Drug Designations to Boost the Rhabdomyosarcoma (RMS) Therapeutics Development

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.

Retinitis Pigmentosa (RP) Therapeutics Pipeline to Witness Remarkable Growth due to Increased Number of Pipeline Candidates by Drug Developers

Retinitis pigmentosa (RP) is a group of hereditary disorders that lead to the breakdown and loss of cells in the retina. Mutation in genes encoding the proteins required by retinal cells for their proper functioning, results in occurrence of RP. The disease results in partial vision loss during the earlier stage of the disease, which may lead to complete blindness with the progression of the disease. Some of patient-reported side effects include night visual impairment, loss of the visual field, and trouble in differentiating colors.

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The aim of diagnosing RP is to determine the type of mutation that has occurred in genes and can be detected by performing tests, including electroretinogram (ERG), visual field testing, and genetic testing.

The available treatment options include retinal implant, increased intake of vitamin A palmitate, and acetazolamide medication. Furthermore, Luxturna (Spark Therapeutics Inc.) is the only approved therapy by the U.S. Food and Drug Administration (USFDA) for the treatment of RP.

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According to the research findings, majority of the pipeline drug candidates are being developed for subretinal administration. It has been observed that this route of administration has high bioavailability and ensures direct effect on targeted cells.

Positive Clinical Results are Expected to Drive the RP Therapeutics Pipeline

Companies that are involved in developing therapeutics for RP have shown positive clinical results in various phases of drug development. For instance, in December 2018, Applied Genetic Technologies Corporation announced positive top-line results from the phase I trial of XLRS gene therapy for the treatment of patients with RP. The results of the study exhibited that 2 of 13 adult patients treated at high dose showed some improvements in retinal structure as measured by optical coherence tomography (OCT), an imaging technology that provides a side view of retina’s layers.

Companies are actively seeking designation grants to accelerate the process of development. For instance, HORA – PDE6B of Horama, was granted Orphan Drug Designation by the USFDA for the treatment of RP in July 2017.

Luxturna is the only approved therapy for the treatment of RP. Therefore, with the emergence of late- and mid-stage pipeline products in the market, the overall RP therapeutic market is expected to rise significantly in the upcoming years.

Acucela Inc., HORAMA S.A., GenSight Biologics S.A., Novartis AG, Allergan Inc., Spark Therapeutics Inc., Sanofi-aventis Groupe, Ocugen Inc., Applied Genetic Technologies Corporation, Eyevensys, MeiraGTx Holdings plc, and Biogen Inc. are some of the key players involved in the development of drugs indicated for the treatment of RP.

RP 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 RP. 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 RP 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 RP.

Atypical Hemolytic Uremic Syndrome (aHUS) Therapeutics – Pipeline Analysis 2018

Atypical hemolytic uremic syndrome (aHUS), also known as non-diarrhea – associated hemolytic uremic syndrome, is a rare disease characterized by hemolytic anemia, thrombocytopenia, acute kidney failure and uremia.

The signs and symptoms of aHUS include formation of tiny blood clots in various small blood vessels of the body. These clots reduce or prevent proper blood flow to various organs of the body, especially the kidneys. aHUS is a complex disorder; and certain genetic, environmental and immunologic factors plays significant role in its development.

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Company lile ChemoCentryx Inc. is in the process of developing avacopan, an orally-administered drug candidate, that inhibits the complement C5a receptor for the treatment of aHUS. Omeros Corporation, Greenovation Biotech GmbH, and Laboratoris Sanifit S.L. are some other companies developing therapeutic agents for aHUS.

Aurora Kinase Inhibitor Therapeutics Pipeline Analysis

Aurora kinase are proteins that function as mitotic regulators and are aberrantly expressed in cancerous cells. Aurora kinase inhibitors target these aberrantly expressed regulators, provides genetic stability and prevents tumorigenesis.

Several studies have demonstrated overexpression and amplification of aurora kinase in various haematological and solid cancers. Aurora kinase inhibitors when combined with other anti-cancer agents gives more positive results in variety of cancers. Better evaluation of aurora kinase inhibitors is required which raises the need of biomarkers, effective and novel therapeutic agents. Also, aurora kinase inhibitors selectivity targets cancer cells rather than healthy tissues, providing potential opportunities for more effective drug development with reduced adverse effects.

AstraZeneca PLC is in the process of developing AZD-2811 as a small molecule which acts as an aurora B inhibitor for the treatment of solid tumors. Some of the other companies having pipeline of aurora kinase inhibitors include AbbVie Inc., Merck & Co., Inc., and CASI Pharmaceuticals Inc.

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The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with detailed analysis of pipeline and clinical trials. Pipeline analysis of drugs by phases includes product description and development activities including information about clinical results, designations, collaborations, licencing, grants, technology and others.