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Orphan Indications - Biotech, Pharma and Life Science Channel

01:34 EST 24th January 2019 | BioPortfolio

Examples of Ophan Drug Indications include:

Anaemia, sickle cell
Cystic fibrosis (CF)
Duchenne muscular dystrophy
Glioma
Graft vs host disease (GvHD)
Hepatoma, liver cancer
Hodgkin Lymphoma
Leukaemia, acute lymphocytic (ALL)
Leukaemia, acute myeloid (AML)
Leukaemia, chronic lymphocytic (CLL)
Leukaemia, chronic myeloid (CML)
Multiple myeloma
Myelodysplastic syndrome
Non-Hodgkin lymphoma (NHL)
Ovarian cancer
Pancreatic cancer
Pulmonary fibrosis, idiopathic
Pulmonary hypertension
Renal cell carcinoma (RCC)
Soft tissue sarcoma

News Articles [195 Associated News Articles listed on BioPortfolio]

TaiwanJ Pharmaceuticals Announces Positive Results from Phase 2 Orphan Drug Designation Study of JKB-122 on Autoimmune Hepatitis (AIH)

HSINCHU, Taiwan, Jan. 22, 2019 /PRNewswire/ -- TaiwanJ Pharmaceuticals, a specialty pharmaceutical company dedicated to the development and commercialization of small molecules for unmet medical nee...

Albireo Granted Orphan Drug Designation by FDA For Lead Product Candidate A4250 for Treatment of Biliary Atresia

Expands A4250 development potential in rare cholestatic liver diseases Biliary atresia is among the most common rare pediatric liver diseases and is the most common pediatric liver disease for whic...

Millendo Therapeutics Reports Inducement Grants Under NASDAQ Listing Rule 5635(c)(4)

Millendo Therapeutics, Inc. (“Millendo” or the “Company”) (NASDAQ: MLND), a clinical-stage biopharmaceutical company developing novel treatments for orphan endocrin...

Bridge Biotherapeutics Announces FDA Orphan Drug Designation for BBT-877 in Idiopathic Pulmonary Fibrosis (IPF)

The U.S. FDA grants an orphan drug designation to BBT-877 in IPF The first-in-human clinical study is set to begin next month SEONGNAM, South Korea, Jan. 16, 2019 /PRNewswire/ -- Bridge Bioth...

FDA Grants Cerecor’s Three Substrate Replacement Therapies Orphan Drug Designation

BALTIMORE, Jan. 16, 2019 (GLOBE NEWSWIRE) -- Cerecor Inc. (NASDAQ: CERC), a biopharmaceutical company focused on becoming a leader in development and commercialization of therapies for rare and orp...

OncoImmune Announces Completion of Phase II Trial

OncoImmune, Inc. today announced that is has completed its Phase IIa trial of CD24Fc for the Prevention of Acute GVHD Following Myeloablative Allogeneic HSCT. The ...

ChemomAb Doses First Patient in Phase 1b Clinical trial of CM-101 in Patients With Non-Alcoholic Fatty Liver Disease

ChemomAb, a clinical-stage biopharmaceutical company focused on the development of novel therapies for fibrotic-inflammatory diseases, announced dosing of the first patient in a P...

EMA Grants Orphan Drug Designation to Rafael Pharmaceuticals’ devimistat (CPI-613®) for Treatment of Patients with Relapsed or Refractory Acute Myeloid Leukemia (AML)

Newark, NJ, Jan. 10, 2019 (GLOBE NEWSWIRE) -- Rafael Pharmaceuticals, Inc., a leader in the growing field of cancer metabolism-based therapeutics, today announced that the European Medicines Agency...

EMA Grants Orphan Drug Designation to Rafael Pharmaceuticals’ devimistat (CPI-613®) for Treatment of Metastatic Pancreatic Cancer

Newark, NJ, Jan. 10, 2019 (GLOBE NEWSWIRE) -- Rafael Pharmaceuticals, Inc., a leader in the growing field of cancer metabolism-based therapeutics, today announced that the European Medicines Agency...

CANbridge Pharmaceuticals and WuXi Biologics Expand Rare Disease Therapeutics Partnership

CANbridge Pharmaceuticals Inc., a biopharmaceutical company developing innovative drug candidates to treat underserved medical conditions in China and other markets, and WuXi Biol...

Companies [74 Associated Companies listed on BioPortfolio]

CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. is a China-based biopharmaceutical company accelerating development and commercialization of specialty healthcare products for orphan diseases and t...

ChemomAb

ChemomAb is a clinical stage biopharmaceutical company, specializes in the development of proprietary monoclonal antibodies, directed towards novel targets, for the treatment of f...

Escient Pharmaceuticals, Inc.

Escient Pharmaceuticals is a privately held biotechnology company that is advancing first-in-class G Protein-Coupled Receptor (GPCR)-targeted drugs to address serious, unserved me...

Rebiotix Inc.

Rebiotix Inc, which is part of the Ferring Pharmaceuticals Group, is a late-stage clinical microbiome company focused on harnessing the power of the human microbiome to revolution...

Lumos Pharma, Inc.

Lumos Pharma, Inc. is a clinical stage biopharmaceutical company focused on development and commercialization of therapeutics for rare and neglected diseases. Lumos Pharma was fou...

SCOUT

SCOUT is a leading healthcare marketing firm with special expertise in rare diseases, orphan drugs and specialty pharmaceuticals. With offices in Atlanta, Chicago, San Diego and N...

Advicenne

Advicenne (Euronext: ADVIC) is a pharmaceutical company developing pediatric-friendly therapeutics for the treatment of orphan renal and neurological diseases. The Company’s ...

AcuityBio Inc. and Cook Biotech Inc.

AcuityBio has developed a platform technology for localized, sustained, safe drug-delivery to be used in the prevention of post-surgical lung cancer recurrence. ABC103 combines ap...

ConSynance Therapeutics, Inc.

ConSynance Therapeutics is a clinical-stage drug development company based in Rensselaer, New York. The company’s pipeline includes innovative treatments for nonalcoholic ste...

Pharnext SAS

Pharnext is an advanced clinical stage biopharmaceutical company founded by renowned scientists and entrepreneurs including Professor Daniel Cohen, a pioneer in modern genomics. P...

Clinical Trials [46 Associated Clinical Trials listed on BioPortfolio]

A Study of Pazopanib and Durvalumab for Metastatic Soft Tissue Sarcoma

Pazopanib is an angiogenesis inhibitor targeting VEGFR-1, -2, and -3; PDGFR-α and -β; and the receptor c-Kit, and is indicated for the treatment of subjects with advanced renal cell carc...

Multicenter Study of Seliciclib (R-roscovitine) for Cushing Disease

This phase 2 multicenter, open-label clinical trial will evaluate safety and efficacy of 4 weeks of oral seliciclib in patients with newly diagnosed, persistent, or recurrent Cushing disea...

Development of In Vitro Functional Assays From Primary Cells of Patients With Monogenic Diseases

The objective of this project is to build a collection of biological samples consisting of fibroblasts and primary keratinocytes from patients with orphan monogenic diseases and to use the...

RORC Genetic Polymorphism of Rheumatoid Arthritis

Three candidate single nucleotide polymorphisms in the RORC2 gene, rs9826 A/G, rs3790515 C/T and rs3828057 C/T were examined together with estimation of nuclear hormone retinoic acid recep...

Cabozantinib in Advanced Adrenocortical Carcinoma

Adrenocortical carcinoma is an orphan malignant disease that has a dismal prognosis in advanced stages. Mitotane is the only approved treatment but is limited by severe toxicity. Efficacy ...

Evaluation of NICHE Programme Activities in Kenya

This study evaluates the impact of additional cash and nutritional counselling on nutrition outcomes in pregnant women and children up to the age of two years in CT-OVC (cash transfer- orp...

Adoptive Transfer of Tumor Infiltrating Lymphocytes for Metastatic Uveal Melanoma

This is a Phase 2 study in which the efficacy of a non-myeloablative lymphodepleting preparative regimen followed by infusion of autologous TIL and high-dose aldesleukin in patients with m...

Safety and Feasibility of Normothermic Machine Perfusion to Rescue Orphan Livers

Orphan livers are organs that have been declined for clinical use by all centers due to their marginality. The current standard of care of liver preservation before transplant is cold stor...

Orphan Europe Carbaglu® Surveillance Protocol

The purpose of this study is to conduct post-marketing surveillance of carglumic acid (Carbaglu) to obtain long-term clinical safety information. Carglumic acid was approved by the United ...

A Study of Protein Metabolism, Microbiome and Investigational Probiotic Use in Patients With ALS

ALS, also known as "Lou Gehrig's" disease, is a neurodegenerative disease which is fatal. Treatment for ALS is limited and currently consists of primary symptom relief or support. In addit...

PubMed Articles [109 Associated PubMed Articles listed on BioPortfolio]

New phylogenomic analysis of the enigmatic phylum Telonemia further resolves the eukaryote tree of life.

The resolution of the broad-scale tree of eukaryotes is constantly improving, but the evolutionary origin of several major groups remains unknown. Resolving the phylogenetic position of these 'orphan'...

Discovery of Potent, Selective, and Orally Bioavailable Estrogen-related Receptor Gamma Inverse Agonists to Restore the Sodium Iodide Symporter Function in Anaplastic Thyroid Cancer.

Inverse agonist of estrogen-related receptor gamma (ERRγ), an orphan nuclear receptor (NR) encoded by Esrrg, enhances NIS-mediated radioiodine uptake in anaplastic thyroid cancer (ATC) cells, thereby...

Oxysterols and nuclear receptors.

Oxysterols are derivatives of cholesterol and an important regulator of cholesterol metabolism, in part due to their role as ligands for nuclear receptors, such as the liver x receptors. Oxysterols ar...

Orphan drugs: major development challenges at the clinical stage.

Biotechnology has provided powerful tools to assist in research and development (R&D) for rare diseases. However, orphan drug development presents several major challenges and obstacles, such as low d...

Effects of early adversity on the brain: Larger-volume anterior cingulate cortex in AIDS orphans.

Multiple studies have revealed that adolescent AIDS orphans have more psychosocial problems than healthy adolescents. However, little is known about whether and how the brain structures of adolescent ...

Comparative genomics of Rhizophagus irregularis, R. cerebriforme, R. diaphanus and Gigaspora rosea highlights specific genetic features in Glomeromycotina.

• Glomeromycotina is a lineage of early diverging Fungi establishing arbuscular mycorrhizal (AM) symbiosis with land plants. Despite their major ecological role, genetic bases of their obligate mutu...

Symbiotic organs shaped by distinct modes of genome evolution in cephalopods.

Microbes have been critical drivers of evolutionary innovation in animals. To understand the processes that influence the origin of specialized symbiotic organs, we report the sequencing and analysis ...

A Nervous System-Specific Model of Creatine Transporter Deficiency Recapitulates the Cognitive Endophenotype of the Disease: a Longitudinal Study.

Mutations in creatine (Cr) transporter (CrT) gene lead to cerebral creatine deficiency syndrome-1 (CTD), an orphan neurodevelopmental disorder presenting with brain Cr deficiency, intellectual disabil...

Knockdown of Orphan Transporter SLC22A18 Impairs Lipid Metabolism and Increases Invasiveness of HepG2 Cells.

The aim of this work is to investigate the roles of solute carrier family 22 member 18 (SLC22A18) in lipid metabolism and in establishing the tumor phenotype of HepG2 cells.

Significant differences on submission lag following regulation reform for registration of novel therapeutic drugs in Taiwan.

Drug lag, which delays patients' access to medicinal products, is typically associated with pharmaceutical regulations. To shorten drug lag, health authorities may establish new policies to liberalize...

Medical and Biotech [MESH] Definitions

An orphan nuclear receptor that is implicated in regulation of steroidogenic pathways. It is unlike most orphan nuclear receptors in that it appears to lack an essential DNA-binding domain and instead acts as a transcriptional co-repressor. Mutations in the gene Dax-1 cause congenital adrenal hypoplasia.

A family of cell surface receptors that were originally identified by their structural homology to neurotropic TYROSINE KINASES and referred to as orphan receptors because the associated ligand and signaling pathways were unknown. Evidence for the functionality of these proteins has been established by experiments showing that disruption of the orphan receptor genes results in developmental defects.

A DNA-binding orphan nuclear receptor that has specificity for directly repeated (DR) AGGTCA sequences. It binds DNA as either as a homodimer or as a heterodimer with the closely-related orphan nuclear receptor NUCLEAR RECEPTOR SUBFAMILY 2, GROUP C, MEMBER 2. The protein was originally identified as a PROSTATE-specific protein and is involved in the regulation of variety of cellular processes, including CELL DIFFERENTIATION; CELL PROLIFERATION; and APOPTOSIS.

An orphan nuclear receptor that has specificity for hormone response elements found in the promoters of target genes. It binds DNA either as a homodimer or as heterodimer with the closely-related orphan nuclear receptor NUCLEAR RECEPTOR SUBFAMILY 2, GROUP C, MEMBER 1. The protein was originally identified as a TESTES-specific protein and is involved in the regulation of variety of cellular processes, including CELL DIFFERENTIATION; CELL PROLIFERATION; and APOPTOSIS.

Production of drugs or biologicals which are unlikely to be manufactured by private industry unless special incentives are provided by others.

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