Annamycin from Moleculin receives orphan drug status for the Treatment of Acute Myeloid Leukemia

Moleculin Biotech, Inc, a preclinical pharmaceutical company focused on the development of anti-cancer drug candidates, some of which are based on license agreements with The University of Texas System on behalf of the M.D. Anderson Cancer Center, announced that company’s novel product, Annamycin (also known as “Liposomal Annamycin”), an anthracycline has been designated as orphan drug by the U.S. Food and Drug Administration (FDA) for the treatment of acute myeloid leukemia (AML). Leukemia is a cancer of the white blood cells and the acute forms of leukemia can manifest quickly and leave patients with limited treatment options. AML is the most common type of acute leukemia in adults.

Orphan Drug Designation is granted to drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity upon marketing approval for the designated indication, as well as with tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and the waiver of prescription drug user 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 orphan drug status given to Exelixis, Inc. (EXEL) for the Treatment of Hepatocellular Carcinoma

US Food and Drug Administration have granted orphan drug designation to Exelixis’ novel product cabozantinib for the treatment of hepatocellular carcinoma (HCC). A pivotal phase 3 trial (CELESTIAL) of cabozantinib is in process with patients suffering from advanced HCC.

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

 

Opko receives orphan drug designation for CUR-1916 in Dravet Syndrome

Opko Health Inc., a leading pharmaceutical company has announced that US Food and Drug Administration has granted orphan drug designation for company’s oligonucleotide-based AntagoNAT (CUR-1916) for the treatment of Dravet Syndrome. Presently, the syndrome does not own any of the approved treatment. Oligonucleotides are synthetic chemical compounds consisting of mixtures of modified DNAs and RNAs.

Orphan drug status is granted to treatments for diseases that affect fewer than 200,000 people in the U.S. and provides certain incentives for medications intended for the treatment, diagnosis or prevention of rare diseases. At present, these incentives include seven years of marketing exclusivity for the orphan indication, certain federal grants, tax credits and waiver of certain FDA 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 orphan status for VAL001

Respitorius, a leading pharmaceutical firm has announced that US Food and Drug Administration has granted orphan drug status for VAL001 (valproic acid) for the treatment of diffuse large b-cell lymphoma. The drug is presently undergoing 1/2a clinical phase trial.

Orphan Drug Designation is granted to drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity upon marketing approval for the designated indication, as well as with tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and the waiver of prescription drug user 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 Sapience Therapeutics (ST-36)

Sapience Therapeutics, a leading biotechnonology company dedicated to develop therapies for complicated cancer cases, declared that US Food and Drug Administration (FDA) has awarded company’s novel product protein-based therapeutic ST-36, orphan drug designation for the treatment of glioma in patients.

Gliomas are a form of malignant brain cancer that affect approximately 18,000 to 20,000 people annually in the U.S. Nearly 70 percent of gliomas are glioblastoma, the most deadly form of brain cancer. Orphan Drug Designation is granted to drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity upon marketing approval for the designated indication, as well as with tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and the waiver of prescription drug user 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   

European Union grants orphan drug designation to LSK BioPharma’s Apatinib

LSK BioPharma, a leading biopharmaceutical company announced that company’s novel small-molecule angiogenesis inhibitor, Apatinib is awarded with orphan drug designation from European Commission for the effective treatment of gastric cancer.

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   

 

Prometic’s PBI-4050 receives FDA orphan drug status for Alström Syndrome

US Food and Drug Administration has announced orphan drug designation to orally active, anti-fibrotic, lead drug candidate, PBI-4050 Drug, for the treatment of Alstrӧm Syndrome (AS).  The disease is chronically debilitating as it can cause permanent blindness, deafness, type 2 diabetes and life-threatening due to progressive organ failure. Presently, there is no satisfactory method to treat patients with AS. The company is investigating drug for its effect on multiple organs of patient’s body.

Orphan Drug Designation is granted to drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity upon marketing approval for the designated indication, as well as with tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and the waiver of prescription drug user 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 designation to Miragen Therapeutics

Miragen Therapeutics, a leading biopharmaceutical company has announced that US Food and Drug Administration have granted orphan drug designation to its novel product, MRG-106 for the treatment of mycosis fungoides.

The orphan drug designation is granted to investigational drugs intended to treat rare diseases affecting fewer than 200,000 people nationwide. The status has the potential to provide several benefits to developers, including tax credits for clinical trials costs, exemptions from certain FDA application fees and seven years of market exclusivity upon 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   

 

EMA grants orphan drug designation to aTyr Pharma for the Treatment of Limb Girdle Muscular Dystrophy with Resolaris™

European Medicine Agency has granted orphan drug designation to aTyr Pharma, Inc. (LIFE), a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address severe, rare diseases, for company’s novel product, Resolaris™ for the treatment of limb girdle muscular dystrophy (LGMD) patients.

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

FDA announces orphan drug status for aNK Natural Killer Cell Therapy in Merkel Cell Carcinoma

NantKwest, Inc. has recently received orphan drug designation from US Food and Drug Administration (FDA) for its leading drug candidate, activated natural killer (aNK) cell therapy for treatment of patients with advanced Merkel cell carcinoma. NantKwest, Inc. is a pioneering, next generation, clinical-stage Immunotherapy Company focused on harnessing the unique power of our immune system using natural killer (NK) cells to treat cancer, infectious diseases and inflammatory diseases.

Presently the drug is undergoing its clinical Phase 2 trial at clinical sites in the United States treating subjects with metastatic or locally advanced Merkel cell carcinoma. FDA Orphan Drug Designation is granted to investigational therapies that address rare medical diseases or conditions that affect fewer than 200,000 people in the U.S. Orphan Drug status provides benefits to drug developers including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of marketing 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