NIEMANN-PICK DISEASE DRUG TYPE C TREATMENT MARKET ANALYSIS

Niemann-Pick disease type C (NPC) is a rare genetic disorder that is a type of lysosomal storage disorder. NPC leads to accumulation of cholesterol and other fatty substances in different tissues of the body, such as brain and others. The onset of specific symptoms for NPC varies from person to person. In most cases, the symptoms appear in the childhood, which progresses to cause life-threatening complications with increasing age. NPC occurs as a result of mutations in the NPC 1 gene and NPC 2 gene, which is inherited in an autosomal recessive manner.

Global Niemann-Pick Disease Drug Type C Treatment Market - Impact of the Coronavirus (COVID-19) Pandemic

Coronavirus (COVID-19) outbreak was first reported on December 31, 2019, in Wuhan, China. The World Health Organization declared COVID-19 as pandemic on March 11, 2020. According to the Coronavirus (COVID-19) Weekly Epidemiological Update by the World Health Organization, over 625 million cases and 6.56 million deaths due to coronavirus (COVID-19) were reported till October 25, 2022, across the globe.

Impact of COVID-19 on Demand and Supply of Vitamin B12

The COVID-19 pandemic and lockdown in various countries across the globe has impacted the financial status of businesses across all sectors including private healthcare sector. The COVID-19 pandemic has impacted the entire supply chain of the healthcare industry mainly due to strict lockdown in several regions. The COVID-19 pandemic has affected the economy of various regions across the globe in three main ways; 1) by directly affecting the production and demand; 2) by creating disruptions in distribution channels; and 3) through its financial impact on companies and financial markets. Several countries such as Thailand, Indonesia, and Singapore are facing problems with regards to transportation and distribution of healthcare products.

Thus, Coronavirus (COVID-19) pandemic is negatively impacted growth of the global Niemann-pick disease drug type C treatment market during the forecast period. The COVID-19 pandemic caused major disruptions to clinical trial execution in the U.S., impacting key stakeholders across the industry. Investigative site capabilities experienced upheaval, driven by staff furloughs, social-distancing protocols, financial losses, and concerns over patient safety. Sponsors, CROs (clinical research organizations), and other organizations that support drug development shifted to remote working environments. For instance, according to Lancet 2020, an estimated 80% of non- COVID-19 trials were stopped or interrupted as a result of the COVID-19 pandemic.

Global Niemann-Pick Disease Drug Type C Treatment Market is estimated to be valued at US$ 45.40  million in 2022, and is expected to exhibit a CAGR 22.0 % over the forecast period (2022-2030).

Figure 1: Global Niemann-Pick Disease Drug Type C Treatment Market Share (%) Analysis, By Drug Type, 2022

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Global Niemann-Pick Disease Drug Type C Treatment Market – Driver

Increasing number of company collaborations regarding niemann-pick disease drug type C treatment by market players is expected to fuel the market growth over the forecast period. For instance, in March 2020, Evox Therapeutics Limited, a leading exosome therapeutics company, announced the signing of a rare disease-focused partnership with Takeda Pharmaceutical Company Limited, a multinational pharmaceutical company. The collaboration will focus on developing up to five novel protein replacement and mRNA therapies, including Evox's preclinical programme in Niemann-Pick disease type C (NPC) and a second new programme directed at another undisclosed rare disease

Increasing number of product approvals is expected to boost the global niemann-pick disease drug type C treatment market growth over the forecast period. For instance, in March 2022, Azafaros B.V., a company developing new treatment options to help patients with rare metabolic disorders such as lysosomal storage diseases (LSDs), announced that the U.S. Food and Drug Administration (FDA) had granted Orphan Drug Designation (ODD) for AZ-3102, a novel small molecule with a unique dual mode of action, in Niemann-pick disease type C (NP-C). The designation was based on promising preclinical data of AZ-3120 in a NP-C mouse model.

Global Niemann-Pick Disease Drug Type C Treatment Market-Restraint

Termination in clinical trials is expected to hamper the growth of global niemann-pick disease drug type C treatment market over forecast period. For instance, in April 2022, Mandos, LLC, a biopharmaceutical company, terminated a phase II/III trial in Niemann-pick disease type C (In children, adolescents, and adults) in Australia, France, Germany, Italy, New Zealand, Singapore, Spain, Turkey, the U.K. and the U.S. (Intrathecal) (NCT02534844) (EudraCT2015-002548-15). The U.S. Food and Drug Administration (FDA) did not find the drug to be safe and effective for treating Niemann-pick disease type C.

Global Niemann-Pick Disease Drug Type C Treatment Market – Regional Analysis

On the basis of region, the global niemann-pick disease drug type C treatment market is segmented into North America, Latin America, Europe, Asia Pacific, Middle East, and Africa.

North America is expected to be the most lucrative region in the global niemann-pick disease Drug Type C, owing to increasing number of product approvals from regulatory authorities. For instance, on September 16, 2020, Orphazyme A/S, a biopharmaceutical company, received the U.S. Food and Drug Administration (FDA) approval, with priority review for New Drug Application (NDA), for arimoclomol for the treatment of Niemann Pick disease type C (NPC).

Europe is expected to witness significant growth in the forecast period, owing to increasing research and development activities for Niemann-Pick Disease Type C treatment. For instance, on September 30, 2020, IntraBio Inc., a private pharmaceutical company, announced positive results for the Phase II clinical trial, which is carried out for testing the safety and efficacy of drug candidate, IB1001, for the treatment of NPC. The clinical trial showed a decrease in the severity of ataxia, a symptom of NPC and the trial also passed the endpoint for Clinical Impression of Change in Severity (CI-CS).

Figure 2: Global Niemann-Pick Disease Drug Type C Treatment Market Value (US$ Mn), by Region, 2022

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Global Niemann-Pick Disease Drug Type C Treatment Market – Competitive Landscape

Major players operating in the global niemann-pick disease drug type C treatment market include Johnson & Johnson, Intrabio, Cyclo Therapeutics, Inc., Kempharm, Inc., Azafaros B.V., StrideBio. Sarepta Therapeutics, Inc.