Tag Archives: global orphan drug market pipeline

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.

The EMA’s designation of orphan status is granted to encourage new developments and therapies for rare diseases and disorders. Orphan status can be granted to those diseases that are rare. These incentives include protocol assistance, i.e. scientific advice specific for designated orphan medicines, and 10 years of market exclusivity once the medicine is on the market.

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

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.

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.

 

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

FDA grants VX15 from Vaccinex Inc. Orphan Drug designation for Huntington’s disease

VX15 developed by Vaccinex Inc., a pharmaceutical company focusing on the discovery and development of therapeutic monoclonal antibodies to treat patients with cancer and neurodegenerative diseases, has been designated as orphan drug by Food and Drug Association (FDA) for the treatment of Huntington’s disease.

Huntington’s disease (HD) is a hereditary neurodegenerative disease that causes progressive breakdown of nerve cells in the brain. The disease is commonly found in middle aged people followed by severely affecting them in next 5-10 years.

FDA grants orphan drug status for rare and life threatening diseases that affect less than 200,000 people per year in the U.S. Also, FDA provide several beneficiaries 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 Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

Singh Biotechnology receives orphan drug designation for STAT3

Food and Drug association, U.S, has granted orphan drug status to SBT-100, developed by Singh Biotechnology, a start-up biotech company. STAT3 has novel anti-STAT3 (Signal Transducer and Activator of Transcription 3) B VHH13 single domain antibody (SDAB) SBT100, for the treatment of pancreatic cancer. It can be inhibited either by directly targeting the intracellular protein or indirectly, by targeting the upstream regulators of the STAT3 pathway. Pancreatic cancer is a life threatening disease causing death of almost 95% of all the patients suffering.

Orphan designation is granted by the FDA Office of Orphan Products Development to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The drug also qualifies for financial incentives and tax credits clinical investigation and certain FDA application fee exemption and seven year of market exclusivity on regulatory product approval.

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

GLPG1690 from Galapogas, receives orphan drug designation for idiopathic pulmonary fibrosis

Galapogas, a Belgium based drug developing company, has received orphan drug status for GLPG1690 to treat patients suffering from idiopathic pulmonary fibrosis, in Europe. GLPG1690 has unique approach to target a novel molecule, GPR84, a pro-inflammatory protein responsible for chronic low grade inflammatory diseases targets a novel molecule, GPR84, a pro-inflammatory protein responsible for chronic low grade inflammatory diseases.

 The drug yielded a positive result in efficacy, safety, and tolerability in phase 1 clinical investigation.

 Pulmonary fibrosis is a lung damaging disease including thick, stiff and scarred lungs. Affected lungs are not able to pump oxygen all over the body, as a result, the human brain does not receives the required oxygen.

 

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

FDA approves Soricimed’s SOR-C13 as orphan drug for pancreatic cancer

Peptide SOR-C13 of Soricimed Biopharma Inc., known for discovering and developing peptide based cancer therapeautics, has been granted as orphan drug designation for the treatment of pancreatic and ovarian cancer by Food and Drug Association (FDA), U.S.

Pancreatic cancer, being the eleventh most common cancer and third leading cause of cancer related deaths in United States, causes approximately 53,070 deaths per year in U.S and 330,000 per year deaths per year worldwide.

According FDA, orphan drug designation status is given to those drugs or medication which helps in the treatment of rare and severe diseases, keeping in mind the potential benefit of product treating the disease. FDA defines rare diseases as those which affects. Peptide SOR-C13, if approved for commercialization by the FDA, SOR-C13 may qualify for seven years of marketing exclusivity in the United States for this indication. In certain cases, it can be made available for patients before marketing approval of compassionate use.

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

Coversin by Akari therapeutics receives Orphan drug Designation from FDA

Coversin developed by Akari therapeutics, an emerging pharmaceuticals company dedicated to the development and commercialization of innovative therapeutics to treat orphan autoimmune and inflammatory diseases has been granted orphan drug designation for the treatment of Paroxysmal Nocturnal Hemoglobinuria.

PNH is an ultra-rare, life-threatening and debilitating disease of the blood with estimated 8,000-10,000 patients across North America and Europe. The PNH patients suffer from a certain genetic deficiency in which they allow their own complement system to attack and destroy blood cells, leading to life-threatening complications.

According to FDA, orphan drug designation status is given to those drugs or treatments which promise to cure or prevent rare life threatening diseases. By FDA, rare diseases are defined as those which affect less than 200,000 people in US. Also, FDA helps the companies which cultivate such drugs by providing them with incentives to sponsor developing drugs and biologics. The receipt of Orphan Drug Designation status does not change the regulatory requirements or process for obtaining marketing approval and designation does not mean that marketing approval will be received.

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

Theranexus’s THN102 receives orphan drug designation from FDA for treatment of Narcolepsy

US Food and Drug Administration has granted orphan drug designation to THN102 developed by Theraneux, a clinical stage biopharmaceutical company, for the treatment of narcolepsy, a rare and highly debilitating sleep/wake disorder. The company has initiated a clinical phase 2 study for determining the potential of THN102 in patients with narcolepsy.

 

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 BP1001 for AML treatment

Bio-Path Holding Inc. declares that European Medicine Agency has granted orphan drug Designation to BP1001 for treatment of acute myeloid leukemia (AML). BP1001 is introduced by Bio-Path Holding Inc. to fill the void of the treatments and medications for effective treatment of acute myeloid leukemia.

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