint Analysis, Unmet needs)

Key Questions

• Would there be any changes observed in the current treatment approach?
• Will there be any improvements in ocular melanoma management recommendations?
• Would research and development advances pave the way for future tests and therapies for ocular melanoma?
• Would the diagnostic testing space experience a significant impact and lead to a positive shift in the treatment landscape of ocular melanoma?
• What kind of uptake will the new therapies witness in the coming years in ocular melanoma patients?