Tag Archives: global orphan drug market overview

FDA grants orphan drug status to Diazepam Buccal soluble film

MonoSol Rx, a leading pharmaceutical company has announced that U.S Food and Drug Administration (FDA) have granted orphan drug designation to Diazepam Buccal soluble film to treat acute retentive seizures. The agency is planning to initiate a pivotal study of diazepam BSF in adults in early 2017.

Orphan Drug designation is granted to those medications or therapy that are rare, more specifically, affect less than 200,000 patients in the country. These rare diseases are incurable and severe in nature and may cause death. To fulfill the need of treat options for individuals, FDA grants orphan drug designation to drugs which are investigated to be helpful in the treatment of such rare diseases. Orphan drug status is a package of several financial benefits for the drugs and its manufacturers such as certain tax credits and exemption from paying prescription drug user fees normally required from companies submitting products for approval.

 

 

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

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.

In order to get EMA designation of orphan drug, the therapy or treatment must be curing any of the rare life threatening disease with a prevalence of less than 5 in 10,000 people in the European Union. Orphan drug designation provides incentives designed to facilitate development including fee reductions for protocol assistance, scientific advice and importantly, may provide up to ten years of market exclusivity in the EU following product approval.

 

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

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

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.

Orphan Drug designation is granted by the FDA’s Office of Orphan Products Development for drugs that are expected to provide significant therapeutic advantage over existing treatments and that target conditions affecting 200,000 or fewer U.S. patients annually. Orphan Drug designation qualifies a company for several benefits under the Orphan Drug Act 1983. These benefits include assistance with clinical study design in 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

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

Tivorsan receives orphan drug designation for Human Recombinant Biglycan for DMD

Tivorsan pharmaceutical, a company focusing on developing drugs and treatments for all kinds of Duchenne Muscular Dystrophy (DMD) and other neuromuscular disorders, declared that food and Drug Association (FDA) has granted Orphan drug designation to its leading drug candidate, Human Recombinant Biglycan for the treatment of DMD.

Human Recombinant Biglycan, also called as TVN-102, is a small, naturally occurring extracellular matrix protein found in muscle. It has a unique mechanism of action, that of up regulating utrophin, nNOS (neuronal nitric oxide synthase) and other dystrophin-associated proteins in the muscle membrane, as well as controlling MuSK (muscle specific kinase) activity and localization at the nerve-muscle synapse.

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 grants orphan drug designation to OV101 for Angelman Syndrome

Ovid therapeutics, a private pharmaceutical company from New York, developed OV101 for the treatment of angelman syndrome. Food and Drug Association, U.S, has approves OV101 as orphan drug designation.

OV101 is the first potential therapy to target the disruption of tonic inhibition, a key mechanism that allows the brain to fine-tune neurological signaling and accurately decipher excitatory from inhibitory signals, seen in this disorder.

Angelman Syndrome is a rare genetic disorder which causes developmental and neurologic disabilities such as difficulty in speaking, balancing and walking, as well as other symptoms such as anxiety, sleep disturbances and seizures.

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