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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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.

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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.

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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.

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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.

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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

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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.

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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.

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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

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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