Rhabdomyosarcoma (RMS) is a type of cancer that usually affects children. Soft tissues or hollow organs such as bladder or uterus are often affected by this disease. There are mainly two types of RMS: embryonal RMS (ERMS) and alveolar RMS (ARMS). Other types include anaplastic RMS and undifferentiated sarcoma. Depending on the type, RMS is likely to affect head and neck area, vagina, urinary bladder, uterus or testes, arms, and legs. There are certain risk factors associated with the disease, such as genetic disorders, including Li-Fraumeni syndrome, neurofibromatosis, Beckwith-Wiedemann syndrome, and Costello syndrome.
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People affected from RMS may face different signs and symptoms, some of which may not be seen until the tumor becomes large. The most common symptoms observed in 50% of the children with this disease include bleeding from nose, throat, vagina, or rectum (depending on the location of tumor); numbness, pain, or tingling if the tumor affects nerve areas; and loosening of eyelids (may indicate a tumor behind the eyes).
The diagnosis of the disease is reliant on features such as tumor size and where it starts. It can be detected by studying complete medical history of the patient, and imaging tests, such as X-ray, magnetic resonance imaging (MRI), computed tomography (CT) scan, bone scan, and others.
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RMS can be treated by surgery, chemotherapy, radiation therapy, and stem-cell transplantation. Moreover, Cosmegen (Ovation Pharmaceuticals Inc.) and VinCRIStine Sulfate (Hospira Inc.) are amongst the drugs approved by the United States Food and Drug Administration (USFDA).
According to the research findings, majority of pipeline drug candidates are being developed for intravenous route of administration. In this route of administration, entire administered dose reaches the systemic circulation immediately, which increases the bioavailability of drug.
Companies that are actively seeking designation grants to accelerate the process of development. For instance, Taiwan Liposome Company Ltd. was granted Pediatric Disease Designation for TLC178 drug by the USFDA in April 2017 for the treatment of RMS. The company also submitted an investigational new drug (IND) application in June 2018 to initiate a phase I/II clinical study of TLC178 to evaluate pediatric RMS patients.
Cosmegen and VinCRIStine Sulfate are approved drugs for the treatment of RMS. Moreover, with the emergence of late- and mid-stage pipeline products in the market, the overall RMS therapeutic market is expected to rise significantly in the upcoming years.
Taiwan Liposome Company Ltd., Ovation Pharmaceuticals Inc., Hospira Inc., F. Hoffmann-La Roche Ltd., Epizyme Inc., and Novartis AG are some of the significant players involved in the development of RMS therapeutics.
RMS Therapeutics Pipeline Analysis
- By Phase
- By Molecule Type
- By Route of Administration
- By Company
The report comprises detailed pipeline analysis of therapeutics being developed for the treatment of RMS. Comprehensive insights on the pipeline products have been provided, with special focus on strategic developments of key players, information on drug licensing, designations, financing, and grants, technological advancements, patents, and upcoming conferences. In addition, the report highlights the winning strategies of companies involved in the RMS therapeutics development. Detailed regulatory approval procedures in the U.S., Europe, and Japan are also provided in this report. Furthermore, the report contains competitive analysis and extensive information on monotherapies, combination therapies, targets and mechanisms of action, and drug origin with respect to RMS.
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