Tag Archives: orphan drug 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.

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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

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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

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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

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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

 

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

 

SGT-001 declared orphan drug for DMD

Solid Biosciences and Solid GT announces that US Food and Drug Administration (FDA) have granted Orphan drug status to SGT001 for the treatment of Duchenne Muscular Dystrophy (DMD). It is a rare and progressive muscle wasting disease that is usually diagnosed in childhood. SGT001 is the gene therapy mediated to amino-associated viral (AAV) vector. The gene therapy functions by potentially restoring the dystrophin expression in the muscles.

The drug has proved to provide long term systemic expression of comparatively shorter form of dystrophin in the muscles by its clinical studies. The gene therapy also benefits as improved muscle power and prevention from contraction induced damage. The therapy is expected to commercialize in early 2017.

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

 

FDA grants orphan drug status to SER100 in PAH

Serodus announces orphan drug designation for its leading product, SER100 by US Food and Drug Administration (FDA) for the treatment of Pulmonary Arterial Hypertension (PAH). The company is planning for first clinical study in phase IIa or combined phase IIa/III study in patients with PAH.

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