Tag Archives: global orphan drug pipeline

First orphan drug introduced for Friedreich’s Ataxia: FDA

Agilis Biotherapeutics, LLC (Agilis), a biotechnology company dedicated to innovation of DNA therapies for rare genetic diseases damaging the central nervous system, declared that Food and Drug Association (FDA)and United States Food has granted orphan drug designation to AGIL-FA , company’s gene therapy product candidate to treat patients of Friedreich’s Ataxia.

Friedreich’s Ataxia (FA) is an inherited degenerative neuromuscular disorder resulting in loss of motor coordination and strength, hearing, vision, speech and often premature death. It is primarily caused by a defect in the FXN gene that lowers the production of the frataxin protein.

Agilis gene therapy is considered to be the first one in the list of FDA approved therapies for treatment of FA. Also the company holds fourth position in the list of all orphan designated drugs declared by FDA this year. Agilis gene is designed to deliver corrective DNA to specific CNS cells to restore frataxin protein levels in patient’s body.

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 grants orphan drug designation to BIV201 for treatment of Acsites

BioVie Inc., a company dedicated to discovering and developing therapeutic drugs, declares that it’s new drug candidate BIV201 has received orphan drug designation for the treatment of acsites due to chronic liver cirrhosis.

Currently, BioVie is looking forward to complete an investigational new drug (IND) application for BIV201 having potent vasoconstrictor as an active ingredient, which qualifies the drug for effective treatment of various life threatening diseases including type 1 hepatorenal syndrome (HRS), esophageal variceal bleeds, and sepsis.

 

 

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

Orphan drug designation plea for Delta-9-Tetrahydrocannibinol and Cannabidiol

PhytoPain Pharma Inc., a part of GrowPros Cannabis Ventures Inc., declares the submission of applications for orphan drug designation status for Delta-9-Tetrahydrocannibinol and Cannabidiol for the treatment of Complex Regional Pain Syndrome Type 1 (CRPS1) and Central PostStroke Pain (CPSP).  Also, it will help in treatment of neuropathic pain associated with cancer, HIV, CPSP, and CRPS1. The aim is to commercialize the drug for the management of uncontrolled pain related to life threatening diseases.

 

 

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

SB- Fix receives orphan drug designation for Hemophilia B

 FDA announces orphan drug designation status to SB-FIX , zinc finger nuclease (ZFN)-mediated genome editing product for the treatment of hemophilia  from Sangamo Biosciences. The company has aces in therapeutic genome editing.

SB-FIX is designed as a single treatment strategy intended to provide stable, continuous production of Factor IX clotting protein (FIX) for the lifetime of the patient. Sangamo’s ZFN-mediated in vivo genome editing approach makes use of the albumin gene locus, a highly expressing and liver-specific genomic “safe-harbor site,” that can be edited with ZFNs to accept and express therapeutic genes. The approach is designed to enable the patient’s liver to permanently produce circulating therapeutic levels of a corrective protein product.

Hemophilia B is a rare bleeding disorder caused by mutations in genes that encode factors which help the blood clot and stop bleeding when blood vessels are injured. The patients of Hemophilia B experience continuous bleeding after injuries which leads to joint diseases such as arthritis.

FDA grants orphan drug designation for certain rare and life threatening diseases which affects less than 200,000 people in the U.S. Orphan drug status is intended to facilitate drug development for rare diseases and provides several benefits 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

EMA grants BioMarin’s Brinuera orphan drug status for CLN2 disease

BioMarin pharmaceuticals Inc. declares its leading drug candidate, Brinuera as orphan drug as approved by European Medicinal Agency (EMA) for the treatment of a form of Batten disease, CLN2 disease usually found in children. The disease is generally diagnosed in children of 2 to 4 years of age causing them to lose their ability to walk by 6 years of age and the patient may die while reaching to 12 years of age.. Symptoms of CLN2 include language delay and seizures, followed by movement disorders, motor deterioration, dementia and blindness.

The European medicinal Agency (EMA) 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

 

FDA approves SD-809 for orphan drug designation

The US food and drug Administration (FDA) has granted orphan drug status SD-809 (deutetrabenazine) for the treatment of chorea associated with Huntington disease (HD). The compound is being developed by an Israeli drug manufacturer, Teva Pharmaceutical Industries (NYSE: TEVA). The drug is also being tested for the treatment of tardive dyskinesia (TD) and Tourette syndrome (TS).

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

 

Ofev receives orphan drug status for Potential Scleroderma

Ofev, a kinase inhibitor, has been declared as an orphan drug by European Commission and US Food and Drug Administration for the treatment of potential scleroderma and related interstitial lung diseases. The drug is currently in its phase 3 clinical trials.

The FDA and EMA’s designation of orphan status is granted to encourage new developments and therapies for rare diseases and disorders. Orphan Status can be applied to products proven safe and effective in treating conditions that affect a relatively small number of patients, and the designation may entitle up to seven years of US marketing exclusivity upon regulatory approval. Companies whose drugs have an orphan designation also receive certain tax credits and are exempted from paying prescription drug user fees normally required of companies submitting products for approval.

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

Or Visit:  Global Orphan Drug Market Future Outlook 2020

 

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