Monday, 5 July 2021

Gaucher disease – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030

 


Indication name: Gaucher disease

Gaucher disease – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030 (Markets covered: US, Germany, France, Spain, United Kingdom, Italy, Japan & China).

Gaucher disease is a rare, inherited metabolic disorder in which deficiency of the enzyme glucocerebrosidase results in the accumulation of harmful quantities of certain fats (lipids), specifically the glycolipid glucocerebroside, throughout the body especially within the bone marrow, spleen and liver. Gaucher disease is caused by changes (mutations) in the GBA gene.

Gaucher disease divided into 3 different types based on the presence or absence of early-onset brain involvement, including:

Gaucher disease type 1 is also known as non-neuronopathic, experience easy bruising due to low levels of blood clotting cells known as platelets (thrombocytopenia), chronic fatigue due to low levels of circulating red blood cells (anemia), and an abnormally enlarged liver and/or spleen (hepatosplenomegaly).

Gaucher disease type 2, also known as acute neuronopathic Gaucher disease, occurs in newborns and infants and is characterized by neurological complications due to the abnormal accumulation of glucocerebroside in the brain.

Gaucher disease type 3, also known as chronic neuronopathic Gaucher disease, occurs during the first decade of life. Associated neurological complications include mental deterioration; an inability to coordinate voluntary movements (ataxia); and brief, shock-like muscle spasms of the arms, legs or entire body (myoclonic seizures).

Epidemiology-

Gaucher disease can affect anyone, occurring in up to 1 in 40,000 live births in the general population. Literature suggest it is more common among Jews of Ashkenazi (Eastern European) descent, occurring in approximately 1 in 450 within this population. As many as 1 in 10 may carry the mutated gene responsible for Gaucher disease.

Competitive landscape of Gaucher disease includes country specific approved as well as pipeline therapies. Any asset/ product specific designation or review and Accelerated Approval are being tracked and supplemented with analyst commentary.

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

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

Read more: Gaucher disease – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030

S. No     Asset     Company           Stage

1            PR001   Prevail Therapeutics      Phase 2

2            GA-GCB (velaglucerase alfa)        Shire     Phase 2

3            AVR-RD-02         AvroBio Phase 2

4            venglustat (GZ402671)   Genzyme, a Sanofi Company      Phase 3

5            Arimoclomol     Orphazyme        Phase 2

6            Abcertin             ISU Abxis Co., Ltd.           Phase 1

7            Miglustat            Actelion              Phase 3

8            afegostat tartrate           Amicus Therapeutics      Phase 2

9            PRX-112              Protalix Phase 2

Sarcoidosis – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030

 



Indication name: Sarcoidosis

Sarcoidosis – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030 (Markets covered: US, Germany, France, Spain, United Kingdom, Italy, Japan & China).

Sarcoidosis is an inflammatory disease characterized by the formation of granulomas—tiny clumps of inflammatory cells—in one or more organs of the body. Radiologic Staging of Sarcoidosis:

Stage 0- Normal

Stage I-Bilateral hilar lymphadenopathy

Stage II- Bilateral hilar lymphadenopathy and pulmonary infiltrates

Stage III- Pulmonary infiltrates without bilateral hilar lymphadenopathy

Stage IV- Pulmonary fibrosis

Epidemiology-

Thelansis estimated that the prevalence of sarcoidosis in the US ranges between 150,000 and 200,000, with an estimated 1.2 million individuals with sarcoidosis worldwide.

Competitive landscape of Sarcoidosis includes country specific approved as well as pipeline therapies. Any asset/ product specific designation or review and Accelerated Approval are being tracked and supplemented with analyst commentary.

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

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

Read more: Sarcoidosis – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030

S. No        Asset        Company        Stage

1        ATYR1923        aTyr Pharma, Inc.        Phase 2

2        CMK389        Novartis Pharmaceuticals        Phase 2

3        ARA 290        Araim Pharmaceuticals, Inc.        Phase 2

4        Selexipag        Actelion        Phase 2

5        Ambrisentan        Gilead Sciences        Phase 3

6        iNO        Bellerophon        Phase 2

7        Tofacitinib        Pfizer        Phase 1

8        18F-FSPG        GE Healthcare        Phase 2

9        Inhaled Treprostinil        United Therapeutics        Phase 2

10        Ursodeoxycholic Acid        Exalenz Bioscience LTD.        Phase 2

11        Bardoxolone methyl        Reata Pharmaceuticals, Inc.        Phase 2

Medulloblastoma – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030

 



Indication name: Medulloblastoma

Medulloblastoma – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030 (Markets covered: US, Germany, France, Spain, United Kingdom, Italy, Japan & China).

Medulloblastoma is a cancerous tumor—also called cerebellar primitive neuroectodermal tumor (PNET)- that starts in the region of the brain at the base of the skull, called the posterior fossa. Tumors tend to spread to other parts of the brain and to the spinal cord. There’s a small percent of childhood medulloblastomas that are related to gene changes that can be passed down through families. There’s no known cause for adults diagnosed with medulloblastomas.

The 4 molecular subgroups currently recognised in the WHO classification have been named WNT, SHH, Group 3, and Group 4.

Medulloblastoma - wingless (WNT) subgroup tumours are the least common medulloblastoma subgroup and have by far the best prognosis. They are usually seen in children and, to a lesser degree, adults. They typically arise from the region surrounding the foramen of Luschka and middle cerebellar peduncle.

WNT tumours account for only 11% of all medulloblastomas, and have, unlike group 3 and 4 tumours, no particular predilection for males, with ~1:1 male to female ratio

Medulloblastoma - sonic hedgehog (SHH) subgroup tumours are malignant tumours of the central nervous system. They are the second most common medulloblastoma subgroup (after group 4) and are approximately as common as group 3. They are found most commonly in adults and infants, but infrequently in children. Although they can arise from the vermis of the cerebellum, as is typical for group 3 and 4, they are most frequently located laterally within the cerebellar hemispheres.

SHH subgroup tumours account for approximately 27% of all medulloblastomas, and, unlike group 3 and 4, have no particular predilection for males, with a ~1:1 male to female ratio

Medulloblastoma - group 3 tumours are malignant tumours of the central nervous system, and one of the most common paediatric tumours. They typically arise from the vermis of the cerebellum and present in childhood and infancy. They have the worst prognosis of all medulloblastoma subgroups.

Group 3 tumours account for approximately 27% of all medulloblastomas, and have a predilection for males, with a 2:1 male to female ratio.

Medulloblastoma - group 4 tumours are malignant tumours of the central nervous system, and one of the most common paediatric tumours. They are the most common medulloblastoma subgroup (followed by group 3, SHH, and WNT), and typically arise from the vermis of the cerebellum.

Group 4 tumours account for approximately 34-40% of all medulloblastomas, and have a predilection for males, with a 2:1 male to female ratio

Epidemiology- Overall medulloblastomas account for 12-25 % of all paediatric CNS tumours, and 30-40% of paediatric posterior fossa tumours 1,7. They are also seen in adults but only account for 0.4-1.0% of adult brain tumours 1. Since there are many more adults than children, 14-30% of all medulloblastomas are found in adults.

Competitive landscape of Medulloblastoma includes country specific approved as well as pipeline therapies. Any asset/ product specific designation or review and Accelerated Approval are being tracked and supplemented with analyst commentary.

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

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

Read more: Medulloblastoma – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030

S. No        Asset        Company        Stage

1        177Lu-DTPA-omburtamab        Y-mAbs Therapeutics        Phase 2

2        LDE225        Novartis Pharmaceuticals        Phase 2

3        AloCELYVIR        Apices Soluciones S.L.        Phase 2

4        TB-403        Oncurious NV        Phase 1

5        Nifurtimox        Bayer        Phase 2

6        TPI 287        Cortice Biosciences, Inc.        Phase 1

7        Cyclophosphamide        Genentech, Inc.        Phase 2

8        Prexasertib        Eli Lilly and Company        Phase 1

9        Pomalidomide        Celgene        Phase 2

10        NKTR-214        Nektar Therapeutics        Phase 2

11        VAL-413        Valent Technologies, Inc.        Phase 1

12        ZSP1602        Guangdong Zhongsheng Pharmaceutical Co., Ltd.        Phase 1"

Phenylketonuria (PKU) – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030

 



Indication name: Phenylketonuria (PKU)

"Phenylketonuria (PKU) – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030 (Markets covered: US, Germany, France, Spain, United Kingdom, Italy, Japan & China)

Phenylketonuria (PKU) is an autosomal recessive inborn error of phenylalanine (Phe) metabolism resulting from deficiency of phenylalanine hydroxylase (PAH). Mutations in the PAH gene cause phenylketonuria. The PAH gene provides instructions for making an enzyme called phenylalanine hydroxylase

Epidemiology- In the United States, PKU occurs in 1 in 10,000 to 15,000 newborns.

Competitive landscape of Phenylketonuria (PKU) includes country specific approved as well as pipeline therapies. Any asset/ product specific designation or review and Accelerated Approval are being tracked and supplemented with analyst commentary.

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

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

S. No     Asset     Company             Stage

1            SYNB1618           Synlogic              Phase 2

2            BMN165             BioMarin Pharmaceutical            Phase 3

3            RTX-134              Rubius Therapeutics       Phase 1

4            CDX 6114            Nestlé   Phase 1

5            HMI-102             Homology Medicines, Inc            Phase 2

6            CNSA-001           PTC Therapeutics            Phase 2"

Read more: Phenylketonuria (PKU) – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030

Pro-opiomelanocortin (POMC) – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030

 



Indication name: Pro-opiomelanocortin (POMC)

"Pro-opiomelanocortin (POMC) – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030 (Markets covered: US, Germany, France, Spain, United Kingdom, Italy, Japan & China).

Pro-opiomelanocortin (POMC) deficiency is a form of monogenic obesity resulting in severe early-onset obesity, adrenal insufficiency, red hair and pale skin. POMC deficiency is caused by mutations in the POMC gene, which provides instructions for making the proopiomelanocortin protein. The condition is inherited in an autosomal recessive pattern

Epidemiology- POMC deficiency is a rare condition; approximately 50 cases have been reported in the medical literature.

Competitive landscape of Pro-opiomelanocortin (POMC) includes country specific approved as well as pipeline therapies. Any asset/ product specific designation or review and Accelerated Approval are being tracked and supplemented with analyst commentary.

KOLs insights of Pro-opiomelanocortin (POMC) across 8 MM market from center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm and Unmet needs.

Pro-opiomelanocortin (POMC) Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard) which 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.

Read more: Pro-opiomelanocortin (POMC) – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030

S. No        Asset        Company        Stage

1        Setmelanotide        Rhythm Pharmaceuticals, Inc.        Phase 3

2        Adrenocorticotropic Hormone        Mallinckrodt        Phase 1

3        LCI699        Novartis Pharmaceuticals        Phase 2"

Friedreich ataxia (FA) – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030

 



Indication name: Friedreich ataxia (FA)

"Friedreich ataxia (FA) – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030

Friedreich ataxia (FA) is a rare inherited disease that causes progressive nervous system damage and movement problems.  It usually begins in childhood and leads to impaired muscle coordination (ataxia) that worsens over time. The gene responsible for FRDA has been designated FXN. The FXN gene codes for frataxin, a protein that is required for proper functioning of mitochondria, which are the energy producing parts of our cells. In most affected individuals, the FXN gene contains a very specific type of error called an expanded GAA trinucleotide repeat.

Epidemiology-

Based on epidemiology study of Thelansis 18,660 patients are likely to be diagnosed patient in 6 Major markets (USA and EU5), out of which 16,800 patients will able to meet the treatment for the year 2021 for the same 6 major markets.

Geography Covered

North America- United States and Canada

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

Other countries- Japan & China

Study Period: 2020-2030

Competitive landscape of Friedreich ataxia (FA) includes country specific approved as well as pipeline therapies. Any asset/ product specific designation or review and Accelerated Approval are being tracked and supplemented with analyst commentary.

KOLs insights of Friedreich ataxia (FA) across 8 MM market from center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm and Unmet needs.

Friedreich ataxia (FA) Market Forecasting: Patient Based Forecast Model (MS. Excel Based Automated Dashboard) which 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.

Read more: Friedreich ataxia (FA) – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030

S. No        Asset        Company            Stage

1        Omaveloxolone Capsules        Reata Pharmaceuticals, Inc.        Phase 2

2        Vatiquinone        PTC Therapeutics        Phase 3

3        deferiprone        ApoPharma        Phase 2

4        cohort        Retrotope, Inc.        Phase 2

5        MIB-626        Metro International Biotech, LLC        Phase 2

6        CTI-1601        Larimar Therapeutics, Inc.        Phase 1

7        MIN-102        Minoryx Therapeutics, S.L.        Phase 2

8        TAK-831        Takeda        Phase 2

9        interferon γ-1b        Horizon Pharma Ireland, Ltd., Dublin Ireland        Phase 3

10        Idebenone        Santhera Pharmaceuticals        Phase 3

11        VP 20629        Shire        Phase 1

12        Lu AA24493        H. Lundbeck A/S        Phase 2

13        alpha-tocopherolquinone (A0001)        Penwest Pharmaceuticals Co.        Phase 2

14        EGb 761 120 mg        Ipsen        Phase 2

15        (+)-Epicatechin        Cardero Therapeutics, Inc.        Phase 2

16        EPI-743        Edison Pharmaceuticals Inc        Phase 2"

PD-1 and PD-L1 Evolution, Navigating through Lines of Therapy; Market Outlook, Competitive Landscape, Drug Utilization, and Market Forecast by cancer Types – 2021 to 2030

 


Indication name: PD-1 and PD-L1

"Market Outlook - Evolutions of PD-1 and PD-L1, Navigating through Lines of Therapy; Competitive Landscape, Drug Utilization, and Market Forecast by cancer Types; Non-Small Cell Lung Cancer (NSCLC), Squamous cell carcinoma of the head and neck (SCCHN), Renal Cell Carcinoma (RCC), Bladder Cancer (BC), Melanoma, Hepatocellular carcinoma (HCC), Small Cell Lung Cancer (SCLC), Gastric Cancer, Breast cancer, Colorectal cancer, and Other Cancer types – 2021 to 2030

Background: PD-1 is a receptor expressed on mature T cells in peripheral tissues and the TIME, as well as on non-T cell immune cells such as B cells, DCs, and NK cells. PD-L1 is found on the surface of tumor cells, DCs and macrophages. PD-1 on T cells interact with its ligand PD-L1 (B7-H1/CD274) and PD-L2 (B7-DC/CD273), it leads to an immunosuppressive response such as decreased production of inflammatory cytokines and cell survival proteins in the T cells. Also, PD-L1 is able to interact with CD80 to provide a secondary mechanism of T cell suppression. In this instance, CD80 acts as the receptor and provides additional inhibitory signals. Clinically, this interaction has become one of the most successful targets in the current I/O therapies.

Key segments: T-Cell Targeted Immunotherapy, Cell Therapy, Cancer Vaccines, Oncolytic Virus, CD3-targeted antibody, and Other Immunomodulators.

PD-1/ PD-L1 achieved approximately 22% to 45% of overall response rates (ORR) in melanoma, 25% to 62% in NSCLC, ~19% in SCLC, 43% to 71% in lymphoma, 21% to 50% in urothelial carcinoma, ~20% in hepatocellular carcinoma, 32% to 50% in high microsatellite instability (MSI-H) tumors, and over 50% in Merkel cell or renal cell carcinomas. Solid commercial success has also been attained with the clinical achievements, as Keytruda alone has generated over $11BN and Opdivo generated another $8.5BN in sales worldwide in 2020.

Potential patient pool and Utilization of Immunotherapy

Cancer segment: Non-Small Cell Lu      nm,,mkmmng Cancer (NSCLC), Squamous cell carcinoma of the head and neck (SCCHN), Bladder Cancer (BC), Hepatocellular carcinoma (HCC), Gastric Cancer, Colorectal cancer, and Other cancer types, Melanoma, PMBCL, urothelial carcinoma, microsatellite instability-high cancer, and endometrial carcinoma

Patient Segmentation: Incidence, Prevalence (Survival adjusted), Stage wise segmentation (Localized, Regional, Distant, and Unknown), Treated population-based o Lines of Therapy (LoT1, LoT2, LoT3).

Endpoints: I/ O Treated pool LoT 1L+, Responder, Non- responder, Overall Response Rate

Investment risk analysis:

Novel biologic development is inherent with a risk of non-standardized nature. Clinical trials always carry a risk of failure in demonstrate significantly enough levels of efficacy or safety in current or future clinical trials. Risk analysis; Clinical development risk, Regulatory and approval risk, Commercialization risks, Competitive risk, financial risk (NPV, eNPV and IRR)

Competitive Landscape:

Competitive landscape includes country specific approved (Indications and Key Efficacy (ORR, CR, mDOR (months), mPFS (months), mOS (months)) as well as pipeline therapies; PD-1 & PD-L1 Agents in the Current Global Drug Development Pipeline. 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 being tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and key product positioning includes 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 in determining the potential of the key assets as well as their probable filing and launch date.

Market Access and Payers perspective:

From the payer’s perspective majorly focused on wide range of treatment options off course cost savings is the major concern. In coming year, the PD-1 and PD-L1 could change dramatically treatment landscape dramatically based on strong clinical outcomes data which have great evidence on managing the clinical outcomes

Geography Covered

North America- United States and Canada

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

Other countries- Japan & China

Study Period: 2020-2030

Current Clinical Practice and Treatment Algorithm

Country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs are being covered under this section of the study. Retrospective analysis and bench-marking of clinical study outcomes are being presented in terms of Pr-treatment & post-treatment clinical and demographic patient characteristics. Essentially the evolution of the current competitive landscape and its impact on the future treatment paradigm is being well covered under this section.

KOL Insights:

KOLs across 8 MM market from 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

Competitive Landscape: 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 being tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and key product positioning includes 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 in determining the potential of the key assets as well as 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 important unmet needs identified through our research, include decreased cost burden on patients, improved administration convenience, and improved patient compliance."

Read more: PD-1 and PD-L1 Evolution, Navigating through Lines of Therapy; Market Outlook, Competitive Landscape, Drug Utilization, and Market Forecast by cancer Types – 2021 to 2030

Post-traumatic stress disorder (PTSD) – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030

  Indication name: Post-traumatic stress disorder (PTSD) Post-traumatic stress disorder (PTSD) – Market outlook, Epidemiology, Market For...