Monthly Archives: November 2016

FDA announces orphan drug designation for Veliparib as NSCLC treatment

US Food and drug Association (FDA) has declared Veliparib, an oral poly (adenosine diphosphate [ADP]-ribose) polymerase (PARP) inhibitor, developed by Abbvie (ABBV). Currently, Veliparib is undergoing investigation in combination with chemotherapies, such as carboplatin and paclitaxel, or radiation for the treatment of advanced squamous non-small cell lung cancer (NSCLC).

PARP is the naturally occurring enzyme in the body that repairs damage to DNA in the cells. The process is useful to maintain the integrity of healthy cells, and can help in repairing DNA cancer cells, causing them to survive. Veliparib is currently under investigation that its combination with DNA damaging therapies, such as chemotherapy or radiation, may lessen the repair of DNA damage in cancer cells, eventually causing some cells to die.

NSCLC is the common type of lung cancer in the US accounting for 80 to 85% of diagnosed cancer cases. Survival from lung cancer depends on the stage at which cancer is diagnosed.

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

 

FDA approves orphan drug status for Dusquetide for Macrophage Activation Syndrome

Soligenix Inc., a pharmaceutical company, specialized in developing and commercializing medicines to treat rare diseases declared that Food and Drug association (FDA) has granted its leading candidate, Dusquetide, orphan drug designation for the treatment of patients with Macrophage Activation Syndrome (MAS). Perviously, Dusquetide was granted orphan drug status for treatment of Acute Radiation Syndrome (ARS).

Dusquetide is an innate defense regulator (IDR) belonging to a new class of short, synthetic peptides that accelerates bacterial clearance and resolution of tissue damage while modulating inflammation following exposure to a variety of agents including bacterial pathogens, trauma, radiation and/or chemotherapy.

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

 

GS030 by GenSight Biologics receives orphan drug designation from EMA for Retinitis Pigmentosa

Gensight Biologics, a biopharma company specialized in developing innovative gene therapies for neurodegenerative retinal diseases and central nervous system disorders declared its leading candidate GS030 as Orphan drug designation, as approved by European Medicine Agency (EMA) for the treatment of retinitis pigmentosa. GS030 has also been classified as Advanced Therapy Medicinal product by EMA.

Currently, GS030 is undergoing preclinical development and laboratory practices are expected to initiate in September, 2017, regulatory toxicity study in non-human primates, prior to entering the clinic with a Phase I/II clinical trial in retinitis pigmentosa patients in Q3 2017, subject to toxicity results and future regulatory review.

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

 

FDA announces orphan drug designation for DTX401 for the treatment of Glycogen Storage Disease Type Ia (GSDIa)

Dimension Therapeutics Inc., a biopharmaceutical company specialized in developing gene therapies targeting liver related diseases, declared that US Food and Drug Association (FDA) granted orphan drug status for DTX401 for the treatment of patients with Glycogen Storage Disease Type Ia (GSDIa). DTX401 is the first AAV gene therapy to enter IND-enabling studies for the treatment of GSDIa.

DTX401 has been designed to serve underlying genetic effect for which it delivers stable expression and activity of the enzyme glucose-6-phosphatase (G6Pase). It improves G6Pase activity and reduces hepatic glycogen levels, a well-described biomarker of disease progression, in multiple in vivo preclinical models.

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

 

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

 

EMA grants orphan drug status to GLPG1690 for idiopathic pulmonary fibrosis

Galapagos, a bio-technology company dedicated to produce small molecule medicines with novel modes of action, declared that its novel medication GLPG1690 has been approved as orphan drug by European Commission for the treatment of idiopathic pulmonary fibrosis (IPF).

IPF is a chronic fibrotic disorder of lungs affecting people above the age of 40 years. IPF is a rare disease as its prevalence has been estimated to be 30 per 100,000 people in Europe as well as US. Presently, there are no medications available to cure IPF.

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

 

Firdapse gets orphan drug status for treatment of Myasthenia Gravis

Catalyst pharmaceuticals Inc., a biopharmaceutical company, declare its novel therapy Firdapse (amifampridine phosphate) as orphan drug for the treatment of patients with Myasthenia Gravis, as approved by Food and Drug Association (FDA). The company focuses on developing and commercializing new therapies for the treatment of rare debilitating diseases.

Myasthenia Gravis is the rare neuromuscular disease antibodies to the muscle-specific kinase (MuSK-MG) which inflicts approximately 5-8% of Myasthenia Gravis patients which is equal to 4,500 patients in the US. The characteristics of myasthenia gravis are predominance in women, prominent bulbar involvement, sever clinical conditions and resistant to treatment. Presently, there are anticholinesterase inhibitors or immunosuppressant available for the treatment of the disease.

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

 

FDA announces orphan drug designation for TG-1101 for NMO and NMOSD

TG Therapeutics Inc., a biopharmaceutical company  focusing on developing and commercializing the medicines or treatments for B-cell malignancies and auto-immune diseases, developed TG-1101 (ublituximab), the company’s novel products has been designated as orphan drug by Food and Drug Association (FDA) for the treatment of Neuromyelitis Optica (NMO) and Neuromyelitis Optica Spectrum Disorder (NMOSD). Presently, there are no other FDA approved treatment options available in the market for NMO and NMSD. TG-1101 is the glycol-engineered anti-CD20 monoclonal antibody and a non-oncology indication providing additional protection.

Neuromyelitis Optica (NMO) and Neuromyelitis Optica Spectrum Disorder (NMOSD) are also called as Devic’s disease. It is an immune related chronic inflammatory disorder involving different parts of the central nervous system, primarily damaging optic nerve and spinal cord. The affected optic nerve and spinal cord causes loss of swelling and inflammation in eyes, vision and pain weakness or paralysis in the legs or arms, loss of sensation, and problems with bladder and bowel function. The disease rarely possess its progressive phase.

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

 

TiGenix’s Cx601 granted as orphan drug designation in Switzerland

TiGenix NV, a biopharmaceutical company, focused on developing novel therapeutics from its proprietary platform of allogeneic expanded stem cells, declared that its leading product Cx601, has been granted orphan drug designation status in Switzerland, for the treatment of complex perianal fistulas in Crohn’s disease patients. Cx601 is developed by TiGenix and licensed to Takeda Pharmaceutical Company Limited for development and commercialization outside the US.

Cx601 is the suspension of allogeneic adipose-derived stem cells (eASC) injected within a lesion for curing complex perianal fistulas in patients with Crohn’s disease which is irresponsive to at least one of the conventional or biologic treatment. Crohn’s disease is the inflammatory disease of intestines. Patients suffering with crohn’s disease develop complex perianal fistulas for which there not treatments options available.

Swiss agency declared orphan drug status for therapeutic products regarding the application dossier for Cx601, or eASCs, adipose-derived stem cells for the rare disease anal fistulas. This was based on the orphan drug designation by European commission in 2009, which defines proof of quality, efficacy and safety and rarity of the disease as well.

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

 

EMA announces orphan drug designation for ABO-102 gene therapy

Abeona Therapeutics Inc., a biopharmaceutical company dedicated to develop gene therapies for life threatening rare diseases declared that leading therapy ABO-102 has been designated as orphan drug b European Medicines Agency (EMA) for the treatment of Sanfilippo syndrome type A (MPS IIIA), a rare autosomal recessive disease causing neurocognitive decline, speech of loss, premature death in children and loss of mobility.

Abeona’s MPS IIIA program has been previously granted the designation of orphan drug by Food and Drug Association (FDA) in the US and received the Rare Pediatric Disease Designation as a pre-requisite part of the Priority Review Voucher (PRV) process.

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