Tag Archives: Orphan drug clinical pipeline

EU grants orphan drug status to Strekin’s STR001 for Sudden Sensorineural Hearing Loss

European Medicine Agency (EMA) has granted orohan drug designation to Sterkin AG, a clinical stage biopharmaceutical company’s development candidate STR001 for the treatment of patients with Sudden Sensorineural Hearing Loss.

FDA and EMA grants orphan drug designation to those products like drugs and therapies that treat rare diseases. The administration defines rare diseases as those affecting fewer than 200,000 people in United States. Orphan drug designation provides certain financial benefits such as incentives to the developing company. These benefits include market exclusivity for the marketing application, if regulatory approval is received for the designated indication, potential tax credits for certain activities and waiver of certain administrative fees.

“Global Orphan Drug Clinical Pipeline Insight” reports by kuick research gives comprehensive insight on various clinical and non-clinical aspects associated with ongoing clinical trials of 808 orphan designated drugs across the globe. The in-depth clinical insight presented in the report helps the reader to analyze and identify the various stakeholders involved in the clinical development and commercialization of orphan designated drugs in the global market. Currently there are more than 300 orphan designated drugs commercially available in the global market and around 800 drugs in clinical development phase.

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com   

NantCell receives FDA orphan drug status for Ganitumab Monoclonal Antibody Therapy in Ewing Sarcoma

NantCell, Inc., part of the NantWorks ecosystem of companies, has been granted orphan drug designation from US Food and Drug Administration (FDA) for the company’s Ganitumab, Insulin Growth Factor-1R (IGF-1R) Monoclonal Antibody Therapy for the treatment of Ewing Sarcoma. Ganitumab is a fully human monoclonal antibody directed against IGF-1R that is being investigated in a Phase III human clinical trial.

Mechanism of action of the monoclonal antibody is described as IGF-1R plays a role in the development and progression of many cancers, possibly due to its anti-apoptotic properties, which allow cancerous cells to resist the cytotoxic properties of chemotherapeutic drugs or radiation therapy. Ganitumab inhibits cancer cell proliferation through disruption of the P13K/Akt and MAPK pathways. Signaling through IGF-1R plays an important role in the regulation of cell growth and survival, and has been shown to be a critical promoter of anchorage independent growth, a well-recognized mechanism for malignancy.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.

“Global Orphan Drug Clinical Pipeline Insight” reports by kuick research gives comprehensive insight on various clinical and non-clinical aspects associated with ongoing clinical trials of 808 orphan designated drugs across the globe. The in-depth clinical insight presented in the report helps the reader to analyze and identify the various stakeholders involved in the clinical development and commercialization of orphan designated drugs in the global market. Currently there are more than 300 orphan designated drugs commercially available in the global market and around 800 drugs in clinical development phase.

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com   

Tasquinimod from Active Biotech receives orphan drug status for multiple myeloma

US Food and Drug Administrations has granted orphan drug designation to Tasquinimod, investigational novel agent developed by Active Biotech, a leading biopharmaceutical company for the effective treatment for patients with multiple myeloma. Tasquinimod is an immunomodulatory, anti-metastatic and anti-angiogenic compound that affects the tumor’s ability to grow and spread. Multiple myeloma is an incurable form of blood cancer where the plasma cells in the bone marrow grow uncontrollably while other blood forming cells such as white and red blood cells and blood platelets are suppressed.

FDA grants orphan drug designation to those products like drugs and therapies that treat rare diseases. The administration defines rare diseases as those affecting fewer than 200,000 people in United States. Orphan drug designation provides certain financial benefits such as incentives to the developing company. These benefits include market exclusivity for the marketing application, if regulatory approval is received for the designated indication, potential tax credits for certain activities and waiver of certain administrative fees.

“Global Orphan Drug Clinical Pipeline Insight” reports by kuick research gives comprehensive insight on various clinical and non-clinical aspects associated with ongoing clinical trials of 808 orphan designated drugs across the globe. The in-depth clinical insight presented in the report helps the reader to analyze and identify the various stakeholders involved in the clinical development and commercialization of orphan designated drugs in the global market. Currently there are more than 300 orphan designated drugs commercially available in the global market and around 800 drugs in clinical development phase.

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com   

 

Minoryx therapeutics announces orphan drug designation for MIN-102

Minoryx Theapeutics, a leading drug development company focused on developing orphan drugs for rare diseases recently, announced that US Food and Drug Administration (FDA) has granted orphan drug designation to its leading product candidate, MIN-102 for the treatment of patients with life threatening CNS disease with high unmet medical need. MIN-102 contains superior profile with selective PPAR gamma agonist for diseases linked with central nervous system. MIN-102 targets X-linked adrenoleukodystrophy (X-ALD), a rare and chronically debilitating life threatening neurodegenerative disease. Additionally, the drug has been previously awarded with orphan drug designation by European Medicine Agency (EMA).

FDA and EMA grants orphan drug designation to those products like drugs and therapies that treat rare diseases. The administration defines rare diseases as those affecting fewer than 200,000 people in United States. Orphan drug designation provides certain financial benefits such as incentives to the developing company. These benefits include market exclusivity for the marketing application, if regulatory approval is received for the designated indication, potential tax credits for certain activities and waiver of certain administrative fees.

“Global Orphan Drug Clinical Pipeline Insight” reports by kuick research gives comprehensive insight on various clinical and non-clinical aspects associated with ongoing clinical trials of 808 orphan designated drugs across the globe. The in-depth clinical insight presented in the report helps the reader to analyze and identify the various stakeholders involved in the clinical development and commercialization of orphan designated drugs in the global market. Currently there are more than 300 orphan designated drugs commercially available in the global market and around 800 drugs in clinical development phase.

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com   

Protalex receives orphan drug designation for ITP

Protalex, a biopharmaceutical company dedicated to develop orphan drug or therapies for the effective treatment of patients with rare diseases, has recently announced that its novel drug candidate, PRTX 100 has been granted orphan drug designation by the US Food and Drug Administration (FDA) for the treatment of persistent, chronic immune thrombocytopenia (ITP).

PRTX-100 is currently being evaluated in its Phase ½ trial in patients who have previously been treated with a thrombopoietin receptor agonist and at least one additional ITP therapy. Each patient in the study will be given 4 weekly intravenous doses of PRTX-100 and is monitored for up to 48 weeks thereafter. PRTX-100 is a purified form of SpA, an immunomodulatory protein known to modify aspects of the human immune system. It is designed to bind to human B-lymphocytes and macrophages and to modulate immune processes.

FDA grants orphan drug designation to those products like drugs and therapies that treat rare diseases. The administration defines rare diseases as those affecting fewer than 200,000 people in United States. Orphan drug designation provides certain financial benefits such as incentives to the developing company. These benefits include market exclusivity for the marketing application, if regulatory approval is received for the designated indication, potential tax credits for certain activities and waiver of certain administrative fees.

“Global Orphan Drug Clinical Pipeline Insight” reports by kuick research gives comprehensive insight on various clinical and non-clinical aspects associated with ongoing clinical trials of 808 orphan designated drugs across the globe. The in-depth clinical insight presented in the report helps the reader to analyze and identify the various stakeholders involved in the clinical development and commercialization of orphan designated drugs in the global market. Currently there are more than 300 orphan designated drugs commercially available in the global market and around 800 drugs in clinical development phase.

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com   

 

FDA grants orphan drug status to ZW25 for Gastric Cancer Treatment

US Food and Drug Administration has awarded orphan drug designation to Zymework’s leading product candidate, ZW25 for the treatment of gastric cancer and cancer of the gastroesophageal junction (GEJ). Presently, the dug is being examined under Phase 1 clinical trial in patients with advanced HER2 expressing cancers.

ZW25 is a bio assisted antibody formulated to tie up with two non-overlapping epitopes of HER2 resulting in dual HER2 signal blockade, followed by its removal from cell surface and enhanced effector function.

FDA grants orphan drug designation to those products like drugs and therapies that treat rare diseases. The administration defines rare diseases as those affecting fewer than 200,000 people in United States. Orphan drug designation provides certain financial benefits such as incentives to the developing company. These benefits include market exclusivity for the marketing application, if regulatory approval is received for the designated indication, potential tax credits for certain activities and waiver of certain administrative fees.

“Global Orphan Drug Clinical Pipeline Insight” reports by kuick research gives comprehensive insight on various clinical and non-clinical aspects associated with ongoing clinical trials of 808 orphan designated drugs across the globe. The in-depth clinical insight presented in the report helps the reader to analyze and identify the various stakeholders involved in the clinical development and commercialization of orphan designated drugs in the global market. Currently there are more than 300 orphan designated drugs commercially available in the global market and around 800 drugs in clinical development phase.

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com   

Immune design receives orphan drug status for G100 Intratumoral therapy Candidate

Immune Design, a biopharmaceutical company focused on developing drugs and therapies for oncology has declared receipt of orphan drug designation by US Food and Drug Administration (FDA) for its investigational therapy G100 for the treatment of follicular non-Hodgkin’s lymphoma. The product is currently in its discovery state containing potent synthetic small molecule toll-like receptor-4 (TLR-4) agonist, Glucopyranosyl Lipid-A (GLA).

FDA grants orphan drug designation to those products like drugs and therapies that treat rare diseases. The administration defines rare diseases as those affecting fewer than 200,000 people in United States. Orphan drug designation provides certain financial benefits such as incentives to the developing company. These benefits include market exclusivity for the marketing application, if regulatory approval is received for the designated indication, potential tax credits for certain activities and waiver of certain administrative fees.

“Global Orphan Drug Clinical Pipeline Insight” reports by kuick research gives comprehensive insight on various clinical and non-clinical aspects associated with ongoing clinical trials of 808 orphan designated drugs across the globe. The in-depth clinical insight presented in the report helps the reader to analyze and identify the various stakeholders involved in the clinical development and commercialization of orphan designated drugs in the global market. Currently there are more than 300 orphan designated drugs commercially available in the global market and around 800 drugs in clinical development phase.

 

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com   

 

AstraZeneca’s NMOSD Drug receives orphan drug designation

European Medicine Agency has Announced orphan drug designation for inebilizumab (earlier known as MEDI-551) developed by AstraZeneca PLC (AZN) for the treatment of neuromyelitis optica spectrum disorder (NMOSD).  The drug has already received orphan drug status from US Food and Drug Administration (FDA). NMOSD, also called Devic’s disease, is a rare, autoimmune disorder of the central nervous system (CNS) that affects the functioning of optic nerves and spinal cord. It leads to blindness, bladder and bowel problems, loss of sensation, severe muscle weakness, paralysis, and other related symptoms. Autoimmune diseases are the ones in which the body’s immune system attacks healthy cells.

EMA orphan designation is intended to encourage development of medicines for the diagnosis, treatment, or prevention of life-threatening or chronically debilitating conditions that affect no more than 5 in 10,000 people in the EU. Medicines that receive orphan designation are eligible for a number of incentives, including assistance with development of the medicine; reduced fees for marketing-authorization applications; and extended market exclusivity once the medicine is authorized. Orphan designation is conferred following a positive opinion by the EMA’s Committee for Orphan Medicinal Products (COMP).

“Global Orphan Drug Clinical Pipeline Insight” reports by kuick research gives comprehensive insight on various clinical and non-clinical aspects associated with ongoing clinical trials of 808 orphan designated drugs across the globe. The in-depth clinical insight presented in the report helps the reader to analyze and identify the various stakeholders involved in the clinical development and commercialization of orphan designated drugs in the global market. Currently there are more than 300 orphan designated drugs commercially available in the global market and around 800 drugs in clinical development phase.

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com   

 

DLBCL receives FDA orphan drug designation for eFT508

US Food and Drug Administration has announced orphan drug designation to eFT508, a highly selective inhibitor of MNK1 and MNK2 for the treatment of diffuse large B-cell lymphoma. The drug is developed by eFFECTOR Therapeutics. The company has initiated to evaluate the safety, pharmacokinetics, pharmacodynamics and antitumor activity of the agent in patients with B-cell hematologic malignancies. The company also is conducting a phase 1/phase 2 dose-escalation trial of eFT508 in patients with solid tumors.

The FDA grants orphan drug designation to drugs and biologics that are intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. Orphan drug status is intended to facilitate drug development for rare diseases and may provide several benefits to drug developers, including tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.

“Global Orphan Drug Clinical Pipeline Insight” reports by kuick research gives comprehensive insight on various clinical and non-clinical aspects associated with ongoing clinical trials of 808 orphan designated drugs across the globe. The in-depth clinical insight presented in the report helps the reader to analyze and identify the various stakeholders involved in the clinical development and commercialization of orphan designated drugs in the global market. Currently there are more than 300 orphan designated drugs commercially available in the global market and around 800 drugs in clinical development phase.

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com   

 

FDA grants orphan drug designation to SB-913 Genome Editing Treatment for MPS II

Sangamo Therapeutics, Inc., a leading pharmaceutical company dedicated to develop gene therapies and therapeutic genome editing has recived orphan drug designation from US Food and Drug Administration for company’s genome editing product candidate, SB-913,  for the treatment of Mucopolysaccharidosis Type II (MPS II), a rare lysosomal storage disorder. MPS II is caused by mutations in the gene encoding iduronate 2-sulfatase (IDS) enzyme. Using Sangamo’s zinc finger nuclease (ZFN) genome editing technology, SB-913 is designed as a single treatment strategy intended to provide stable, continuous production of the IDS enzyme for the lifetime of the patient.

The FDA grants orphan drug designation to drugs and biologics that are intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. Orphan drug status is intended to facilitate drug development for rare diseases and may provide several benefits to drug developers, including tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.

“Global Orphan Drug Clinical Pipeline Insight” reports by kuick research gives comprehensive insight on various clinical and non-clinical aspects associated with ongoing clinical trials of 808 orphan designated drugs across the globe. The in-depth clinical insight presented in the report helps the reader to analyze and identify the various stakeholders involved in the clinical development and commercialization of orphan designated drugs in the global market. Currently there are more than 300 orphan designated drugs commercially available in the global market and around 800 drugs in clinical development phase.

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com