Neurofibromatosis Type 1 (NF1) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Neurofibromatosis Type 1 (NF1) Market Outlook

Thelansis’s “Neurofibromatosis Type 1 (NF1) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Neurofibromatosis Type 1 treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Neurofibromatosis Type 1 (NF1) Overview

Neurofibromatosis type 1 (NF1) is a neurocutaneous genetic disorder known for its diverse clinical manifestations, including café-au-lait spots, Lisch nodules in the iris, freckling in axillary and inguinal regions, and multiple neurofibromas. NF1 condition exhibits significant variability in clinical features, even among individuals within the same family. Almost all patients display multiple café-au-lait macules, some present at birth and most emerging within the first year of life. Intertriginous freckling typically begins around the age of 5. In adults, cutaneous and subcutaneous neurofibromas develop and continue to increase in number and size with age. It's important to note that these cutaneous neurofibromas do not turn malignant. However, plexiform neurofibromas, which grow along nerves and their branches, may cause disfigurement, pain, and functional impairments. They are usually present at birth and may become malignant later in life. Ocular manifestations of NF1 include optic pathway gliomas and iris hamartomas (Lisch nodules). Optic pathway gliomas usually appear before age 6 and rarely progress beyond that age. Additionally, NF1 can lead to various skeletal issues, such as osteopenia, osteoporosis, bone overgrowth, short stature, microcephaly, scoliosis, and various skeletal dysplasias. Other associated features encompass hypertension, vasculopathy, intracranial tumors, malignant peripheral nerve sheath tumors (MPNST), and, occasionally, seizures or hydrocephalus. While intellectual development is typically not severely affected, cognitive deficits and learning difficulties are common in 50% to 75% of cases. The overall cancer risk in NF1 is higher than in the general population, with a lifetime risk of 10-12% for MPNST, primarily occurring between ages 20 and 40, and an elevated risk of breast cancer before age 50. Variants of NF1, such as familial spinal and segmental forms and Watson syndrome, exist within the NF1 spectrum. Neurofibromatosis-Noonan syndrome represents a variant in 99% of cases. To diagnose NF1 formally, specific criteria have been established. Two or more of the following are diagnostic: more than 5 café-au-lait macules, 2 or more neurofibromas or one plexiform neurofibroma, optic glioma, freckling, 2 or more Lisch nodules, specific bone dysplasias, or a first-degree relative with NF1. Magnetic resonance imaging can assess the extent of plexiform neurofibromas, while molecular genetic testing is rarely necessary. Legius syndrome, observed in about 2% of individuals meeting NF1 diagnostic criteria, often closely resembles NF1 clinically. However, some individuals with NF1 may not develop non-pigmentary manifestations, resembling Legius syndrome patients. Constitutional mismatch repair deficiency syndrome should be considered a differential diagnosis in certain cases. Other differential diagnoses include McCune-Albright syndrome, Noonan syndrome with lentigines, and Proteus syndrome. Most instances of multiple non-ossifying fibromatosis can be attributed to NF1. The overall prognosis for NF1 is generally favorable, but significant morbidity is common. MPNST, in particular, carries a poor prognosis. Malignancy and vascular disease are the leading causes of early mortality. Currently, no treatment to reverse NF1 exists, but its signs and symptoms can be managed and addressed.

• NF1 is considered one of the most common genetic conditions, affecting an estimated 1 in 3,000 people worldwide, with up to 30% to 50% of affected individuals having no family history.

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2023-2033

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- Data Inputs with sourcing

- Market Event and Product Event

- Country-specific Forecast Model

- Market uptake and patient share uptake

- Attribute Analysis

- Analog Analysis

- Disease burden and pricing scenario

- Summary and Insights

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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