Latest Published 4 Rare Neurological Disorders Market Reports by DelveInsight: Amyotrophic Lateral Sclerosis, Developmental and Epileptic Encephalopathies, Primary Ciliary Dyskinesia, and Chronic Inflammatory Demyelinating Polyneuropathy

With over 600 recognized rare neurological disorders, many patients face misdiagnosis or lengthy diagnostic journeys before proper care is provided. Only 10% of these conditions have FDA-approved treatments, but breakthroughs are on the horizon, bringing hope to those battling these unique and challenging diseases.

New York, USA, Jan. 09, 2025 (GLOBE NEWSWIRE) — Latest Published 4 Rare Neurological Disorders Market Reports by DelveInsight: Amyotrophic Lateral Sclerosis, Developmental and Epileptic Encephalopathies, Primary Ciliary Dyskinesia, and Chronic Inflammatory Demyelinating Polyneuropathy

With over 600 recognized rare neurological disorders, many patients face misdiagnosis or lengthy diagnostic journeys before proper care is provided. Only 10% of these conditions have FDA-approved treatments, but breakthroughs are on the horizon, bringing hope to those battling these unique and challenging diseases.

Rare neurological disorders encompass a wide range of conditions that affect the nervous system, often leading to severe disability or death. These disorders, though infrequent, can have a significant patient burden, as they are typically complex, progressive, and difficult to diagnose. Many individuals with rare neurological conditions experience debilitating symptoms such as cognitive decline, motor impairments, seizures, and sensory abnormalities. 

The patient burden is often compounded by emotional and psychological challenges, as patients and their families may face uncertainty about prognosis and limited treatment options. Treatment for rare neurological disorders is often challenging due to a lack of specific therapies, with most treatments focused on symptom management rather than disease modification. 

However, advances in genetic therapies, personalized medicine, and clinical trials are slowly improving the outlook for some conditions. Early diagnosis and multidisciplinary care are critical in managing symptoms and improving the quality of life for patients living with these conditions.

DelveInsight has expertise in the rare disease market with an experienced team handling the rare disease domain proficiently. DelveInsight has recently released a series of epidemiology-based market reports on rare neurological disorders including Amyotrophic Lateral Sclerosis, Developmental and Epileptic Encephalopathies, Primary Ciliary Dyskinesia, and Chronic Inflammatory Demyelinating Polyneuropathy. These reports include a comprehensive understanding of current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034  segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan].

Additionally, the reports feature an examination of prominent companies working with their lead candidates in different stages of clinical development. Let’s deep dive into the assessment of these rare neurological disorders markets individually.

Amyotrophic Lateral Sclerosis Market

Amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease, is a progressive neurodegenerative condition that impacts the nerve cells in the brain and spinal cord. This leads to muscle weakness, paralysis, and eventually respiratory failure. Although the exact cause is not fully understood, it is believed that both genetic and environmental factors play a role in its development.

Among the 7MM, the US accounted for the highest ALS prevalent cases in 2023, with around 26,000 cases; these cases are expected to increase during the forecast period. The major sites of onset of ALS are the bulbar, and spinal, along with other uncertain regions. In 2023, the patients with the spinal as a site of onset accounted highest cases, i.e., 15,000, followed by the bulbar site of onset.

At present, there is no cure for ALS, nor is there an effective treatment to halt or slow the progression of the disease. However, treatments such as physical therapy, speech therapy, dietary counseling, heat or whirlpool therapy, and other supportive measures are available within the ALS treatment landscape.

Currently, the medications used in the treatment of ALS include EXSERVAN (Riluzole oral film), RELYVRIO, QALSODY, RADICAVA (edaravone injection), RADICAVA ORS (edaravone oral suspension), NEUDEXTA, TIGLUTEK, RILUTEK, as well as other symptomatic treatments like anti-epileptic drugs, opioids, NSAIDs, diuretics, SSRIs, antidepressants, and more.

As per DelveInsight analysis, the ALS market size in the 7MM was ~USD 1 billion in 2023. This is further expected to rise owing to the improvement in the diagnosis methodologies, raising awareness of the diseases, incremental healthcare spending across the world, and also expected launch of emerging therapies during the forecasted period of 2024–2034.

Amyotrophic Lateral Sclerosis Pipeline Therapies and Key Companies

• MASITINIB: AB SCIENCE
• LATOZINEMAB (AL001/GSK4527223): ALECTOR/GSK
• NUROWN (MSC-NTF CELLS): BRAINSTORM CELL THERAPEUTICS
• ULEFNERSEN (ION363): IONIS PHARMACEUTICALS
• IBUDILAST: MEDICINOVA
• DNL343: DENALI THERAPEUTICS
• ABBV-CLS-7262: ABBVIE/CALICO LIFE SCIENCES
• CNM-AU8: CLENE NANOMEDICINE BIOSCIENCES
• SLS-005 (TREHALOSE): SEELOS THERAPEUTICS
• PRIDOPIDINE: PRILENIA THERAPEUTICS
• RAPA-501: RAPA THERAPEUTICS
• PRIMEC: NEUROSENSE THERAPEUTICS
• TPN-101: TRANSPOSON THERAPEUTICS
• RNS60: REVALESIO CORPORATION
• ANX005: ANNEXON BIOSCIENCES
• DAZUCORILANT (CORT113176): CORCEPT THERAPEUTICS
• AP-101: AL-S PHARMA
• SAR443820/DNL788: SANOFI/DENALI THERAPEUTICS
• AIT-101 (LAM-002A): ORPHAI THERAPEUTICS

Discover more about ALS drugs in development @ Amyotrophic Lateral Sclerosis Clinical Trials

Developmental and Epileptic Encephalopathies Market

Developmental and epileptic encephalopathy (DEE) is a category of severe epilepsies marked by persistent, often drug-resistant seizures and encephalopathy, a condition that involves significant developmental delays or a loss of previously acquired developmental skills.

The total number of diagnosed prevalent cases of DEE in the 7MM was nearly 284,000 cases in 2023 and are projected to increase during the forecasted period. Lennox-Gastaut syndrome (LGS) and Tuberous sclerosis complex (TSC) associated epilepsy are the largest contributors to the overall DEE pool.

In the last ten years, substantial efforts have been made to develop new treatments for DEEs. Recently, several therapies have been approved, including EPIDIOLEX (cannabidiol), which has received orphan drug status for Dravet syndrome, Tuberous Sclerosis Complex (TSC), and Lennox-Gastaut syndrome (LGS). Furthermore, everolimus shows potential as an antiepileptogenic and precision medicine for TSC due to its effects on the mammalian target of the rapamycin pathway.

Currently, ZETALMY is the only FDA-approved treatment for CDK5L deficiency disorder, with market exclusivity. However, this exclusivity is expected to be challenged within the next 2–3 years as FINTEPLA, another promising candidate, is expected to be launched. This shift points to an evolving treatment landscape for rare genetic disorders, offering renewed hope for patients and their caregivers.

At present, most therapies for DEE target specific conditions such as Dravet syndrome, LGS, CDKL5 deficiency disorder, and TSC. However, these represent only a fraction of the DEE spectrum, with over 20 other subtypes largely unexplored, presenting a significant untapped market with considerable potential for companies developing new treatments.

As per DelveInsight analysis, the total market size in the US for DEE was estimated to be nearly USD 1.6 billion in 2023, which is expected to increase due to the launch of emerging therapies and label expansion of current therapies.

Developmental and Epileptic Encephalopathies Pipeline Therapies and Companies 

• Soticlestat (OV935/TAK-935): Takeda/Ovid Therapeutics
• Zorevunersen (STK-001): Stoke Therapeutics
• EPX-100 (Clemizole Hydrochloride): Harmony Biosciences
• Bexicaserin (LP352): Longboard Pharmaceuticals
• Relutrigine (PRAX-562): Praxis Precision Medicines
• Lorcaserin (E2023): Eisai
• Carisbamate (YKP509): SK Life Science
• NBI-921352: Neurocrine Biosciences
• Tricaprilin (CER-0001): Cerecin Neurosciences
• ETX101: Encoded Therapeutics
• Elsunersen (PRAX-222): Praxis Precision Medicines

Dive deeper for rich insights into the Developmental and Epileptic Encephalopathies Clinical Trials

Primary Ciliary Dyskinesia Market

Primary ciliary dyskinesia (PCD) is a genetically diverse recessive condition that impacts motile cilia, resulting in a range of clinical symptoms, including respiratory issues in full-term newborns, persistent oto-sino-pulmonary conditions, male infertility, and organ laterality abnormalities in about 50% of cases.

In 2023, the 7MM accounted for nearly 70,000 total 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 are currently no approved therapies specifically for PCD that restore ciliary function, and treatment is primarily based on those used for cystic fibrosis and non-CF bronchiectasis. Both PCD and cystic fibrosis are rare genetic disorders that impact the respiratory system. For cystic fibrosis, treatments like CFTR modulators (such as Trikafta and Kalydeco) have been developed. 

In PCD, treatment mainly focuses on symptom management and preventing complications. Airway clearance methods, including chest physiotherapy (CPT) and Positive Expiratory Pressure (PEP) devices, are commonly used to loosen and expel mucus from the lungs. Inhaled bronchodilators may also be prescribed to open the airways and help with mucus removal.

Antibiotic therapy plays a crucial role in managing PCD, especially in preventing and treating infections. Some patients may take low-dose antibiotics regularly as a preventive measure against infections. When infections occur, particularly with bacteria like Pseudomonas aeruginosa, targeted antibiotics are essential to effectively manage the condition.

According to the DelveInsight assessment, the market size of primary ciliary dyskinesia in 2023 was approximately USD 101 million in the 7MM. These numbers are further predicted to surge due to the increasing awareness and early diagnosis, advancements in genetic testing, and the rising prevalence of respiratory disorders linked to PCD. Additionally, the development of targeted therapies, such as gene editing and innovative drug treatments, is contributing to market growth. Moreover, improving healthcare infrastructure and patient support systems further drive the demand for effective PCD treatments.

Primary Ciliary Dyskinesia Pipeline Therapies and Companies 

• P-1037: PARION SCIENCES
• RCT1100: RECODE THERAPEUTICS
• ETH 42: ETHRIS
• CIL-05: CILA THERAPEUTICS
• ETH 43: ETHRIS
• ETH 44: ETHRIS
• RESEARCH PROGRAMME: PCD GENE 2: RECODE THERAPEUTICS
• RESEARCH PROGRAMME: PCD GENE 3: RECODE THERAPEUTICS

For a deeper understanding of the primary ciliary dyskinesia market landscape, explore the Primary Ciliary Dyskinesia Market Outlook

Chronic Inflammatory Demyelinating Polyneuropathy Market

Chronic inflammatory demyelinating polyneuropathy is a rare neurological condition marked by inflammation and injury to the myelin sheath, which is the protective layer surrounding nerves. This damage interferes with nerve-muscle communication, resulting in gradual weakness and sensory dysfunction.

According to DelveInsight’s estimates, in 2023, there were approximately 45K diagnosed prevalent cases of chronic inflammatory demyelinating polyneuropathy in the 7MM. Of these, the United States accounted for 66% of the cases.

The treatment approach for CIDP has evolved to incorporate a comprehensive strategy that blends medications with supportive care. Corticosteroids, such as prednisone and dexamethasone, are commonly used as first-line treatments. Although effective in inducing remission, these drugs pose a significant risk of long-term side effects like osteoporosis, weight gain, and heightened vulnerability to infections. 

As a result, combination therapies with steroid-sparing agents are often required, though these may not work equally well for all patients. Several medications are now approved for CIDP treatment, highlighting the emphasis on immune modulation therapies. Notable options include PANZYGA, a human immunoglobulin co-developed by Pfizer and Octapharma, and PRIVIGEN, a room-temperature-stored liquid IVIg from CSL Behring, stabilized with proline.

According to DelveInsight’s analysis, the CIDP market in the 7MM was valued at USD 1.7 million in 2023. Over the forecast period from 2024 to 2034, this market is projected to grow at a CAGR of 7.2%. This expansion across the 7MM will be driven by the launch of innovative therapies, including riliprubart, nipocalimab, and batoclimab.

CIDP Pipeline Therapies and Companies 

• Riliprubart (SAR445088): Sanofi
• Nipocalimab: Janssen Research and Development, LLC
• Batoclimab (HL161): Immunovant Sciences GmbH/HanAll Pharma/Roivant Sciences, Inc.

To access a complete analysis of the CIDP market, visit Chronic Inflammatory Demyelinating Polyneuropathy Market Assessment

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