Primary Ciliary Dyskinesia Market Insight, Epidemiology and Market Forecast - 2034

Key Highlights

• The market size of Primary Ciliary Dyskinesia in 2023 was approximately USD 101 million in the 7MM.
• In 2023, the 7MM accounted for nearly 70,000 total prevalent cases of Primary Ciliary Dyskinesia.
• The market size of Primary Ciliary Dyskinesia in 2023 was approximately USD 36 million in the United States.
• In 2023, the United States accounted for nearly 1,800 diagnosed prevalent cases of Primary Ciliary Dyskinesia.
• The age-specific cases of Primary Ciliary Dyskinesia include 0-5, 6-20, 21-40 and >40 age groups. In 2023, among all age groups, 6-20 accounted for the highest number of prevalent cases in the United States.
• There is no single, standard treatment for Primary Ciliary Dyskinesia. Primary Ciliary Dyskinesia treatment is multifaceted and tailored to the individual's symptoms and disease progression. The main focus remains on managing respiratory symptoms, preventing infections, and preserving lung function.
• In June 2024, the US Food and Drug Administration (FDA) granted Orphan Drug Designation for RCT1100 for the treatment of primary ciliary dyskinesia (PCD).
• In April 2023, Parion Sciences announced receipt of a positive opinion from the Paediatric Committee (PDCO) of the European Medicines Agency (EMA) for the Paediatric Investigation Plan (PIP) for Idrevloride Inhalation Solution, for the treatment of primary ciliary dyskinesia (PCD).

This report delivers an in-depth understanding of Primary Ciliary Dyskinesia, historical and forecasted epidemiology as well as the Primary Ciliary Dyskinesia market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The Primary Ciliary Dyskinesia market report provides current treatment practices, emerging drugs, Primary Ciliary Dyskinesia market share of individual therapies, and current and forecasted Primary Ciliary Dyskinesia market size from 2020 to 2034, segmented by seven major markets. The report also covers current Primary Ciliary Dyskinesia treatment practices/algorithms and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.

Geography Covered

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

Primary Ciliary Dyskinesia Understanding and Treatment Algorithm

Primary Ciliary Dyskinesia Overview

Primary ciliary dyskinesia (PCD) is a genetically heterogeneous recessive disorder affecting motile cilia, leading to various clinical manifestations such as respiratory distress in term neonates, chronic oto-sino-pulmonary disease, male infertility, and organ laterality defects in approximately 50% of cases. Historically, this syndrome was identified by a triad of chronic sinusitis, bronchiectasis, and situs inversus, known as Kartagener syndrome. Later, Afzelius discovered that patients with this syndrome had "immotile" cilia with defective ciliary ultrastructure. It was eventually recognized that most patients exhibited stiff, uncoordinated, and ineffective ciliary beats, leading to the term "primary ciliary dyskinesia" to distinguish this genetic disorder from secondary or acquired ciliary defects.

Primary Ciliary Dyskinesia Diagnosis

Diagnosing primary ciliary dyskinesia is a multi-step process that requires a combination of clinical evaluation, specialized tests, and often genetic analysis. Each test provides different pieces of information that, when combined, can lead to a definitive diagnosis. Due to the variability in clinical presentation and the fact that some tests may not provide conclusive results, healthcare providers often need to use multiple diagnostic methods to accurately diagnose PCD and differentiate it from other similar conditions. The diagnostic process usually begins with a detailed physical examination and medical history assessment. Healthcare providers look for symptoms consistent with PCD, such as chronic respiratory issues (e.g., sinusitis, bronchitis), recurrent ear infections, and situs inversus (a condition where organs are mirrored from their normal positions).

Primary Ciliary Dyskinesia Treatment

There is no single, standard treatment for Primary Ciliary Dyskinesia. Primary Ciliary Dyskinesia treatment is multifaceted and tailored to the individual's symptoms and disease progression. The main focus remains on managing respiratory symptoms, preventing infections, and preserving lung function. As research continues, newer therapies like gene therapy and precision medicine offer hope for more targeted and effective treatments in the future. Early diagnosis and a comprehensive treatment plan are key to improving outcomes and quality of life for PCD patients. Effective airway clearance is crucial for preventing mucus build-up, which can lead to infections and lung damage. Chest Physiotherapy (CPT) includes methods like postural drainage, chest percussion, and vibration. These techniques help to dislodge mucus from the airways, making it easier to cough up. Postural drainage involves positioning the patient in ways that allow gravity to assist in draining mucus from different parts of the lungs. Chest percussion and vibration are often performed with the help of a caregiver or therapist.

Primary Ciliary Dyskinesia Epidemiology

The Primary Ciliary Dyskinesia epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by the Total Prevalent Cases of Primary Ciliary Dyskinesia, Total Diagnosed Prevalent Cases of Primary Ciliary Dyskinesia, Age-specific Cases of Primary Ciliary Dyskinesia, Total Prevalence of Primary Ciliary Dyskinesia by Pathogenic variants in gene, and Prevalent Cases of Primary Ciliary Dyskinesia by clinical manifestations in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2020 to 2034.

• In 2023, the United States accounted for nearly 1,800 diagnosed prevalent cases of Primary Ciliary Dyskinesia.
• The prevalent cases of Primary Ciliary Dyskinesia by age are segmented in three categories i.e., 0-5, 6-20, 21-40 and >40. Among all age groups, the 6-20 category accounted for the highest number of cases in the United States.
• The prevalent cases of Primary Ciliary Dyskinesia by clinical manifestations is segmented into Pulmonary Disease, Situs Abnormality, Infertility, Eye Disorders, and other clinical manifestations. Among all, Pulmonary Disease accounted for the highest number of cases in the United States.

Primary Ciliary Dyskinesia Drug Chapters

The drug chapter segment of the Primary Ciliary Dyskinesia report encloses a detailed analysis of the mid-stage pipeline drugs, and early-stage drugs. The current key players include Parion Sciences and ReCode Therapeutics. The drug chapter also helps understand the Primary Ciliary Dyskinesia clinical trial details, pharmacological action, agreements and collaborations, approval, and patent details, and the latest news and press releases.

Emerging Drugs

P-1037: Parion Sciences

P-1037 (Idrevloride/VX-371) is an inhaled investigational epithelial sodium channel (ENaC) inhibitor being developed by Parion for the treatment of PCD. ENaC inhibitors are designed to block the sodium channels on the airway surface, thus both blocking the absorption of water and stimulating fluid secretion in people with functional cystic fibrosis transmembrane conductance regulator (CFTR). Inhaled Idrevloride Solution is formulated to hydrate the mucus in the lung, thereby improving clearance of mucus by cough, thus improving lung function in people with PCD and other respiratory diseases who accumulate excessively concentrated mucus in their lungs. Idrevloride has been well-tolerated in multiple clinical trials in healthy volunteers and patients with muco-obstructive lung diseases, including primary ciliary dyskinesia. Currently, P-1037 is being investigated in a Phase II trial, for treating Primary ciliary dyskinesia.

Primary Ciliary Dyskinesia Market Outlook

Primary ciliary dyskinesia (PCD) is a genetically heterogeneous recessive disorder affecting motile cilia. It leads to various clinical manifestations, including respiratory distress in term neonates, chronic oto-sino-pulmonary disease, male infertility, and organ laterality defects. There are three main types of PCD: Motionless respiratory cilia, Ineffective Ciliary Movement, and Ciliary Aplasia. To date, pathogenic variants in 45 genes are known to cause PCD. PCD is inherited in an autosomal recessive manner with the exception of FOXJ1-PCD (which is autosomal dominant) and PIH1D3-PCD and OFD1-PCD (which are X-linked). However, 20%-30% of individuals with well-characterized PCD do not have identifiable pathogenic variants in any of the associated genes.

There are currently no PCD-specific approved therapies that restore ciliary function, and treatment is drawn from those used for cystic fibrosis and non-CF bronchiectasis. Like PCD, CF affects the respiratory system and is a rare genetic disorder. Drugs, such as CFTR modulators (e.g., Trikafta and Kalydeco), have been developed for its treatment. Treatment for PCD primarily focuses on managing symptoms and preventing complications. Airway clearance techniques are a key component, often involving chest physiotherapy (CPT) and Positive Expiratory Pressure (PEP) devices to loosen mucus in the lungs, which can then be expelled. Inhaled bronchodilators may also be prescribed to open the airways and facilitate mucus clearance.

Antibiotic therapy is essential in managing PCD, particularly to prevent and treat infections. Some patients may be on regular, low-dose prophylactic antibiotics to reduce the risk of infections. When infections do occur, particularly with bacteria like Pseudomonas aeruginosa, targeted antibiotic therapy is crucial to manage these episodes effectively.

Currently, several emerging therapies, such as P-1037, RCT1100, and others, are being evaluated in mid-stage and early-stage clinical trials.

Primary Ciliary Dyskinesia Drug Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2024-2034, which depends on the competitive landscape, safety, and efficacy data along with order of entry. It is important to understand that the key players evaluating their novel therapies in the pivotal and confirmatory trials should remain vigilant when selecting appropriate comparators to stand the greatest chance of a positive opinion from regulatory bodies, leading to approval, smooth launch, and rapid uptake.

Primary Ciliary Dyskinesia Pipeline Development Activities

The report provides insights into therapeutic candidates in different stages. It also analyzes key players involved in developing targeted therapeutics. Companies like Parion Sciences and ReCode Therapeutics, and others actively engage in late and mid-stage research and development efforts for Primary Ciliary Dyskinesia. The pipeline of Primary Ciliary Dyskinesia possesses a few potential drugs. However, there is a positive outlook for the therapeutics market, with expectations of growth during the forecast period (2024-2034).

Pipeline Development Activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for Primary Ciliary Dyskinesia emerging therapy.

KOL Views

To keep up with current market trends, we take KOLs and SMEs’ opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts contacted for insights on Primary Ciliary Dyskinesia’ evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake, along with challenges related to accessibility, including MDs, radiologists, oncologists, and others.

The analysts connected with 20+ KOLs to gather insights; however, interviews were conducted with 10+ KOLs in the 7MM. The opinion helps understand and validate current and emerging therapy treatment patterns or Primary Ciliary Dyskinesia market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT 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.

Market Access and Reimbursement

The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of currently used 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.

Scope of the Report

• The report covers a segment of key events, an executive summary, and a descriptive overview of Primary Ciliary Dyskinesia, explaining their causes, signs, symptoms, pathogenesis, and currently used therapies.
• Comprehensive insight into the epidemiology segments and forecasts, disease progression, and treatment guidelines has been provided.
• Additionally, an all-inclusive account of the emerging therapies and the elaborative profiles of late-stage and prominent therapies will impact the current treatment landscape.
• A detailed review of the Primary Ciliary Dyskinesia market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies by understanding trends through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help shape and drive Primary Ciliary Dyskinesia.

Primary Ciliary Dyskinesia Report Insights

• Patient Population
• Therapeutic Approaches
• Primary Ciliary Dyskinesia Pipeline Analysis
• Primary Ciliary Dyskinesia Market Size and Trends
• Existing and Future Market Opportunity

Primary Ciliary Dyskinesia Report Key Strengths

• Eleven Years Forecast
• The 7MM Coverage
• Primary Ciliary Dyskinesia Epidemiology Segmentation
• Key Cross Competition
• Drugs Uptake and Key Market Forecast Assumptions

Primary Ciliary Dyskinesia Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT and Conjoint Analysis)

FAQs

• What was the Primary Ciliary Dyskinesia market size, the market size by therapies, market share (%) distribution in 2023, and what would it look like by 2034? What are the contributing factors for this growth?
• What can be the future treatment paradigm for Primary Ciliary Dyskinesia?
• What are the disease risks, burdens, and unmet needs of Primary Ciliary Dyskinesia? What will be the growth opportunities across the 7MM concerning the patient population with Primary Ciliary Dyskinesia?
• What are the current options for the treatment of Primary Ciliary Dyskinesia? What are the current guidelines for treating Primary Ciliary Dyskinesia in the 7MM?
• What are the recent novel therapies, targets, mechanisms of action, and technologies being developed to overcome the limitations of existing therapies?
• What is the patient's share of Primary Ciliary Dyskinesia?

Reasons to Buy

• The report will help develop business strategies by understanding the latest trends and changing treatment dynamics driving Primary Ciliary Dyskinesia.
• Insights on patient burden/disease prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
• Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
• Detailed analysis ranking of class-wise potential current and emerging therapies under the analyst view section to provide visibility around leading classes.
• Highlights of access and reimbursement policies of current therapies, barriers to accessibility of expensive off-label therapies, and patient assistance programs.
• To understand Key Opinion Leaders’ perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.