Diffuse Cutaneous Systemic Sclerosis - Pipeline Insight, 2024

This “Diffuse Cutaneous Systemic Sclerosis- Pipeline Insight, 2024” report provides comprehensive insights about 15+ companies and 15+ pipeline drugs in Diffuse Cutaneous Systemic Sclerosis pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

Diffuse Cutaneous Systemic Sclerosis: Understanding

Diffuse Cutaneous Systemic Sclerosis: Overview

Diffuse cutaneous systemic sclerosis (dcSSc) is a subtype of systemic scleroderma (systemic sclerosis) and is characterized by skin hardening (fibrosis) and problems in many organs of the body. The prevalence is estimated at about 1/25,000 adults. Women are predominantly affected (F/M sex ratio around 4:1). The disease usually manifests at between 40 and 50 years of age. Pediatric onset can occur but is extremely rare. Raynaud's phenomenon is often the first sign of the disease. The other signs usually appear a few months later. Skin hardening first occurs on the fingers and face, but rapidly becomes generalized. Telangiectasias are sometimes present on the thorax, face, lips, tongue, and fingers. Tendon friction rubs are observed. Esophageal dysmotility is common and provokes gastroesophageal reflux and sometimes dysphagia. Gastrointestinal malabsorption and dysmotility may also be present and are associated with weight loss, vomiting, diarrhea or occlusion. Dry mouth and dental involvement can occur. Arthralgias and acroosteolysis are frequent. Muscular involvement can lead to muscular pain and weakness, and cramps. Severe life-threatening renal involvement may occur (about 2% of cases). Pulmonary fibrosis is frequently seen (60% of cases). Pulmonary hypertension may also occur (10-15% of cases). The exact cause of diffuse cutaneous SSc is unknown. The disease originates from an autoimmune reaction which leads to overproduction of collagen. In some cases, the condition is associated with exposure to chemicals (including silica, solvents and hydrocarbons). Diagnosis is based on typical clinical manifestations and on evidence of specific microangiopathy with giant loops on nailfold capillaroscopy. Skin biopsy is usually not needed. Blood tests must be performed, and show typical antitopoisomerase autoantibodies in 30-40% of cases. The extent of the disease should be evaluated by computed tomography (CT), electrocardiogram, echocardiography, radiography of the hands and esophageal and gastric fibroscopy if needed. The prognosis is severe (10-year survival rate of 60-80%) because of the high risk of life-threatening complications: renal crisis, severe digestive involvement, severe lung fibrosis, and, sometimes, severe heart involvement and pulmonary arterial hypertension.

Diffuse Cutaneous Systemic Sclerosis- Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Diffuse Cutaneous Systemic Sclerosis pipeline landscape is provided which includes the disease overview and Diffuse Cutaneous Systemic Sclerosis treatment guidelines. The assessment part of the report embraces, in depth Diffuse Cutaneous Systemic Sclerosis commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Diffuse Cutaneous Systemic Sclerosis collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

• The companies and academics are working to assess challenges and seek opportunities that could influence Diffuse Cutaneous Systemic Sclerosis R&D. The therapies under development are focused on novel approaches to treat/improve Diffuse Cutaneous Systemic Sclerosis.

Diffuse Cutaneous Systemic Sclerosis Emerging Drugs Chapters

This segment of the Diffuse Cutaneous Systemic Sclerosis report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Diffuse Cutaneous Systemic Sclerosis Emerging Drugs

FCR 001: Talaris TherapeuticsFCR001 is an investigational, allogeneic cell therapy developed by Talaris Therapeutics to induce or restore patients’ immune tolerance. FCR001 builds on over 30 years of research by the company’s founder, Dr. Suzanne Ildstad, into the means by which durable immune tolerance can be induced in a patient who receives a transplanted organ or can be restored in patients with certain immune-mediated or blood disorders. FCR001 has received both Orphan Drug Designation and Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration. Currently, the drug is being developed in the Phase I/II stage of clinical trial evaluation for the treatment of Diffuse Cutaneous Systemic Sclerosis.

Belumosudil: Kadmon PharmaceuticalsBelumosudil, is an orally administered, selective inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2). Kadmon’s research has helped define the role of ROCK in the pathogenesis of immune and fibrotic diseases. Kadmon’s research has demonstrated that belumosudil helps to resolve immune dysregulation by down-regulating pro-inflammatory Th17 cells and increasing regulatory T (Treg) cells. Belumosudil, Kadmon’s ROCK2 inhibitor, has demonstrated activity in preclinical sclerodermatous models. Currently, the drug is being developed in the Phase II stage of clinical trial evaluation for the treatment of Diffuse Cutaneous Systemic Sclerosis.

Diffuse Cutaneous Systemic Sclerosis: Therapeutic Assessment

This segment of the report provides insights about the different Diffuse Cutaneous Systemic Sclerosis drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Diffuse Cutaneous Systemic Sclerosis

There are approx. 15+ key companies which are developing the therapies for Diffuse Cutaneous Systemic Sclerosis. The companies which have their Diffuse Cutaneous Systemic Sclerosis drug candidates in the most advanced stage, i.e. phase II include, Kadmon Pharmaceuticals.

Phases

This report covers around 15+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Diffuse Cutaneous Systemic Sclerosis pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Diffuse Cutaneous Systemic Sclerosis: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Diffuse Cutaneous Systemic Sclerosis therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Diffuse Cutaneous Systemic Sclerosis drugs.

Diffuse Cutaneous Systemic Sclerosis Report Insights

• Diffuse Cutaneous Systemic Sclerosis Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Diffuse Cutaneous Systemic Sclerosis Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Diffuse Cutaneous Systemic Sclerosis drugs?
• How many Diffuse Cutaneous Systemic Sclerosis drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Diffuse Cutaneous Systemic Sclerosis?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Diffuse Cutaneous Systemic Sclerosis therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Diffuse Cutaneous Systemic Sclerosis and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Kadmon Pharmaceuticals
• Talaris Therapeutics
• Horizon Therapeutics
• Mitsubishi Tanabe Pharma
• Takeda Oncology
• Seagen

Key Products

• FCR 001
• Belumosudil
• HZN 825
• Tepezza
• MT-7117
• Brentuximab vedotin

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