Tag Archives: orphan drug status

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

 

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

 

Lundbeck’s carnexiv receives FDA orphan drug status

Lundbeck declares orphan drug designation by Food and Drug Association (FDA) for its leading product, Carnexiv injection for the treatment of oral carbamazepine formulations in adults with certain types of seizures when oral administration is temporary not feasible. Carnexiv is the first intravenous formulation of antiepileptic drug (AED) carbamazepine. The pharmaceutical company plans to introduce the drug commercially in early 2017 in the United States.

Carnexiv is a short term alternative therapy for oral carbamazepine formulations that delivers constant care for adult patients who are unable to take the drug by mouth. The oral dosage of the drug consist of certain side effects as dermatologic reactions such as toxic epidermal neurolysis and stevens-johnson syndrome as well as risk of aplastic anemia and agranulocytosis.

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

 

Eloxx pharmaceuticals receives orphan drug designation for ELX-02 in Mucopolysacchardisis Type 1 (MPS 1) in US

Eloxx Pharmaceuticals, a biotechnology firm focusing on developing and commercializing medicines and treatments for rare diseases, announces that Food and Drug Administration in US and European Medicine Agency (EMA) has granted orphan drug status to its leading product candidate, ELX-02 for the treatment of patients with Mucopolysacchardisis Type 1 (MPS 1).

ELX-02 is a synthetic designer aminoglycoside optimized as a translational read-though drug (TRID) for the treatment of genetic diseases caused by nonsense mutations. The compound is currently under investigation in Phase 1 first in human (FIH) clinical study in healthy volunteers in Israel.

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

 

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

 

Invasive Aspergillus infections recieves orphan drug as SCY-078 by SCYNEXIS

Scynexis, a pharmaceutical company specialized in drug development, declared its triterpenoid broad-spectrum antifungal agent, SCY-078 as orphan drug as approved by Food and Drug Administration for the treatment of invasive Aspergillus infections. Previously, the company announced that SCY-078 has received orphan drug designation for the treatment of invasive Candida infections, including candidemia.

 The company was previously granted Qualified Infectious Disease Product (QIDP) designation for both the IV and oral formulations of SCY-078, which provides an additional five years of exclusivity. Together, these designations provide SCYNEXIS with a potential 12 years of market exclusivity in the U.S. upon FDA approval.

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

 

Solid Biosciences receives orphan drug designation from FDA & EMA for Duchenne Muscular Dystrophy

Solid Bioscience and its subsidiaries declares that Food and Drug Administration and European Medicine Agency has granted orphan drug designation to company’s gene therapy candidate, SGT-001, for the treatment of patients with Duchenne muscular dystrophy (DMD).

Duchenne muscular dystrophy (DMD) is a very rare and progressive muscle damaging disease affecting approximately 1 in 3,500-5,000 boys born worldwide and is the most common fatal genetic disorder diagnosed in childhood. The disease is mainly caused by the absence of dystrophin protein, an important mediator between cardiac and skeletal muscle function. The gene therapy is developed or designed to protect and maintain functional dystrophin expression in the muscles.

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