Monthly Archives: March 2017

FDA grants orphan drug designation to Sangamo’s SB-318

Sangamo Biosciences Inc., a biopharmaceutical company announced that US Food and Drug Administration has granted orphan drug designation to its genome editing candidate, SB-319 for the treatment of Mucopolysaccharidosis Type I (MPS I), a rare lysosomal storage disorder.

FDA’s Orphan Drug Designation program provides certain incentives for companies developing therapeutics to treat rare diseases or conditions, defined as those affecting less than 200,000 individuals in the U.S. A drug candidate and its sponsor must meet several key criteria in order to qualify for, and obtain, orphan drug status. Once a drug has received orphan drug designation, the developer qualifies for a range of benefits, including federal grants, tax credits, reduction in certain regulatory fees, and the potential for seven years of market exclusivity for the drug following FDA marketing approval.

 

 

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

FDA grants orphan drug status to Vicore

US Food and Drug Administration has awarded Vicore Pharmaceuticals with orphan Drug designation for the treatment of Idiopathic Pulmonary Fibrosis (IPF). FDA’s Orphan Drug Designation program provides certain incentives for companies developing therapeutics to treat rare diseases or conditions, defined as those affecting less than 200,000 individuals in the U.S. A drug candidate and its sponsor must meet several key criteria in order to qualify for, and obtain, orphan drug status. Once a drug has received orphan drug designation, the developer qualifies for a range of benefits, including federal grants, tax credits, reduction in certain regulatory fees, and the potential for seven years of market exclusivity for the drug following FDA marketing approval.

 

 

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

NH Therapeutics receives orphan drug designation for congenital hypogonadotropic hypogonadism

NH Therapeutics, a biopharmaceutical company that focuses on developing drugs and therapies for effective treatment of rare neuroendocrine disorders, recently announced that European Medicine Agency has awarded its leading drug candidate NH-901 with orphan drug designation for the treatment of congenital hypogonadotropic hypogonadism. NH901 is a reformulation of a GnRH analogue, which has been repurposed to increase gonadotropins, sex hormones and boost fertility. The Stichting Centre for Human Drug Research (CHDR) in the Netherlands acted as the European-based sponsor on NHT’s behalf.

The European Commission or European Medicine Agency grants orphan drug designation 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 European Union. The orphan drug designation provides Abeona with incentives and benefits in the EU, including protocol assistance, reduced fees and protection from market competition once ABO-101 is approved for the treatment of MPS IIIB patients.

 

 

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

ONCURIOUS NV receives orphan drug status for TB-403 for Medulloblastoma

Oncurious NV, an oncology development company dedicated to develop innovative orphan drugs for the treatment of pediatric tumors, has received orphan drug designation for its novel product, TB-403 from European Medicine Agency (EMA) for the treatment of patients with Medulloblastoma.  TB-403 is currently being examined in a Phase I/II a clinical trial for treatment of medulloblastoma, a pediatric cancer.

TB-403 is a humanized monoclonal antibody against placental growth factor (PlGF). PlGF is expressed in several types of cancer, including medulloblastoma. In medulloblastoma patients, high expression of the PlGF receptor neuropilin 1 has been shown to correlate with poor overall patient survival.

FDA’s Orphan Drug Designation program provides certain incentives for companies developing therapeutics to treat rare diseases or conditions, defined as those affecting less than 200,000 individuals in the U.S. A drug candidate and its sponsor must meet several key criteria in order to qualify for, and obtain, orphan drug status. Once a drug has received orphan drug designation, the developer qualifies for a range of benefits, including federal grants, tax credits, reduction in certain regulatory fees, and the potential for seven years of market exclusivity for the drug following FDA marketing approval.

 

 

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

 

EC grants orphan drug designation to Therabron Therapeutic’s CG367

Therabron Therapeutics has recently announced that European Union has awarded its leading drug candidate, CG367 with orphan drug designation for the treatment of Bronchiolitis Obliterans Syndrome. Previously CG367 has been granted orphan drug status by US Food and Drug Administration.

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

 

Arthritis in Children gets orphan drug by FDA

US Food and Drug Administration has granted orphan drug designation to an oral liquid suspension of celecoxib for the treatment of children suffering from pediatric juvenile idiopathic arthritis. The liquid suspension is developed and formulated by a clinical stage biopharmaceutical company, NuBioPharma, LLC.  Nu-Celecoxib has been developed within flavors for children enabling measurement of precise doses that can easily be administered.

Juvenile Idiopathic Arthritis (JIA) is the most common type of arthritis in children under the age of 17. JIA is a chronic disease causing illness, flu-like symptoms, and serious inflammation in eyes, rashes, joint pain, swelling and stiffness. Prevalence of JIA is recorded to be 141,300 patients in United States.

FDA’s Orphan Drug Designation program provides certain incentives for companies developing therapeutics to treat rare diseases or conditions, defined as those affecting less than 200,000 individuals in the U.S. A drug candidate and its sponsor must meet several key criteria in order to qualify for, and obtain, orphan drug status. Once a drug has received orphan drug designation, the developer qualifies for a range of benefits, including federal grants, tax credits, reduction in certain regulatory fees, and the potential for seven years of market exclusivity for the drug following FDA marketing approval.

 

 

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

Idogen receives orphan drug status for Hemophilia A

European Commission has granted orphan drug designation to Idogen for the treatment of patients with Hemophilia A. The European Commission grants orphan drug designation 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 European Union. The orphan drug designation provides Abeona with incentives and benefits in the EU, including protocol assistance, reduced fees and protection from market competition once ABO-101 is approved for the treatment of MPS IIIB patients.

 

 

 

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

 

Abeona receives orphan drug designation for ABO-101 to treat Sanfilippo Syndrome Type- B

Abeona therapeutics, a clinical stage bio-pharmaceutical company dedicated to develop gene therapies for life threatening diseases, has recently announced that its novel gene therapy candidate, ABO-101 has been awarded with orphan drug status for the treatment of children with Sanfilippo Syndrome Type-B, by the European Medicine Agency (EMA), a committee for orphan medicinal products. Sanfilippo Syndrome Type-B is a rare genetic disease causing neurocognitive decline, speech loss, loss of mobility, and premature death in children.

Previously, the product has been grant with orphan drug designation by US Food and Drug Administration (FDA). The organization has allowed the drug for clinical phase ½ trial and enrollments are due in the second quarter of 2017.

The European Commission grants orphan drug designation 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 European Union. The orphan drug designation provides Abeona with incentives and benefits in the EU, including protocol assistance, reduced fees and protection from market competition once ABO-101 is approved for the treatment of MPS IIIB patients.

 

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

 

FDA grants orphan drug designation to sickle cell treatment

Imara therapeutics, a leading biopharmaceutical announced that US Food and Drug Administration have granted orphan drug designation to its novel drug, IMR-657 to treat patients with Sickle cell disease. Presently, IMR-657 is stepping towards its first clinical phase to investigate its safety and pharmo-kinetics in patients with sickle cell. The study is currently being examined over patients between the ages of 18-55 years having sickle cell.

IMR-687 is an orally-administered selective phosphodiesterase 9 (PDE9) inhibitor. Pre-clinical data has demonstrated that IMR-687 reduces both the sickling of red blood cells and blood vessel obstruction that cause sapping pain, organ damage, and early mortality in affected sickle cell patients. Sickle cell is a rare genetic disorder causing hard, sticky and C-shaped red blood cells which further blocks smaller cell resulting into pain as well as increases the risk of infection, acute chest syndrome and stroke.

FDA’s Orphan Drug Designation program provides certain incentives for companies developing therapeutics to treat rare diseases or conditions, defined as those affecting less than 200,000 individuals in the U.S. A drug candidate and its sponsor must meet several key criteria in order to qualify for, and obtain, orphan drug status. Once a drug has received orphan drug designation, the developer qualifies for a range of benefits, including federal grants, tax credits, reduction in certain regulatory fees, and the potential for seven years of market exclusivity for the drug following FDA marketing approval.

 

 

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

FDA grants orphan drug designation to lysogene’s LYSGM101 for GM1 Gangliosidosis

US Food and Drug Association has designated LYGSM101 as orphan drug, developed by Lysogene, a clinical stage biotechnology company focused on developing gene therapy for rare central nervous system diseases, for the treatment of GM1 Gangliosidosis. LY-GSM101 is the company’s leading gene therapy candidate.

Action of mechanism for the novel gene therapy, LYGSM101 is to replace the defective genes in the cells of patients suffering from GM1 Gangliosidosis to allow production and functional enzyme and to prevent the progressive nature of neurological damage caused by GM1 in human body. GM1 is an extremely severe, autosomal recessive disease caused by a mutation in the GLB1 gene encoding for the lysosomal acid beta-galactosidase (ßgal) enzyme. The resulting enzymatic deficiency leads to accumulation of GM1-ganglioside in cells.

FDA’s Orphan Drug Designation program provides certain incentives for companies developing therapeutics to treat rare diseases or conditions, defined as those affecting less than 200,000 individuals in the U.S. A drug candidate and its sponsor must meet several key criteria in order to qualify for, and obtain, orphan drug status. Once a drug has received orphan drug designation, the developer qualifies for a range of benefits, including federal grants, tax credits, reduction in certain regulatory fees, and the potential for seven years of market exclusivity for the drug following FDA marketing approval.

 

 

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