Tag Archives: drug

Coversin by Akari therapeutics receives Orphan drug Designation from FDA

Coversin developed by Akari therapeutics, an emerging pharmaceuticals company dedicated to the development and commercialization of innovative therapeutics to treat orphan autoimmune and inflammatory diseases has been granted orphan drug designation for the treatment of Paroxysmal Nocturnal Hemoglobinuria.

PNH is an ultra-rare, life-threatening and debilitating disease of the blood with estimated 8,000-10,000 patients across North America and Europe. The PNH patients suffer from a certain genetic deficiency in which they allow their own complement system to attack and destroy blood cells, leading to life-threatening complications.

According to FDA, orphan drug designation status is given to those drugs or treatments which promise to cure or prevent rare life threatening diseases. By FDA, rare diseases are defined as those which affect less than 200,000 people in US. Also, FDA helps the companies which cultivate such drugs by providing them with incentives to sponsor developing drugs and biologics. The receipt of Orphan Drug Designation status does not change the regulatory requirements or process for obtaining marketing approval and designation does not mean that marketing approval will be received.

 

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

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

 

 

 

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

 

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

 

ELX-02 receives orphan drug status for MPS I

Eloxx pharmaceuticals announces that its leading drug candidate has been approved as an orphan drug for the treatment of patients with novel mucopolysaccharidosis Tx type 1 (MPS I) by the US Food and Drug Administration (FDA). ELX-02 is a synthetic design of aminoglycoside enhanced as translational read through drug (TRID) for the treatment of genetic diseases caused by nonsense mutations. Currently, the drug is undergoing its phase 1 (first in Human) clinical trial.

Mucopolysaccharidosis type I (MPS I) is a chronic, progressive genetic disorder caused by an enzymatic deficiency of alpha-L-iduronidase (IDUA) which interrupts the glycosaminoglycan (GAG) catabolic pathway leading to an intra-lysosomal accumulation of substrates, heparan sulfate (HS) and dermatan sulfate (DS).

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

 

Resunab by Corbus Pharma receives Orphan drug status by EC

Corbus Pharmaceutical Holdings Inc. announces that its leading asset has been granted orphan drug designation by European Commission for the treatment of patients with Cystic Fibrosis. Resunab is the synthetic oral endocannabinoid-mimetic drug.

The pharmaceutical firm previously announced that the drug has been granted orphan drug status and fast track status for the treatment of systemic sclerosis by the US food and Drug Association (FDA). Currently, the drug is undergoing three clinical studies in phase 2 trials for Cystic Fibrosis, Systemic Sclerosis and dermatomyositis. Furthermore, the drug is expected to undergo clinical study for treatment of systemic lupus erythematosus in the first quarter of 2017.

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 status designation provides the company and its drug with certain benefits and incentives in the EU, including a period of market exclusivity.

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

Or Visit:  Global Orphan Drug Market Future Outlook 2020