Tag Archives: orphand rug market

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

 

EC grants orphan drug designation to Philogen SpA for the treatment of soft tissue sarcoma.

Philogen SpA, a clinical stage biological company dedicated to develop and commercialize immunology and immune-oncology protein therapeutics, declared that European commission has granted orphan drug designation to L19TNF – a non-covalent trimer of tumor necrosis factor (TNF) fused to an antibody specific to the extra-domain B of fibronectin in single-chain variable fragment format – for the treatment of soft-tissue sarcoma. Soft tissue sarcoma is estimated to affect approximately 2.8 in every 10,000 people in Europe and possess 5year survival rate of 58% for patients suffering from STS.

Rare diseases are also known as Orphan diseases. These diseases are generally categorized as chronic, degenerative, and life-threatening. The rare diseases normally include certain cancers, metabolic conditions, diseases of the nervous system and musculoskeletal disorders, which are extremely life-threatening. As per the official definition, rare diseases are classified as those that affect fewer than 200,000 in the United States and less than 5 in 10,000 in the EU. These two regions form a dominant portion of the global market for orphan drugs. 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 Free Sample of Global Orphan Drug Clinical Pipeline Reports Contact: avinash@kuickresearch.com

Or Visit:  Global Orphan Drug Market Future Outlook 2020

 

Singh Biotechnology receives orphan drug designation for STAT3

Food and Drug association, U.S, has granted orphan drug status to SBT-100, developed by Singh Biotechnology, a start-up biotech company. STAT3 has novel anti-STAT3 (Signal Transducer and Activator of Transcription 3) B VHH13 single domain antibody (SDAB) SBT100, for the treatment of pancreatic cancer. It can be inhibited either by directly targeting the intracellular protein or indirectly, by targeting the upstream regulators of the STAT3 pathway. Pancreatic cancer is a life threatening disease causing death of almost 95% of all the patients suffering.

Rare diseases are also known as Orphan diseases. These diseases are generally categorized as chronic, degenerative, and life-threatening. The rare diseases normally include certain cancers, metabolic conditions, diseases of the nervous system and musculoskeletal disorders, which are extremely life-threatening. As per the official definition, rare diseases are classified as those that affect fewer than 200,000 in the United States and less than 5 in 10,000 in the EU. These two regions form a dominant portion of the global market for orphan drugs. 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 Free Sample of Global Orphan Drug Clinical Pipeline Reports Contact: avinash@kuickresearch.com

Or Visit:  Global Orphan Drug Market Future Outlook 2020