Tag Archives: orphan drug pipeline

SGT-001 declared orphan drug for DMD

Solid Biosciences and Solid GT announces that US Food and Drug Administration (FDA) have granted Orphan drug status to SGT001 for the treatment of Duchenne Muscular Dystrophy (DMD). It is a rare and progressive muscle wasting disease that is usually diagnosed in childhood. SGT001 is the gene therapy mediated to amino-associated viral (AAV) vector. The gene therapy functions by potentially restoring the dystrophin expression in the muscles.

The drug has proved to provide long term systemic expression of comparatively shorter form of dystrophin in the muscles by its clinical studies. The gene therapy also benefits as improved muscle power and prevention from contraction induced damage. The therapy is expected to commercialize in early 2017.

The FDA Orphan Drug Designation is granted to medicines and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment medicine.

 

 

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

FDA grants orphan drug designation to Synlogic Proprietary Synthetic Biotic

Synlogic, a privately owned bio-pharmaceutical company focusing on developing novel medicines by synthetic biology and microbiome platform, announces that US Food and Drugs Administration grants its investigational program involving a live E. Coli Nissle bacterium modified to assimilate ammonia, for the treatment of urea cycle disorders (UCD). He company accounts for first Live E. Coli Nissle bacterium modified to assimilate ammonia for the potential treatment of UCDs.

UCDs are rare and severe genetic mutation allowing the build-up of toxic levels of ammonia in the bloodstream which causes severe neurological complications and even death. The synlogic’s leading candidate, SYNB1020, is currently undergoing clinical development. The company is planning to file another application for orphan drug status to FDA for its investigational new drug for treatment for UCDs.

The FDA Orphan Drug Designation is granted to medicines and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment medicine.

 

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

GS030 by GenSight Biologics receives orphan drug designation from EMA for Retinitis Pigmentosa

Gensight Biologics, a biopharma company specialized in developing innovative gene therapies for neurodegenerative retinal diseases and central nervous system disorders declared its leading candidate GS030 as Orphan drug designation, as approved by European Medicine Agency (EMA) for the treatment of retinitis pigmentosa. GS030 has also been classified as Advanced Therapy Medicinal product by EMA.

Currently, GS030 is undergoing preclinical development and laboratory practices are expected to initiate in September, 2017, regulatory toxicity study in non-human primates, prior to entering the clinic with a Phase I/II clinical trial in retinitis pigmentosa patients in Q3 2017, subject to toxicity results and future regulatory review.

The EMA’s designation of orphan status is granted to encourage new developments and therapies for rare diseases and disorders. Orphan status can be granted to those diseases that are rare. These incentives include protocol assistance, i.e. scientific advice specific for designated orphan medicines, and 10 years of market exclusivity once the medicine is on the market.

 

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

Tivorsan receives orphan drug designation for Human Recombinant Biglycan for DMD

Tivorsan pharmaceutical, a company focusing on developing drugs and treatments for all kinds of Duchenne Muscular Dystrophy (DMD) and other neuromuscular disorders, declared that food and Drug Association (FDA) has granted Orphan drug designation to its leading drug candidate, Human Recombinant Biglycan for the treatment of DMD.

Human Recombinant Biglycan, also called as TVN-102, is a small, naturally occurring extracellular matrix protein found in muscle. It has a unique mechanism of action, that of up regulating utrophin, nNOS (neuronal nitric oxide synthase) and other dystrophin-associated proteins in the muscle membrane, as well as controlling MuSK (muscle specific kinase) activity and localization at the nerve-muscle synapse.

FDA grants orphan drug status for rare and life threatening diseases that affect less than 200,000 people per year in the U.S. Also, FDA provide several beneficiaries to drug developers including assistance with clinical study design and drug development, tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.

 

 

 

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

FDA approves N-Methanocarbathymidine as orphan drug in U.S

N&N Pharmaceuticals Inc., an upcoming pharmaceuticals company announces N -Methanocarbathymidine receives orphan drug designation for the treatment of neonatal herpes simplex, by FDA.

Almost 25 % of all the pregnant women are seropositive for HSV-2, therefore, 85% of the neonates which are born with HSV-2 infection have one year mortality even with aggressive treatment. Despite of proper treatments the disease may cause death due to presence of virus-associated encephalitis and neutropenia. 

 

 

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

FDA orphan drug designation for ProTmune given to Fate Therapeutics

Fate therapeutics receives Orphan drug designation for prevention of graft-versus-host disease in patients undergoing allogeneic hematopoietic cell transplantation and also includes blood cancer and genetic disorders by Food and Drug Association (FDA). The company is committed to the development of programmed cellular immune therapies for cancer and immune disorders. The company boasts of the drug’s unique therapeutic potential to address life-threatening complications and improve the curative potential of allogeneic HCT.

Graft-versus-host disease (GvHD) is a severe immunological complication that arises when newly-transplanted donor immune cells attack the patient’s tissues and organs, resulting in a potentially fatal immune system reaction. Regardless of repetitive prevention practices, 50% of patients suffer from GvHD and Inspite of systematical treatment provided to patients only half of them responds positively.

ProTmune™ is an investigational programmed cellular immunotherapy undergoing clinical development for the prevention of acute GvHD and cytomegalovirus (CMV) infection in patients undergoing allogeneic HCT. The cell therapy is produced by modulating a donor-sourced, mobilized peripheral blood graft ex vivo with two small molecules (FT1050 and FT4145) to enhance the biological properties and therapeutic function of the graft’s immune cells. The programmed mobilized peripheral blood graft is administered to a patient as a one-time intravenous infusion.

 

 

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

Ra’s RX101494 receives orphan drug designation for PNH by EU

Ra pharmaceuticals announce that European Union has granted orphan drug designation to its leading candidate, RA101495 for the treatment of Paroxysmal Nocturnal Hemoglobinuria.

The European Commission grants orphan medicinal product status to provide incentives to develop medicinal products to treat, prevent or diagnose diseases or conditions that affect no more than five in 10,000 persons in the EU. The orphan medicinal product designation provides the company and its drug with certain benefits and incentives in the EU, including a period of market exclusivity of 10 years after the market approval, free consultation and scientific advice on contact with European Medicinal Agency.

For Free Sample of Global Orphan Drug Clinical Pipeline Reports Contact: avinash@kuickresearch.com

Or Visit:  Global Orphan Drug Market Future Outlook 2020

 

 

 

European Union grants orphan drug status to Crenolanib

Arog Pharmaceuticals declared that its leading drug candidate has received orphan drug designation for the treatment of patients with acute myeloid leukemia and soft tissue sarcoma.

 The European Commission grants orphan medicinal product status to provide incentives to develop medicinal products to treat, prevent or diagnose diseases or conditions that affect no more than five in 10,000 persons in the EU. The orphan medicinal product designation provides the company and its drug with certain benefits and incentives in the EU, including a period of market exclusivity of 10 years after the market approval, free consultation and scientific advice on contact with European Medicinal Agency.

For Free Sample of Global Orphan Drug Clinical Pipeline Reports Contact: avinash@kuickresearch.com

Or Visit:  Global Orphan Drug Market Future Outlook 2020

 

GX-H9 receives orphan designation for growth hormone deficiency

The Food and Drug Association has granted orphan drug designation to Generix’s leading product, GX-H9, for the treatment of patients with growth hormone deficiency.

 The FDA Orphan Drug Designation is granted to medicines and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment medicine.

For Free Sample of Global Orphan Drug Clinical Pipeline Reports Contact: avinash@kuickresearch.com

Or Visit:  Global Orphan Drug Market Future Outlook 2020

 

Cytori’s ECCS- 50 granted as orphan drug status for scleroderma

Cytori pharmaceuticals declared that its leading product ECCS 50 has been granted orphan drug status by Food and Drug Administration (FDA) for the treatment of patients with scleroderma.

The FDA Orphan Drug Designation is granted to medicines and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment medicine.

For Free Sample of Global Orphan Drug Clinical Pipeline Reports Contact: avinash@kuickresearch.com

Or Visit:  Global Orphan Drug Market Future Outlook 2020