Tag Archives: orphan drug development

FDA declares Firdapse as orphan drug for treatment of Myasthenia Gravis

Catalyst Pharmaceuticals, a company dedicated to drug discovering and developing for patients with rare diseases, announces that orphan drug designation has been granted to Firdapse to treat patients suffering from myasthenia gravis, by Food and Drug Association (FDA).

Antibodies to the muscle-specific kinase (MuSK-MG), results in a rare Myasthenia Gravis disease, found to be dominant in females and characterized by prominent bulbar involvement, more severe clinical condition and resistance to treatment. Many patients with Myasthenia Gravis are recorded to be unresponsive to the current treatment with anticholinesterase inhibitors or immunosuppressant.

Orphan Drug designation is granted by the FDA’s Office of Orphan Products Development for drugs that are expected to provide significant therapeutic advantage over existing treatments and that target conditions affecting 200,000 or fewer U.S. patients annually. The benefits apply across all stages of drug development and include an accelerated approval process; seven years of market exclusivity following marketing approval; tax credits on U.S. clinical trials; eligibility for Orphan Drug grants; and waiver of Prescription Drug User Fee Act (PDUFA) and certain other administrative fees.

 

 

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

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

 

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

 

 

 

FDA grants orphan drug status to Singh Biotechnology

Singh Biotechnology, a pharmaceutical company, announces that its SBT-100, its novel anti-STAT3 (Signal Transducer and Activator of Transcription 3) B VHH13 single domain antibody (sdAb) SBT-100, has been granted as orphan drug designation by US Food and Drug Administration (FDA) for the treatment of pancreatic Cancer. It has been investigated that elevated STAT3 is linked with poor prognosis of solid tumors, including tumor of the pancreas. It can be inhibited either by directly targeting the intracellular protein or indirectly, by targeting the upstream regulators of the STAT3 pathway.

 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

 

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

 

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

 

EU grants orphan drug status to Respiratorius’s VAL001

The European Commission has granted orphan drug status to VAL001, the leading product candate from respiratorius pharmaceuticals for the treatment of diffused large b-cell lymphoma.

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