LAS VEGAS, March 4, 2021 /PRNewswire/ -- Rare cancers account for approximately 22% of all the diagnosed cancer cases. The US National Cancer Institute defines rare cancers as those with an incidence of less than 15 cases per 100?000 per year.
However, these cases are not confined to one or the other particular geography, but are disproportionately spread across the world. Many rare human cancers are not curable, and have limited number of treatment options. Rare cancers are more difficult to diagnose and are often misdiagnosed. Rarity of the cancers further complicates the running of clinical trials. Lack of clinical expertise, research, and funding are other barriers in the Rare cancer market.
Therefore, the Rare cancer market calls for continued efforts to speed up the process of drug development. Strategies to enroll patients in cohorts, meta-analyzing of studies, patient evaluation, acknowledging the burden rare cancer poses, innovative approaches to develop therapies, and a go-ahead attitude from pharma players as well as international support organizations shall help
DelveInsight has recently launched several Rare disease market reports with a special focus on Rare cancers. Some of the key highlights from a few of the published rare cancers reports are as follows:
Soft Tissue Sarcoma Market
Soft Tissue Sarcoma (STS) is a rare and heterogeneous group of tumors that account for less than one percent of all adult malignancies. It originates from connective tissue such as muscles, nerves, tendons, blood vessels, fatty and fibrous tissues. The majority of the soft tissue sarcomas cases occur in the lower extremities, making this the commonest site. Other locations are the trunk, the retroperitoneum, and the head and neck. They can be classified based on cell-type, nature of malignancy, and disease's clinical course. As per the data by the WHO, there are 50+ identified histologic subtypes of STSs.
According to DelveInsight, the total incident population of STS (including GIST Cases in the US) in the 7MM (the US, EU5 (the UK, Germany, Italy, Spain, and France) and Japan) was 41,787 in 2020. Among all the seven major markets, the United States accounts for the highest number of cases of STS. Males accounted for most of the STS cases as compared to females.
Soft tissue sarcoma have various subtypes. It was observed that in the United States and European countries, liposarcoma and leiomyosarcoma were the most incident subtypes of STS, which contributed to the majority of the cases. Whereas in Japan, subtypes, fibroblastic sarcomas and liposarcoma were more common.
The Soft tissue sarcoma treatment landscape is dependent on surgery, chemotherapy, radiation therapy, targeted therapy, and antiangiogenesis drugs, or a combination of these therapies. The treatment option for patients with advanced STS has historically been limited. Cytotoxic chemotherapy is the mainstay of the STS treatment market. Active drugs include doxorubicin, ifosfamide, gemcitabine, taxanes, and several others; these may be either administered as single agents or in combination regimens. The most commonly used regimen is the combination of doxorubicin and ifosfamide. The US FDA-approved drugs comprise Votrient (pazopanib), Yondelis (trabectedin), Lartruvo (olaratumab), Vitrakvi (larotrectinib), Rozlytrek (Entrectinib), and others.
However, the histology-driven approach remains the mainstay for the treatment of advanced unresectable or metastatic STSs. There is an urgency for new therapies with improved efficacy and fewer side effects. Several companies are developing combination approaches, targeting the multiple molecular pathways and mechanisms with special attention to antagonistic antibodies, TK inhibitors, and inhibitors of downstream molecules of the PI3K, MAPK, and mTOR pathways.
Several pharmaceutical companies like Blueprint Medicines Corporation, Advenchen Laboratories/Jiangsu Chia-Tai Tianqing Pharmaceutical, Karyopharm Therapeutics, Deciphera Pharmaceuticals, Philogen, GlaxoSmithKline, Epizyme, Eli Lilly, Pfizer, Aadi Bioscience, Sierra Oncology, Nanobiotix, Taiho Oncology, Arog Pharmaceuticals, and Merck are developing novel therapies in the Soft tissue sarcoma market. DelveInisght expects an increase in the Soft tissue sarcoma market size during the study period, 2017-2030 at a CAGR of 6.47% in the 7MM.
Among all the emerging therapies, Fibromun is going to get launched for first line STS in the forecasted period (2021-2030). While, among all the second or higher line of therapies, it is anticipated that the highest market is expected to be captured by AL3818 (anlotinib) by 2030. Few second-line emerging therapies includes, Vigil in combination with irinotecan and temozolomide, AL3818, GSK 3377794, Tazemetostat, Abemaciclib, Pembrolizumab, Crizotinib, and ABI-009.
Key Soft Tissue Sarcoma Pipeline Therapies
- Selinexor (KPT-330): Karyopharm Therapeutics
- Ripretinib (DCC-2618): Deciphera Pharmaceuticals
- GSK3377794: GlaxoSmithKline
- BA3021: BioAtla
- Tazemetostat: Epizyme
Get in touch for comprehensive market trends @ Soft Tissue Sarcoma Market
Synovial sarcoma (SyS) Market
Synovial sarcoma (SyS) is a rare subtype of soft tissue sarcoma. It originates from different types of soft tissue, such as muscle or ligaments. It accounts for 5-10% of soft-tissue tumors. The symptoms of Synovial sarcoma depend on the location of the tumor.
As per DelveInsight epidemiological analysis, Synovial sarcoma incidence in the 7MM was estimated to be 1,610 in 2020, which is expected to further rise in the coming decade.
The primary goal of Synovial sarcoma treatment is to remove the entire tumor with clear margins. To completely remove the residual cancerous cells, radiotherapy may also be used, either before or after surgery. In advanced or metastatic disease, chemotherapy (typically Doxorubicin and/or Ifosfamide) is recommended.
DelveInsight's analysts estimate that the market is expected to show positive growth, primarily attributed to increased prevalence and expected launch of novel therapies. Some of the key pharmaceutical companies taking charge in the Synovial sarcoma market are Advenchen Laboratories, Adaptimmune Therapeutics, and others during the forecast period (2018-2030). Furthermore, besides these, an increase in incidence, the discovery of multiple biomarkers, and vast research in the domain is expected to inflate the market size at a modest hike with a CAGR of 19.14% in the Synovial sarcoma market share in the 7mm for the forecast period 2021-30.
Key Synovial Sarcoma Pipeline Therapies
- AL 3818: Advenchen Laboratories
- ADP-A2M4: Adaptimmune Therapeutics
- GSK3377794: GlaxoSmithKline
Get in touch for a holistic view of the Synovial Sarcoma Market
Cholangiocarcinoma (CCA) Market
Cholangiocarcinoma (CCA) is a heterogeneous malignant neoplasm with epithelial cell origin of the biliary duct and histologic and biochemical features of cholangiocyte differentiation. It may arise from any portion of the bile duct i.e., from terminal ductules (canals of Hering) to the ampulla of Vater, as well as at the peribiliary glands (intramural and extramural) and sometimes from the gallbladder.
Cholangiocarcinoma is a rare disorder, and as per DelveInsight's epidemiological analysis, total CCA incidence was recorded to be approximately 73,442 in 2020 in the 7MM. Further, the analysts believe that Cholangiocarcinoma incidence is expected to increase further during the study period 2018-30.
At present, the CCA treatment market is dominated by surgery with adjuvant chemotherapy and chemoradiation therapy. For advanced and metastatic Cholangiocarcinoma cases, systemic chemotherapy is the only available option. Surgical treatments are the only potentially curative therapeutic options for all the subtypes of CCA. Improvements in molecular understanding have led to the development of targeted therapies. Last year, Incyte corporation received approval for its pemigatinib as the first targeted therapy for adults with previously treated, unresectable locally advanced, or metastatic cholangiocarcinoma.
There is no established second-line systemic therapy that can be given following the progression after first-line treatment although chemotherapeutic agents either in monotherapy or in combination are used. Besides lack of standard treatment, the majority of CCA patients are diagnosed with late-stage disease, and nearly more than one-fourth of patients considered resectable are found to be unresectable during explorative laparotomy. The CCA market also witnesses higher cases of recurrence of tumor. Besides, classification of Cholangiocarcinoma makes it difficult to quantify the true incidence of this highly aggressive malignancy.
To combat the unmet needs, the treatment modalities are focusing more towards a personalized approach, targeting FGFR gene fusions and somatic mutations in IDH 1/2 in iCCA, PRKACA or PRKACB gene fusions in pCCA, and ELF3 mutations in dCCA/ampullary carcinoma. Several pharmaceutical companies and biotech players are investigating novel therapeutic agents to fuel the Cholangiocarcinoma market size growth in the coming decade.
DelveInsight estimates optimal growth of the Cholangiocarcinoma market size in the coming years for the forecast period 2021-30 at a CAGR of 12.71% in the 7MM owing to a better understanding of the molecular pathway of cancer through whole-genome analysis, identified multiple actionable mutations, development of targeted agents with promising results in early clinical trials, increasing incidence, better funding and a newly-shifted focus towards rare disease domain.
Key Cholangiocarcinoma Pipeline Therapies
- Ivosidenib: Agios Pharmaceuticals
- Durvalumab: AstraZeneca
- M7824: Merck/EMD Serono
- Infigratinib: QED Therapeutics
- Nucana: Acelarin
- Telotristat ethyl: Lexicon Pharmaceutical
Know more about in touch for a holistic view of the Cholangiocarcinoma Market
Glioblastoma Multiforme (GBM) Market
Glioblastoma Multiforme (GBM) is a rare tumor that occurs mostly in adults. It is a grade IV glioma, and according to the WHO 2007 classification, it is the most common and lethal type of primary malignancy of the central nervous system.
GBMs can be classified into primary and secondary GBMs. While Primary GBM occurs de novo without evidence of a less malignant precursor, Secondary GBM develops from initially low-grade diffuse astrocytoma (WHO grade II diffuse astrocytoma) or anaplastic astrocytoma (Grade III). In 2020, DelveInsight estimated an occurrence of approximately 29,440 incident cases of GBM and suspects a modest increase in the same in the coming decade.
The mainstay of treatment for GBM patients is surgery, followed by radiation and chemotherapy. Surgery is performed to achieve "maximal safe resection," or removing as much of the tumor as possible without causing lasting neurological damage. However, it is difficult to get rid of all of the cancerous cells therefore, residual cancer cells are targeted with additional treatments, such as chemotherapy or radiation therapy.
Chemotherapy with the drug temozolomide is the current standard of treatment for GBM. Additionally, Bevacizumab is typically used as a second-line treatment for recurrent glioblastomas. It is seen that Lomustine may help improve the efficacy of bevacizumab when both the medications are administered at the same time.
However, despite the availability of multidisciplinary treatments, the median overall survival for patients with GBM is only 14.6 months. This may be attributed to its high degree of intra tumor heterogeneity due to which radical tumor resection is not often curative. Besides these, poor prognosis, limited treatment options, resistance to drugs, inability of therapies to cross BBB (blood-brain-barrier), and a high mortality rate are some other unmet needs.
Nevertheless, advancements in medical science have paved the way for better understanding and thus, development of better therapies. The expected launch of potential therapies in the forecast period 2021-30 is expected to pace up the growth of the GBM market size. The GBM market is expected to experience an influx of the significant positive shift owing to the positive outcomes of the several products during the developmental stage by key players such as Bayer, Diffusion Pharmaceuticals, VBL Therapeutics, AstraZeneca, DNAtrix, DelMar Pharmaceuticals, Oncoceutics, KaryoPharm, VBI Vaccines, Kazia Therapeutics, Aivita Biomedical, Medicenna Therapeutics, Immunomic Therapeutics, and Inovio Pharmaceuticals.
DelveInsight anticipates a positive growth of the Glioblastoma Multiforme market size at a CAGR of 12.9% during the forecast period 2021-30, within the 7MM.
Key Glioblastoma Multiforme Pipeline Therapies
- Ofranergene obadenovec: VBL Therapeutics
- Trans Sodium Crocetinate: Diffusion Pharmaceuticals
- Regorafenib: Bayer
- Durvalumab: Astrazeneca
- DNX-2401: DNAtrix
- ONC201: Oncoceutics
- Selinexor (KPT-330): Karyopharm Therapeutics
- Paxalisib (GDC-0084): Kazia Therapeutics
- AV-GBM-1: Aivita Biomedical
- VBI-1901: VBI Vaccines
- MDNA55: Medicenna Therapeutics
- VAL-083 (Dianhydrogalactitol): DelMar Pharmaceuticals
- pp65-shLAMP DC with GM-CSF (ITI-1000): Immunomic Therapeutics
- Ad-RTS-hIL-12 + Veledimex: Ziopharm
Out of the above-mentioned emerging therapies, several key players are targeting newly diagnosed GBM patient pools, thereby targeting to enter the first line treatment market. These key players include Diffusion Pharmaceuticals, Astrazeneca, DelMar Pharmaceuticals, Kazia Therapeutics, Aivita Biomedical, Immunomic Therapeutics, Inovio Pharmaceuticals and Bayer. This clearly represents high competition among the pipeline therapies targeting first line treatment patterns.
Furthermore, certain key players are also targeting specific mutations among the GBM patient pool such as unmethylated MGMT GBM, H3 K27M-mutant Glioma and others. The approval of such mutation targeting therapies may be able to enhance the treatment regimen of patients suffering from GBM in the forecast period.
For more insights, visit Glioblastoma Multiforme Market
Multiple Myeloma Market
Multiple myeloma is a cancer of plasma cells. It is a heterogenous hematological malignancy. It is a cancer that originates from bone marrow resulting in bone fractures (osteolysis) due to an increase in osteoclastic and decreased osteoblastic activity.
However, rare, it is the second most commonly diagnosed blood cancer, after non-Hodgkin lymphoma, in the United States. It shows a higher predisposition of males than females. As per DelveInsight, the total Multiple Myeloma incident cases in 2020 were estimated to be 91,520 in the 8MM (7MM + China).
The present treatment landscape for Multiple myeloma comprises chemotherapies in conjunction with proteasome inhibitors, monoclonal antibodies, corticosteroids, and bone marrow transplantation. Chemo drugs used to treat multiple myeloma include Melphalan, Vincristine (Oncovin), Cyclophosphamide (Cytoxan), Etoposide (VP-16), Doxorubicin (Adriamycin), among others. Corticosteroids, such as dexamethasone and prednisone, are an important part of the treatment of multiple myeloma. Until now, the conventional treatment for multiple myeloma includes therapy with immunomodulatory drugs such as Revlimid and Thalomid, proteasome inhibitors (which block the action of protein-degrading structures in cells) such as Velcade and Kyprolis, anti-CD38 antibodies such as Darzalex, and autologous stem cell transplant. Patients who fail to respond to these approaches need better treatments. However, heterogeneity of the tumor, lack of proper knowledge of etiology, side effects of the present therapies and cost-burden are the major constraints in the MM market. Therefore, many companies came up with their own CAR-T products for multiple myeloma including BMS, Janssen and many more.
No doubt, over the time, several advancements have been made. Expected entry of novel therapeutic agents coupled with intensified R&D has helped in gaining a better picture of the tumor and thus facilitating the development of novel therapies. DelveInsight estimates a surge in the Multiple Myeloma market size at a CAGR of 2.1% during the forecast period 2021-30 in the 8MM. Launch of BCMA-targeting therapies along with increasing MM incidence is expected to garner much revenue and attention of pharmaceutical stakeholders to explore the domain.
2021 is going to mark itself as the first year with the expected approvals of the two Chimerican antigen receptor therapies (CAR-Ts), idecabtagene vicleucel (Ide-cel; Bristol Myers Squibb) and ciltacabtagene autoleucel (cilta-cel; Janssen) for relapsed/refractory multiple myeloma. Therefore, these CAR-Ts in the multiple relapsed/refractory patient who at this point does not have great options may represent the best ways of getting a response or the highest chances of getting a response in that patient. The infusion of CAR-T cells is based on the fact that there is no typical planned maintenance therapy required. This means that the patients can enjoy a treatment-free, disease-free period, where they can restore their health to a point that even if they do relapse after about a year or so, and they can restart the treatment.
One can look back and observe the evolution in the field of cell therapies. Until a decade ago, where no CAR-Ts were approved, the US FDA now estimated that in 2019 that it had received more than 200 investigational new drug applications per year for cell and gene therapies, and by 2025, it expects to have accelerated to 10 to 20 cell and gene therapy approvals per year.
Key pharmaceutical companies, such as GlaxoSmithKline, Bristol-Myers Squibb, AbbVie, Roche, Janssen Research & Development, Merck Sharp & Dohme Corp., Pfizer, Takeda, Amgen, AstraZeneca, Abbvie, and others are dynamically aspiring to bring novel, standard and effective treatment regimens in the Multiple Myeloma market.
Key Multiple Myeloma Pipeline Therapies
- Idecabtage ne vicleucel: Bristol-Myers Squibb/Bluebird bio
- Venetoclax: AbbVie and Genentech
- JNJ-68284528: Janssen and Legend Biotech
- Melflufen: Oncopeptides AB
- Keytruda: Merck Sharp & Dohme
- Cetrelimab: Janssen
- REGN5458: Regeneron Pharmaceuticals
- Iberdomide: Bristol-Myers Squibb/Celgene
- NY-ESO-1 C259 T Cells: Adaptimmune Therapeutics /GlaxoSmithKline /Merck
- Braftovi: Pfizer (Array Biopharma)
- Felzartamab (MOR202): MorphoSys AG/I-Mab Biopharma
- JCARH125: Juno Therapeutics (BMS)
- Chidamide (Epidaza): Chipscreen Biosciences
Got queries? Reach out to us @ Multiple Myeloma Market
DelveInsight offers the most comprehensive healthcare data, market insights and customized solutions gained with the help of integrated tools and primary and secondary resources to help its clients traverse strategically engaged in Rare disease market.
DelveInsight is offering flat 20% discount on all of its Market insights reports focusing on Rare diseases. Use Code 'Rare20' to avail.
Reach out to us @ Rare disease market insights
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