Tag Archives: orphand rug act

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

 

 

 

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

 

DPX-Survivac receives orphan drug status from EMA

The US Food and Drug Association (FDA) has granted orphan drug designation Immunovaccine’s leading asset, DPX- Survivac for the treatment of patients with ovarian cancer. The EMA grants orphan designation to rare diseases that are defined as life-threatening or chronically debilitating conditions that affect no more than 5 in 10,000 people in the EU. With an estimated 30 million people living in the EU this equates to approximately 250,000 people or less for each rare disease.

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

Or Visit:  Global Orphan Drug Market Future Outlook 2020

 

Iomab-B receives EMA orphan drug status for AML

Iomab-B manufactured by Actinium Pharmaceuticals Inc. has been declared as orphan drug by European Medicines Agency for the treatment of patients with Acute Myeloid Leukemia (AML). The drug has the clinical tested usage in the treatment of patients with relapsed or refractory Acute Myeloid Leukemia (AML) with an age crossing 55 years. The major use of the drug is to prepare patients for bone marrow transplant also called as hematopoietic stem cell transplant (HSCT). Currently, it is undergoing phase III clinical trial with 150 patients in the US.

The EMA grants orphan designation to rare diseases that are defined as life-threatening or chronically debilitating conditions that affect no more than 5 in 10,000 people in the EU. With an estimated 30 million people living in the EU this equates to approximately 250,000 people or less for each rare disease.

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

Or Visit:  Global Orphan Drug Market Future Outlook 2020