Tag Archives: orphand rug designation

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

 

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

 

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

 

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

 

MediciNova declares FDA approval for MN-166 as Orphan Drug

Medicinova, a biopharmaceutical company, announced FDA approved orphan drug designation for its leading medical treatment, MN-166 (ibudilast) for the treatment of Amyotrophic Lateral Sclerosis (ALS). Currently, there is only one approved treatment for ALS that is riluzole, present in the market. MN-166 is presently undergoing clinical trials by researchers at Carolinas HealthCare System’s (CHS).

Amyotrophic Lateral Sclerosis (ALS) is also called as Lou Gehrig’s disease is a progressive neuro-degenerative disease that affects brain and the spinal cord. ALS drains the ability to trigger specific muscles causing weakness in them. It affects the voluntary movement in the patient and causes total paralysis at later stages.

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